Study of Luspatercept for the Treatment of Anemia in Pati... | NCT01749514 | Trialant
NCT01749514
Sponsor
Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
Status
Completed
Last Update Posted
Jul 29, 2024Actual
Enrollment
116Actual
Phase
Phase 2
Conditions
Anemia
Interventions
Luspatercept
Countries
Germany
Protocol Section
Identification Module
NCT ID
Results Section
Participant Flow Module
Pre-assignment Details
Annotation Section
No data available
No data is available for this block.
Document Section
Large Document Module
Document Has No Statistical Analysis Plan (SAP)
Derived Section
Miscellaneous Info Module
Version Holder
NCT01749514
Obsolete or Duplicate NCT IDs
Not provided
Organization Study
A536-03
Secondary IDs
ID
Type
Description
Link
MK-6143-001
Other Identifier
Merck
A536-03
Other Identifier
Acceleron
2012-002523-14
EudraCT Number
Brief Title
Study of Luspatercept for the Treatment of Anemia in Patients With Myelodysplastic Syndrome (MDS) (MK-6143-001)
Official Title
A Phase 2, Open Label, Ascending Dose Study of ACE-536 for the Treatment of Anemia in Patients With Low or Intermediate-1 Risk Myelodysplastic Syndromes (MDS)
Acronym
Not provided
Organization
Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USAINDUSTRY
Status Module
Record Verification Date
Feb 2024
Overall Recruitment Status or Expanded Access Status
Completed
Last Known Status
Not provided
Delayed Posting
Not provided
Why Stopped
Not provided
Expanded Access Info
No
Start Date
Jan 21, 2013Actual
Primary Completion Date
Oct 22, 2018Actual
Completion Date
Oct 22, 2018Actual
First Submitted Date
Dec 10, 2012
First Submission Date that Met QC Criteria
Dec 12, 2012
First Posted Date
Dec 13, 2012Estimated
Results Waived
Not provided
Results First Submitted Date
Apr 11, 2023
Results First Submitted that Met QC Criteria
Feb 8, 2024
Results First Posted Date
Jul 29, 2024Actual
Certification/Extension (aka Delayed Results) First Submitted Date
Not provided
Certification/Extension First Submitted that Passed QC Review
Not provided
Certification/Extension First Posted Date
Not provided
Last Update Submitted Date
Feb 8, 2024
Last Update Posted Date
Jul 29, 2024Actual
Sponsor/Collaborators Module
Responsible Party, by Official Title
Sponsor
Lead Sponsor
Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USAINDUSTRY
Collaborators
Not provided
Oversight Module
Has Data Monitoring Committee (DMC)
No
Is FDA Regulated Drug
Not provided
Is FDA Regulated Device
Not provided
Is Unapproved Device
Not provided
Pediatric Postmarket Surveillance of a Device Product
Not provided
Product Exported from US
Not provided
FDAAA801 Violation
Not provided
Description Module
Brief Summary
The purpose of this study is to evaluate the effects of luspatercept (MK-6143, formerly called ACE-536) on anemia in patients with low or intermediate-1 risk myelodysplastic syndrome (MDS). There is no primary hypothesis in this study.
Detailed Description
Not provided
Conditions Module
Conditions
Anemia
Keywords
Myelodysplastic Syndrome
Design Module
Study Type
Interventional
Number of References to an Expanded Access Study
Not provided
Expanded Access Types
Not provided
Patient Registry
Not provided
Target Follow-Up Duration
Not provided
Phases
Phase 2
Interventional Study Design
Allocation
Biospecimen
No data available
No data is available for this block.
Enrollment
116Actual
Arms/Interventions Module
Arm Groups
Label
Type
Description
Intervention Names
Luspatercept 0.125mg/kg (Cohort 1)
Experimental
Participants receive luspatercept 0.125mg/kg as a subcutaneous (SC) injection every 3 weeks (Q3W) on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
Drug: Luspatercept
Luspatercept 0.25mg/kg (Cohort 2)
Experimental
Participants receive luspatercept titrated up to 0.25mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
Drug: Luspatercept
Luspatercept 0.50mg/kg (Cohort 3)
Experimental
Participants receive luspatercept titrated up to 0.50mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
Drug: Luspatercept
Luspatercept 0.75mg/kg (Cohort 4)
Experimental
Participants receive luspatercept titrated up to 0.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
Drug: Luspatercept
Luspatercept 1.00mg/kg (Cohort 5)
Experimental
Participants receive luspatercept titrated up to 1.00mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
Interventions
Name
Type
Description
Arm Group Labels
Other Names
Luspatercept
Drug
Participants receive luspatercept up to 1.75mg/kg subcutaneously (SC) every 3 weeks for up to 5 cycles (each cycle length = 21 days).
Expansion Cohort
Outcomes Module
Primary Outcomes
Measure
Description
Time Frame
Percentage of Low Transfusion Burden (LTB) Participants With Modified Erythroid Response (mHI-E)
The mHI-E for LTB participants was defined as a hemoglobin increase of ≥1.5 g/dL from baseline for ≥14 days or rolling 2 weeks (in the absence of red blood cell [RBC] transfusions). Hemoglobin measurements within 7 days following RBC transfusion were excluded from analysis. LTB participants were those who received <4 units of RBCs within 8 weeks prior to baseline. Rolling 2 weeks was defined as any consecutive 2 weeks during the study. The percentage of LTB participants with mHI-E were reported.
Any consecutive 2 weeks during the study (up to approximately 75 weeks)
Percentage of High Transfusion Burden (HTB) Participants With mHI-E
The mHI-E for HTB participants was defined as a ≥4 units or ≥50% reduction in RBC transfusion burden during any rolling 8-week window compared to baseline. HTB participants were those who required ≥4 units of RBC transfusions within 8 weeks prior to baseline. Rolling 8 weeks was defined as any consecutive 8 weeks during the study. The percentage of HTB participants with mHI-E were reported.
Any consecutive 8 weeks during the study (up to approximately 75 weeks)
Secondary Outcomes
Measure
Description
Time Frame
Number of Participants Who Experienced an Adverse Event (AE)
An adverse event (AE) was any untoward medical occurrence in a participant or clinical investigation participant administered a study drug, which does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of the study drug whether or not it is considered related to the study drug. The number of participants who experienced an AE were reported.
Other Outcomes
Not provided
Eligibility Module
Eligibility Criteria
Key Inclusion Criteria:
Documented diagnosis of idiopathic/de novo myelodysplastic syndrome (MDS) or non-proliferative chronic myelomonocytic leukemia (CMML), according to WHO criteria (white blood count, 13,000/uL), that meets International Prognostic Scoring System (IPSS) classification of low or intermediate-1 risk disease as determined by microscopic and standard cytogenetic analyses of the bone marrow and peripheral complete blood count (CBC) obtained during screening
Anemia defined as:
Mean hemoglobin concentration <10.0 g/dL of 2 measurements (one performed within one day prior to Cycle 1 Day 1 and the other performed 7-28 days prior to Cycle 1 Day 1, not influenced by red blood cell (RBC) transfusion within 7 days of measurement for non-transfusion dependent patients (defined as having received <4 units of RBCs within 8 weeks prior to Cycle 1 Day 1), or Transfusion dependent, defined as having received ≥4 units of RBCs within 8 weeks prior to Cycle 1 Day 1
Serum erythropoietin levels and prior erythropoiesis-stimulating agent (ESA) treatment:
Dose escalation cohorts and expansion cohort 1 patients: Serum erythropoietin level >500 U/L, OR, if ≤500 U/L, patient is non-responsive, refractory, or intolerant to erythropoiesis-stimulating agents (ESAs), or ESAs are contraindicated or unavailable
Expansion cohort 2 patients: If patient is RS+ (defined as having ≥15% ring sideroblasts in the bone marrow), no prior ESA treatment and serum erythropoietin level ≤ 200 U/L. If a patient is RS- (defined as having <15% ring sideroblasts in the bone marrow), prior ESA treatment and any serum erythropoietin level is allowed
No alternative treatment options, per applicable MDS guidelines, are available and/or appropriate for the patient, at the discretion of the investigator
Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2 (if related to anemia).
Adequate renal (creatinine ≤2 x upper limit of normal [ULN]) and hepatic (total bilirubin <2 x ULN and aspartate aminotransferase (AST) and alanine aminotransferase (ALT) <3 x ULN) function
Adequate transferrin saturation (≥15%), ferritin (≥ 50 µg/L), folate (≥4.5 nmol/L [≥2.0 µg/L]) and vitamin B12 (≥148 pmol/L [≥ 200 pg/mL]) during screening (supplementation and retest during screening is acceptable)
Females of child bearing potential (defined as sexually mature women who have not undergone hysterectomy or bilateral oophorectomy, or are not naturally postmenopausal ≥24 consecutive months) must have negative urine or blood pregnancy test prior to enrollment and use adequate birth control methods (abstinence, oral contraceptives, barrier method with spermicide, or surgical sterilization) during study participation and for 12 weeks following the last dose of luspatercept. Males must agree to use a latex condom during any sexual contact with females of child-bearing potential while participating in the study and for 12 weeks following the last dose of luspatercept, even if he has undergone a successful vasectomy. Patients must be counseled concerning measures to be used to prevent pregnancy and potential toxicities prior to the first dose of luspatercept
Patients are able to adhere to the study visit schedule, understand and comply with all protocol requirements
Patients understand and are able to provide written informed consent
Key Exclusion Criteria:
Prior treatment with azacitidine or decitabine
Treatment within 28 days prior to Cycle 1 Day 1 with:
Erythropoiesis stimulating agent (ESA)
Granulocyte colony-stimulating factor (G-CSF) and granulocyte-macrophage colony stimulating factor (GM-CSF)
Lenalidomide
Iron chelation therapy if initiated within 56 days prior to Cycle 1 Day 1
Treatment with another investigational drug or device, or approved therapy for investigational use ≤28 days prior to Cycle 1 Day 1, or if the half-life of the previous product is known, within 5 times the half-life prior to Cycle 1 Day 1, whichever is longer
Major surgery within 28 days prior to Cycle 1 Day 1. Patients must have completely recovered from any previous surgery prior to Cycle 1 Day 1
Platelet count <30 x 109/L.
Any active infection requiring parenteral antibiotic therapy within 28 days prior to Cycle 1 Day 1 or oral antibiotics within 14 days of Cycle 1 Day 1
History of stroke, deep venous thrombosis (DVT) or arterial embolism within 6 months prior to Cycle 1 Day 1
Known positive for human immunodeficiency virus (HIV), active infectious hepatitis B (HBV) or active infectious hepatitis C (HCV)
Any malignancy other than MDS which has not been in remission and/or has required systemic therapy including radiation, chemotherapy, hormonal therapy or surgery, within the last year prior to Cycle 1 Day 1
Uncontrolled hypertension, defined as systolic blood pressure (BP) ≥ 150 mm Hg or diastolic BP ≥ 100 mm Hg
Pregnant or lactating females
History of severe allergic or anaphylactic reactions or hypersensitivity to recombinant proteins or excipients in the investigational drug
Any other condition not specifically noted above which, in the judgment of the investigator, would preclude the patient from participating in the study
Transfusion event within 7 days prior to Cycle 1 Day 1
Prior treatment with sotatercept (MK-7962, formerly called ACE-011) or luspatercept.
Platzbecker U, Gotze KS, Kiewe P, Germing U, Mayer K, Radsak M, Wolff T, Chromik J, Sockel K, Oelschlagel U, Haase D, Illmer T, Al-Ali HK, Silling G, Reynolds JG, Zhang X, Attie KM, Shetty JK, Giagounidis A. Long-Term Efficacy and Safety of Luspatercept for Anemia Treatment in Patients With Lower-Risk Myelodysplastic Syndromes: The Phase II PACE-MDS Study. J Clin Oncol. 2022 Nov 20;40(33):3800-3807. doi: 10.1200/JCO.21.02476. Epub 2022 Aug 23.
A total of 116 participants were enrolled and received treatment.
Recruitment Details
Not provided
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
FG000
Luspatercept 0.125mg/kg (Cohort 1)
Participants received luspatercept 0.125mg/kg as a subcutaneous (SC) injection every 3 weeks (Q3W) on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
FG001
Luspatercept 0.25mg/kg (Cohort 2)
Participants received luspatercept titrated up to 0.25mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
FG002
Luspatercept 0.50mg/kg (Cohort 3)
Participants received luspatercept titrated up to 0.50mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
FG003
Luspatercept 0.75mg/kg (Cohort 4)
Participants received luspatercept titrated up to 0.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
FG004
Luspatercept 1.00mg/kg (Cohort 5)
Participants received luspatercept titrated up to 1.00mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
FG005
Luspatercept 1.33mg/kg (Cohort 6)
Participants received luspatercept titrated up to 1.33mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
FG006
Luspatercept 1.75mg/kg (Cohort 7)
Participants received luspatercept titrated up to 1.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
FG007
Expansion Cohort
Participants received an initial dose of luspatercept 1.0mg/kg SC on Day 1 of the Cycle 1 (Cycle length = 21 days). For each subsequent cycle (up to 5 cycles), the dose was titrated via Fibonacci scheme up to a maximum dose of 1.75mg/kg based on the safety review team (SRT) recommendations.
Periods
Title
Milestones
Reasons Not Completed
Overall Study
Type
Comment
Milestone Data
STARTED
FG0003 subjects
FG0013 subjects
FG0023 subjects
FG0036 subjects
FG0043 subjects
FG0056 subjects
FG0063 subjects
FG00789 subjects
COMPLETED
FG0003 subjects
FG0013 subjects
FG0023 subjects
FG0036 subjects
FG004
NOT COMPLETED
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG0030 subjects
FG004
Type
Comment
Reasons
Lost to Follow-up
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG003
Baseline Characteristics Module
Baseline Analysis Population Description
All enrolled participants
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
BG000
Luspatercept 0.125mg/kg (Cohort 1)
Participants received luspatercept 0.125mg/kg as a subcutaneous (SC) injection every 3 weeks (Q3W) on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
BG001
Luspatercept 0.25mg/kg (Cohort 2)
Denominators
Units
Counts
Participants
BG000
Baseline Measures
Title
Description
Population Description
Parameter Type
Dispersion Type
Unit of Measure
Calculate Percentage
Denominator Units Selected
Denominators
Classes
Age, Continuous
Mean
Outcome Measures Module
Outcome Measures
Type
Title
Description
Population Description
Reporting Status
Anticipated Posting Date
Parameter Type
Dispersion Type
Unit of Measure
Calculate Percentage
Time Frame
Units Analyzed
Denominator Units Selected
Arm/Group Information
Denominators
Classes
Analyses
Primary
Percentage of Low Transfusion Burden (LTB) Participants With Modified Erythroid Response (mHI-E)
The mHI-E for LTB participants was defined as a hemoglobin increase of ≥1.5 g/dL from baseline for ≥14 days or rolling 2 weeks (in the absence of red blood cell [RBC] transfusions). Hemoglobin measurements within 7 days following RBC transfusion were excluded from analysis. LTB participants were those who received <4 units of RBCs within 8 weeks prior to baseline. Rolling 2 weeks was defined as any consecutive 2 weeks during the study. The percentage of LTB participants with mHI-E were reported.
All enrolled LTB participants who received at least one dose of luspatercept and received <4 units of RBCs within 8 weeks prior to baseline.
Posted
Number
95% Confidence Interval
Percentage of participants
Any consecutive 2 weeks during the study (up to approximately 75 weeks)
ID
Title
Description
OG000
Luspatercept 0.125mg/kg (Cohort 1)
Adverse Events Module
Frequency Threshold
5
Time Frame
Up to approximately 75 weeks
Description
All-cause mortality was reported on all enrolled participants. Serious and non-serious AEs were reported among participants who received at least one dose of study treatment.
All-Cause Mortality Comment
Not provided
Arm/Groups
ID
Title
Description
Deaths (Affected)
Deaths (At Risk)
Serious Events (Affected)
Serious Events (At Risk)
Other Events (Affected)
Other Events (At Risk)
EG000
Luspatercept 0.125 mg/kg (Cohort 1)
Participants received luspatercept 0.125mg/kg as a subcutaneous (SC) injection every 3 weeks (Q3W) on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days)
Serious Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Anaemia
Blood and lymphatic system disorders
MedDRA 20.0
Systematic Assessment
Other Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Anaemia
Blood and lymphatic system disorders
MedDRA 20.0
Systematic Assessment
More Info Module
Limitations and Caveats
Not provided
Certain Agreements
Are all PI(s) employees of the sponsor?
No
Restriction Type
Point of Contact
Title
Organization
Phone
Extension
Email
Senior Vice President, Global Clinical Development
Participants receive luspatercept titrated up to 1.33mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
Drug: Luspatercept
Luspatercept 1.75mg/kg (Cohort 7)
Experimental
Participants receive luspatercept titrated up to 1.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
Drug: Luspatercept
Expansion Cohort
Experimental
Participants receive an initial dose of luspatercept 1.0mg/kg SC on Day 1 of the Cycle 1 (Cycle length = 21 days). For each subsequent cycle (up to 5 cycles), the dose was titrated via Fibonacci scheme up to a maximum dose of 1.75mg/kg based on the safety review team (SRT) recommendations
Drug: Luspatercept
Luspatercept 0.125mg/kg (Cohort 1)
Luspatercept 0.25mg/kg (Cohort 2)
Luspatercept 0.50mg/kg (Cohort 3)
Luspatercept 0.75mg/kg (Cohort 4)
Luspatercept 1.00mg/kg (Cohort 5)
Luspatercept 1.33mg/kg (Cohort 6)
Luspatercept 1.75mg/kg (Cohort 7)
MK-6143
A536
Up to approximately 24 weeks
Number of Participants Who Discontinued Study Treatment Due To an AE
An adverse event (AE) was any untoward medical occurrence in a participant or clinical investigation participant administered a study drug, which does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of the study drug whether or not it is considered related to the study drug. The number of participants who discontinued study treatment due to an AE were reported.
Up to approximately 12 weeks
Rate of Erythroid Response (HI-E) Per International Working Group (IWG) 2006 Response Criteria
Per IWG 2006 response criteria, rate of HI-E is defined as the percentage of participants for whom the mean of all hemoglobin value from baseline during any rolling 8-week increased ≥1.5 g/dL in the absence of transfusion for LTB participants, or a reduction by ≥4 units of RBCs transfusion over any rolling 8-week window for HTB patients. LTB participants were those who received <4 units of RBCs within 8 weeks prior to baseline. HTB participants were those who required ≥4 units of RBC transfusions within 8 weeks prior to baseline. Rolling 8 weeks was defined as any consecutive 8 weeks during the study.
Any consecutive 8 Weeks during the study treatment (up to approximately 75 weeks)
Rate of Platelet Response (HI-P) Per IWG 2006 Criteria
Per IWG 2006 response criteria, HI-P was defined for participants with baseline value ≥20 x 10^9/L as the platelet increase in any rolling 8 weeks ≥30 x 10^9 and for participants with baseline value <20 x 10^9/L as the platelet increase in any rolling 8 weeks >20 x 10^9/L with increase of at least 100%. The rolling 8-week was defined as any consecutive 8 weeks during the study. The percentage of participants with HI-P were reported.
Any consecutive 8 Weeks during the study treatment (up to approximately 75 weeks)
Rate of Neutrophil Response (HI-N) Per IWG 2006 Criteria
Per IWG 2006 response criteria, HI-N was defined as the neutrophil increase in any rolling 8 weeks is at last 100% and an absolute increase > 0.5 x 10^9/L. The rolling 8-week was defined as any consecutive 8 weeks during the study. The percentage of participants with HI-N response were reported.
Any consecutive 8 Weeks during the study treatment (up to approximately 75 weeks)
Duration of HI-E Per IWG 2006 Response Criteria
Per IWG 2006 response criteria, duration of HI-E response was defined as the time from the first day of the first rolling 8-week interval of showing response to the last day of the last consecutive rolling 8-week interval of showing response. HI-E response for LTB participants was defined as hemoglobin increase ≥ 1.5 g/dL during any rolling 8-week window and for HTB HI-E was defined as RBC transfusion reduction ≥4 units during any rolling 8 weeks. LTB participants were those who received <4 units of RBCs within 8 weeks prior to baseline. HTB participants were those who required ≥4 units of RBC transfusions within 8 weeks prior to baseline. The rolling 8-week was defined as any consecutive 8 weeks during the study.
up to approximately 75 weeks
Time to HI-E Per IWG 2006 Response Criteria
Per IWG 2006 response criteria, the time to HI-E response was defined as the first date of the rolling 8-week interval of showing response minus first dose date plus 1. HI-E response for LTB participants was defined as hemoglobin increase ≥ 1.5 g/dL during any rolling 8 weeks window and for HTB HI-E was defined as RBC transfusion reduction ≥4 units during any rolling 8 weeks. LTB participants were those who received <4 units of RBCs within 8 weeks prior to baseline. HTB participants were those who required ≥4 units of RBC transfusions within 8 weeks prior to baseline. The rolling 8-week was defined as any consecutive 8 weeks during the study.
Any consecutive 8 weeks during the study (up to approximately 75 weeks)
Mean Change From Baseline to Day 113 in Frequency of RBC Transfusions
Frequency of RBC transfusion was defined as the total number of RBC transfusions received. Per protocol, the mean change from baseline to Day 113 in frequency of RBC transfusion was reported in the HTB participants (those who required ≥4 units of RBC transfusions within 8 weeks prior to baseline).
Baseline (prior to first dose of luspatercept) and Day 113
Rate of RBC Transfusion Independence (RBC-TI)
Rate of RBC-TI was defined as percentage of participants with ≥2 units of RBC transfusions at baseline who experienced transfusion independence which was defined as ≥8 weeks without a transfusion while on treatment. Per protocol, rate of RBC-TI was reported in HTB participants (those who required ≥4 units of RBC transfusions within 8 weeks prior to baseline).
Up to approximately 16 weeks
Time to Maximum Concentration (Tmax) of Luspatercept
Blood samples were collected at specified intervals for the determination of Tmax. Tmax was defined as time to the maximum concentration of luspatercept reached. Tmax was based on non-compartmental analysis and a mean Tmax value was presented.
Cycle 1 Day 1: Predose, Cycle 1: Days 8, 11, 15: Postdose; Cycle 2 Day 1, Cycle 2 Day 8, Cycle 4 Day 1, Cycle 5 Day 1, and Cycle 5 Days 8, 15: Postdose (each cycle length = 21 days)
Terminal Half-Life (t ½) of Luspatercept
Blood samples were collected at specified intervals for the determination of t½. t½ was defined as the time required to divide the luspatercept plasma concentration by two after reaching pseudo-equilibrium, following a single dose of luspatercept. t½ was based on non-compartmental analysis and a mean t1/2 value was presented.
Cycle 1 Day 1: Predose, Cycle 1: Days 8, 11, 15: Postdose; Cycle 2 Day 1, Cycle 2 Day 8, Cycle 4 Day 1, Cycle 5 Day 1, and Cycle 5 Days 8, 15: Postdose (each cycle length = 21 days)
Maximum Concentration (Cmax) of Luspatercept
Blood samples were collected at specified intervals for the determination of Cmax. Cmax was defined as the maximum concentration of luspatercept reached. Cmax was based on non-compartmental analysis and a mean Cmax value was presented.
Cycle 1 Day 1: Predose, Cycle 1: Days 8, 11, 15: Postdose; Cycle 2 Day 1, Cycle 2 Day 8, Cycle 4 Day 1, Cycle 5 Day 1, and Cycle 5 Days 8, 15: Postdose (each cycle length = 21 days)
Area Under the Concentration-Time Curve of Luspatercept From Time 0 to Day 21 (AUC0-21)
Blood samples were collected at specified intervals for the determination of AUC0-21. AUC0-21 was defined as the area under the concentration-time curve of luspatercept from time zero to Study Day 21. AUC0-21 was based on non-compartmental analysis and a mean AUC0-21 value was presented.
Cycle 1 Day 1: Predose, Cycle 1 Days 8, 11, 15 and Cycle 2 Day 1: Postdose (each cycle length = 21 days)
Percent Change From Baseline to Day 113 in Concentration of Serum Iron
Blood samples were to be collected at pre-specified time intervals to determine serum iron concentration. Baseline was prespecified to be the last measurement prior to the first dose of study drug.
Baseline (prior to first dose of luspatercept) and Day 113
Percent Change From Baseline to Day 113 in Total Iron Binding Capacity (TIBC)
Blood samples were collected at pre-specified time intervals to determine TIBC. The percentage change from baseline in TIBC was reported.
Baseline (prior to first dose of luspatercept) and Day 113
Percent Change From Baseline to Day 113 in Concentration of Transferrin
Blood samples were to be collected at pre-specified time intervals to determine concentration of transferrin. Baseline was prespecified to be the last measurement prior to the first dose of study drug.
Baseline (prior to first dose of luspatercept) and Day 113
Percent Change From Baseline to Day 113 in Concentration of Soluble Transferrin Receptor
Blood samples were collected at pre-specified time intervals to determine soluble transferrin receptor concentration. The percentage change from baseline in concentration of soluble transferrin receptor was reported.
Baseline (prior to first dose of luspatercept) and Day 113
Percent Change From Baseline to Day 113 in Concentration of Serum Ferritin
Blood samples were collected at pre-specified time intervals to determine serum ferritin concentration. The percentage change from baseline in concentration of serum ferritin was reported.
Baseline (prior to first dose of luspatercept) and Day 113
Percent Change From Baseline to Day 113 in Concentration of Non-Transferrin Bound Iron (NTBI)
Blood samples were to be collected at pre-specified time intervals to determine concentration of NTBI. Baseline was prespecified to be the last measurement prior to the first dose of study drug.
Baseline (prior to first dose of luspatercept) and Day 113
Percent Change From Baseline to Day 113 in Concentration of Serum Hepcidin
Blood samples were collected at pre-specified time intervals to determine soluble hepcidin concentration. The percentage change from baseline in concentration of soluble hepcidin was reported.
Baseline (prior to first dose of luspatercept) and Day 113
Percent Change From Baseline to Day 113 in Concentration of Serum Erythropoietin
Blood samples were collected at pre-specified time intervals to determine serum erythropoietin concentration. The percentage change from baseline in concentration of serum erythropoietin was reported.
Baseline (prior to first dose of luspatercept) and Day 113
Percent Change From Baseline to Day 113 in Reticulocyte Count
Blood samples were collected at pre-specified time intervals to determine reticulocyte count. The percentage change from baseline in mean reticulocyte count was reported.
Baseline (prior to first dose of luspatercept) and Day 113
Percent Change From Baseline to Day 113 in Direct Bilirubin Level
Blood samples were collected at pre-specified time intervals to determine serum direct bilirubin concentration. The percentage change from baseline in mean concentration direct bilirubin was reported.
Baseline (prior to first dose of luspatercept) and Day 113
Percent Change From Baseline to Day 113 in Total Bilirubin Level
Blood samples were collected at pre-specified time intervals to determine total bilirubin concentration. The percentage change from baseline in mean concentration total bilirubin was reported.
Baseline (prior to first dose of luspatercept) and Day 113
Percent Change From Baseline to Day 113 in Lactate Dehydrogenase Level
Blood samples were collected at pre-specified time intervals to determine serum lactate dehydrogenase level. The percentage change from baseline in mean concentration lactate dehydrogenase level was reported.
Baseline (prior to first dose of luspatercept) and Day 113
Percent Change From Baseline to Day 113 in Nucleated RBC (nRBC)
Blood samples were to be collected at pre-specified time intervals to determine concentration of nRBC. Baseline was prespecified to be the last measurement prior to the first dose of study drug.
Baseline (prior to first dose of luspatercept) and Day 113
Percent Change From Baseline to Day 113 in Concentration of Serum Bone-Specific Alkaline Phosphatase (BSAP)
Blood samples were collected at pre-specified time intervals to determine serum BSAP concentration. The percentage change from baseline in concentration BSAP was reported.
Baseline (prior to first dose of luspatercept) and Day 113
Percent Change From Baseline to Day 113 in Concentration of Serum Cross-Linked C-Telopeptide of Type I Collagen (CTX)
Blood samples were collected at pre-specified time intervals to determine serum CTX. The percentage change from baseline in concentration of CTX was reported.
Baseline (prior to first dose of luspatercept) and Day 113
Derived
Platzbecker U, Germing U, Gotze KS, Kiewe P, Mayer K, Chromik J, Radsak M, Wolff T, Zhang X, Laadem A, Sherman ML, Attie KM, Giagounidis A. Luspatercept for the treatment of anaemia in patients with lower-risk myelodysplastic syndromes (PACE-MDS): a multicentre, open-label phase 2 dose-finding study with long-term extension study. Lancet Oncol. 2017 Oct;18(10):1338-1347. doi: 10.1016/S1470-2045(17)30615-0. Epub 2017 Sep 1.
3 subjects
FG0055 subjects
FG0063 subjects
FG00782 subjects
0 subjects
FG0051 subjects
FG0060 subjects
FG0077 subjects
0 subjects
FG0040 subjects
FG0050 subjects
FG0060 subjects
FG0071 subjects
Withdrawal by Subject
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG0030 subjects
FG0040 subjects
FG0051 subjects
FG0060 subjects
FG0071 subjects
Not Reported
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG0030 subjects
FG0040 subjects
FG0050 subjects
FG0060 subjects
FG0075 subjects
Participants received luspatercept titrated up to 0.25mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
BG002
Luspatercept 0.50mg/kg (Cohort 3)
Participants received luspatercept titrated up to 0.50mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
BG003
Luspatercept 0.75mg/kg (Cohort 4)
Participants received luspatercept titrated up to 0.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
BG004
Luspatercept 1.00mg/kg (Cohort 5)
Participants received luspatercept titrated up to 1.00mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
BG005
Luspatercept 1.33mg/kg (Cohort 6)
Participants received luspatercept titrated up to 1.33mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
BG006
Luspatercept 1.75mg/kg (Cohort 7)
Participants received luspatercept titrated up to 1.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
BG007
Expansion Cohort
Participants received an initial dose of luspatercept 1.0mg/kg SC on Day 1 of the Cycle 1 (Cycle length = 21 days). For each subsequent cycle (up to 5 cycles), the dose was titrated via Fibonacci scheme up to a maximum dose of 1.75mg/kg based on the safety review team (SRT) recommendations.
BG008
Total
Total of all reporting groups
3
BG0013
BG0023
BG0036
BG0043
BG0056
BG0063
BG00789
BG008116
Standard Deviation
years
Title
Denominators
Categories
ParticipantsBG0003
ParticipantsBG0013
ParticipantsBG0023
ParticipantsBG0036
ParticipantsBG0043
ParticipantsBG0056
ParticipantsBG0063
ParticipantsBG00789
ParticipantsBG008116
Title
Measurements
BG00070.0± 19.1
BG00159.0± 27.8
BG00266.3± 5.1
BG003
Sex: Female, Male
Count of Participants
Participants
Title
Denominators
Categories
ParticipantsBG0003
ParticipantsBG0013
ParticipantsBG0023
ParticipantsBG0036
ParticipantsBG0043
ParticipantsBG0056
ParticipantsBG0063
ParticipantsBG00789
ParticipantsBG008116
Title
Measurements
Female
BG0003
BG0013
BG0022
BG003
Ethnicity (NIH/OMB)
Count of Participants
Participants
Title
Denominators
Categories
ParticipantsBG0003
ParticipantsBG0013
ParticipantsBG0023
ParticipantsBG0036
ParticipantsBG0043
ParticipantsBG0056
ParticipantsBG0063
ParticipantsBG00789
ParticipantsBG008116
Title
Measurements
Hispanic or Latino
BG0000
BG0010
BG0020
BG003
Race (NIH/OMB)
Count of Participants
Participants
Title
Denominators
Categories
ParticipantsBG0003
ParticipantsBG0013
ParticipantsBG0023
ParticipantsBG0036
ParticipantsBG0043
ParticipantsBG0056
ParticipantsBG0063
ParticipantsBG00789
ParticipantsBG008116
Title
Measurements
American Indian or Alaska Native
BG0000
BG0010
BG0020
BG003
Transfusion Status
Low Transfusion Burden (LTB) participants are defined as those who received <4 units of red blood cells (RBCs) within 8 weeks prior to baseline (Cycle 1 Day 1). High Transfusion Burden (HTB) participants are defined as those who required ≥4 units of RBC transfusions within 8 weeks prior to baseline.
Count of Participants
Participants
Title
Denominators
Categories
HTB
ParticipantsBG0003
ParticipantsBG0013
ParticipantsBG0023
ParticipantsBG0036
ParticipantsBG0043
ParticipantsBG0056
ParticipantsBG0063
ParticipantsBG00789
ParticipantsBG008116
Title
Measurements
BG0002
BG0012
BG0023
BG003
LTB
ParticipantsBG0003
ParticipantsBG0013
ParticipantsBG0023
ParticipantsBG0036
Hemoglobin level
Hemoglobin level of participants in peripheral blood was reported.
All enrolled participants with baseline hemoglobin level data available. Data were not collected from 3 participants at baseline.
Mean
Standard Deviation
g/dl
Title
Denominators
Categories
ParticipantsBG0003
ParticipantsBG0013
ParticipantsBG0023
ParticipantsBG0036
ParticipantsBG0043
ParticipantsBG0055
ParticipantsBG0063
ParticipantsBG00787
ParticipantsBG008113
Title
Measurements
BG00010.30± 0.98
BG0018.91± 0.81
BG0028.83± 0.31
BG003
Participants received luspatercept 0.125mg/kg as a subcutaneous (SC) injection every 3 weeks (Q3W) on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG001
Luspatercept 0.25mg/kg (Cohort 2)
Participants received luspatercept titrated up to 0.25mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG002
Luspatercept 0.50mg/kg (Cohort 3)
Participants received luspatercept titrated up to 0.50mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG003
Luspatercept 0.75mg/kg (Cohort 4)
Participants received luspatercept titrated up to 0.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG004
Luspatercept 1.00mg/kg (Cohort 5)
Participants received luspatercept titrated up to 1.00mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG005
Luspatercept 1.33mg/kg (Cohort 6)
Participants received luspatercept titrated up to 1.33mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG006
Luspatercept 1.75mg/kg (Cohort 7)
Participants received luspatercept titrated up to 1.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG007
Expansion Cohort
Participants received an initial dose of luspatercept 1.0mg/kg SC on Day 1 of the Cycle 1 (Cycle length = 21 days). For each subsequent cycle (up to 5 cycles), the dose was titrated via Fibonacci scheme up to a maximum dose of 1.75mg/kg based on the safety review team (SRT) recommendations.
Units
Counts
Participants
OG0001
OG0011
OG0020
OG0033
OG0040
OG0050
OG0062
OG00758
Title
Denominators
Categories
Title
Measurements
OG0000(0 to 97.5)
OG0010(0 to 97.5)
OG00366.7(9.4 to 99.2)
OG006100(15.8 to 100)
OG00769.0(55.5 to 80.5)
Primary
Percentage of High Transfusion Burden (HTB) Participants With mHI-E
The mHI-E for HTB participants was defined as a ≥4 units or ≥50% reduction in RBC transfusion burden during any rolling 8-week window compared to baseline. HTB participants were those who required ≥4 units of RBC transfusions within 8 weeks prior to baseline. Rolling 8 weeks was defined as any consecutive 8 weeks during the study. The percentage of HTB participants with mHI-E were reported.
All enrolled HTB participants who received at least one dose of luspatercept and who required ≥4 units of RBC transfusions within 8 weeks prior to baseline.
Posted
Number
95% Confidence Interval
Percentage of Participants
Any consecutive 8 weeks during the study (up to approximately 75 weeks)
ID
Title
Description
OG000
Luspatercept 0.125mg/kg (Cohort 1)
Participants received luspatercept 0.125mg/kg as a subcutaneous (SC) injection every 3 weeks (Q3W) on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG001
Luspatercept 0.25mg/kg (Cohort 2)
Participants received luspatercept titrated up to 0.25mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG002
Luspatercept 0.50mg/kg (Cohort 3)
Participants received luspatercept titrated up to 0.50mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG003
Luspatercept 0.75mg/kg (Cohort 4)
Participants received luspatercept titrated up to 0.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG004
Luspatercept 1.00mg/kg (Cohort 5)
Participants received luspatercept titrated up to 1.00mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG005
Luspatercept 1.33mg/kg (Cohort 6)
Participants received luspatercept titrated up to 1.33mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG006
Luspatercept 1.75mg/kg (Cohort 7)
Participants received luspatercept titrated up to 1.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG007
Expansion Cohort
Participants received an initial dose of luspatercept 1.0mg/kg SC on Day 1 of the Cycle 1 (Cycle length = 21 days). For each subsequent cycle (up to 5 cycles), the dose was titrated via Fibonacci scheme up to a maximum dose of 1.75mg/kg based on the safety review team (SRT) recommendations.
Units
Counts
Participants
OG0002
OG0012
OG0023
OG003
Title
Denominators
Categories
Title
Measurements
OG00050.0(1.26 to 98.7)
OG00150.0(1.26 to 98.7)
OG00233.3(0.84 to 90.6)
OG003
Secondary
Number of Participants Who Experienced an Adverse Event (AE)
An adverse event (AE) was any untoward medical occurrence in a participant or clinical investigation participant administered a study drug, which does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of the study drug whether or not it is considered related to the study drug. The number of participants who experienced an AE were reported.
All enrolled participants who received at least one dose of luspatercept.
Posted
Count of Participants
Participants
Up to approximately 24 weeks
ID
Title
Description
OG000
Luspatercept 0.125mg/kg (Cohort 1)
Participants received luspatercept 0.125mg/kg as a subcutaneous (SC) injection every 3 weeks (Q3W) on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG001
Luspatercept 0.25mg/kg (Cohort 2)
Participants received luspatercept titrated up to 0.25mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG002
Luspatercept 0.50mg/kg (Cohort 3)
Participants received luspatercept titrated up to 0.50mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG003
Luspatercept 0.75mg/kg (Cohort 4)
Participants received luspatercept titrated up to 0.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG004
Luspatercept 1.00mg/kg (Cohort 5)
Participants received luspatercept titrated up to 1.00mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG005
Luspatercept 1.33mg/kg (Cohort 6)
Participants received luspatercept titrated up to 1.33mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG006
Luspatercept 1.75mg/kg (Cohort 7)
Participants received luspatercept titrated up to 1.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG007
Expansion Cohort
Participants received an initial dose of luspatercept 1.0mg/kg SC on Day 1 of the Cycle 1 (Cycle length = 21 days). For each subsequent cycle (up to 5 cycles), the dose was titrated via Fibonacci scheme up to a maximum dose of 1.75mg/kg based on the safety review team (SRT) recommendations.
Units
Counts
Participants
OG0003
OG0013
OG0023
OG003
Title
Denominators
Categories
Title
Measurements
OG0001
OG0012
OG0023
OG003
Secondary
Number of Participants Who Discontinued Study Treatment Due To an AE
An adverse event (AE) was any untoward medical occurrence in a participant or clinical investigation participant administered a study drug, which does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of the study drug whether or not it is considered related to the study drug. The number of participants who discontinued study treatment due to an AE were reported.
All enrolled participants who received at least one dose of luspatercept.
Posted
Count of Participants
Participants
Up to approximately 12 weeks
ID
Title
Description
OG000
Luspatercept 0.125mg/kg (Cohort 1)
Participants received luspatercept 0.125mg/kg as a subcutaneous (SC) injection every 3 weeks (Q3W) on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG001
Luspatercept 0.25mg/kg (Cohort 2)
Participants received luspatercept titrated up to 0.25mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG002
Luspatercept 0.50mg/kg (Cohort 3)
Participants received luspatercept titrated up to 0.50mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG003
Luspatercept 0.75mg/kg (Cohort 4)
Participants received luspatercept titrated up to 0.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG004
Luspatercept 1.00mg/kg (Cohort 5)
Participants received luspatercept titrated up to 1.00mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG005
Luspatercept 1.33mg/kg (Cohort 6)
Participants received luspatercept titrated up to 1.33mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG006
Luspatercept 1.75mg/kg (Cohort 7)
Participants received luspatercept titrated up to 1.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG007
Expansion Cohort
Participants received an initial dose of luspatercept 1.0mg/kg SC on Day 1 of the Cycle 1 (Cycle length = 21 days). For each subsequent cycle (up to 5 cycles), the dose was titrated via Fibonacci scheme up to a maximum dose of 1.75mg/kg based on the safety review team (SRT) recommendations.
Units
Counts
Participants
OG0003
OG0013
OG0023
OG003
Title
Denominators
Categories
Title
Measurements
OG0000
OG0010
OG0020
OG003
Secondary
Rate of Erythroid Response (HI-E) Per International Working Group (IWG) 2006 Response Criteria
Per IWG 2006 response criteria, rate of HI-E is defined as the percentage of participants for whom the mean of all hemoglobin value from baseline during any rolling 8-week increased ≥1.5 g/dL in the absence of transfusion for LTB participants, or a reduction by ≥4 units of RBCs transfusion over any rolling 8-week window for HTB patients. LTB participants were those who received <4 units of RBCs within 8 weeks prior to baseline. HTB participants were those who required ≥4 units of RBC transfusions within 8 weeks prior to baseline. Rolling 8 weeks was defined as any consecutive 8 weeks during the study.
All enrolled participants who received at least one dose of luspatercept.
Posted
Number
95% Confidence Interval
Percentage of participants
Any consecutive 8 Weeks during the study treatment (up to approximately 75 weeks)
ID
Title
Description
OG000
Luspatercept 0.125mg/kg (Cohort 1)
Participants received luspatercept 0.125mg/kg as a subcutaneous (SC) injection every 3 weeks (Q3W) on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG001
Luspatercept 0.25mg/kg (Cohort 2)
Participants received luspatercept titrated up to 0.25mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG002
Luspatercept 0.50mg/kg (Cohort 3)
Participants received luspatercept titrated up to 0.50mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG003
Luspatercept 0.75mg/kg (Cohort 4)
Participants received luspatercept titrated up to 0.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG004
Luspatercept 1.00mg/kg (Cohort 5)
Participants received luspatercept titrated up to 1.00mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG005
Luspatercept 1.33mg/kg (Cohort 6)
Participants received luspatercept titrated up to 1.33mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG006
Luspatercept 1.75mg/kg (Cohort 7)
Participants received luspatercept titrated up to 1.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG007
Expansion Cohort
Participants received an initial dose of luspatercept 1.0mg/kg SC on Day 1 of the Cycle 1 (Cycle length = 21 days). For each subsequent cycle (up to 5 cycles), the dose was titrated via Fibonacci scheme up to a maximum dose of 1.75mg/kg based on the safety review team (SRT) recommendations.
Units
Counts
Participants
OG0003
OG0013
OG0023
OG003
Title
Denominators
Categories
Title
Measurements
OG00033.3(0.8 to 90.6)
OG0010(0.0 to 70.8)
OG00233.3(0.8 to 90.6)
OG003
Secondary
Rate of Platelet Response (HI-P) Per IWG 2006 Criteria
Per IWG 2006 response criteria, HI-P was defined for participants with baseline value ≥20 x 10^9/L as the platelet increase in any rolling 8 weeks ≥30 x 10^9 and for participants with baseline value <20 x 10^9/L as the platelet increase in any rolling 8 weeks >20 x 10^9/L with increase of at least 100%. The rolling 8-week was defined as any consecutive 8 weeks during the study. The percentage of participants with HI-P were reported.
All enrolled participants who received at least one dose of luspatercept and had baseline platelet count <100x10^9/L.
Posted
Number
95% Confidence Interval
Percentage of participants
Any consecutive 8 Weeks during the study treatment (up to approximately 75 weeks)
ID
Title
Description
OG000
Luspatercept 0.125mg/kg (Cohort 1)
Participants received luspatercept 0.125mg/kg as a subcutaneous (SC) injection every 3 weeks (Q3W) on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG001
Luspatercept 0.25mg/kg (Cohort 2)
Participants received luspatercept titrated up to 0.25mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG002
Luspatercept 0.50mg/kg (Cohort 3)
Participants received luspatercept titrated up to 0.50mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG003
Luspatercept 0.75mg/kg (Cohort 4)
Participants received luspatercept titrated up to 0.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG004
Luspatercept 1.00mg/kg (Cohort 5)
Participants received luspatercept titrated up to 1.00mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG005
Luspatercept 1.33mg/kg (Cohort 6)
Participants received luspatercept titrated up to 1.33mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG006
Luspatercept 1.75mg/kg (Cohort 7)
Participants received luspatercept titrated up to 1.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG007
Expansion Cohort
Participants received an initial dose of luspatercept 1.0mg/kg SC on Day 1 of the Cycle 1 (Cycle length = 21 days). For each subsequent cycle (up to 5 cycles), the dose was titrated via Fibonacci scheme up to a maximum dose of 1.75mg/kg based on the safety review team (SRT) recommendations.
Units
Counts
Participants
OG0000
OG0010
OG0021
OG003
Title
Denominators
Categories
Title
Measurements
OG0020± 0.0(0.0 to 97.5)
OG0040± 0.0(0.0 to 84.2)
OG0050± 0.0(0.0 to 97.5)
Secondary
Rate of Neutrophil Response (HI-N) Per IWG 2006 Criteria
Per IWG 2006 response criteria, HI-N was defined as the neutrophil increase in any rolling 8 weeks is at last 100% and an absolute increase > 0.5 x 10^9/L. The rolling 8-week was defined as any consecutive 8 weeks during the study. The percentage of participants with HI-N response were reported.
All enrolled participants who received at least one dose of luspatercept and had baseline neutrophil count <1.0×10^9/L.
Posted
Number
95% Confidence Interval
Percentage of Participants
Any consecutive 8 Weeks during the study treatment (up to approximately 75 weeks)
ID
Title
Description
OG000
Luspatercept 0.125mg/kg (Cohort 1)
Participants received luspatercept 0.125mg/kg as a subcutaneous (SC) injection every 3 weeks (Q3W) on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG001
Luspatercept 0.25mg/kg (Cohort 2)
Participants received luspatercept titrated up to 0.25mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG002
Luspatercept 0.50mg/kg (Cohort 3)
Participants received luspatercept titrated up to 0.50mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG003
Luspatercept 0.75mg/kg (Cohort 4)
Participants received luspatercept titrated up to 0.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG004
Luspatercept 1.00mg/kg (Cohort 5)
Participants received luspatercept titrated up to 1.00mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG005
Luspatercept 1.33mg/kg (Cohort 6)
Participants received luspatercept titrated up to 1.33mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG006
Luspatercept 1.75mg/kg (Cohort 7)
Participants received luspatercept titrated up to 1.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG007
Expansion Cohort
Participants received an initial dose of luspatercept 1.0mg/kg SC on Day 1 of the Cycle 1 (Cycle length = 21 days). For each subsequent cycle (up to 5 cycles), the dose was titrated via Fibonacci scheme up to a maximum dose of 1.75mg/kg based on the safety review team (SRT) recommendations.
Units
Counts
Participants
OG0001
OG0010
OG0021
OG003
Title
Denominators
Categories
Title
Measurements
OG0000(0.0 to 97.5)
OG002100(2.5 to 100)
OG0030(0.0 to 97.5)
OG005
Secondary
Duration of HI-E Per IWG 2006 Response Criteria
Per IWG 2006 response criteria, duration of HI-E response was defined as the time from the first day of the first rolling 8-week interval of showing response to the last day of the last consecutive rolling 8-week interval of showing response. HI-E response for LTB participants was defined as hemoglobin increase ≥ 1.5 g/dL during any rolling 8-week window and for HTB HI-E was defined as RBC transfusion reduction ≥4 units during any rolling 8 weeks. LTB participants were those who received <4 units of RBCs within 8 weeks prior to baseline. HTB participants were those who required ≥4 units of RBC transfusions within 8 weeks prior to baseline. The rolling 8-week was defined as any consecutive 8 weeks during the study.
All enrolled participants who received at least one dose of luspatercept. Per protocol, the participants with response were pooled into low and high dose groups to increase the accuracy of the analyses.
Participants received luspatercept 0.125mg/kg up to 0.5mg/kg as a SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days)
Participants receive luspatercept 0.75mg/kg up to 1.75mg/kg as a SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
Units
Counts
Participants
OG0009
OG001107
Title
Denominators
Categories
Title
Measurements
OG00078± 7.8
OG00188± 22.8
Secondary
Time to HI-E Per IWG 2006 Response Criteria
Per IWG 2006 response criteria, the time to HI-E response was defined as the first date of the rolling 8-week interval of showing response minus first dose date plus 1. HI-E response for LTB participants was defined as hemoglobin increase ≥ 1.5 g/dL during any rolling 8 weeks window and for HTB HI-E was defined as RBC transfusion reduction ≥4 units during any rolling 8 weeks. LTB participants were those who received <4 units of RBCs within 8 weeks prior to baseline. HTB participants were those who required ≥4 units of RBC transfusions within 8 weeks prior to baseline. The rolling 8-week was defined as any consecutive 8 weeks during the study.
All enrolled participants who received at least one dose of luspatercept. Per protocol, the participants with response were pooled into low and high dose groups to increase the accuracy of the analyses.
Posted
Mean
Standard Deviation
Days
Any consecutive 8 weeks during the study (up to approximately 75 weeks)
Participants received luspatercept 0.125mg/kg up to 0.5mg/kg as a SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days)
Participants receive luspatercept 0.75mg/kg up to 1.75mg/kg as a SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
Units
Counts
Participants
OG0009
OG001107
Title
Denominators
Categories
Title
Measurements
OG00035± 48.1
OG00117± 18.3
Secondary
Mean Change From Baseline to Day 113 in Frequency of RBC Transfusions
Frequency of RBC transfusion was defined as the total number of RBC transfusions received. Per protocol, the mean change from baseline to Day 113 in frequency of RBC transfusion was reported in the HTB participants (those who required ≥4 units of RBC transfusions within 8 weeks prior to baseline).
All enrolled HTB participants who received at least one dose of luspatercept and who required ≥4 units of RBC transfusions within 8 weeks prior to baseline.
Posted
Mean
Standard Deviation
Number of RBC transfusions
Baseline (prior to first dose of luspatercept) and Day 113
ID
Title
Description
OG000
Luspatercept 0.125mg/kg (Cohort 1)
Participants received luspatercept 0.125mg/kg as a subcutaneous (SC) injection every 3 weeks (Q3W) on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG001
Luspatercept 0.25mg/kg (Cohort 2)
Participants received luspatercept titrated up to 0.25mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG002
Luspatercept 0.50mg/kg (Cohort 3)
Participants received luspatercept titrated up to 0.50mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG003
Luspatercept 0.75mg/kg (Cohort 4)
Participants received luspatercept titrated up to 0.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG004
Luspatercept 1.00mg/kg (Cohort 5)
Participants received luspatercept titrated up to 1.00mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG005
Luspatercept 1.33mg/kg (Cohort 6)
Participants received luspatercept titrated up to 1.33mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG006
Luspatercept 1.75mg/kg (Cohort 7)
Participants received luspatercept titrated up to 1.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG007
Expansion Cohort
Participants received an initial dose of luspatercept 1.0mg/kg SC on Day 1 of the Cycle 1 (Cycle length = 21 days). For each subsequent cycle (up to 5 cycles), the dose was titrated via Fibonacci scheme up to a maximum dose of 1.75mg/kg based on the safety review team (SRT) recommendations.
Units
Counts
Participants
OG0002
OG0012
OG0023
OG003
Title
Denominators
Categories
Baseline
Title
Measurements
OG0002.50± 0.71
OG0012.50± 2.12
OG0024.00± 0.00
OG003
Secondary
Rate of RBC Transfusion Independence (RBC-TI)
Rate of RBC-TI was defined as percentage of participants with ≥2 units of RBC transfusions at baseline who experienced transfusion independence which was defined as ≥8 weeks without a transfusion while on treatment. Per protocol, rate of RBC-TI was reported in HTB participants (those who required ≥4 units of RBC transfusions within 8 weeks prior to baseline).
All enrolled HTB participants who received at least one dose of luspatercept and had ≥2 units of RBC transfusions at baseline.
Posted
Number
95% Confidence Interval
Percentage of participants
Up to approximately 16 weeks
ID
Title
Description
OG000
Luspatercept 0.125mg/kg (Cohort 1)
Participants received luspatercept 0.125mg/kg as a subcutaneous (SC) injection every 3 weeks (Q3W) on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG001
Luspatercept 0.25mg/kg (Cohort 2)
Participants received luspatercept titrated up to 0.25mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG002
Luspatercept 0.50mg/kg (Cohort 3)
Participants received luspatercept titrated up to 0.50mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG003
Luspatercept 0.75mg/kg (Cohort 4)
Participants received luspatercept titrated up to 0.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG004
Luspatercept 1.00mg/kg (Cohort 5)
Participants received luspatercept titrated up to 1.00mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG005
Luspatercept 1.33mg/kg (Cohort 6)
Participants received luspatercept titrated up to 1.33mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG006
Luspatercept 1.75mg/kg (Cohort 7)
Participants received luspatercept titrated up to 1.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG007
Expansion Cohort
Participants received an initial dose of luspatercept 1.0mg/kg SC on Day 1 of the Cycle 1 (Cycle length = 21 days). For each subsequent cycle (up to 5 cycles), the dose was titrated via Fibonacci scheme up to a maximum dose of 1.75mg/kg based on the safety review team (SRT) recommendations.
Units
Counts
Participants
OG0002
OG0012
OG0023
OG003
Title
Denominators
Categories
Title
Measurements
OG00050.0(1.3 to 98.7)
OG0010.0(0.0 to 84.2)
OG0020.0(0.0 to 70.8)
OG003
Secondary
Time to Maximum Concentration (Tmax) of Luspatercept
Blood samples were collected at specified intervals for the determination of Tmax. Tmax was defined as time to the maximum concentration of luspatercept reached. Tmax was based on non-compartmental analysis and a mean Tmax value was presented.
All participants who have received at least one dose of luspatercept and had sufficient pharmacokinetic samples collected and assayed.
Posted
Geometric Mean
Geometric Coefficient of Variation
Days
Cycle 1 Day 1: Predose, Cycle 1: Days 8, 11, 15: Postdose; Cycle 2 Day 1, Cycle 2 Day 8, Cycle 4 Day 1, Cycle 5 Day 1, and Cycle 5 Days 8, 15: Postdose (each cycle length = 21 days)
ID
Title
Description
OG000
Luspatercept 0.125mg/kg (Cohort 1)
Participants received luspatercept 0.125mg/kg as a subcutaneous (SC) injection every 3 weeks (Q3W) on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG001
Luspatercept 0.25mg/kg (Cohort 2)
Participants received luspatercept titrated up to 0.25mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG002
Luspatercept 0.50mg/kg (Cohort 3)
Participants received luspatercept titrated up to 0.50mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG003
Luspatercept 0.75mg/kg (Cohort 4)
Participants received luspatercept titrated up to 0.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG004
Luspatercept 1.00mg/kg (Cohort 5)
Participants received luspatercept titrated up to 1.00mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG005
Luspatercept 1.33mg/kg (Cohort 6)
Participants received luspatercept titrated up to 1.33mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG006
Luspatercept 1.75mg/kg (Cohort 7)
Participants received luspatercept titrated up to 1.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG007
Expansion Cohort
Participants received an initial dose of luspatercept 1.0mg/kg SC on Day 1 of the Cycle 1 (Cycle length = 21 days). For each subsequent cycle (up to 5 cycles), the dose was titrated via Fibonacci scheme up to a maximum dose of 1.75mg/kg based on the safety review team (SRT) recommendations.
Units
Counts
Participants
OG0003
OG0013
OG0023
OG003
Title
Denominators
Categories
Title
Measurements
OG0009.93± 35.73
OG0017.23± 20.68
OG00210.16± 39.56
OG003
Secondary
Terminal Half-Life (t ½) of Luspatercept
Blood samples were collected at specified intervals for the determination of t½. t½ was defined as the time required to divide the luspatercept plasma concentration by two after reaching pseudo-equilibrium, following a single dose of luspatercept. t½ was based on non-compartmental analysis and a mean t1/2 value was presented.
All participants who received at least one dose of luspatercept and had sufficient pharmacokinetic samples collected and assayed.
Posted
Geometric Mean
Geometric Coefficient of Variation
Days
Cycle 1 Day 1: Predose, Cycle 1: Days 8, 11, 15: Postdose; Cycle 2 Day 1, Cycle 2 Day 8, Cycle 4 Day 1, Cycle 5 Day 1, and Cycle 5 Days 8, 15: Postdose (each cycle length = 21 days)
ID
Title
Description
OG000
Luspatercept 0.125mg/kg (Cohort 1)
Participants received luspatercept 0.125mg/kg as a subcutaneous (SC) injection every 3 weeks (Q3W) on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG001
Luspatercept 0.25mg/kg (Cohort 2)
Participants received luspatercept titrated up to 0.25mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG002
Luspatercept 0.50mg/kg (Cohort 3)
Participants received luspatercept titrated up to 0.50mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG003
Luspatercept 0.75mg/kg (Cohort 4)
Participants received luspatercept titrated up to 0.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG004
Luspatercept 1.00mg/kg (Cohort 5)
Participants received luspatercept titrated up to 1.00mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG005
Luspatercept 1.33mg/kg (Cohort 6)
Participants received luspatercept titrated up to 1.33mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG006
Luspatercept 1.75mg/kg (Cohort 7)
Participants received luspatercept titrated up to 1.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG007
Expansion Cohort
Participants received an initial dose of luspatercept 1.0mg/kg SC on Day 1 of the Cycle 1 (Cycle length = 21 days). For each subsequent cycle (up to 5 cycles), the dose was titrated via Fibonacci scheme up to a maximum dose of 1.75mg/kg based on the safety review team (SRT) recommendations.
Units
Counts
Participants
OG0002
OG0013
OG0023
OG003
Title
Denominators
Categories
Title
Measurements
OG00023.78± 22.29
OG0019.15± 44.83
OG00215.14± 5.94
OG003
Secondary
Maximum Concentration (Cmax) of Luspatercept
Blood samples were collected at specified intervals for the determination of Cmax. Cmax was defined as the maximum concentration of luspatercept reached. Cmax was based on non-compartmental analysis and a mean Cmax value was presented.
All participants who received at least one dose of luspatercept and had sufficient pharmacokinetic samples collected and assayed.
Posted
Geometric Mean
Geometric Coefficient of Variation
ug/mL
Cycle 1 Day 1: Predose, Cycle 1: Days 8, 11, 15: Postdose; Cycle 2 Day 1, Cycle 2 Day 8, Cycle 4 Day 1, Cycle 5 Day 1, and Cycle 5 Days 8, 15: Postdose (each cycle length = 21 days)
ID
Title
Description
OG000
Luspatercept 0.125mg/kg (Cohort 1)
Participants received luspatercept 0.125mg/kg as a subcutaneous (SC) injection every 3 weeks (Q3W) on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG001
Luspatercept 0.25mg/kg (Cohort 2)
Participants received luspatercept titrated up to 0.25mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG002
Luspatercept 0.50mg/kg (Cohort 3)
Participants received luspatercept titrated up to 0.50mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG003
Luspatercept 0.75mg/kg (Cohort 4)
Participants received luspatercept titrated up to 0.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG004
Luspatercept 1.00mg/kg (Cohort 5)
Participants received luspatercept titrated up to 1.00mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG005
Luspatercept 1.33mg/kg (Cohort 6)
Participants received luspatercept titrated up to 1.33mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG006
Luspatercept 1.75mg/kg (Cohort 7)
Participants received luspatercept titrated up to 1.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG007
Expansion Cohort
Participants received an initial dose of luspatercept 1.0mg/kg SC on Day 1 of the Cycle 1 (Cycle length = 21 days). For each subsequent cycle (up to 5 cycles), the dose was titrated via Fibonacci scheme up to a maximum dose of 1.75mg/kg based on the safety review team (SRT) recommendations.
Units
Counts
Participants
OG0003
OG0013
OG0023
OG003
Title
Denominators
Categories
Title
Measurements
OG0000.64± 34.88
OG0010.96± 92.99
OG0022.33± 27.16
OG003
Secondary
Area Under the Concentration-Time Curve of Luspatercept From Time 0 to Day 21 (AUC0-21)
Blood samples were collected at specified intervals for the determination of AUC0-21. AUC0-21 was defined as the area under the concentration-time curve of luspatercept from time zero to Study Day 21. AUC0-21 was based on non-compartmental analysis and a mean AUC0-21 value was presented.
All participants who received at least one dose of luspatercept and had sufficient pharmacokinetic samples collected and assayed.
Posted
Geometric Mean
Geometric Coefficient of Variation
day*ug/m
Cycle 1 Day 1: Predose, Cycle 1 Days 8, 11, 15 and Cycle 2 Day 1: Postdose (each cycle length = 21 days)
ID
Title
Description
OG000
Luspatercept 0.125mg/kg (Cohort 1)
Participants received luspatercept 0.125mg/kg as a subcutaneous (SC) injection every 3 weeks (Q3W) on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG001
Luspatercept 0.25mg/kg (Cohort 2)
Participants received luspatercept titrated up to 0.25mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG002
Luspatercept 0.50mg/kg (Cohort 3)
Participants received luspatercept titrated up to 0.50mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG003
Luspatercept 0.75mg/kg (Cohort 4)
Participants received luspatercept titrated up to 0.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG004
Luspatercept 1.00mg/kg (Cohort 5)
Participants received luspatercept titrated up to 1.00mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG005
Luspatercept 1.33mg/kg (Cohort 6)
Participants received luspatercept titrated up to 1.33mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG006
Luspatercept 1.75mg/kg (Cohort 7)
Participants received luspatercept titrated up to 1.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG007
Expansion Cohort
Participants received an initial dose of luspatercept 1.0mg/kg SC on Day 1 of the Cycle 1 (Cycle length = 21 days). For each subsequent cycle (up to 5 cycles), the dose was titrated via Fibonacci scheme up to a maximum dose of 1.75mg/kg based on the safety review team (SRT) recommendations.
Units
Counts
Participants
OG0003
OG0013
OG0022
OG003
Title
Denominators
Categories
Title
Measurements
OG0009.29± 32.16
OG00112.56± 94.99
OG00236.90± 4.19
OG003
Secondary
Percent Change From Baseline to Day 113 in Concentration of Serum Iron
Blood samples were to be collected at pre-specified time intervals to determine serum iron concentration. Baseline was prespecified to be the last measurement prior to the first dose of study drug.
No data were collected for the percent change from baseline to day 113 in concentration of serum iron.
Posted
Baseline (prior to first dose of luspatercept) and Day 113
ID
Title
Description
OG000
Luspatercept 0.125mg/kg (Cohort 1)
Participants received luspatercept 0.125mg/kg as a subcutaneous (SC) injection every 3 weeks (Q3W) on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG001
Luspatercept 0.25mg/kg (Cohort 2)
Participants received luspatercept titrated up to 0.25mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG002
Luspatercept 0.50mg/kg (Cohort 3)
Participants received luspatercept titrated up to 0.50mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG003
Luspatercept 0.75mg/kg (Cohort 4)
Participants received luspatercept titrated up to 0.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG004
Luspatercept 1.00mg/kg (Cohort 5)
Participants received luspatercept titrated up to 1.00mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG005
Luspatercept 1.33mg/kg (Cohort 6)
Participants received luspatercept titrated up to 1.33mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG006
Luspatercept 1.75mg/kg (Cohort 7)
Participants received luspatercept titrated up to 1.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG007
Expansion Cohort
Participants received an initial dose of luspatercept 1.0mg/kg SC on Day 1 of the Cycle 1 (Cycle length = 21 days). For each subsequent cycle (up to 5 cycles), the dose was titrated via Fibonacci scheme up to a maximum dose of 1.75mg/kg based on the safety review team (SRT) recommendations.
Units
Counts
Participants
OG0000
OG0010
OG0020
OG003
Secondary
Percent Change From Baseline to Day 113 in Total Iron Binding Capacity (TIBC)
Blood samples were collected at pre-specified time intervals to determine TIBC. The percentage change from baseline in TIBC was reported.
All enrolled participants who received at least one dose of luspatercept and had data available for TIBC.
Posted
Mean
Standard Deviation
Percent change
Baseline (prior to first dose of luspatercept) and Day 113
ID
Title
Description
OG000
Luspatercept 0.125mg/kg (Cohort 1)
Participants received luspatercept 0.125mg/kg as a subcutaneous (SC) injection every 3 weeks (Q3W) on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG001
Luspatercept 0.25mg/kg (Cohort 2)
Participants received luspatercept titrated up to 0.25mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG002
Luspatercept 0.50mg/kg (Cohort 3)
Participants received luspatercept titrated up to 0.50mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG003
Luspatercept 0.75mg/kg (Cohort 4)
Participants received luspatercept titrated up to 0.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG004
Luspatercept 1.00mg/kg (Cohort 5)
Participants received luspatercept titrated up to 1.00mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG005
Luspatercept 1.33mg/kg (Cohort 6)
Participants received luspatercept titrated up to 1.33mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG006
Luspatercept 1.75mg/kg (Cohort 7)
Participants received luspatercept titrated up to 1.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG007
Expansion Cohort
Participants received an initial dose of luspatercept 1.0mg/kg SC on Day 1 of the Cycle 1 (Cycle length = 21 days). For each subsequent cycle (up to 5 cycles), the dose was titrated via Fibonacci scheme up to a maximum dose of 1.75mg/kg based on the safety review team (SRT) recommendations.
Units
Counts
Participants
OG0003
OG0013
OG0023
OG003
Title
Denominators
Categories
Title
Measurements
OG000-1.49± 8.940
OG0011.61± 1.392
OG002-6.70± 6.050
OG003
Secondary
Percent Change From Baseline to Day 113 in Concentration of Transferrin
Blood samples were to be collected at pre-specified time intervals to determine concentration of transferrin. Baseline was prespecified to be the last measurement prior to the first dose of study drug.
No data were collected for the percent change from baseline to day 113 in concentration of transferrin.
Posted
Baseline (prior to first dose of luspatercept) and Day 113
ID
Title
Description
OG000
Luspatercept 0.125mg/kg (Cohort 1)
Participants received luspatercept 0.125mg/kg as a subcutaneous (SC) injection every 3 weeks (Q3W) on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG001
Luspatercept 0.25mg/kg (Cohort 2)
Participants received luspatercept titrated up to 0.25mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG002
Luspatercept 0.50mg/kg (Cohort 3)
Participants received luspatercept titrated up to 0.50mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG003
Luspatercept 0.75mg/kg (Cohort 4)
Participants received luspatercept titrated up to 0.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG004
Luspatercept 1.00mg/kg (Cohort 5)
Participants received luspatercept titrated up to 1.00mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG005
Luspatercept 1.33mg/kg (Cohort 6)
Participants received luspatercept titrated up to 1.33mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG006
Luspatercept 1.75mg/kg (Cohort 7)
Participants received luspatercept titrated up to 1.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG007
Expansion Cohort
Participants received an initial dose of luspatercept 1.0mg/kg SC on Day 1 of the Cycle 1 (Cycle length = 21 days). For each subsequent cycle (up to 5 cycles), the dose was titrated via Fibonacci scheme up to a maximum dose of 1.75mg/kg based on the safety review team (SRT) recommendations.
Units
Counts
Participants
OG0000
OG0010
OG0020
OG003
Secondary
Percent Change From Baseline to Day 113 in Concentration of Soluble Transferrin Receptor
Blood samples were collected at pre-specified time intervals to determine soluble transferrin receptor concentration. The percentage change from baseline in concentration of soluble transferrin receptor was reported.
All enrolled participants who received at least one dose of luspatercept and had data available for soluble transferrin receptor.
Posted
Mean
Standard Deviation
Percent change
Baseline (prior to first dose of luspatercept) and Day 113
ID
Title
Description
OG000
Luspatercept 0.125mg/kg (Cohort 1)
Participants received luspatercept 0.125mg/kg as a subcutaneous (SC) injection every 3 weeks (Q3W) on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG001
Luspatercept 0.25mg/kg (Cohort 2)
Participants received luspatercept titrated up to 0.25mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG002
Luspatercept 0.50mg/kg (Cohort 3)
Participants received luspatercept titrated up to 0.50mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG003
Luspatercept 0.75mg/kg (Cohort 4)
Participants received luspatercept titrated up to 0.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG004
Luspatercept 1.00mg/kg (Cohort 5)
Participants received luspatercept titrated up to 1.00mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG005
Luspatercept 1.33mg/kg (Cohort 6)
Participants received luspatercept titrated up to 1.33mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG006
Luspatercept 1.75mg/kg (Cohort 7)
Participants received luspatercept titrated up to 1.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG007
Expansion Cohort
Participants received an initial dose of luspatercept 1.0mg/kg SC on Day 1 of the Cycle 1 (Cycle length = 21 days). For each subsequent cycle (up to 5 cycles), the dose was titrated via Fibonacci scheme up to a maximum dose of 1.75mg/kg based on the safety review team (SRT) recommendations.
Units
Counts
Participants
OG0003
OG0012
OG0022
OG003
Title
Denominators
Categories
Title
Measurements
OG00060.88± 76.427
OG001-31.46± 5.135
OG002-4.62± 6.527
OG003
Secondary
Percent Change From Baseline to Day 113 in Concentration of Serum Ferritin
Blood samples were collected at pre-specified time intervals to determine serum ferritin concentration. The percentage change from baseline in concentration of serum ferritin was reported.
All enrolled participants who received at least one dose of luspatercept and had data available for serum ferritin.
Posted
Mean
Standard Deviation
Percent change
Baseline (prior to first dose of luspatercept) and Day 113
ID
Title
Description
OG000
Luspatercept 0.125mg/kg (Cohort 1)
Participants received luspatercept 0.125mg/kg as a subcutaneous (SC) injection every 3 weeks (Q3W) on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG001
Luspatercept 0.25mg/kg (Cohort 2)
Participants received luspatercept titrated up to 0.25mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG002
Luspatercept 0.50mg/kg (Cohort 3)
Participants received luspatercept titrated up to 0.50mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG003
Luspatercept 0.75mg/kg (Cohort 4)
Participants received luspatercept titrated up to 0.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG004
Luspatercept 1.00mg/kg (Cohort 5)
Participants received luspatercept titrated up to 1.00mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG005
Luspatercept 1.33mg/kg (Cohort 6)
Participants received luspatercept titrated up to 1.33mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG006
Luspatercept 1.75mg/kg (Cohort 7)
Participants received luspatercept titrated up to 1.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG007
Expansion Cohort
Participants received an initial dose of luspatercept 1.0mg/kg SC on Day 1 of the Cycle 1 (Cycle length = 21 days). For each subsequent cycle (up to 5 cycles), the dose was titrated via Fibonacci scheme up to a maximum dose of 1.75mg/kg based on the safety review team (SRT) recommendations.
Units
Counts
Participants
OG0003
OG0013
OG0023
OG003
Title
Denominators
Categories
Title
Measurements
OG000-19.55± 32.845
OG00158.63± 24.342
OG00274.31± 160.308
OG003
Secondary
Percent Change From Baseline to Day 113 in Concentration of Non-Transferrin Bound Iron (NTBI)
Blood samples were to be collected at pre-specified time intervals to determine concentration of NTBI. Baseline was prespecified to be the last measurement prior to the first dose of study drug.
No data were collected for the percent change from baseline to day 113 in concentration of NTBI.
Posted
Baseline (prior to first dose of luspatercept) and Day 113
ID
Title
Description
OG000
Luspatercept 0.125mg/kg (Cohort 1)
Participants received luspatercept 0.125mg/kg as a subcutaneous (SC) injection every 3 weeks (Q3W) on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG001
Luspatercept 0.25mg/kg (Cohort 2)
Participants received luspatercept titrated up to 0.25mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG002
Luspatercept 0.50mg/kg (Cohort 3)
Participants received luspatercept titrated up to 0.50mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG003
Luspatercept 0.75mg/kg (Cohort 4)
Participants received luspatercept titrated up to 0.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG004
Luspatercept 1.00mg/kg (Cohort 5)
Participants received luspatercept titrated up to 1.00mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG005
Luspatercept 1.33mg/kg (Cohort 6)
Participants received luspatercept titrated up to 1.33mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG006
Luspatercept 1.75mg/kg (Cohort 7)
Participants received luspatercept titrated up to 1.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG007
Expansion Cohort
Participants received an initial dose of luspatercept 1.0mg/kg SC on Day 1 of the Cycle 1 (Cycle length = 21 days). For each subsequent cycle (up to 5 cycles), the dose was titrated via Fibonacci scheme up to a maximum dose of 1.75mg/kg based on the safety review team (SRT) recommendations.
Units
Counts
Participants
OG0000
OG0010
OG0020
OG003
Secondary
Percent Change From Baseline to Day 113 in Concentration of Serum Hepcidin
Blood samples were collected at pre-specified time intervals to determine soluble hepcidin concentration. The percentage change from baseline in concentration of soluble hepcidin was reported.
All enrolled participants who received at least one dose of luspatercept and had data available for serum hepcidin.
Posted
Mean
Standard Deviation
Percent change
Baseline (prior to first dose of luspatercept) and Day 113
ID
Title
Description
OG000
Luspatercept 0.125mg/kg (Cohort 1)
Participants received luspatercept 0.125mg/kg as a subcutaneous (SC) injection every 3 weeks (Q3W) on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG001
Luspatercept 0.25mg/kg (Cohort 2)
Participants received luspatercept titrated up to 0.25mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG002
Luspatercept 0.50mg/kg (Cohort 3)
Participants received luspatercept titrated up to 0.50mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG003
Luspatercept 0.75mg/kg (Cohort 4)
Participants received luspatercept titrated up to 0.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG004
Luspatercept 1.00mg/kg (Cohort 5)
Participants received luspatercept titrated up to 1.00mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG005
Luspatercept 1.33mg/kg (Cohort 6)
Participants received luspatercept titrated up to 1.33mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG006
Luspatercept 1.75mg/kg (Cohort 7)
Participants received luspatercept titrated up to 1.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG007
Expansion Cohort
Participants received an initial dose of luspatercept 1.0mg/kg SC on Day 1 of the Cycle 1 (Cycle length = 21 days). For each subsequent cycle (up to 5 cycles), the dose was titrated via Fibonacci scheme up to a maximum dose of 1.75mg/kg based on the safety review team (SRT) recommendations.
Units
Counts
Participants
OG0003
OG0013
OG0023
OG003
Title
Denominators
Categories
Title
Measurements
OG000-42.40± 5.907
OG0016.56± 35.740
OG002-14.55± 39.855
OG003
Secondary
Percent Change From Baseline to Day 113 in Concentration of Serum Erythropoietin
Blood samples were collected at pre-specified time intervals to determine serum erythropoietin concentration. The percentage change from baseline in concentration of serum erythropoietin was reported.
All participants who received at least one dose of luspatercept and had data available for serum erythropoietin.
Posted
Mean
Standard Deviation
Percent change
Baseline (prior to first dose of luspatercept) and Day 113
ID
Title
Description
OG000
Luspatercept 0.125mg/kg (Cohort 1)
Participants received luspatercept 0.125mg/kg as a subcutaneous (SC) injection every 3 weeks (Q3W) on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG001
Luspatercept 0.25mg/kg (Cohort 2)
Participants received luspatercept titrated up to 0.25mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG002
Luspatercept 0.50mg/kg (Cohort 3)
Participants received luspatercept titrated up to 0.50mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG003
Luspatercept 0.75mg/kg (Cohort 4)
Participants received luspatercept titrated up to 0.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG004
Luspatercept 1.00mg/kg (Cohort 5)
Participants received luspatercept titrated up to 1.00mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG005
Luspatercept 1.33mg/kg (Cohort 6)
Participants received luspatercept titrated up to 1.33mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG006
Luspatercept 1.75mg/kg (Cohort 7)
Participants received luspatercept titrated up to 1.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG007
Expansion Cohort
Participants received an initial dose of luspatercept 1.0mg/kg SC on Day 1 of the Cycle 1 (Cycle length = 21 days). For each subsequent cycle (up to 5 cycles), the dose was titrated via Fibonacci scheme up to a maximum dose of 1.75mg/kg based on the safety review team (SRT) recommendations.
Units
Counts
Participants
OG0003
OG0013
OG0022
OG003
Title
Denominators
Categories
Title
Measurements
OG000144.48± 232.336
OG001110.51± 85.751
OG002122.85± 163.500
OG003
Secondary
Percent Change From Baseline to Day 113 in Reticulocyte Count
Blood samples were collected at pre-specified time intervals to determine reticulocyte count. The percentage change from baseline in mean reticulocyte count was reported.
All enrolled participants who received at least one dose of luspatercept and had data available for reticulocyte count.
Posted
Mean
Standard Deviation
Percent change
Baseline (prior to first dose of luspatercept) and Day 113
ID
Title
Description
OG000
Luspatercept 0.125mg/kg (Cohort 1)
Participants received luspatercept 0.125mg/kg as a subcutaneous (SC) injection every 3 weeks (Q3W) on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG001
Luspatercept 0.25mg/kg (Cohort 2)
Participants received luspatercept titrated up to 0.25mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG002
Luspatercept 0.50mg/kg (Cohort 3)
Participants received luspatercept titrated up to 0.50mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG003
Luspatercept 0.75mg/kg (Cohort 4)
Participants received luspatercept titrated up to 0.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG004
Luspatercept 1.00mg/kg (Cohort 5)
Participants received luspatercept titrated up to 1.00mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG005
Luspatercept 1.33mg/kg (Cohort 6)
Participants received luspatercept titrated up to 1.33mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG006
Luspatercept 1.75mg/kg (Cohort 7)
Participants received luspatercept titrated up to 1.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG007
Expansion Cohort
Participants received an initial dose of luspatercept 1.0mg/kg SC on Day 1 of the Cycle 1 (Cycle length = 21 days). For each subsequent cycle (up to 5 cycles), the dose was titrated via Fibonacci scheme up to a maximum dose of 1.75mg/kg based on the safety review team (SRT) recommendations.
Units
Counts
Participants
OG0003
OG0011
OG0022
OG003
Title
Denominators
Categories
Title
Measurements
OG000110.82± 127.489
OG0018.10± NANA=SD could not be calculated due to insufficient number of participants in the study.
OG00279.77± 45.816
Secondary
Percent Change From Baseline to Day 113 in Direct Bilirubin Level
Blood samples were collected at pre-specified time intervals to determine serum direct bilirubin concentration. The percentage change from baseline in mean concentration direct bilirubin was reported.
All enrolled participants who received at least one dose of luspatercept and had data available for direct bilirubin.
Posted
Mean
Standard Deviation
Percent change
Baseline (prior to first dose of luspatercept) and Day 113
ID
Title
Description
OG000
Luspatercept 0.125mg/kg (Cohort 1)
Participants received luspatercept 0.125mg/kg as a subcutaneous (SC) injection every 3 weeks (Q3W) on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG001
Luspatercept 0.25mg/kg (Cohort 2)
Participants received luspatercept titrated up to 0.25mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG002
Luspatercept 0.50mg/kg (Cohort 3)
Participants received luspatercept titrated up to 0.50mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG003
Luspatercept 0.75mg/kg (Cohort 4)
Participants received luspatercept titrated up to 0.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG004
Luspatercept 1.00mg/kg (Cohort 5)
Participants received luspatercept titrated up to 1.00mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG005
Luspatercept 1.33mg/kg (Cohort 6)
Participants received luspatercept titrated up to 1.33mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG006
Luspatercept 1.75mg/kg (Cohort 7)
Participants received luspatercept titrated up to 1.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG007
Expansion Cohort
Participants received an initial dose of luspatercept 1.0mg/kg SC on Day 1 of the Cycle 1 (Cycle length = 21 days). For each subsequent cycle (up to 5 cycles), the dose was titrated via Fibonacci scheme up to a maximum dose of 1.75mg/kg based on the safety review team (SRT) recommendations.
Units
Counts
Participants
OG0003
OG0012
OG0021
OG003
Title
Denominators
Categories
Title
Measurements
OG000-18.45± 6.569
OG00112.38± 38.465
OG0022.33± NANA = SD could not be calculated due to low number of participants with direct bilirubin values
Secondary
Percent Change From Baseline to Day 113 in Total Bilirubin Level
Blood samples were collected at pre-specified time intervals to determine total bilirubin concentration. The percentage change from baseline in mean concentration total bilirubin was reported.
All enrolled participants who received at least one dose of luspatercept and had data available for total bilirubin.
Posted
Mean
Standard Deviation
Percent change
Baseline (prior to first dose of luspatercept) and Day 113
ID
Title
Description
OG000
Luspatercept 0.125mg/kg (Cohort 1)
Participants received luspatercept 0.125mg/kg as a subcutaneous (SC) injection every 3 weeks (Q3W) on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG001
Luspatercept 0.25mg/kg (Cohort 2)
Participants received luspatercept titrated up to 0.25mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG002
Luspatercept 0.50mg/kg (Cohort 3)
Participants received luspatercept titrated up to 0.50mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG003
Luspatercept 0.75mg/kg (Cohort 4)
Participants received luspatercept titrated up to 0.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG004
Luspatercept 1.00mg/kg (Cohort 5)
Participants received luspatercept titrated up to 1.00mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG005
Luspatercept 1.33mg/kg (Cohort 6)
Participants received luspatercept titrated up to 1.33mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG006
Luspatercept 1.75mg/kg (Cohort 7)
Participants received luspatercept titrated up to 1.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG007
Expansion Cohort
Participants received an initial dose of luspatercept 1.0mg/kg SC on Day 1 of the Cycle 1 (Cycle length = 21 days). For each subsequent cycle (up to 5 cycles), the dose was titrated via Fibonacci scheme up to a maximum dose of 1.75mg/kg based on the safety review team (SRT) recommendations.
Units
Counts
Participants
OG0003
OG0013
OG0023
OG003
Title
Denominators
Categories
Title
Measurements
OG000-25.41± 5.596
OG0014.32± 26.094
OG00247.43± 51.076
OG003
Secondary
Percent Change From Baseline to Day 113 in Lactate Dehydrogenase Level
Blood samples were collected at pre-specified time intervals to determine serum lactate dehydrogenase level. The percentage change from baseline in mean concentration lactate dehydrogenase level was reported.
All enrolled participants who received at least one dose of luspatercept and had data available for lactate dehydrogenase.
Posted
Mean
Standard Deviation
Percent change
Baseline (prior to first dose of luspatercept) and Day 113
ID
Title
Description
OG000
Luspatercept 0.125mg/kg (Cohort 1)
Participants received luspatercept 0.125mg/kg as a subcutaneous (SC) injection every 3 weeks (Q3W) on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG001
Luspatercept 0.25mg/kg (Cohort 2)
Participants received luspatercept titrated up to 0.25mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG002
Luspatercept 0.50mg/kg (Cohort 3)
Participants received luspatercept titrated up to 0.50mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG003
Luspatercept 0.75mg/kg (Cohort 4)
Participants received luspatercept titrated up to 0.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG004
Luspatercept 1.00mg/kg (Cohort 5)
Participants received luspatercept titrated up to 1.00mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG005
Luspatercept 1.33mg/kg (Cohort 6)
Participants received luspatercept titrated up to 1.33mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG006
Luspatercept 1.75mg/kg (Cohort 7)
Participants received luspatercept titrated up to 1.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG007
Expansion Cohort
Participants received an initial dose of luspatercept 1.0mg/kg SC on Day 1 of the Cycle 1 (Cycle length = 21 days). For each subsequent cycle (up to 5 cycles), the dose was titrated via Fibonacci scheme up to a maximum dose of 1.75mg/kg based on the safety review team (SRT) recommendations.
Units
Counts
Participants
OG0003
OG0013
OG0023
OG003
Title
Denominators
Categories
Title
Measurements
OG00013.09± 28.850
OG001-10.28± 6.589
OG0020.03± 32.694
OG003
Secondary
Percent Change From Baseline to Day 113 in Nucleated RBC (nRBC)
Blood samples were to be collected at pre-specified time intervals to determine concentration of nRBC. Baseline was prespecified to be the last measurement prior to the first dose of study drug.
No data were collected for the percent change from baseline to day 113 in concentration of nRBC.
Posted
Baseline (prior to first dose of luspatercept) and Day 113
ID
Title
Description
OG000
Luspatercept 0.125mg/kg (Cohort 1)
Participants received luspatercept 0.125mg/kg as a subcutaneous (SC) injection every 3 weeks (Q3W) on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG001
Luspatercept 0.25mg/kg (Cohort 2)
Participants received luspatercept titrated up to 0.25mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG002
Luspatercept 0.50mg/kg (Cohort 3)
Participants received luspatercept titrated up to 0.50mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG003
Luspatercept 0.75mg/kg (Cohort 4)
Participants received luspatercept titrated up to 0.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG004
Luspatercept 1.00mg/kg (Cohort 5)
Participants received luspatercept titrated up to 1.00mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG005
Luspatercept 1.33mg/kg (Cohort 6)
Participants received luspatercept titrated up to 1.33mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG006
Luspatercept 1.75mg/kg (Cohort 7)
Participants received luspatercept titrated up to 1.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG007
Expansion Cohort
Participants received an initial dose of luspatercept 1.0mg/kg SC on Day 1 of the Cycle 1 (Cycle length = 21 days). For each subsequent cycle (up to 5 cycles), the dose was titrated via Fibonacci scheme up to a maximum dose of 1.75mg/kg based on the safety review team (SRT) recommendations.
Units
Counts
Participants
OG0000
OG0010
OG0020
OG003
Secondary
Percent Change From Baseline to Day 113 in Concentration of Serum Bone-Specific Alkaline Phosphatase (BSAP)
Blood samples were collected at pre-specified time intervals to determine serum BSAP concentration. The percentage change from baseline in concentration BSAP was reported.
All enrolled participants who received at least one dose of luspatercept and had data available for BSAP.
Posted
Mean
Standard Deviation
Percent change
Baseline (prior to first dose of luspatercept) and Day 113
ID
Title
Description
OG000
Luspatercept 0.125mg/kg (Cohort 1)
Participants received luspatercept 0.125mg/kg as a subcutaneous (SC) injection every 3 weeks (Q3W) on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG001
Luspatercept 0.25mg/kg (Cohort 2)
Participants received luspatercept titrated up to 0.25mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG002
Luspatercept 0.50mg/kg (Cohort 3)
Participants received luspatercept titrated up to 0.50mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG003
Luspatercept 0.75mg/kg (Cohort 4)
Participants received luspatercept titrated up to 0.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG004
Luspatercept 1.00mg/kg (Cohort 5)
Participants received luspatercept titrated up to 1.00mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG005
Luspatercept 1.33mg/kg (Cohort 6)
Participants received luspatercept titrated up to 1.33mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG006
Luspatercept 1.75mg/kg (Cohort 7)
Participants received luspatercept titrated up to 1.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG007
Expansion Cohort
Participants received an initial dose of luspatercept 1.0mg/kg SC on Day 1 of the Cycle 1 (Cycle length = 21 days). For each subsequent cycle (up to 5 cycles), the dose was titrated via Fibonacci scheme up to a maximum dose of 1.75mg/kg based on the safety review team (SRT) recommendations.
Units
Counts
Participants
OG0002
OG0013
OG0023
OG003
Title
Denominators
Categories
Title
Measurements
OG000-6.41± 3.698
OG001-36.70± 9.430
OG002-24.60± 16.413
OG003
Secondary
Percent Change From Baseline to Day 113 in Concentration of Serum Cross-Linked C-Telopeptide of Type I Collagen (CTX)
Blood samples were collected at pre-specified time intervals to determine serum CTX. The percentage change from baseline in concentration of CTX was reported.
All enrolled participants who received at least one dose of luspatercept and had data available for CTX.
Posted
Mean
Standard Deviation
Percent change
Baseline (prior to first dose of luspatercept) and Day 113
ID
Title
Description
OG000
Luspatercept 0.125mg/kg (Cohort 1)
Participants received luspatercept 0.125mg/kg as a subcutaneous (SC) injection every 3 weeks (Q3W) on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG001
Luspatercept 0.25mg/kg (Cohort 2)
Participants received luspatercept titrated up to 0.25mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG002
Luspatercept 0.50mg/kg (Cohort 3)
Participants received luspatercept titrated up to 0.50mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG003
Luspatercept 0.75mg/kg (Cohort 4)
Participants received luspatercept titrated up to 0.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG004
Luspatercept 1.00mg/kg (Cohort 5)
Participants received luspatercept titrated up to 1.00mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG005
Luspatercept 1.33mg/kg (Cohort 6)
Participants received luspatercept titrated up to 1.33mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG006
Luspatercept 1.75mg/kg (Cohort 7)
Participants received luspatercept titrated up to 1.75mg/kg via Fibonacci scheme, SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
OG007
Expansion Cohort
Participants received an initial dose of luspatercept 1.0mg/kg SC on Day 1 of the Cycle 1 (Cycle length = 21 days). For each subsequent cycle (up to 5 cycles), the dose was titrated via Fibonacci scheme up to a maximum dose of 1.75mg/kg based on the safety review team (SRT) recommendations.
Units
Counts
Participants
OG0001
OG0010
OG0021
OG003
Title
Denominators
Categories
Title
Measurements
OG000-4.42± NANA = SD could not be calculated due to low number of participants with CTX values
OG00216.26± NANA = SD could not be calculated due to low number of participants with CTX values
OG003
0
3
1
3
1
3
EG001
Luspatercept 0.25 mg/kg (Cohort 2)
Participants received luspatercept titrated up to 0.25mg/kg SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
0
3
0
3
2
3
EG002
Luspatercept 0.50 mg/kg (Cohort 3)
Participants received luspatercept titrated up to 0.50mg/kg SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
0
3
0
3
3
3
EG003
Luspatercept 0.75 mg/kg (Cohort 4)
Participants received luspatercept titrated up to 0.75mg/kg SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
0
6
0
6
5
6
EG004
Luspatercept 1.0 mg/kg (Cohort 5)
Participants received luspatercept titrated up to 1mg/kg SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
0
3
1
3
3
3
EG005
Luspatercept 1.33 mg/kg (Cohort 6)
Participants received luspatercept titrated up to 1.33mg/kg SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
0
6
3
6
3
6
EG006
Luspatercept 1.75 mg/kg (Cohort 7)
Participants received luspatercept titrated up to 1.75mg/kg SC injection Q3W on Day 1 of each cycle for up to 5 cycles (each cycle length = 21 days).
0
3
1
3
2
3
EG007
Expansion Cohort
Participants received an initial dose of luspatercept 1mg/kg SC on Day 1 of the Cycle 1 (Cycle length = 21 days). For each subsequent cycle (up to 5 cycles), the dose was titrated up to a maximum dose of 1.75mg/kg based on the safety review team (SRT) recommendations.
0
89
14
89
62
89
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0051 events1 affected6 at risk
EG0060 events0 affected3 at risk
EG0071 events1 affected89 at risk
Aortic valve stenosis
Cardiac disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0071 events1 affected89 at risk
Colitis
Gastrointestinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0071 events1 affected89 at risk
General physical health deterioration
General disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0072 events1 affected89 at risk
Cholecystitis acute
Hepatobiliary disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0041 events1 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0070 events0 affected89 at risk
Clostridium difficile colitis
Infections and infestations
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0051 events1 affected6 at risk
EG0060 events0 affected3 at risk
EG0070 events0 affected89 at risk
Haematoma infection
Infections and infestations
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0071 events1 affected89 at risk
Infection
Infections and infestations
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0071 events1 affected89 at risk
Lung infection
Infections and infestations
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0071 events1 affected89 at risk
Pneumonia
Infections and infestations
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0071 events1 affected89 at risk
Contusion
Injury, poisoning and procedural complications
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0071 events1 affected89 at risk
Rib fracture
Injury, poisoning and procedural complications
MedDRA 20.0
Systematic Assessment
EG0001 events1 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0070 events0 affected89 at risk
Bursitis
Musculoskeletal and connective tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0051 events1 affected6 at risk
EG0060 events0 affected3 at risk
EG0070 events0 affected89 at risk
Haemarthrosis
Musculoskeletal and connective tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0061 events1 affected3 at risk
EG0070 events0 affected89 at risk
Intervertebral disc protrusion
Musculoskeletal and connective tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0061 events1 affected3 at risk
EG0070 events0 affected89 at risk
Myalgia
Musculoskeletal and connective tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0071 events1 affected89 at risk
Diffuse large B-cell lymphoma
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0071 events1 affected89 at risk
Myelodysplastic syndrome
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0071 events1 affected89 at risk
Transformation to acute myeloid leukaemia
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0071 events1 affected89 at risk
Normal pressure hydrocephalus
Nervous system disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0071 events1 affected89 at risk
Hypertensive crisis
Vascular disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0071 events1 affected89 at risk
Temporal arteritis
Vascular disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0071 events1 affected89 at risk
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0061 events1 affected3 at risk
EG0075 events3 affected89 at risk
Leukopenia
Blood and lymphatic system disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0041 events1 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0070 events0 affected89 at risk
Red blood cell abnormality
Blood and lymphatic system disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0041 events1 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0070 events0 affected89 at risk
Splenomegaly
Blood and lymphatic system disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0031 events1 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0071 events1 affected89 at risk
Atrial fibrillation
Cardiac disorders
MedDRA 20.0
Systematic Assessment
EG0001 events1 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0070 events0 affected89 at risk
Hypertensive heart disease
Cardiac disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0051 events1 affected6 at risk
EG0060 events0 affected3 at risk
EG0070 events0 affected89 at risk
Vertigo
Ear and labyrinth disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0031 events1 affected6 at risk
EG0040 events0 affected3 at risk
EG0051 events1 affected6 at risk
EG0060 events0 affected3 at risk
EG0072 events2 affected89 at risk
Abdominal pain lower
Gastrointestinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0041 events1 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0070 events0 affected89 at risk
Abdominal pain upper
Gastrointestinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0021 events1 affected3 at risk
EG0030 events0 affected6 at risk
EG0041 events1 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0074 events4 affected89 at risk
Constipation
Gastrointestinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0031 events1 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0071 events1 affected89 at risk
Diarrhoea
Gastrointestinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0011 events1 affected3 at risk
EG0021 events1 affected3 at risk
EG0034 events1 affected6 at risk
EG0040 events0 affected3 at risk
EG0051 events1 affected6 at risk
EG0060 events0 affected3 at risk
EG0075 events5 affected89 at risk
Flatulence
Gastrointestinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0031 events1 affected6 at risk
EG0041 events1 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0070 events0 affected89 at risk
Inguinal hernia
Gastrointestinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0041 events1 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0070 events0 affected89 at risk
Nausea
Gastrointestinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0021 events1 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0062 events1 affected3 at risk
EG0072 events2 affected89 at risk
Toothache
Gastrointestinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0062 events1 affected3 at risk
EG0071 events1 affected89 at risk
Vomiting
Gastrointestinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0011 events1 affected3 at risk
EG0020 events0 affected3 at risk
EG0032 events1 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0073 events3 affected89 at risk
Fatigue
General disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0054 events3 affected6 at risk
EG0060 events0 affected3 at risk
EG00712 events11 affected89 at risk
Injection site erythema
General disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0032 events2 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0072 events2 affected89 at risk
Injection site pain
General disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0031 events1 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0070 events0 affected89 at risk
Injection site pruritus
General disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0011 events1 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0071 events1 affected89 at risk
Injection site rash
General disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0031 events1 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0070 events0 affected89 at risk
Injection site swelling
General disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0031 events1 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0071 events1 affected89 at risk
Non-cardiac chest pain
General disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0051 events1 affected6 at risk
EG0060 events0 affected3 at risk
EG0070 events0 affected89 at risk
Oedema peripheral
General disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0021 events1 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0077 events6 affected89 at risk
Peripheral swelling
General disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0061 events1 affected3 at risk
EG0070 events0 affected89 at risk
Cholelithiasis
Hepatobiliary disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0041 events1 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0070 events0 affected89 at risk
Hepatic steatosis
Hepatobiliary disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0031 events1 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0070 events0 affected89 at risk
Bronchitis
Infections and infestations
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0041 events1 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0077 events6 affected89 at risk
Catheter site infection
Infections and infestations
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0021 events1 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0070 events0 affected89 at risk
Conjunctivitis
Infections and infestations
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0031 events1 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0070 events0 affected89 at risk
Infection
Infections and infestations
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0021 events1 affected3 at risk
EG0030 events0 affected6 at risk
EG0041 events1 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0072 events2 affected89 at risk
Nasopharyngitis
Infections and infestations
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0031 events1 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0070 events0 affected89 at risk
Sinusitis
Infections and infestations
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0041 events1 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0070 events0 affected89 at risk
Subcutaneous abscess
Infections and infestations
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0041 events1 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0070 events0 affected89 at risk
Viral upper respiratory tract infection
Infections and infestations
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0011 events1 affected3 at risk
EG0020 events0 affected3 at risk
EG0031 events1 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG00712 events11 affected89 at risk
Fall
Injury, poisoning and procedural complications
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0051 events1 affected6 at risk
EG0060 events0 affected3 at risk
EG0077 events3 affected89 at risk
Lumbar vertebral fracture
Injury, poisoning and procedural complications
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0042 events2 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0070 events0 affected89 at risk
Thoracic vertebral fracture
Injury, poisoning and procedural complications
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0041 events1 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0070 events0 affected89 at risk
Tooth fracture
Injury, poisoning and procedural complications
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0051 events1 affected6 at risk
EG0060 events0 affected3 at risk
EG0070 events0 affected89 at risk
Blood bilirubin increased
Investigations
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0052 events1 affected6 at risk
EG0060 events0 affected3 at risk
EG0072 events2 affected89 at risk
C-reactive protein increased
Investigations
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0051 events1 affected6 at risk
EG0060 events0 affected3 at risk
EG0071 events1 affected89 at risk
Decreased appetite
Metabolism and nutrition disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0031 events1 affected6 at risk
EG0041 events1 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0070 events0 affected89 at risk
Hyperkalaemia
Metabolism and nutrition disorders
MedDRA 20.0
Systematic Assessment
EG0001 events1 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0070 events0 affected89 at risk
Arthralgia
Musculoskeletal and connective tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0061 events1 affected3 at risk
EG0076 events5 affected89 at risk
Bone pain
Musculoskeletal and connective tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0023 events1 affected3 at risk
EG0030 events0 affected6 at risk
EG0044 events2 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0079 events5 affected89 at risk
Bursitis
Musculoskeletal and connective tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0052 events1 affected6 at risk
EG0060 events0 affected3 at risk
EG0070 events0 affected89 at risk
Muscle spasms
Musculoskeletal and connective tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0022 events2 affected3 at risk
EG0030 events0 affected6 at risk
EG0041 events1 affected3 at risk
EG0050 events0 affected6 at risk
EG0061 events1 affected3 at risk
EG0071 events1 affected89 at risk
Muscular weakness
Musculoskeletal and connective tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0021 events1 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0070 events0 affected89 at risk
Musculoskeletal pain
Musculoskeletal and connective tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0061 events1 affected3 at risk
EG0071 events1 affected89 at risk
Myalgia
Musculoskeletal and connective tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0011 events1 affected3 at risk
EG0021 events1 affected3 at risk
EG0030 events0 affected6 at risk
EG0041 events1 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0077 events6 affected89 at risk
Osteopenia
Musculoskeletal and connective tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0051 events1 affected6 at risk
EG0060 events0 affected3 at risk
EG0070 events0 affected89 at risk
Osteoporosis
Musculoskeletal and connective tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0041 events1 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0070 events0 affected89 at risk
Pain in extremity
Musculoskeletal and connective tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0051 events1 affected6 at risk
EG0060 events0 affected3 at risk
EG0071 events1 affected89 at risk
Myelofibrosis
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0051 events1 affected6 at risk
EG0060 events0 affected3 at risk
EG0070 events0 affected89 at risk
Transformation to acute myeloid leukaemia
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0041 events1 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0070 events0 affected89 at risk
Dizziness
Nervous system disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0062 events1 affected3 at risk
EG0077 events4 affected89 at risk
Headache
Nervous system disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0031 events1 affected6 at risk
EG0040 events0 affected3 at risk
EG0051 events1 affected6 at risk
EG0060 events0 affected3 at risk
EG00712 events9 affected89 at risk
Migraine
Nervous system disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0061 events1 affected3 at risk
EG0070 events0 affected89 at risk
Polyneuropathy
Nervous system disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0021 events1 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0070 events0 affected89 at risk
Insomnia
Psychiatric disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0031 events1 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0071 events1 affected89 at risk
Dysuria
Renal and urinary disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0021 events1 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0070 events0 affected89 at risk
Nocturia
Renal and urinary disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0031 events1 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0070 events0 affected89 at risk
Dyspnoea
Respiratory, thoracic and mediastinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0021 events1 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0061 events1 affected3 at risk
EG0074 events3 affected89 at risk
Dyspnoea exertional
Respiratory, thoracic and mediastinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0021 events1 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0071 events1 affected89 at risk
Epistaxis
Respiratory, thoracic and mediastinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0051 events1 affected6 at risk
EG0060 events0 affected3 at risk
EG0073 events3 affected89 at risk
Oropharyngeal pain
Respiratory, thoracic and mediastinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0011 events1 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0071 events1 affected89 at risk
Rhinorrhoea
Respiratory, thoracic and mediastinal disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0031 events1 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0070 events0 affected89 at risk
Pruritus
Skin and subcutaneous tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0022 events1 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0075 events2 affected89 at risk
Pruritus generalised
Skin and subcutaneous tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0011 events1 affected3 at risk
EG0020 events0 affected3 at risk
EG0031 events1 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0070 events0 affected89 at risk
Rash
Skin and subcutaneous tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0021 events1 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0074 events4 affected89 at risk
Urticaria
Skin and subcutaneous tissue disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0061 events1 affected3 at risk
EG0070 events0 affected89 at risk
Hot flush
Vascular disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0011 events1 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0061 events1 affected3 at risk
EG0071 events1 affected89 at risk
Hypertension
Vascular disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0020 events0 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0051 events1 affected6 at risk
EG0060 events0 affected3 at risk
EG00725 events19 affected89 at risk
Hypotension
Vascular disorders
MedDRA 20.0
Systematic Assessment
EG0000 events0 affected3 at risk
EG0010 events0 affected3 at risk
EG0021 events1 affected3 at risk
EG0030 events0 affected6 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected6 at risk
EG0060 events0 affected3 at risk
EG0071 events1 affected89 at risk
OTHER
Results Disclosure Restriction on PI(s)?
Yes
Other Details
The publication or disclosure of study results will be permitted as per the agreement between the sponsor and the investigator(s).
64.8
± 11.3
BG00473.7± 3.8
BG00568.2± 7.1
BG00663.7± 19.6
BG00771.6± 9.6
BG00870.4± 10.7
3
BG0040
BG0051
BG0062
BG00730
BG00844
Male
BG0000
BG0010
BG0021
BG0033
BG0043
BG0055
BG0061
BG00759
BG00872
0
BG0040
BG0050
BG0060
BG0070
BG0080
Not Hispanic or Latino
BG0003
BG0012
BG0023
BG0036
BG0043
BG0055
BG0063
BG00780
BG008105
Unknown or Not Reported
BG0000
BG0011
BG0020
BG0030
BG0040
BG0051
BG0060
BG0079
BG00811
0
BG0040
BG0050
BG0060
BG0070
BG0080
Asian
BG0000
BG0010
BG0020
BG0030
BG0040
BG0050
BG0060
BG0070
BG0080
Native Hawaiian or Other Pacific Islander
BG0000
BG0010
BG0020
BG0030
BG0040
BG0050
BG0060
BG0070
BG0080
Black or African American
BG0000
BG0010
BG0020
BG0030
BG0040
BG0050
BG0060
BG0070
BG0080
White
BG0003
BG0013
BG0023
BG0036
BG0043
BG0056
BG0063
BG00789
BG008116
More than one race
BG0000
BG0010
BG0020
BG0030
BG0040
BG0050
BG0060
BG0070
BG0080
Unknown or Not Reported
BG0000
BG0010
BG0020
BG0030
BG0040
BG0050
BG0060
BG0070
BG0080
3
BG0043
BG0056
BG0061
BG00731
BG00851
Participants
BG004
3
ParticipantsBG0056
ParticipantsBG0063
ParticipantsBG00789
ParticipantsBG008116
Title
Measurements
BG0001
BG0011
BG0020
BG0033
BG0040
BG0050
BG0062
BG00758
BG00865
8.55
± 0.75
BG0048.37± 1.10
BG0058.42± 0.58
BG0069.53± 0.06
BG0078.79± 1.01
BG0088.81± 0.98
3
OG0043
OG0056
OG0061
OG00731
33.3
(0.84 to 90.6)
OG00433.3(0.84 to 90.6)
OG00550.0(11.8 to 88.2)
OG006100(2.5 to 100)
OG00754.8(36.0 to 72.7)
6
OG0043
OG0056
OG0063
OG00789
5
OG0043
OG0054
OG0062
OG00775
6
OG0043
OG0056
OG0063
OG00789
0
OG0040
OG0050
OG0060
OG0075
6
OG0043
OG0056
OG0063
OG00789
33.3
(4.3 to 77.7)
OG00433.3(0.8 to 90.6)
OG00550.0(11.8 to 88.2)
OG006100(29.2 to 100)
OG00753.9(43.0 to 64.6)
0
OG0042
OG0051
OG0060
OG00717
OG00723.5± 6.8(6.8 to 49.9)
1
OG0040
OG0053
OG0061
OG00718
66.7
(9.4 to 99.2)
OG006100(2.5 to 100)
OG00725.0(8.7 to 49.1)
3
OG0043
OG0056
OG0061
OG00731
2.00
± 0.00
OG0044.00± 1.00
OG0052.83± 0.75
OG0063.00± NANA = SD could not be calculated due to low number of participants with RBC transfusion.
OG0073.06± 1.34
Baseline to Day 113
Title
Measurements
OG000-0.70± 0.71
OG0010.11± 0.69
OG0020.38± 2.08
OG0030.40± 2.12
OG004-0.31± 1.21
OG005-0.36± 1.03
OG006-2.46± NANA = SD could not be calculated due to low number of participants with RBC transfusion.
OG007-0.85± 1.14
6
OG0043
OG0056
OG0061
OG00757
66.7
(22.3 to 95.7)
OG0040(0.0 to 70.8)
OG00533.3(4.3 to 77.7)
OG006100(2.5 to 100)
OG00743.9(30.7 to 57.6)
6
OG0043
OG0056
OG0063
OG00789
7.16
± 5.46
OG0047.23± 20.68
OG0058.47± 31.58
OG0066.65± 8.92
OG0077.77± 21.76
6
OG0043
OG0055
OG0061
OG00762
14.45
± 16.02
OG00413.75± 7.37
OG00514.76± 24.43
OG00626.56± 9.19
OG00714.74± 44.35
6
OG0043
OG0056
OG0063
OG00789
3.76
± 42.29
OG0044.35± 12.87
OG0057.46± 14.55
OG0069.66± 7.52
OG0075.73± 26.93
6
OG0043
OG0056
OG0063
OG00787
51.82
± 35.93
OG00462.46± 25.20
OG005112.56± 17.00
OG006137.56± 1.13
OG00780.02± 27.77
0
OG0040
OG0050
OG0060
OG0070
5
OG0043
OG0055
OG0062
OG00786
10.91
± 11.806
OG0047.46± 11.727
OG005-12.27± 22.144
OG006-4.16± 2.435
OG007-2.40± 9.916
0
OG0040
OG0050
OG0060
OG0070
5
OG0040
OG0055
OG0062
OG00777
36.35
± 21.718
OG005-17.11± 32.861
OG00619.25± 7.793
OG00735.35± 44.228
5
OG0043
OG0055
OG0062
OG00787
1.16
± 23.487
OG00419.38± 25.666
OG0058.56± 41.532
OG006-16.07± 27.172
OG007-1.51± 46.269
0
OG0040
OG0050
OG0060
OG0070
4
OG0043
OG0055
OG0061
OG00764
-15.62
± 20.814
OG004-5.54± 49.144
OG00565.41± 179.249
OG006-64.51± NANA = SD could not be calculated due to low number of participants with hepcidin values
OG00730.15± 143.118
5
OG0042
OG0055
OG0062
OG00784
1.81
± 48.208
OG004749.10± 902.000
OG005146.49± 160.087
OG00629.07± 80.426
OG007238.85± 974.226
5
OG0043
OG0053
OG0062
OG00774
OG00328.29± 28.434
OG00476.16± 54.003
OG0050.45± 19.770
OG00684.06± 108.298
OG00741.37± 73.445
4
OG0041
OG0053
OG0061
OG00764
OG0037.22± 20.592
OG004-5.00± NANA = SD could not be calculated due to low number of participants with direct bilirubin values
OG005-7.41± 12.830
OG0066.78± NANA = SD could not be calculated due to low number of participants with direct bilirubin values
OG007-0.50± 27.541
5
OG0043
OG0055
OG0062
OG00785
4.02
± 10.439
OG004-25.08± 17.240
OG005-25.84± 19.893
OG00610.68± 20.258
OG00713.26± 37.239
5
OG0043
OG0055
OG0062
OG00785
2.76
± 7.848
OG004-15.20± 25.355
OG005-2.52± 18.669
OG006-11.91± 35.318
OG00720.58± 46.589
0
OG0040
OG0050
OG0060
OG0070
5
OG0043
OG0055
OG0061
OG00723
47.48
± 42.431
OG00414.15± 13.191
OG005-6.43± 25.559
OG00615.27± NANA = SD could not be calculated due to low number of participants with BSAP values
OG0078.31± 23.800
5
OG0043
OG0054
OG0061
OG00724
26.37
± 24.669
OG00415.94± 35.018
OG00513.04± 62.513
OG006-70.97± NANA = SD could not be calculated due to low number of participants with CTX values