Study to Evaluate Imetelstat (GRN163L) in Participants Wi... | NCT02598661 | Trialant
NCT02598661
Sponsor
Geron Corporation
Status
Active, not recruiting
Last Update Posted
Jan 5, 2026Actual
Enrollment
289Actual
Phase
Phase 2Phase 3
Conditions
Myelodysplastic Syndromes
Interventions
Imetelstat Sodium
Placebo
Countries
United States
Belgium
Canada
Czechia
France
Germany
Israel
Italy
Netherlands
Poland
Russia
South Korea
Spain
Switzerland
Turkey (Türkiye)
Ukraine
United Kingdom
Protocol Section
Identification Module
NCT ID
NCT02598661
Obsolete or Duplicate NCT IDs
Not provided
Organization Study
CR107947
Secondary IDs
ID
Type
Description
Link
63935937MDS3001
Other Identifier
Geron
EU CTIS number
Other Identifier
2024-511348-25-00
2015-002874-19
EudraCT Number
Brief Title
Study to Evaluate Imetelstat (GRN163L) in Participants With International Prognostic Scoring System (IPSS) Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)
Official Title
A Study to Evaluate Imetelstat (GRN163L) in Transfusion-Dependent Subjects With IPSS Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS) That is Relapsed/Refractory to Erythropoiesis-Stimulating Agent (ESA) Treatment
Acronym
Not provided
Organization
Geron CorporationINDUSTRY
Status Module
Record Verification Date
Sep 2025
Overall Recruitment Status or Expanded Access Status
Active, not recruiting
Last Known Status
Not provided
Delayed Posting
Not provided
Why Stopped
Not provided
Expanded Access Info
No
Start Date
Jan 12, 2016Actual
Primary Completion Date
Oct 13, 2023Actual
Completion Date
Oct 13, 2026Estimated
First Submitted Date
Oct 27, 2015
First Submission Date that Met QC Criteria
Nov 4, 2015
First Posted Date
Nov 6, 2015Estimated
Results Waived
Not provided
Results First Submitted Date
Sep 24, 2024
Results First Submitted that Met QC Criteria
Jan 22, 2025
Results First Posted Date
Feb 14, 2025Actual
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
Dec 12, 2025
Last Update Posted Date
Jan 5, 2026Actual
Sponsor/Collaborators Module
Responsible Party, by Official Title
Sponsor
Lead Sponsor
Geron CorporationINDUSTRY
Collaborators
Not provided
Oversight Module
Has Data Monitoring Committee (DMC)
Yes
Is FDA Regulated Drug
Yes
Is FDA Regulated Device
No
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 efficacy and safety of imetelstat sodium in transfusion-dependent participants with low or intermediate-1 risk myelodysplastic syndrome (MDS) that is relapsed/refractory to erythropoiesis-stimulating agent (ESA) treatment in Phase 2 study and to compare the efficacy, in terms of red blood cell (RBC) transfusion independence (TI), of imetelstat sodium to placebo in transfusion-dependent participants with low or intermediate-1 risk MDS that is relapsed/refractory to ESA treatment in Phase 3 study.
A separate Ventricular Repolarization Substudy (QTc Substudy) will evaluate the effect of imetelstat sodium on ventricular repolarization.
An Extension Phase has been included to allow continued treatment for those participants who are benefitting from imetelstat sodium and to continue to evaluate the long-term safety, overall survival (OS), and disease progression, including progression to acute myeloid leukemia (AML) in transfusion-dependent participants with low or immediate-1 risk MDS that is relapsed/refractory to ESA treatment.
Detailed Description
This is a Phase 2/3, multicenter study of imetelstat sodium in which 289 participants were enrolled.
Phase 2 is an open-label, single-arm design to assess the efficacy and safety of imetelstat sodium. A total of 57 participants were enrolled in Phase 2, including the expansion cohort.
Phase 3 is a double-blind, randomized design to compare the efficacy of imetelstat sodium with placebo. In the Phase 3 study, 178 participants were enrolled and randomized in a 2:1 ratio to receive either imetelstat sodium or placebo, respectively.
In a separate Ventricular Repolarization (VR) Substudy (QTc Substudy), 54 participants were enrolled and randomized 2:1 to receive either imetelstat sodium or placebo. If after a minimum of 2 treatment cycles in the VR substudy, a participant has no significant change to packed red blood cell (pRBC) transfusion burden or evidence of clinical benefit per Investigator, after discussion with the Sponsor the participant may be unblinded. If the participant was on placebo treatment, he/she may be permitted to start treatment with imetelstat sodium.
The Extension Phase was initiated at the end of the Phase 3 study (24 months after the last participant was randomized in the Phase 3) and will continue until participants who entered Phase 3 study participated in the study for up to 5 years from the first dose of imetelstat sodium (including treatment and follow-up), or 3 years of post-treatment follow-up from the last dose of study treatment, whichever occurs later, or until death, withdrawal of consent, study termination, or until a participant is lost to follow-up. Participants ongoing on imetelstat sodium and considered to be benefiting from treatment per Investigator in the Phase 3 Study or Ventricular Repolarization Substudy, have the option to continue receiving imetelstat sodium in the Extension Phase. Participants in the follow-up phase for the Phase 3 study or Ventricular Repolarization Substudy have the option to continue the follow-up in the Extension Phase.
The Phase 2, Phase 3, and VR Substudy all consist of 3 phases: a Screening phase (up to 28 days); a treatment phase; and a post-treatment follow-up phase which will continue until death, lost to follow-up, withdrawal of consent, or the End of the Study (whichever occurs first). The Extension Phase of the study will consist of an extended treatment phase and an extended follow-up phase which will continue until death, lost to follow-up, withdrawal of consent, or the End of the Study (whichever occurs first).
Conditions Module
Conditions
Myelodysplastic Syndromes
Keywords
Myelodysplastic Syndromes
Imetelstat Sodium
GRN163L
Relapsed/refractory to ESAs
Transfusion dependent
IMerge
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 2Phase 3
Interventional Study Design
Allocation
Biospecimen
No data available
No data is available for this block.
Enrollment
289Actual
Arms/Interventions Module
Arm Groups
Label
Type
Description
Intervention Names
Phase 2: Imetelstat Sodium
Experimental
Imetelstat sodium administered intravenously (IV), at a starting dose of 7.5 milligrams per kilogram (mg/kg), every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first. Dose escalation to 9.4 mg/kg was allowed before Protocol Amendment 2.
Drug: Imetelstat Sodium
Phase 3: Imetelstat Sodium
Experimental
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
Drug: Imetelstat Sodium
Phase 3: Placebo
Placebo Comparator
Imetelstat sodium-matching placebo administered IV, every 4 weeks (on a 28-day cycle), until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
Drug: Placebo
QTc Substudy: Imetelstat Sodium
Experimental
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
Drug: Imetelstat Sodium
QTc Substudy: Placebo
Interventions
Name
Type
Description
Arm Group Labels
Other Names
Imetelstat Sodium
Drug
Imetelstat sodium IV infusion.
Extension Phase: Imetelstat Sodium
Phase 2: Imetelstat Sodium
Phase 3: Imetelstat Sodium
Outcomes Module
Primary Outcomes
Measure
Description
Time Frame
Phase 2: Percentage of Participants Without Any Red Blood Cell (RBC) Transfusion During Any Consecutive 8-Weeks Period (All Participants)
Percentage of participants without any RBC transfusion during any consecutive 8 weeks (56 days) starting from Study Day 1 until subsequent anti-cancer therapy if any were reported. Study Day 1 is defined as the day of the first dose for participants enrolled in Phase 2. The 95% confidence interval (CI) was calculated using Clopper-Pearson method. Percentages were rounded off to the nearest single decimal place.
Up to 5 years in Phase 2
Phase 2: Percentage of Participants Without Any RBC Transfusion During Any Consecutive 8-Weeks Period in Target Population
Percentage of participants without any RBC transfusion during any consecutive 8 weeks (56 days) starting from Study Day 1 until subsequent anti-cancer therapy if any were reported. Study Day 1 is defined as the day of the first dose for participants enrolled in Phase 2. The 95% confidence interval (CI) was calculated using Clopper-Pearson method. Percentages were rounded off to the nearest single decimal place.
Up to 5 years in Phase 2
Phase 3: Percentage of Participants Without Any RBC Transfusion During Any Consecutive 8-Weeks Period
Percentage of participants without any RBC transfusion during any consecutive 8 weeks (56 days) starting from Study Day 1 until subsequent anti-cancer therapy if any were reported. Study Day 1 is defined as the day of the first dose for participants enrolled in Phase 2. The 95% CI was calculated using Clopper-Pearson method. Percentages were rounded off to the nearest single decimal place.
Up to 3.7 years in Phase 3
Secondary Outcomes
Measure
Description
Time Frame
Phase 2 and Phase 3: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Deaths
TEAEs were defined as those events that 1) occurred after the first dose of study drug, through the treatment phase, and for 30 days following the last dose of study drug or until subsequent anti-cancer therapy if earlier; 2) any event that was considered study drug-related regardless of the start date of the event; or 3) any event that was presented at baseline but worsened in severity or was subsequently considered drug-related by the investigator. Serious TEAEs are any TEAEs that result in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, is a suspected transmission of any infectious agent via a medicinal product, is medically important. Any clinically significant vital sign measurements, clinical laboratory values, and electrocardiogram (ECG) findings were reported as TEAEs. TEAEs included both serious and non-serious TEAEs.
Other Outcomes
Measure
Description
Time Frame
Extension Phase: Number of Participants With AEs
An adverse event (AE) was any untoward medical occurrence in a clinical study participant administered a medicinal product. An AE does not necessarily have a causal relationship with the treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal finding), symptom, or disease temporally associated with the use of a medicinal product, whether or not related to that medicinal product. AEs included clinically significant vital signs measurements, clinical laboratory values and electrocardiograms (ECGs) changes.
Eligibility Module
Eligibility Criteria
Inclusion Criteria:
Man or woman greater than or equal to (≥) 18 years of age
Diagnosis of myelodysplastic syndrome (MDS) according to World Health Organization (WHO) criteria confirmed by bone marrow aspirate and biopsy within 12 weeks prior to Cycle 1 Day 1 (C1D1) (Phase 2) or randomization (Phase 3). In Ventricular Repolarization Substudy, diagnosis of MDS or myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T) according to WHO criteria confirmed by bone marrow aspirate and biopsy within 12 weeks prior to C1D1
International Prognostic Scoring System (IPSS) low Risk or intermediate-1 risk MDS
Red blood cell (RBC) transfusion dependent, defined as requiring at least 4 RBC units transfused over an 8-week period during the 16 weeks prior to Study Entry; pre-transfusion hemoglobin (Hb) should be less than or equal to (≤) 9.0 gram per deciliter (g/dL) to count towards the 4 units total
Eastern Cooperative Oncology Group (ECOG) performance status 0, 1 or 2
Exclusion Criteria:
Participant has known allergies, hypersensitivity, or intolerance to imetelstat sodium or its excipients
Participant has received an investigational drug or used an invasive investigational medical device within 30 days prior to Study Entry or is currently enrolled in an investigational study
Prior treatment with imetelstat sodium
Have received corticosteroids greater than (>) 30 milligram per day (mg/day) prednisone or equivalent, or growth factor treatment within 4 weeks prior to study entry
Has received an erythropoiesis-stimulating agent (ESA) or any chemotherapy, immunomodulatory, or immunosuppressive therapy within 4 weeks prior to study entry (8 weeks for long-acting ESAs)
Phase 3: a) Prior treatment with a hypomethylating agent (example [eg], azacitidine, decitabine); b) Prior treatment with lenalidomide
Additional Exclusion Criteria for the Ventricular Repolarization Substudy:
Concurrent therapy with medications known to prolong the QT interval and have been associated with Torsade de pointes arrhythmia (TdP)
Cardiac function abnormalities on screening ECG as follows:
Resting heart rate outside of 50 to 100 beats per minute
QT interval by Fridericia's correction method (QTcF) >470 millisecond (msec) (or QTcF >490 msec in the presence of a right bundle branch block or ventricular conduction delay [QRS >119 msec]), determined by central assessment based on the average value of a triplicate set of ECGs
Diagnosed or suspected congenital long QT syndrome
Family history of sudden unexpected death from cardiac-related causes if indicative of a pathogenic mutation of cardiac ion channels
Family history of congenital long QT syndrome
History of Mobitz II second degree or third degree heart block
Implantable pacemaker or automatic implantable cardioverter defibrillator
Complete left bundle branch block
Chronic or persistent atrial arrhythmia including atrial fibrillation and atrial flutter
History or presence of clinically relevant heart rhythm disturbances including atrial, junctional, re-entry, and ventricular tachycardia
Unusual T-wave morphology (i.e., bifid T-wave) likely to interfere with QT measurements
History or evidence for any of the following: severe or unstable angina, myocardial infarction, symptomatic congestive heart failure, arterial or venous thromboembolic events (example, pulmonary embolism, cerebrovascular accident including transient ischemic attacks) within 12 months prior to Cycle 1 Day 1, New York Heart Association (NYHA) Class II to IV heart disease
Presence of uncontrolled hypertension (persistent systolic blood pressure [BP] ≥160 mmHg or diastolic BP ≥100 mmHg). Participants with a history of hypertension are permitted, provided that BP is controlled to within these limits by anti-hypertensive treatment
Any skin condition likely to interfere with electrocardiographic electrode placement or adhesion
History of thoracic surgery likely to cause abnormality of the electrical conduction through thoracic tissues
Sekeres MA, Zeidan AM, Santini V, Komrokji RS, Fenaux P, Savona MR, Madanat YF, Valcarcel D, Regnault A, Creel K, Sun L, Wan Y, Navada S, Berry T, Feller F, Platzbecker U, Diez-Campelo M, Oliva EN. Imetelstat improves patient-reported outcomes and quality of life in lower-risk myelodysplastic syndromes: results from the phase III IMerge study. Haematologica. 2026 Jul 1;111(7):2385-2396. doi: 10.3324/haematol.2025.288956. Epub 2026 Jan 22.
57 participants with MDS received Imetelstat Sodium in Phase 2, followed by Phase 3 where 178 participants were randomized to receive either Imetelstat Sodium or Imetelstat sodium-matching placebo. 54 participants enrolled in QTc Substudy to receive either Imetelstat Sodium or Imetelstat sodium-matching placebo.
Recruitment Details
Participants were enrolled in Phase 2 at 48 study sites and in the Phase 3 Study at 77 study sites. Data is reported for the Phase 2, Phase 3 and QTc substudy up to the primary completion date (PCD) of 13 October 2023. Enrolment in Extension phase started after October 2023 and analyses for the participants in extension phase is still ongoing, hence data for extension phase will be reported at study completion date in 2026.
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
FG000
Phase 2: Imetelstat Sodium
Imetelstat sodium administered intravenously (IV), at a starting dose of 7.5 milligrams per kilogram (mg/kg), every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first. Dose escalation to 9.4 mg/kg was allowed before Protocol Amendment 2.
Periods
Title
Milestones
Reasons Not Completed
Overall Study
Type
Comment
Milestone Data
STARTED
Baseline Characteristics Module
Baseline Analysis Population Description
Outcome Measures Module
Outcome Measures
Adverse Events Module
Frequency Threshold
5
More Info Module
Limitations and Caveats
Not provided
Annotation Section
No data available
No data is available for this block.
Document Section
Large Document Module
Document Has No Statistical Analysis Plan (SAP)
Not provided
Uploaded Document Information
Type
Includes Protocol
Includes SAP
Includes ICF
Document Label
Document Date
Document Uploaded Date
Document File Name
Prot
Yes
No
No
Study Protocol
Dec 13, 2022
Sep 24, 2024
Derived Section
Miscellaneous Info Module
Version Holder
Jul 10, 2026
Removed Countries
Brazil
Mexico
Submission Tracking
No data available
No data is available for this block.
Condition Browse Module
MeSH Terms
Intervention Browse Module
MeSH Terms
Randomized
Intervention Model
Parallel Assignment
Intervention Model Description
Not provided
Primary Purpose
Treatment
Observational Model
Not provided
Time Perspective
Not provided
Masking Info
Masking
Double
Masking Description
Not provided
Who Masked
ParticipantInvestigator
Placebo Comparator
Imetelstat sodium-matching placebo administered IV, every 4 weeks (on a 28-day cycle), until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first. If after a minimum of 2 treatment cycles a participant has no significant change to pRBC transfusion burden or evidence of clinical benefit per Investigator, after discussion with the Sponsor the participant, he/she may be permitted to start treatment with imetelstat sodium.
Drug: Placebo
Extension Phase: Imetelstat Sodium
Experimental
Participants randomized to the imetelstat sodium arm in the Phase 3 and the VR QTc Substudy, based on the response will continue to receive imetelstat sodium IV, at the dose they were receiving in the Phase 3 or VR QTc Substudy, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, study termination, or up to 3 years whichever occurs first.
Drug: Imetelstat Sodium
Extension Phase: Extended Follow-up
Experimental
Participants randomized to the placebo arm in the Phase 3 study will enter the Extended Follow-up part of the Extension Phase and continue in follow up until death, lost to follow-up, withdrawal of consent, study termination, or whichever occurs first up to approximately 3 years.
Drug: Placebo
QTc Substudy: Imetelstat Sodium
GRN163L
Placebo
Drug
Imetelstat sodium-matching placebo IV infusion.
Extension Phase: Extended Follow-up
Phase 3: Placebo
QTc Substudy: Placebo
Adverse events: Up to 5 years in Phase 2 and up to 3.7 years in Phase 3; Deaths: Up to 6.6 years in Phase 2 and up to 4 years in Phase 3
Phase 2 and Phase 3: Percentage of Participants Without Any RBC Transfusion During Any Consecutive 24-Weeks Period
Percentage of participants without any RBC transfusion during any consecutive 24 weeks (168 days) starting from Study Day 1 until subsequent anti-cancer therapy if any were reported. Study Day 1 is defined as the day of the first dose for participants enrolled in Phase 2 and the day of randomization for participants enrolled in Phase 3. The 95% CI was calculated using Clopper-Pearson method. Percentages were rounded off to the nearest single decimal place.
Up to 5 years in Phase 2 and up to 3.7 years in Phase 3
Phase 2 and Phase 3: Time to the 8-Weeks RBC Transfusion Independence (TI)
Time to 8-week RBC TI was defined as the interval from Study Day 1 to the first day of the first 8-week RBC TI period.
Up to 5 years in Phase 2 and up to 3.7 years in Phase 3
Phase 2 and Phase 3: Time to the 24-Weeks RBC TI
Time to 24-week RBC TI was defined as the interval from Study Day 1 to the first day of the first 24-weeks RBC TI period.
Up to 5 years in Phase 2 and up to 3.7 years in Phase 3
Phase 2 and Phase 3: Duration of RBC TI
Duration of RBC TI was defined as the first day of the longest RBC TI period to the date of the first RBC transfusion after the TI period started. The 95% CI was based on Kaplan-Meier product limit estimates.
Up to 5 years in Phase 2 and up to 3.7 years in Phase 3
Phase 2 and Phase 3: Percentage of Participants With Hematologic Improvement Including Erythroid Response (HI-E) as Per International Working Group (IWG) Response Criteria 2006
As per IWG Response Criteria 2006: HI-E was defined as a hemoglobin (Hb) increase by greater than or equal to (≥)1.5 grams per deciliter (g/dL) relevant reduction of units of RBC transfusions by an absolute number of at least 4 RBC transfusion units/8 weeks compared with the pretreatment transfusion number in the previous 8 weeks. Only RBC transfusions given for a Hb of less than or equal to (≤)9 g/dL pretreatment were counted in the RBC transfusion response evaluation. The 95% CI was calculated using Clopper-Pearson method. Percentages were rounded off to the nearest single decimal place.
Up to 5 years in Phase 2 and up to 3.7 years in Phase 3
Phase 2: Percentage of Participants With Complete Remission (CR), Partial Remission (PR), or Marrow Complete Remission (mCR) as Per International Working Group (IWG) Response Criteria 2006 as Assessed by the Investigator
As per the IWG Response Criteria 2006, CR was defined as Bone marrow: ≤5% myeloblasts with normal maturation of all cell lines; Peripheral blood (PB): Hb ≥11 g/dL; platelets ≥100 x 10^9/dL; neutrophils ≥1.0 x 10^9/liter; blasts: 0%. PR was defined as Bone marrow: ≤5% myeloblasts and decrease by ≥50% over pretreatment; cellularity and morphology not relevant. mCR was defined as Bone marrow: ≤5% myeloblasts and decreased by ≥50% over pre-treatment PB. Percentages were rounded off to the nearest single decimal place. All participants in Phase 2 were evaluated by the Investigator for CR/PR/mCR regardless of bone marrow blasts at baseline.
Up to 5 years in Phase 2
Phase 3: Percentage of Participants With CR, PR, or mCR as Per IWG Response Criteria 2006 as Assessed by the Independent Review Committee (IRC)
As per the IWG Response Criteria 2006, CR was defined as Bone marrow: ≤5% myeloblasts with normal maturation of all cell lines; PB: Hb ≥11 g/dL; platelets ≥100 x 10^9/dL; neutrophils ≥1.0 x 10^9/liter; blasts: 0%. PR was defined as Bone marrow: ≤5% myeloblasts and decrease by ≥50% over pretreatment; cellularity and morphology not relevant. mCR was defined as Bone marrow: ≤5% myeloblasts and decreased by ≥50% over pre-treatment PB. CR, PR and mCR were assessed by IRC in Phase 3 and participants were required to fit at least one of the following criteria (participants with >5% baseline blasts per central pathology reviewer assessment; participants with CR, PR, mCR, or cytogenetic response as assessed by the investigator) and have at least one post-baseline assessment.
Up to 3.7 years in Phase 3
Phase 2 and Phase 3: Overall Survival (OS)
OS was defined as the interval from Study Day 1 to death from any cause. Survival time of living participants was censored on the last date a participant is known to be alive or lost to follow-up. The Kaplan-Meier method was used to estimate overall survival.
Up to 6.6 years in Phase 2 and up to 4 years in Phase 3
Phase 2 and Phase 3: Progression Free Survival (PFS)
Progression free survival was defined as the time interval from study Day 1 to the first date of disease progression or death from any cause, whichever occurs first. Disease progression as per IWG criteria was defined as: at least one of the following: at least 50 % decrement from maximum response levels in granulocytes or platelets; reduction in hemoglobin by ≥1.5 g/dL; transfusion dependence.
Up to 6.6 years in Phase 2 and up to 4 years in Phase 3
Phase 2 and Phase 3: Time to Progression to Acute Myeloid Leukemia (AML)
Time to progression to AML was defined as the interval from Study Day 1 to the date of AML diagnosis. Participants who did not progress to AML and were still alive at the cutoff date for the analysis or who withdrew from the study (withdrawal of consent or lost to follow-up), data was censored at the date of the last disease evaluation.
Up to 6.6 years in Phase 2 and up to 4 years in Phase 3
Phase 2 and Phase 3: Amount of RBC Transfusions in the Best 8-week Interval
Amount of RBC transfusions for 8-week interval was defined as the total number of RBC transfusion units in a given 8-week interval during study. The best 8-week interval is a post-baseline 8-week interval where the participant had the fewest post-Study Day 1 RBC transfusion units. A valid 8-week period must start before the date of last dose of study drug + 30 days or end of treatment (EOT) visit whichever occurs first and ends before the first transfusion in post-treatment follow-up or the first day of subsequent anti-cancer therapy whichever occurs first.
Up to 5 years in Phase 2 and up to 3.7 years in Phase 3
Phase 2 and Phase 3: Percent Change in RBC Transfusions Relative to Prior Transfusion Burden
Relative percent change in RBC transfusions per 8-week = (amount of RBC transfusions per 8-week - prior RBC transfusion burden) / prior RBC transfusion burden multiplied by 100%. Prior RBC transfusion burden was defined as the maximum number of RBC units transfused over any consecutive 8 weeks prior to study entry.
Up to 5 years in Phase 2 and up to 3.7 years in Phase 3
Phase 2 and Phase 3: Percentage of Participants Who Received Any Myeloid Growth Factors
Percentage of participants who received any myeloid growth factors starting from Study Day 1 were reported. Percentages were rounded off to the nearest single decimal place.
Up to 5 years in Phase 2 and up to 3.7 years in Phase 3
Phase 2 and Phase 3: Duration of Myeloid Growth Factors Administration
Duration of myeloid growth factor administered starting from Study Day 1 was reported.
Up to 5 years in Phase 2 and up to 3.7 years in Phase 3
Phase 2 and Phase 3: Maximum Observed Plasma Concentration (Cmax)
As pre-specified in the statistical analysis plan (SAP), participants in the imetelstat sodium arm group of Phase 2 and Phase 3 were pooled for pharmacokinetic (PK) data collection and analyses in this outcome measure. PK parameters were determined by population PK model.
Pre-dose on Cycle 1 Day 1 and pre-dose on Day 1 of every 3 cycles from Cycle 4 up to Cycle 66 in Phase 2 and Cycle 34 in Phase 3 (each cycle length= 28 days)
Phase 2 and Phase 3: Area Under the Drug Concentration-Plasma Time Curve From Time Zero to Time 28 Days (AUC0-28d)
As pre-specified in the statistical analysis plan (SAP), participants in the imetelstat sodium arm group of Phase 2 and Phase 3 were pooled for PK data collection and analyses in this outcome measure. PK parameters were determined by population PK model.
Cycle 1 (Days 1 to 28) (Cycle duration= 28 days)
Phase 2 and Phase 3: Percentage of Participants With Anti-drug Antibodies (ADA) to Imetelstat Sodium
As pre-specified in the SAP, participants in the imetelstat sodium arm group of Phase 2 and Phase 3 were pooled for immunogenicity data collection and analyses in this outcome measure. Percentages were rounded off to the nearest single decimal place.
Pre-dose on Cycle 1 Day 1 and pre-dose on Day 1 of every 3 cycles from Cycle 4 up to Cycle 66 in Phase 2 and Cycle 34 in Phase 3 (each cycle length= 28 days)
Phase 3: Medical Resource Utilization Assessed Based on Percentage of Participants With Outpatient Medical Encounters
Outpatient medical encounters included various sites of care: a) emergency room (ER) visits, b) hospital outpatient visits, c) home care visit, d) visit to lab, e) visit to doctor's office, f) other visits. Percentages were rounded off to the nearest single decimal place.
Up to 3.7 years in Phase 3
Phase 3: Medical Resource Utilization Assessed Based on Duration of Hospitalization
Hospitalization included any medical encounter defined as hospice, hospital inpatient department, and intensive care unit (ICU). Hospitalizations without end dates were not counted in the calculation of length of stay. If any participant had multiple readmissions, duration was calculated as the sum of all hospital stays.
Up to 3.7 years in Phase 3
QTc Substudy: Change From Baseline in QT Interval by Fridericia's Correction Method (ΔQTcF)
Baseline was defined as the mean of the measured ECG intervals collected at 3 time points (-1 hour, -0.5 hour, and 0 hour) prior to treatment administration on Cycle 1 Day 1 (Cycle length= 28 days)
Baseline, Cycle 1, Day 1: 0.5, 1, 2, 4, 6, and 8 hours post-dose (cycle length= 28 days)
Up to approximately 3 years in the extension phase
Extension Phase: Overall Survival
Up to approximately 3 years in the extension phase
Progression free survival will be assessed as the time interval from the end of the Phase 3 study until death, withdrawal of consent, study termination, or until a subject is lost to follow-up. As per IWG criteria disease progression is defined as: at least one of the following: at least 50 percent (%) decrement from maximum response levels in granulocytes or platelets; reduction in hemoglobin by greater than or equal to ≥1.5 g/dL; transfusion dependence.
Up to approximately 3 years in the extension phase
Tucson
Arizona
85715
United States
CBCC Global Research, Inc.
Bakersfield
California
93309
United States
UCLA Ronald Regan Medical Center
Los Angeles
California
90095
United States
Yale-New Haven Hospital (YNHH) - Smilow Cancer Hospital
New Haven
Connecticut
06510-3220
United States
BRCR Medical Center
Plantation
Florida
33326
United States
University of South Florida (USF) - H. Lee Moffitt Cancer Center
Tampa
Florida
33612
United States
Franciscan Health
Indianapolis
Indiana
46237-8601
United States
St. Agnes Healthcare, Inc
Baltimore
Maryland
21229
United States
Center for Cancer and Blood Disorders
Bethesda
Maryland
20817
United States
Washington University School of Medicine
St Louis
Missouri
63110
United States
University of New Mexico Cancer Center
Albuquerque
New Mexico
87131
United States
Icahn School of Medicine at Mount Sinai Program for the Protection of Human Subjects
New York
New York
10029
United States
Columbia University Medical Center
New York
New York
10032
United States
Weill Cornell Medical College-New York Presbyterian Hospital
New York
New York
10065
United States
Cleveland Clinic Taussig Cancer
Cleveland
Ohio
44195
United States
The Ohio State Comprehensive Cancer Center
Columbus
Ohio
43210
United States
Prairie lakes Healthcare system, Inc
Watertown
South Dakota
57201
United States
Vanderbilt University Medical - Hematology-Oncology
Nashville
Tennessee
37232-6307
United States
Texas Oncology/Methodist Charlton Cancer Center
Dallas
Texas
75237
United States
Simmons Comprehensive Cancer Center
Dallas
Texas
75390
United States
Fred Hutchinson Cancer Research Center (FHCRC)
Seattle
Washington
98109-1024
United States
ZAS Middelheim
Antwerp
Antwerpen
2020
Belgium
ZAS Cadix
Antwerp
Antwerpen
2030
Belgium
GZA Ziekenhuizen - Campus Sint
Wilrijk
Antwerpen
2610
Belgium
AZ Sint-Jan Burgge-Oostende
Bruges
West-Vlaanderen
8000
Belgium
Az Groeninge
Kortrijk
West-Vlaanderen
8500
Belgium
AZ Klina
Brasschaat
2930
Belgium
Universitair Ziekenhuis Gent
Ghent
9000
Belgium
UZ Leuven - Campus Gasthuisberg
Leuven
3000
Belgium
Tom Baker Cancer Centre
Calgary
Alberta
T2N 4N2
Canada
University of Alberta Hospital - Hematology Research
Edmonton
Alberta
T6G 2R3
Canada
The Ottawa Hospital
Ottawa
Ontario
K1G 8L6
Canada
Sunnybrook Health Sciences Centre
Toronto
Ontario
M4N 3M5
Canada
Princess Margaret Hospital
Toronto
Ontario
M5G 2L7
Canada
Jewish General Hospital
Montreal
Quebec
H3T 1E2
Canada
Fakultni nemocnice Brno
Brno
Brno-město
625 00
Czechia
FN Hradec Kralove
Hradec Králové
Hradec Králové
500 05
Czechia
FN Kralovske Vinohrady
Prague
100 34
Czechia
Vseobecna fakultni nemocnice v Praze
Prague
128 08
Czechia
Hopital de l'Archet
Nice
Alpes-Maritimes
6202
France
CHU Tours
Tours
Centre-Val de Loire
37044
France
CHU de Limoges, Hopital Dupuytren
Limoges
Haute-Vienne
87042
France
CHU de Grenoble - Hôpital Albe
La Tronche
Isère
38700
France
CHRU Nancy Brabois
Vandœuvre-lès-Nancy
Meurthe-et-Moselle
54511
France
CH Le Mans - HAEMATOLOGY
Le Mans
Sarthe
72037
France
CHU de Poitiers
Poitiers
Vienne
86021
France
Centre Hospitalier Universitai
Angers
49100
France
CHRU de Lille - Hopital Claude Huriez - Maladies du Sang
Lille
59037
France
CHU - Hôpital Saint Louis - H
Paris
Île-de-France Region
75010
France
University Hospital Freiburg
Freiburg im Breisgau
Baden-Wurttemberg
79106
Germany
Fachärztliche Gemeinschaftspraxis mit Schwerpunkt
Dresden
Saxony
1307
Germany
University Hospital Leipzig
Leipzig
Saxony
4107
Germany
Studienzentrum für Hämatologie, Onkologie,Diabetologie, Endoskopie und Fußambulanz
Aschaffenburg
63739
Germany
University Hospital Bonn
Bonn
53127
Germany
Universitatsklinikum Carl Gustav Carcus Dresden
Dresden
01307
Germany
Universitätsklinikum Düsseldorf
Düsseldorf
40225
Germany
Johannes Gutenberg Universität
Mainz
55131
Germany
The Edith Wolfson Medical Center
H̱olon
Central District
58100
Israel
Meir Medical Center
Kfar Saba
Central District
44281
Israel
Kaplan Medical Center
Rehovot
Hagalil Saint
7610001
Israel
Hadassah Medical Organization
Jerusalem
Jerusalem
9112001
Israel
Ha'Emek Medical Center
Afula
Northern District
1834111
Israel
Tel Aviv Sourasky Medical Center
Tel Aviv
Tel Aviv
49372
Israel
The Chaim Sheba Medical Center
Tel Litwinsky
Tel Aviv
5265601
Israel
Carmel MC
Haifa
3436212
Israel
Rabin Medical Center, Beilinson Hospital
Petah Tikva
4941492
Israel
A.O. Ospedale Niguarda Ca' Granda
Milan
Lombardy
20162
Italy
Istituto Clinico Humanitas Rozzano, IRCCS
Rozzano
Milano
20089
Italy
Irccs Crob
Rionero in Vulture
Potenza
85028
Italy
A.O. Universitaria Policlinico Tor Vergata
Roma
Roma
00133
Italy
AOU Ospedali Riuniti Umberto I G.M. Lancisi G. Salesi
Ancona
60020
Italy
AOU di Bologna Policlinico S. Orsola Malpighi
Bologna
40138
Italy
Azienda Ospedaliera Universitaria Careggi di Firenze
Florence
50134
Italy
Grande Ospedale Metropolitano 'Bianchi-Melacrino-Morelli' Reggio Calabria
Reggio Calabria
89124
Italy
AO S. Andrea, Università degli Studi di Roma La Sapienza
Roma
189
Italy
Ospedale di Circolo, PO Varese
Varese
21100
Italy
Radboud Umcn
Nijmegen
Gelderland
6525 GA
Netherlands
Meander Medisch Centrum
Amersfoort
3813 TZ
Netherlands
VU Medisch Centrum
Amsterdam
1081 HV
Netherlands
Universitair Medisch Centrum Groningen
Groningen
9713 GZ
Netherlands
Uniwersytecki Szpital Kliniczny im. J. Mikulicza-Radeckiego
Wroclaw
Lower Silesian Voivodeship
50-367
Poland
Wojewódzki Szpital Specjalistyczny sp.z o.o.
Słupsk
Pomeranian Voivodeship
76-200
Poland
PRATIA Poznań
Poznan
Ul. Gryfińska 1
60-192
Poland
SPZOZ MSWiA z Warminsko - Mazurskim Centrum Onkologii
Olsztyn
Warmian-Masurian Voivodeship
10-228
Poland
Ars Medical sp. z o.o.
Piła
Wielkopolskie Województwo
64-920
Poland
Clinics of Samarskiy GMU
Samara
Volga
443079
Russia
Emergency Hospital of Dzerzhinsk
Dzerzhinsk
606019
Russia
City Clinical Hospital
Moscow
129301
Russia
Nizhniy Novgorod Region Clinical Hospital
Nizhny Novgorod
603126
Russia
Ryazan Regional Clinical Hospital
Ryazan
390039
Russia
FGU-Russian Research Institut
Saint Petersburg
191024
Russia
Oncologic Dispensary No.2
Sochi
354057
Russia
Pusan National University Hospital - Hematology and Oncology
Seogu
Incheon
42941
South Korea
Gachon University Gil Medical Center - oncology
Incheon
Incheon Gwang'Yeogsi
21565
South Korea
Chonnam National University Hwasun Hospital
Hwasun
Jeollanam-do
58128
South Korea
Seoul National University Hospital
Seoul
Seoul Teugbyeolsi
03080
South Korea
Asan Medical Center
Seoul
05505
South Korea
Samsung Medical Center
Seoul
06351
South Korea
The Catholic University of Korea Seoul St. Mary's Hospital
Seoul
06591
South Korea
Severance Hospital, Yonsei Uni
Seoul
3722
South Korea
H.U.Pta.del Mar
Cadiz
Cádiz
11009
Spain
Hospital Universitario Puerta de Hierro Majadahonda
Instytut patolohii krovi ta transfusiynoi medytsyny NAMN Ukr
Lviv
Lviv Oblast
79044
Ukraine
The Leeds Teaching Hospitals NHS Trust
Leeds
Leeds
LS9 7TF
United Kingdom
Nottingham City Hospital - Clinical Haematology
Nottingham
Nottinghamshire
NG5 1PB
United Kingdom
Southampton University Hospital
Southampton
Southampton
SO16 6YD
United Kingdom
Aberdeen Royal Infirmary
Aberdeen
AB252ZL
United Kingdom
Derived
Kim N, Pulte ED, Ehrlich LA, Ionan AC, Haupert S, Vallejo J, Green F, Zheng N, Wang Y, Liu J, Blanco JG, Dorff SE, Booth B, Choe M, Gehrke B, Bhatnagar V, Theoret M, Pazdur R, De Claro RA, Norsworthy KJ. US Food and Drug Administration Approval Summary: Imetelstat for Selected Patients With Low- to Intermediate-1 Risk Myelodysplastic Syndromes With Transfusion-Dependent Anemia. J Clin Oncol. 2025 Dec 10;43(35):3760-3768. doi: 10.1200/JCO-25-01369. Epub 2025 Oct 24.
Platzbecker U, Santini V, Fenaux P, Sekeres MA, Savona MR, Madanat YF, Diez-Campelo M, Valcarcel D, Illmer T, Jonasova A, Belohlavkova P, Sherman LJ, Berry T, Dougherty S, Shah S, Xia Q, Sun L, Wan Y, Huang F, Ikin A, Navada S, Feller F, Komrokji RS, Zeidan AM. Imetelstat in patients with lower-risk myelodysplastic syndromes who have relapsed or are refractory to erythropoiesis-stimulating agents (IMerge): a multinational, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet. 2024 Jan 20;403(10423):249-260. doi: 10.1016/S0140-6736(23)01724-5. Epub 2023 Dec 1.
Steensma DP, Fenaux P, Van Eygen K, Raza A, Santini V, Germing U, Font P, Diez-Campelo M, Thepot S, Vellenga E, Patnaik MM, Jang JH, Varsos H, Bussolari J, Rose E, Sherman L, Sun L, Wan Y, Dougherty S, Huang F, Feller F, Rizo A, Platzbecker U. Imetelstat Achieves Meaningful and Durable Transfusion Independence in High Transfusion-Burden Patients With Lower-Risk Myelodysplastic Syndromes in a Phase II Study. J Clin Oncol. 2021 Jan 1;39(1):48-56. doi: 10.1200/JCO.20.01895. Epub 2020 Oct 27.
FG001
Phase 3: Imetelstat Sodium
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
FG002
Phase 3: Placebo
Imetelstat sodium-matching placebo administered IV, every 4 weeks (on a 28-day cycle), until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
FG003
QTc Substudy: Imetelstat Sodium
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
FG004
QTc Substudy: Placebo
Imetelstat sodium-matching placebo administered IV, every 4 weeks (on a 28-day cycle), until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first. If after a minimum of 2 treatment cycles a participant has no significant change to pRBC transfusion burden or evidence of clinical benefit per Investigator, after discussion with the Sponsor the participant, he/she may be permitted to start treatment with imetelstat sodium.
FG00057 subjects
FG001118 subjects
FG00260 subjects
FG00335 subjects
FG00419 subjects
Crossover to Imetelstat
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG0030 subjects
FG00414 subjects
Target Population
The Target Population included the participants without prior hypomethylating agent (HMA) or lenalidomide use and non del(5q) in karyotype at baseline.
FG00038 subjects
FG0010 subjects
FG0020 subjects
FG0030 subjects
FG0040 subjects
COMPLETED
FG00015 subjects
FG00157 subjects
FG00229 subjects
FG00333 subjects
FG00417 subjects
NOT COMPLETED
FG00042 subjects
FG00161 subjects
FG00231 subjects
FG0032 subjects
FG0042 subjects
Type
Comment
Reasons
Death
FG00027 subjects
FG00134 subjects
FG00214 subjects
FG0031 subjects
FG0040 subjects
Lost to Follow-up
FG0002 subjects
FG0013 subjects
FG0022 subjects
FG0030 subjects
FG004
Withdrawal by Subject
FG00013 subjects
FG00124 subjects
FG00214 subjects
FG0031 subjects
FG004
Adverse Event
FG0000 subjects
FG0010 subjects
FG0021 subjects
FG0030 subjects
FG004
Randomized, Never Treated
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG0030 subjects
FG004
Phase 2: All Treated Analysis Set included all participants who received at least one dose of study drug. Phase 3: Intent-to-Treat (ITT) Analysis Set included all participants randomized into the phase 3 study. The QTc Substudy Analysis Set included all participants who participated in QTc Substudy and received at least one dose of study drug.
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
BG000
Phase 2: Imetelstat Sodium
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first. Dose escalation to 9.4 mg/kg was allowed before Protocol Amendment 2.
BG001
Phase 3: Imetelstat Sodium
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
BG002
Phase 3: Placebo
Imetelstat sodium-matching placebo administered IV, every 4 weeks (on a 28-day cycle), until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
BG003
QTc Substudy: Imetelstat Sodium
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
BG004
QTc Substudy: Placebo
Imetelstat sodium-matching placebo administered IV, every 4 weeks (on a 28-day cycle), until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first. If after a minimum of 2 treatment cycles a participant has no significant change to pRBC transfusion burden or evidence of clinical benefit per Investigator, after discussion with the Sponsor the participant, he/she may be permitted to start treatment with imetelstat sodium.
BG005
Total
Total of all reporting groups
Denominators
Units
Counts
Participants
BG00057
BG001118
BG00260
BG00335
BG00418
BG005288
Baseline Measures
Title
Description
Population Description
Parameter Type
Dispersion Type
Unit of Measure
Calculate Percentage
Denominator Units Selected
Denominators
Classes
Age, Continuous
Mean
Full Range
years
Title
Denominators
Categories
Title
Measurements
BG00070.1(46 to 83)
BG00170.4(44 to 87)
BG00271.7(39 to 85)
BG003
Sex: Female, Male
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
Female
BG00025
BG00147
BG002
Ethnicity (NIH/OMB)
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
Hispanic or Latino
BG0001
BG0016
BG002
Race (NIH/OMB)
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
American Indian or Alaska Native
BG0000
BG0010
BG002
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
Phase 2: Percentage of Participants Without Any Red Blood Cell (RBC) Transfusion During Any Consecutive 8-Weeks Period (All Participants)
Percentage of participants without any RBC transfusion during any consecutive 8 weeks (56 days) starting from Study Day 1 until subsequent anti-cancer therapy if any were reported. Study Day 1 is defined as the day of the first dose for participants enrolled in Phase 2. The 95% confidence interval (CI) was calculated using Clopper-Pearson method. Percentages were rounded off to the nearest single decimal place.
Phase 2: All Treated Analysis Set included all participants who received at least one dose of study drug.
Posted
Number
95% Confidence Interval
percentage of participants
Up to 5 years in Phase 2
ID
Title
Description
OG000
Phase 2: Imetelstat Sodium
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first. Dose escalation to 9.4 mg/kg was allowed before Protocol Amendment 2.
Units
Counts
Participants
OG00057
Title
Denominators
Categories
Title
Measurements
OG00036.8(24.45 to 50.66)
Primary
Phase 2: Percentage of Participants Without Any RBC Transfusion During Any Consecutive 8-Weeks Period in Target Population
Percentage of participants without any RBC transfusion during any consecutive 8 weeks (56 days) starting from Study Day 1 until subsequent anti-cancer therapy if any were reported. Study Day 1 is defined as the day of the first dose for participants enrolled in Phase 2. The 95% confidence interval (CI) was calculated using Clopper-Pearson method. Percentages were rounded off to the nearest single decimal place.
Phase 2: Target Population included participants without prior hypomethylating agent (HMA) or lenalidomide use and non del(5q) in karyotype at baseline.
Posted
Number
95% Confidence Interval
percentage of participants
Up to 5 years in Phase 2
ID
Title
Description
OG000
Phase 2: Imetelstat Sodium
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first. Dose escalation to 9.4 mg/kg was allowed before Protocol Amendment 2.
Units
Counts
Participants
OG000
Primary
Phase 3: Percentage of Participants Without Any RBC Transfusion During Any Consecutive 8-Weeks Period
Percentage of participants without any RBC transfusion during any consecutive 8 weeks (56 days) starting from Study Day 1 until subsequent anti-cancer therapy if any were reported. Study Day 1 is defined as the day of the first dose for participants enrolled in Phase 2. The 95% CI was calculated using Clopper-Pearson method. Percentages were rounded off to the nearest single decimal place.
Phase 3: The ITT Analysis Set included all participants randomized into the Phase 3 study.
Posted
Number
95% Confidence Interval
percentage of participants
Up to 3.7 years in Phase 3
ID
Title
Description
OG000
Phase 3: Imetelstat Sodium
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
OG001
Phase 3: Placebo
Imetelstat sodium-matching placebo administered IV, every 4 weeks (on a 28-day cycle), until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
Units
Counts
Secondary
Phase 2 and Phase 3: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Deaths
TEAEs were defined as those events that 1) occurred after the first dose of study drug, through the treatment phase, and for 30 days following the last dose of study drug or until subsequent anti-cancer therapy if earlier; 2) any event that was considered study drug-related regardless of the start date of the event; or 3) any event that was presented at baseline but worsened in severity or was subsequently considered drug-related by the investigator. Serious TEAEs are any TEAEs that result in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, is a suspected transmission of any infectious agent via a medicinal product, is medically important. Any clinically significant vital sign measurements, clinical laboratory values, and electrocardiogram (ECG) findings were reported as TEAEs. TEAEs included both serious and non-serious TEAEs.
Phase 2 and Phase 3: Safety Analysis Set included all participants who received at least one dose of study drug.
Posted
Count of Participants
Participants
Adverse events: Up to 5 years in Phase 2 and up to 3.7 years in Phase 3; Deaths: Up to 6.6 years in Phase 2 and up to 4 years in Phase 3
ID
Title
Description
OG000
Phase 2: Imetelstat Sodium (No Dose Escalation)
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
Secondary
Phase 2 and Phase 3: Percentage of Participants Without Any RBC Transfusion During Any Consecutive 24-Weeks Period
Percentage of participants without any RBC transfusion during any consecutive 24 weeks (168 days) starting from Study Day 1 until subsequent anti-cancer therapy if any were reported. Study Day 1 is defined as the day of the first dose for participants enrolled in Phase 2 and the day of randomization for participants enrolled in Phase 3. The 95% CI was calculated using Clopper-Pearson method. Percentages were rounded off to the nearest single decimal place.
Phase 2: All Treated Analysis Set included all participants who received at least one dose of study drug. Phase 3: ITT Analysis Set included all participants randomized into the Phase 3 study.
Posted
Number
95% Confidence Interval
percentage of participants
Up to 5 years in Phase 2 and up to 3.7 years in Phase 3
ID
Title
Description
OG000
Phase 2: Imetelstat Sodium
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first. Dose escalation to 9.4 mg/kg was allowed before Protocol Amendment 2.
OG001
Phase 3: Imetelstat Sodium
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
Secondary
Phase 2 and Phase 3: Time to the 8-Weeks RBC Transfusion Independence (TI)
Time to 8-week RBC TI was defined as the interval from Study Day 1 to the first day of the first 8-week RBC TI period.
Phase 2: All Treated 8-week TI Responder Analysis Set included participants in the All Treated Analysis Set who achieved 8-week RBC TI.
Phase 3: ITT 8-week TI Responder Analysis Set included all participants in the ITT Analysis Set who achieved 8-week RBC TI.
Posted
Median
Full Range
weeks
Up to 5 years in Phase 2 and up to 3.7 years in Phase 3
ID
Title
Description
OG000
Phase 2: Imetelstat Sodium
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first. Dose escalation to 9.4 mg/kg was allowed before Protocol Amendment 2.
OG001
Phase 3: Imetelstat Sodium
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
OG002
Phase 3: Placebo
Imetelstat sodium-matching placebo administered IV, every 4 weeks (on a 28-day cycle), until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
Secondary
Phase 2 and Phase 3: Time to the 24-Weeks RBC TI
Time to 24-week RBC TI was defined as the interval from Study Day 1 to the first day of the first 24-weeks RBC TI period.
Phase 2: All Treated 24-week TI Responder Analysis Set included participants in the All Treated Analysis Set who achieved 24-week RBC TI.
Phase 3: ITT 24-week TI Responder Analysis Set included all participants in the ITT Analysis Set who achieved 24-week RBC TI.
Posted
Median
Full Range
weeks
Up to 5 years in Phase 2 and up to 3.7 years in Phase 3
ID
Title
Description
OG000
Phase 2: Imetelstat Sodium
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first. Dose escalation to 9.4 mg/kg was allowed before Protocol Amendment 2.
OG001
Phase 3: Imetelstat Sodium
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
OG002
Phase 3: Placebo
Imetelstat sodium-matching placebo administered IV, every 4 weeks (on a 28-day cycle), until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
Secondary
Phase 2 and Phase 3: Duration of RBC TI
Duration of RBC TI was defined as the first day of the longest RBC TI period to the date of the first RBC transfusion after the TI period started. The 95% CI was based on Kaplan-Meier product limit estimates.
Phase 2: All Treated 8-week TI Responder Analysis Set included participants in the All Treated Analysis Set who achieved 8-week RBC TI.
Phase 3: ITT 8-week TI Responder Analysis Set included all participants in the ITT Analysis Set who achieved 8-week RBC TI.
Posted
Median
95% Confidence Interval
weeks
Up to 5 years in Phase 2 and up to 3.7 years in Phase 3
ID
Title
Description
OG000
Phase 2: Imetelstat Sodium
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first. Dose escalation to 9.4 mg/kg was allowed before Protocol Amendment 2.
OG001
Phase 3: Imetelstat Sodium
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
OG002
Phase 3: Placebo
Secondary
Phase 2 and Phase 3: Percentage of Participants With Hematologic Improvement Including Erythroid Response (HI-E) as Per International Working Group (IWG) Response Criteria 2006
As per IWG Response Criteria 2006: HI-E was defined as a hemoglobin (Hb) increase by greater than or equal to (≥)1.5 grams per deciliter (g/dL) relevant reduction of units of RBC transfusions by an absolute number of at least 4 RBC transfusion units/8 weeks compared with the pretreatment transfusion number in the previous 8 weeks. Only RBC transfusions given for a Hb of less than or equal to (≤)9 g/dL pretreatment were counted in the RBC transfusion response evaluation. The 95% CI was calculated using Clopper-Pearson method. Percentages were rounded off to the nearest single decimal place.
Phase 2: All Treated Analysis Set included all participants who received at least one dose of study drug.
Phase 3: ITT Analysis Set included all participants randomized into the Phase 3 study.
Posted
Number
95% Confidence Interval
percentage of participants
Up to 5 years in Phase 2 and up to 3.7 years in Phase 3
ID
Title
Description
OG000
Phase 2: Imetelstat Sodium
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first. Dose escalation to 9.4 mg/kg was allowed before Protocol Amendment 2.
OG001
Phase 3: Imetelstat Sodium
Secondary
Phase 2: Percentage of Participants With Complete Remission (CR), Partial Remission (PR), or Marrow Complete Remission (mCR) as Per International Working Group (IWG) Response Criteria 2006 as Assessed by the Investigator
As per the IWG Response Criteria 2006, CR was defined as Bone marrow: ≤5% myeloblasts with normal maturation of all cell lines; Peripheral blood (PB): Hb ≥11 g/dL; platelets ≥100 x 10^9/dL; neutrophils ≥1.0 x 10^9/liter; blasts: 0%. PR was defined as Bone marrow: ≤5% myeloblasts and decrease by ≥50% over pretreatment; cellularity and morphology not relevant. mCR was defined as Bone marrow: ≤5% myeloblasts and decreased by ≥50% over pre-treatment PB. Percentages were rounded off to the nearest single decimal place. All participants in Phase 2 were evaluated by the Investigator for CR/PR/mCR regardless of bone marrow blasts at baseline.
Phase 2: All Treated Analysis Set included all participants who received at least one dose of study drug.
Posted
Number
percentage of participants
Up to 5 years in Phase 2
ID
Title
Description
OG000
Phase 2: Imetelstat Sodium
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first. Dose escalation to 9.4 mg/kg was allowed before Protocol Amendment 2.
Units
Counts
Secondary
Phase 3: Percentage of Participants With CR, PR, or mCR as Per IWG Response Criteria 2006 as Assessed by the Independent Review Committee (IRC)
As per the IWG Response Criteria 2006, CR was defined as Bone marrow: ≤5% myeloblasts with normal maturation of all cell lines; PB: Hb ≥11 g/dL; platelets ≥100 x 10^9/dL; neutrophils ≥1.0 x 10^9/liter; blasts: 0%. PR was defined as Bone marrow: ≤5% myeloblasts and decrease by ≥50% over pretreatment; cellularity and morphology not relevant. mCR was defined as Bone marrow: ≤5% myeloblasts and decreased by ≥50% over pre-treatment PB. CR, PR and mCR were assessed by IRC in Phase 3 and participants were required to fit at least one of the following criteria (participants with >5% baseline blasts per central pathology reviewer assessment; participants with CR, PR, mCR, or cytogenetic response as assessed by the investigator) and have at least one post-baseline assessment.
Phase 3: ITT Analysis Set included all participants randomized into the Phase 3 study. Overall number of participants analyzed is the number of participants with >5% baseline bone marrow aspirate blasts per central pathology reviewer's assessment.
Posted
Number
percentage of participants
Up to 3.7 years in Phase 3
ID
Title
Description
OG000
Phase 3: Imetelstat Sodium
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
OG001
Phase 3: Placebo
Secondary
Phase 2 and Phase 3: Overall Survival (OS)
OS was defined as the interval from Study Day 1 to death from any cause. Survival time of living participants was censored on the last date a participant is known to be alive or lost to follow-up. The Kaplan-Meier method was used to estimate overall survival.
Phase 2: All Treated Analysis Set included all participants who received at least one dose of study drug.
Phase 3: ITT Analysis Set included all participants randomized into the Phase 3 study.
Posted
Median
95% Confidence Interval
months
Up to 6.6 years in Phase 2 and up to 4 years in Phase 3
ID
Title
Description
OG000
Phase 2: Imetelstat Sodium
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first. Dose escalation to 9.4 mg/kg was allowed before Protocol Amendment 2.
OG001
Phase 3: Imetelstat Sodium
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
OG002
Phase 3: Placebo
Imetelstat sodium-matching placebo administered IV, every 4 weeks (on a 28-day cycle), until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
Secondary
Phase 2 and Phase 3: Progression Free Survival (PFS)
Progression free survival was defined as the time interval from study Day 1 to the first date of disease progression or death from any cause, whichever occurs first. Disease progression as per IWG criteria was defined as: at least one of the following: at least 50 % decrement from maximum response levels in granulocytes or platelets; reduction in hemoglobin by ≥1.5 g/dL; transfusion dependence.
Phase 2: All Treated Analysis Set included all participants who received at least one dose of study drug.
Phase 3: ITT Analysis Set included all participants randomized into the Phase 3 study.
Posted
Median
95% Confidence Interval
months
Up to 6.6 years in Phase 2 and up to 4 years in Phase 3
ID
Title
Description
OG000
Phase 2: Imetelstat Sodium
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first. Dose escalation to 9.4 mg/kg was allowed before Protocol Amendment 2.
OG001
Phase 3: Imetelstat Sodium
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
OG002
Secondary
Phase 2 and Phase 3: Time to Progression to Acute Myeloid Leukemia (AML)
Time to progression to AML was defined as the interval from Study Day 1 to the date of AML diagnosis. Participants who did not progress to AML and were still alive at the cutoff date for the analysis or who withdrew from the study (withdrawal of consent or lost to follow-up), data was censored at the date of the last disease evaluation.
Phase 2: All Treated Analysis Set included all participants who received at least one dose of study drug.
Phase 3: ITT Analysis Set included all participants randomized into the Phase 3 study.
Posted
Median
95% Confidence Interval
months
Up to 6.6 years in Phase 2 and up to 4 years in Phase 3
ID
Title
Description
OG000
Phase 2: Imetelstat Sodium
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first. Dose escalation to 9.4 mg/kg was allowed before Protocol Amendment 2.
OG001
Phase 3: Imetelstat Sodium
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
OG002
Phase 3: Placebo
Secondary
Phase 2 and Phase 3: Amount of RBC Transfusions in the Best 8-week Interval
Amount of RBC transfusions for 8-week interval was defined as the total number of RBC transfusion units in a given 8-week interval during study. The best 8-week interval is a post-baseline 8-week interval where the participant had the fewest post-Study Day 1 RBC transfusion units. A valid 8-week period must start before the date of last dose of study drug + 30 days or end of treatment (EOT) visit whichever occurs first and ends before the first transfusion in post-treatment follow-up or the first day of subsequent anti-cancer therapy whichever occurs first.
Phase 2: All Treated Analysis Set included all participants who received at least one dose of study drug.
Phase 3: ITT Analysis Set included all participants randomized into the Phase 3 study.
Posted
Mean
Standard Deviation
number of RBC transfusions units
Up to 5 years in Phase 2 and up to 3.7 years in Phase 3
ID
Title
Description
OG000
Phase 2: Imetelstat Sodium
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first. Dose escalation to 9.4 mg/kg was allowed before Protocol Amendment 2.
OG001
Phase 3: Imetelstat Sodium
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
Secondary
Phase 2 and Phase 3: Percent Change in RBC Transfusions Relative to Prior Transfusion Burden
Relative percent change in RBC transfusions per 8-week = (amount of RBC transfusions per 8-week - prior RBC transfusion burden) / prior RBC transfusion burden multiplied by 100%. Prior RBC transfusion burden was defined as the maximum number of RBC units transfused over any consecutive 8 weeks prior to study entry.
Phase 2: All Treated Analysis Set included all participants who received at least one dose of study drug.
Phase 3: ITT Analysis Set included all participants randomized into the Phase 3 study.
Posted
Mean
Standard Deviation
percent change
Up to 5 years in Phase 2 and up to 3.7 years in Phase 3
ID
Title
Description
OG000
Phase 2: Imetelstat Sodium
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first. Dose escalation to 9.4 mg/kg was allowed before Protocol Amendment 2.
OG001
Phase 3: Imetelstat Sodium
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
OG002
Phase 3: Placebo
Secondary
Phase 2 and Phase 3: Percentage of Participants Who Received Any Myeloid Growth Factors
Percentage of participants who received any myeloid growth factors starting from Study Day 1 were reported. Percentages were rounded off to the nearest single decimal place.
Phase 2: All Treated Analysis Set included all participants who received at least one dose of study drug.
Phase 3: ITT Analysis Set included all participants randomized into the Phase 3 study.
Posted
Number
percentage of participants
Up to 5 years in Phase 2 and up to 3.7 years in Phase 3
ID
Title
Description
OG000
Phase 2: Imetelstat Sodium
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first. Dose escalation to 9.4 mg/kg was allowed before Protocol Amendment 2.
OG001
Phase 3: Imetelstat Sodium
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
OG002
Phase 3: Placebo
Imetelstat sodium-matching placebo administered IV, every 4 weeks (on a 28-day cycle), until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
Secondary
Phase 2 and Phase 3: Duration of Myeloid Growth Factors Administration
Duration of myeloid growth factor administered starting from Study Day 1 was reported.
Phase 2: All Treated Analysis Set included all participants who received at least one dose of study drug.
Phase 3: ITT Analysis Set included all participants randomized into the Phase 3 study. Overall number of participants analyzed is the number of participants who had at least 1 dose of myeloid growth factors in Phase 2 and Phase 3.
Posted
Median
Full Range
weeks
Up to 5 years in Phase 2 and up to 3.7 years in Phase 3
ID
Title
Description
OG000
Phase 2: Imetelstat Sodium
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first. Dose escalation to 9.4 mg/kg was allowed before Protocol Amendment 2.
OG001
Phase 3: Imetelstat Sodium
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
OG002
Phase 3: Placebo
Imetelstat sodium-matching placebo administered IV, every 4 weeks (on a 28-day cycle), until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
Secondary
Phase 2 and Phase 3: Maximum Observed Plasma Concentration (Cmax)
As pre-specified in the statistical analysis plan (SAP), participants in the imetelstat sodium arm group of Phase 2 and Phase 3 were pooled for pharmacokinetic (PK) data collection and analyses in this outcome measure. PK parameters were determined by population PK model.
PK Parameter Analysis Set included all densely, and sparsely sampled participants who had received at least 1 dose of imetelstat sodium and who had sufficient data to calculate PK parameters for plasma imetelstat sodium. Overall number of participants analyzed signifies those participants who were evaluable for this outcome measure.
Posted
Mean
Standard Deviation
micrograms per milliliter (mcg/mL)
Pre-dose on Cycle 1 Day 1 and pre-dose on Day 1 of every 3 cycles from Cycle 4 up to Cycle 66 in Phase 2 and Cycle 34 in Phase 3 (each cycle length= 28 days)
ID
Title
Description
OG000
Phase 2 and Phase 3: Imetelstat Sodium
For Phase 2: Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first. Dose escalation to 9.4 mg/kg was allowed before Protocol Amendment 2.
For Phase 3: Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
Secondary
Phase 2 and Phase 3: Area Under the Drug Concentration-Plasma Time Curve From Time Zero to Time 28 Days (AUC0-28d)
As pre-specified in the statistical analysis plan (SAP), participants in the imetelstat sodium arm group of Phase 2 and Phase 3 were pooled for PK data collection and analyses in this outcome measure. PK parameters were determined by population PK model.
PK Parameter Analysis Set included all densely, and sparsely sampled participants who had received at least 1 dose of imetelstat sodium and who had sufficient data to calculate PK parameters for plasma imetelstat sodium. Overall number of participants analyzed signifies those participants who were evaluable for this outcome measure.
Posted
Mean
Standard Deviation
hour*micrograms per milliliter(h*mcg/mL)
Cycle 1 (Days 1 to 28) (Cycle duration= 28 days)
ID
Title
Description
OG000
Phase 2 and Phase 3: Imetelstat Sodium
For Phase 2: Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first. Dose escalation to 9.4 mg/kg was allowed before Protocol Amendment 2.
For Phase 3: Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
Units
Counts
Secondary
Phase 2 and Phase 3: Percentage of Participants With Anti-drug Antibodies (ADA) to Imetelstat Sodium
As pre-specified in the SAP, participants in the imetelstat sodium arm group of Phase 2 and Phase 3 were pooled for immunogenicity data collection and analyses in this outcome measure. Percentages were rounded off to the nearest single decimal place.
The Pharmacodynamic (PD) Analysis Set included all participants who had at least one quantifiable post-dose determination on biomarker, efficacy or safety parameters as defined in the related Pharmacodynamics analysis plan. Overall number of participants analysed signifies those participants who were evaluable for this outcome measure.
Posted
Number
percentage of participants
Pre-dose on Cycle 1 Day 1 and pre-dose on Day 1 of every 3 cycles from Cycle 4 up to Cycle 66 in Phase 2 and Cycle 34 in Phase 3 (each cycle length= 28 days)
ID
Title
Description
OG000
Phase 2 and Phase 3: Imetelstat Sodium
For Phase 2: Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first. Dose escalation to 9.4 mg/kg was allowed before Protocol Amendment 2.
For Phase 3: Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
Secondary
Phase 3: Medical Resource Utilization Assessed Based on Percentage of Participants With Outpatient Medical Encounters
Outpatient medical encounters included various sites of care: a) emergency room (ER) visits, b) hospital outpatient visits, c) home care visit, d) visit to lab, e) visit to doctor's office, f) other visits. Percentages were rounded off to the nearest single decimal place.
Phase 3: ITT Analysis Set included all participants randomized into the Phase 3 study.
Posted
Number
percentage of participants
Up to 3.7 years in Phase 3
ID
Title
Description
OG000
Phase 3: Imetelstat Sodium
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
OG001
Phase 3: Placebo
Imetelstat sodium-matching placebo administered IV, every 4 weeks (on a 28-day cycle), until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
Units
Counts
Participants
Secondary
Phase 3: Medical Resource Utilization Assessed Based on Duration of Hospitalization
Hospitalization included any medical encounter defined as hospice, hospital inpatient department, and intensive care unit (ICU). Hospitalizations without end dates were not counted in the calculation of length of stay. If any participant had multiple readmissions, duration was calculated as the sum of all hospital stays.
Phase 3: ITT Analysis Set included all participants randomized into the Phase 3 study. Overall number of participants analyzed is the number of participants who were hospitalized.
Posted
Median
Full Range
days
Up to 3.7 years in Phase 3
ID
Title
Description
OG000
Phase 3: Imetelstat Sodium
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
OG001
Phase 3: Placebo
Imetelstat sodium-matching placebo administered IV, every 4 weeks (on a 28-day cycle), until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
Units
Counts
Secondary
QTc Substudy: Change From Baseline in QT Interval by Fridericia's Correction Method (ΔQTcF)
Baseline was defined as the mean of the measured ECG intervals collected at 3 time points (-1 hour, -0.5 hour, and 0 hour) prior to treatment administration on Cycle 1 Day 1 (Cycle length= 28 days)
The QTc substudy analysis set included all participants who participated in QTc substudy and received at least one dose of study drug, with measurements at baseline as well as on-treatment with at least 1 post-dose time point with a ΔQTcF value. By-time Point Analysis Set included each post-baseline time point that had a non-missing change-from-baseline observation. Number analysed is the number of participants with data available for analysis at specified timepoints.
Posted
Least Squares Mean
Standard Error
milliseconds (ms)
Baseline, Cycle 1, Day 1: 0.5, 1, 2, 4, 6, and 8 hours post-dose (cycle length= 28 days)
ID
Title
Description
OG000
QTc substudy: Imetelstat Sodium
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
OG001
QTc substudy: Placebo
Imetelstat sodium-matching placebo administered IV, every 4 weeks (on a 28-day cycle), until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first. If after a minimum of 2 treatment cycles a participant has no significant change to pRBC transfusion burden or evidence of clinical benefit per Investigator, after discussion with the Sponsor the participant, he/she may be permitted to start treatment with imetelstat sodium.
Other Pre-specified
Extension Phase: Number of Participants With AEs
An adverse event (AE) was any untoward medical occurrence in a clinical study participant administered a medicinal product. An AE does not necessarily have a causal relationship with the treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal finding), symptom, or disease temporally associated with the use of a medicinal product, whether or not related to that medicinal product. AEs included clinically significant vital signs measurements, clinical laboratory values and electrocardiograms (ECGs) changes.
Not Posted
Oct 2027
Up to approximately 3 years in the extension phase
Participants
Other Pre-specified
Extension Phase: Overall Survival
Not Posted
Oct 2027
Up to approximately 3 years in the extension phase
Progression free survival will be assessed as the time interval from the end of the Phase 3 study until death, withdrawal of consent, study termination, or until a subject is lost to follow-up. As per IWG criteria disease progression is defined as: at least one of the following: at least 50 percent (%) decrement from maximum response levels in granulocytes or platelets; reduction in hemoglobin by greater than or equal to ≥1.5 g/dL; transfusion dependence.
Not Posted
Oct 2027
Up to approximately 3 years in the extension phase
Participants
Time Frame
Adverse events: Up to 5 years in Phase 2; up to 3.7 years in Phase 3; up to 1.3 years in QTc substudy; All-cause mortality: Up to 6.6 years in Phase 2; up to 4 years in Phase 3; up to 1.6 years in QTc substudy
Description
Safety Analysis Set=all participants who received at least 1 dose of study drug. Safety data for Phase 2 arm has been reported separately for the participants without dose escalation and with dose escalation from 7.5 mg/kg to 9.4 mg/kg. QTc Substudy: Crossover arm includes AEs data after crossover from placebo to Imetelstat. Placebo arm included AE data for participants randomized to placebo prior to crossover.
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
Phase 2: Imetelstat Sodium (No Dose Escalation)
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
24
50
26
50
50
50
EG001
Phase 2: Imetelstat Sodium (Escalated to 9.4 mg/kg)
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) with dose escalation to 9.4 mg/kg allowed before Protocol Amendment 2, until death, lost to follow up, withdrawal of consent, or study termination, whichever occurs first.
3
7
1
7
6
7
EG002
Phase 3: Imetelstat Sodium
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
35
118
41
118
117
118
EG003
Phase 3: Placebo
Imetelstat sodium-matching placebo administered IV, every 4 weeks (on a 28-day cycle), until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
15
59
13
59
58
59
EG004
QTc Substudy: Imetelstat Sodium
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
1
35
8
35
33
35
EG005
QTc Substudy: Crossover Imetelstat Sodium
After a minimum of 2 treatment cycles (28-day cycle) if a participant had no significant change to pRBC transfusion burden or evidence of clinical benefit per Investigator, after discussion with the Sponsor the participant was crossed over from placebo to receive imetelstat sodium 7.5 mg/kg.
0
14
3
14
12
14
EG006
QTc Substudy: Placebo
Imetelstat sodium matching placebo administered IV, every 4 weeks (on a 28-day cycle), until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first. If after a minimum of 2 treatment cycles a participant has no significant change to pRBC transfusion burden or evidence of clinical benefit per Investigator, after discussion with the Sponsor the participant, he/she may be permitted to start treatment with imetelstat sodium.
0
18
0
18
13
18
Serious Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Pneumonia
Infections and infestations
MedDRA (25.0)
Systematic Assessment
EG0002 affected50 at risk
EG0010 affected7 at risk
EG0023 affected118 at risk
EG0031 affected59 at risk
EG0041 affected35 at risk
EG0050 affected14 at risk
EG0060 affected18 at risk
Atypical pneumonia
Infections and infestations
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Bronchitis
Infections and infestations
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
COVID-19 pneumonia
Infections and infestations
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0022 affected118 at risk
EG003
Cellulitis
Infections and infestations
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Cellulitis staphylococcal
Infections and infestations
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Corynebacterium infection
Infections and infestations
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Dermo-hypodermitis
Infections and infestations
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Disseminated tuberculosis
Infections and infestations
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Escherichia bacteraemia
Infections and infestations
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Escherichia urinary tract infection
Infections and infestations
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Sepsis
Infections and infestations
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0022 affected118 at risk
EG003
Urinary tract infection
Infections and infestations
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0022 affected118 at risk
EG003
Urosepsis
Infections and infestations
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Clostridium difficile infection
Infections and infestations
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0021 affected118 at risk
EG003
Enterococcal sepsis
Infections and infestations
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0021 affected118 at risk
EG003
Erysipelas
Infections and infestations
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0021 affected118 at risk
EG003
Escherichia sepsis
Infections and infestations
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0021 affected118 at risk
EG003
Gastroenteritis clostridial
Infections and infestations
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0021 affected118 at risk
EG003
Infection
Infections and infestations
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0021 affected118 at risk
EG003
Neutropenic sepsis
Infections and infestations
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0021 affected118 at risk
EG003
Pneumonia bacterial
Infections and infestations
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0021 affected118 at risk
EG003
Pseudomembranous colitis
Infections and infestations
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0021 affected118 at risk
EG003
Abscess limb
Infections and infestations
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Arthritis bacterial
Infections and infestations
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Gastroenteritis
Infections and infestations
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Listeriosis
Infections and infestations
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Diarrhoea
Gastrointestinal disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Gastric haemorrhage
Gastrointestinal disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Gastrointestinal pain
Gastrointestinal disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Inguinal hernia
Gastrointestinal disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Intestinal haemorrhage
Gastrointestinal disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Oesophageal varices haemorrhage
Gastrointestinal disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0021 affected118 at risk
EG003
Rectal haemorrhage
Gastrointestinal disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0011 affected7 at risk
EG0020 affected118 at risk
EG003
Thrombosis mesenteric vessel
Gastrointestinal disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Faecaloma
Gastrointestinal disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0021 affected118 at risk
EG003
Gastrointestinal disorder
Gastrointestinal disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0021 affected118 at risk
EG003
Gastrointestinal haemorrhage
Gastrointestinal disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0021 affected118 at risk
EG003
Diverticular perforation
Gastrointestinal disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Small intestinal haemorrhage
Gastrointestinal disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Femur fracture
Injury, poisoning and procedural complications
MedDRA (25.0)
Systematic Assessment
EG0003 affected50 at risk
EG0010 affected7 at risk
EG0022 affected118 at risk
EG003
Brain contusion
Injury, poisoning and procedural complications
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Extradural haematoma
Injury, poisoning and procedural complications
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Hip fracture
Injury, poisoning and procedural complications
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0021 affected118 at risk
EG003
Spinal compression fracture
Injury, poisoning and procedural complications
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Toxicity to various agents
Injury, poisoning and procedural complications
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Humerus fracture
Injury, poisoning and procedural complications
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0021 affected118 at risk
EG003
Joint dislocation
Injury, poisoning and procedural complications
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0021 affected118 at risk
EG003
Thrombocytopenia
Blood and lymphatic system disorders
MedDRA (25.0)
Systematic Assessment
EG0002 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Anaemia
Blood and lymphatic system disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0023 affected118 at risk
EG003
Cytopenia
Blood and lymphatic system disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Febrile neutropenia
Blood and lymphatic system disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0021 affected118 at risk
EG003
Neutropenia
Blood and lymphatic system disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Cerebrovascular accident
Nervous system disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Headache
Nervous system disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Neurodegenerative disorder
Nervous system disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Sciatica
Nervous system disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Syncope
Nervous system disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0022 affected118 at risk
EG003
Cardiac failure
Cardiac disorders
MedDRA (25.0)
Systematic Assessment
EG0002 affected50 at risk
EG0010 affected7 at risk
EG0023 affected118 at risk
EG003
Cardiac failure congestive
Cardiac disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0021 affected118 at risk
EG003
Atrial fibrillation
Cardiac disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0021 affected118 at risk
EG003
Angina pectoris
Cardiac disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0021 affected118 at risk
EG003
Myocardial infarction
Cardiac disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0021 affected118 at risk
EG003
Aortic valve incompetence
Cardiac disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Atrial flutter
Cardiac disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Cholangiocarcinoma
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Hepatic cancer
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Hepatocellular carcinoma
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Renal cell carcinoma
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Transitional cell carcinoma
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Bladder papilloma
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0021 affected118 at risk
EG003
Lung neoplasm malignant
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0021 affected118 at risk
EG003
Haematoma
Vascular disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Hypertension
Vascular disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Hypertensive emergency
Vascular disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Peripheral ischaemia
Vascular disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Deep vein thrombosis
Vascular disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0021 affected118 at risk
EG003
Hypertensive crisis
Vascular disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0021 affected118 at risk
EG003
Aortic stenosis
Vascular disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Extremity necrosis
Vascular disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Back pain
Musculoskeletal and connective tissue disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Osteoarthritis
Musculoskeletal and connective tissue disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Arthralgia
Musculoskeletal and connective tissue disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Joint effusion
Musculoskeletal and connective tissue disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Biliary colic
Hepatobiliary disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Cholecystitis
Hepatobiliary disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Hyperglycaemia
Metabolism and nutrition disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Haemochromatosis
Metabolism and nutrition disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Prostatitis
Reproductive system and breast disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Benign prostatic hyperplasia
Reproductive system and breast disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0021 affected118 at risk
EG003
Pleural effusion
Respiratory, thoracic and mediastinal disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Dyspnoea
Respiratory, thoracic and mediastinal disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0021 affected118 at risk
EG003
Pulmonary oedema
Respiratory, thoracic and mediastinal disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0021 affected118 at risk
EG003
Pyrexia
General disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0022 affected118 at risk
EG003
Asthenia
General disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0021 affected118 at risk
EG003
Influenza like illness
General disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0021 affected118 at risk
EG003
Haematuria
Renal and urinary disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0021 affected118 at risk
EG003
Kidney congestion
Renal and urinary disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0021 affected118 at risk
EG003
Nephrolithiasis
Renal and urinary disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0021 affected118 at risk
EG003
Device occlusion
Product Issues
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0021 affected118 at risk
EG003
Diabetic foot
Skin and subcutaneous tissue disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0021 affected118 at risk
EG003
Autoimmune haemolytic anaemia
Blood and lymphatic system disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Hypochromic anaemia
Blood and lymphatic system disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Joint abscess
Infections and infestations
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Staphylococcal bacteraemia
Infections and infestations
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Staphylococcal sepsis
Infections and infestations
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Lung infiltration
Respiratory, thoracic and mediastinal disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Pulmonary hypertension
Respiratory, thoracic and mediastinal disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Cerebral infarction
Nervous system disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0021 affected118 at risk
EG003
Subdural haematoma
Injury, poisoning and procedural complications
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0021 affected118 at risk
EG003
Other Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Neutropenia
Blood and lymphatic system disorders
MedDRA (25.0)
Systematic Assessment
EG00036 affected50 at risk
EG0012 affected7 at risk
EG00289 affected118 at risk
EG0035 affected59 at risk
EG00426 affected35 at risk
EG0058 affected14 at risk
EG0062 affected18 at risk
Thrombocytopenia
Blood and lymphatic system disorders
MedDRA (25.0)
Systematic Assessment
EG00033 affected50 at risk
EG0012 affected7 at risk
EG00289 affected118 at risk
EG003
Anaemia
Blood and lymphatic system disorders
MedDRA (25.0)
Systematic Assessment
EG00012 affected50 at risk
EG0011 affected7 at risk
EG00223 affected118 at risk
EG003
Leukopenia
Blood and lymphatic system disorders
MedDRA (25.0)
Systematic Assessment
EG00011 affected50 at risk
EG0010 affected7 at risk
EG00212 affected118 at risk
EG003
Pyrexia
General disorders
MedDRA (25.0)
Systematic Assessment
EG00010 affected50 at risk
EG0011 affected7 at risk
EG00210 affected118 at risk
EG003
Oedema peripheral
General disorders
MedDRA (25.0)
Systematic Assessment
EG0007 affected50 at risk
EG0011 affected7 at risk
EG00215 affected118 at risk
EG003
Fatigue
General disorders
MedDRA (25.0)
Systematic Assessment
EG0005 affected50 at risk
EG0012 affected7 at risk
EG0029 affected118 at risk
EG003
Asthenia
General disorders
MedDRA (25.0)
Systematic Assessment
EG0004 affected50 at risk
EG0012 affected7 at risk
EG00221 affected118 at risk
EG003
Influenza like illness
General disorders
MedDRA (25.0)
Systematic Assessment
EG0002 affected50 at risk
EG0012 affected7 at risk
EG0020 affected118 at risk
EG003
Non-cardiac chest pain
General disorders
MedDRA (25.0)
Systematic Assessment
EG0003 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Urinary tract infection
Infections and infestations
MedDRA (25.0)
Systematic Assessment
EG00010 affected50 at risk
EG0010 affected7 at risk
EG0027 affected118 at risk
EG003
Nasopharyngitis
Infections and infestations
MedDRA (25.0)
Systematic Assessment
EG0006 affected50 at risk
EG0011 affected7 at risk
EG0020 affected118 at risk
EG003
Bronchitis
Infections and infestations
MedDRA (25.0)
Systematic Assessment
EG0006 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Cellulitis
Infections and infestations
MedDRA (25.0)
Systematic Assessment
EG0003 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Upper respiratory tract infection
Infections and infestations
MedDRA (25.0)
Systematic Assessment
EG0003 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
COVID-19
Infections and infestations
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG00219 affected118 at risk
EG003
Chest pain
General disorders
MedDRA (25.0)
Systematic Assessment
EG0002 affected50 at risk
EG0010 affected7 at risk
EG0022 affected118 at risk
EG003
Back pain
Musculoskeletal and connective tissue disorders
MedDRA (25.0)
Systematic Assessment
EG00010 affected50 at risk
EG0010 affected7 at risk
EG00211 affected118 at risk
EG003
Muscle spasms
Musculoskeletal and connective tissue disorders
MedDRA (25.0)
Systematic Assessment
EG0005 affected50 at risk
EG0011 affected7 at risk
EG0020 affected118 at risk
EG003
Muscular weakness
Musculoskeletal and connective tissue disorders
MedDRA (25.0)
Systematic Assessment
EG0002 affected50 at risk
EG0011 affected7 at risk
EG0020 affected118 at risk
EG003
Arthralgia
Musculoskeletal and connective tissue disorders
MedDRA (25.0)
Systematic Assessment
EG0002 affected50 at risk
EG0010 affected7 at risk
EG00210 affected118 at risk
EG003
Myalgia
Musculoskeletal and connective tissue disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0011 affected7 at risk
EG0026 affected118 at risk
EG003
Pain in extremity
Musculoskeletal and connective tissue disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0011 affected7 at risk
EG0021 affected118 at risk
EG003
Diarrhoea
Gastrointestinal disorders
MedDRA (25.0)
Systematic Assessment
EG0007 affected50 at risk
EG0012 affected7 at risk
EG00214 affected118 at risk
EG003
Constipation
Gastrointestinal disorders
MedDRA (25.0)
Systematic Assessment
EG0008 affected50 at risk
EG0010 affected7 at risk
EG00210 affected118 at risk
EG003
Nausea
Gastrointestinal disorders
MedDRA (25.0)
Systematic Assessment
EG0007 affected50 at risk
EG0010 affected7 at risk
EG0029 affected118 at risk
EG003
Vomiting
Gastrointestinal disorders
MedDRA (25.0)
Systematic Assessment
EG0006 affected50 at risk
EG0011 affected7 at risk
EG0020 affected118 at risk
EG003
Abdominal pain
Gastrointestinal disorders
MedDRA (25.0)
Systematic Assessment
EG0005 affected50 at risk
EG0011 affected7 at risk
EG0028 affected118 at risk
EG003
Abdominal pain upper
Gastrointestinal disorders
MedDRA (25.0)
Systematic Assessment
EG0003 affected50 at risk
EG0012 affected7 at risk
EG0022 affected118 at risk
EG003
Gastritis
Gastrointestinal disorders
MedDRA (25.0)
Systematic Assessment
EG0002 affected50 at risk
EG0011 affected7 at risk
EG0020 affected118 at risk
EG003
Alanine aminotransferase increased
Investigations
MedDRA (25.0)
Systematic Assessment
EG0007 affected50 at risk
EG0013 affected7 at risk
EG00213 affected118 at risk
EG003
Aspartate aminotransferase increased
Investigations
MedDRA (25.0)
Systematic Assessment
EG0006 affected50 at risk
EG0012 affected7 at risk
EG00211 affected118 at risk
EG003
Blood creatinine increased
Investigations
MedDRA (25.0)
Systematic Assessment
EG0004 affected50 at risk
EG0010 affected7 at risk
EG0022 affected118 at risk
EG003
Headache
Nervous system disorders
MedDRA (25.0)
Systematic Assessment
EG00011 affected50 at risk
EG0012 affected7 at risk
EG00215 affected118 at risk
EG003
Dizziness
Nervous system disorders
MedDRA (25.0)
Systematic Assessment
EG0004 affected50 at risk
EG0011 affected7 at risk
EG0027 affected118 at risk
EG003
Decreased appetite
Metabolism and nutrition disorders
MedDRA (25.0)
Systematic Assessment
EG0004 affected50 at risk
EG0011 affected7 at risk
EG0020 affected118 at risk
EG003
Hypokalaemia
Metabolism and nutrition disorders
MedDRA (25.0)
Systematic Assessment
EG0003 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Hyponatraemia
Metabolism and nutrition disorders
MedDRA (25.0)
Systematic Assessment
EG0003 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Hyperferritinaemia
Metabolism and nutrition disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0026 affected118 at risk
EG003
Hyperkalaemia
Metabolism and nutrition disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0021 affected118 at risk
EG003
Iron overload
Metabolism and nutrition disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0011 affected7 at risk
EG0021 affected118 at risk
EG003
Cough
Respiratory, thoracic and mediastinal disorders
MedDRA (25.0)
Systematic Assessment
EG0006 affected50 at risk
EG0010 affected7 at risk
EG0022 affected118 at risk
EG003
Dyspnoea
Respiratory, thoracic and mediastinal disorders
MedDRA (25.0)
Systematic Assessment
EG0005 affected50 at risk
EG0011 affected7 at risk
EG00211 affected118 at risk
EG003
Epistaxis
Respiratory, thoracic and mediastinal disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0011 affected7 at risk
EG0029 affected118 at risk
EG003
Hypertension
Vascular disorders
MedDRA (25.0)
Systematic Assessment
EG0005 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Haematoma
Vascular disorders
MedDRA (25.0)
Systematic Assessment
EG0003 affected50 at risk
EG0011 affected7 at risk
EG0027 affected118 at risk
EG003
Hypotension
Vascular disorders
MedDRA (25.0)
Systematic Assessment
EG0003 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Haematuria
Renal and urinary disorders
MedDRA (25.0)
Systematic Assessment
EG0005 affected50 at risk
EG0011 affected7 at risk
EG0020 affected118 at risk
EG003
Contusion
Injury, poisoning and procedural complications
MedDRA (25.0)
Systematic Assessment
EG0003 affected50 at risk
EG0010 affected7 at risk
EG0021 affected118 at risk
EG003
Fall
Injury, poisoning and procedural complications
MedDRA (25.0)
Systematic Assessment
EG0003 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Hyperbilirubinaemia
Hepatobiliary disorders
MedDRA (25.0)
Systematic Assessment
EG0005 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Hepatomegaly
Hepatobiliary disorders
MedDRA (25.0)
Systematic Assessment
EG0002 affected50 at risk
EG0011 affected7 at risk
EG0020 affected118 at risk
EG003
Atrial fibrillation
Cardiac disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0026 affected118 at risk
EG003
Pruritus
Skin and subcutaneous tissue disorders
MedDRA (25.0)
Systematic Assessment
EG0002 affected50 at risk
EG0010 affected7 at risk
EG0027 affected118 at risk
EG003
Lymphopenia
Blood and lymphatic system disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Dry mouth
Gastrointestinal disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Gastrointestinal pain
Gastrointestinal disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Gastrooesophageal reflux disease
Gastrointestinal disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Mouth ulceration
Gastrointestinal disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Hyperglycaemia
Metabolism and nutrition disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Gout
Metabolism and nutrition disorders
MedDRA (25.0)
Systematic Assessment
EG0002 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Gait disturbance
General disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Infusion site discomfort
General disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Pain
General disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Peripheral swelling
General disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Gamma-glutamyltransferase increased
Investigations
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Blood creatine phosphokinase increased
Investigations
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Serum ferritin increased
Investigations
MedDRA (25.0)
Systematic Assessment
EG0002 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Troponin T increased
Investigations
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Limb discomfort
Musculoskeletal and connective tissue disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Gastroenteritis
Infections and infestations
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Pneumonia
Infections and infestations
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0011 affected7 at risk
EG0020 affected118 at risk
EG003
Burning sensation
Nervous system disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Hypoaesthesia
Nervous system disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Taste disorder
Nervous system disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Dyspnoea exertional
Respiratory, thoracic and mediastinal disorders
MedDRA (25.0)
Systematic Assessment
EG0002 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Rash
Skin and subcutaneous tissue disorders
MedDRA (25.0)
Systematic Assessment
EG0002 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Skin ulcer
Skin and subcutaneous tissue disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Hyperhidrosis
Skin and subcutaneous tissue disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Purpura
Skin and subcutaneous tissue disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Rash maculo-papular
Skin and subcutaneous tissue disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Urticaria
Skin and subcutaneous tissue disorders
MedDRA (25.0)
Systematic Assessment
EG0002 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Hypertransaminasaemia
Hepatobiliary disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Palpitations
Cardiac disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Sinus tachycardia
Cardiac disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Vision blurred
Eye disorders
MedDRA (25.0)
Systematic Assessment
EG0002 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Flushing
Vascular disorders
MedDRA (25.0)
Systematic Assessment
EG0002 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Hot flush
Vascular disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Peripheral artery stenosis
Vascular disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Acute kidney injury
Renal and urinary disorders
MedDRA (25.0)
Systematic Assessment
EG0002 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Pollakiuria
Renal and urinary disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Animal bite
Injury, poisoning and procedural complications
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Skin abrasion
Injury, poisoning and procedural complications
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Skin laceration
Injury, poisoning and procedural complications
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Confusional state
Psychiatric disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Basal cell carcinoma
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Dyspepsia
Gastrointestinal disorders
MedDRA (25.0)
Systematic Assessment
EG0002 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Stomatitis
Gastrointestinal disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Abdominal discomfort
Gastrointestinal disorders
MedDRA (25.0)
Systematic Assessment
EG0002 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Chills
General disorders
MedDRA (25.0)
Systematic Assessment
EG0002 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Presbyacusis
Ear and labyrinth disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Ear congestion
Ear and labyrinth disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Sleep disorder
Psychiatric disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Melanoderma
Skin and subcutaneous tissue disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Nasal congestion
Respiratory, thoracic and mediastinal disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Wound infection
Infections and infestations
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Tooth infection
Infections and infestations
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Eye infection
Infections and infestations
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Weight decreased
Investigations
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Cardiac murmur
Investigations
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Osteonecrosis
Musculoskeletal and connective tissue disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0010 affected7 at risk
EG0020 affected118 at risk
EG003
Dry skin
Skin and subcutaneous tissue disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0011 affected7 at risk
EG0020 affected118 at risk
EG003
Dysuria
Renal and urinary disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0011 affected7 at risk
EG0020 affected118 at risk
EG003
Viral upper respiratory tract infection
Infections and infestations
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0011 affected7 at risk
EG0020 affected118 at risk
EG003
Musculoskeletal pain
Musculoskeletal and connective tissue disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0011 affected7 at risk
EG0020 affected118 at risk
EG003
Flank pain
Musculoskeletal and connective tissue disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0011 affected7 at risk
EG0020 affected118 at risk
EG003
Groin pain
Musculoskeletal and connective tissue disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0011 affected7 at risk
EG0020 affected118 at risk
EG003
Muscle contracture
Musculoskeletal and connective tissue disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0011 affected7 at risk
EG0020 affected118 at risk
EG003
Haematochezia
Gastrointestinal disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0011 affected7 at risk
EG0020 affected118 at risk
EG003
Haemorrhoids
Gastrointestinal disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0011 affected7 at risk
EG0020 affected118 at risk
EG003
Food poisoning
Gastrointestinal disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0011 affected7 at risk
EG0020 affected118 at risk
EG003
Gastric antral vascular ectasia
Gastrointestinal disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0011 affected7 at risk
EG0020 affected118 at risk
EG003
Gastric polyps
Gastrointestinal disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0011 affected7 at risk
EG0020 affected118 at risk
EG003
Gastrointestinal angiectasia
Gastrointestinal disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0011 affected7 at risk
EG0020 affected118 at risk
EG003
Rectal haemorrhage
Gastrointestinal disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0011 affected7 at risk
EG0020 affected118 at risk
EG003
Somnolence
Nervous system disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0011 affected7 at risk
EG0020 affected118 at risk
EG003
Thrombosis
Vascular disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0011 affected7 at risk
EG0020 affected118 at risk
EG003
Vasculitis
Vascular disorders
MedDRA (25.0)
Systematic Assessment
EG0000 affected50 at risk
EG0011 affected7 at risk
EG0020 affected118 at risk
EG003
Neck Pain
Musculoskeletal and connective tissue disorders
MedDRA (25.0)
Systematic Assessment
EG0001 affected50 at risk
EG0011 affected7 at risk
EG0020 affected118 at risk
EG003
Certain Agreements
Are all PI(s) employees of the sponsor?
No
Restriction Type
OTHER
Results Disclosure Restriction on PI(s)?
Yes
Other Details
Consistent with Good Publication Practices and ICMJE guidelines, the sponsor shall have right to publish such primary (multicenter) data and information without approval from investigator. Investigator has right to publish study site-specific data after primary data published. If an investigator wishes to publish information from study, a copy of manuscript must be provided to sponsor for review at least 60 days before submission for publication or presentation.
The p-value was calculated based on Cochran-Mantel-Haenszel (CMH) controlling for prior RBC transfusion burden (≤6 versus[vs.]>6 units RBC) & international prognostic scoring system (IPSS) risk group (low vs. intermediate-1) applied to randomization.
Percentage Difference
24.8
2-Sided
95
9.90
36.89
The 95% CI was calculated based on the Wilson Score method.
Superiority
OG001
Phase 2: Imetelstat Sodium (Escalated to 9.4 mg/kg)
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) with dose escalation to 9.4 mg/kg allowed before Protocol Amendment 2, until death, lost to follow up, withdrawal of consent, or study termination, whichever occurs first.
OG002
Phase 3: Imetelstat Sodium
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
OG003
Phase 3: Placebo
Imetelstat sodium-matching placebo administered IV, every 4 weeks (on a 28-day cycle), until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
Units
Counts
Participants
OG00050
OG0017
OG002118
OG00359
Title
Denominators
Categories
Participants with AEs
Title
Measurements
OG00050
OG0016
OG002117
OG00359
Participants who died
Title
Measurements
OG00024
OG0013
OG00235
OG003
OG002
Phase 3: Placebo
Imetelstat sodium-matching placebo administered IV, every 4 weeks (on a 28-day cycle), until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
Units
Counts
Participants
OG00057
OG001118
OG00260
Title
Denominators
Categories
Title
Measurements
OG00024.6(14.13 to 37.76)
OG00128.0(20.10 to 36.98)
OG0023.3(0.41 to 11.53)
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG001
OG002
Cochran-Mantel-Haenszel
<0.001
The p-value was calculated based on CMH controlling for prior RBC transfusion burden (≤6 vs. >6 units RBC) and IPSS risk group (low vs. intermediate-1) applied to randomization.
Percentage Difference
24.6
2-Sided
95
12.64
34.18
The 95% CI was calculated based on the Wilson Score method.
Superiority
Units
Counts
Participants
OG00021
OG00147
OG0029
Title
Denominators
Categories
Title
Measurements
OG0008.29(0.1 to 100.6)
OG0019.29(0.1 to 64.7)
OG0028.29(0.1 to 46.9)
Units
Counts
Participants
OG00014
OG00133
OG0022
Title
Denominators
Categories
Title
Measurements
OG0008.79(3.3 to 100.6)
OG0018.43(2.1 to 37.6)
OG0023.79(0.3 to 7.3)
Imetelstat sodium-matching placebo administered IV, every 4 weeks (on a 28-day cycle), until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
Units
Counts
Participants
OG00021
OG00147
OG0029
Title
Denominators
Categories
Title
Measurements
OG00069.6(17.00 to 92.43)
OG00151.6(26.86 to 80.43)
OG00213.3(8.00 to 24.86)
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG001
OG002
Log Rank
<0.001
The p-value (2-sided) was calculated using the stratified log-rank test for superiority of imetelstat sodium versus placebo in hazard ratio.
Hazard Ratio (HR)
0.25
2-Sided
95
0.105
0.586
Hazard ratio and 95% CI were calculated using the Cox proportional hazard model, stratified by prior RBC transfusion burden (≤ 6 vs. > 6 units RBC) and IPSS risk group (low vs. intermediate-1), with treatment as the only covariate.
Superiority
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
OG002
Phase 3: Placebo
Imetelstat sodium-matching placebo administered IV, every 4 weeks (on a 28-day cycle), until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
Units
Counts
Participants
OG00057
OG001118
OG00260
Title
Denominators
Categories
Title
Measurements
OG00061.4(47.57 to 74.00)
OG00163.6(54.20 to 72.22)
OG00251.7(38.39 to 64.77)
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG001
OG002
Cochran-Mantel-Haenszel
0.112
The p-value was calculated based on CMH controlling for prior RBC transfusion burden (≤ 6 vs. > 6 units RBC) and IPSS risk group (low vs. intermediate-1) applied to randomization.
Percentage Difference
11.9
2-Sided
95
-4.10
27.56
95% CI was calculated based on the Wilson Score Method.
Superiority
Participants
OG00057
Title
Denominators
Categories
Percentage of participants with CR
Title
Measurements
OG0008.8
Percentage of participants with PR
Title
Measurements
OG0000
Percentage of participants with mCR
Title
Measurements
OG00012.3
Imetelstat sodium-matching placebo administered IV, every 4 weeks (on a 28-day cycle), until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
Units
Counts
Participants
OG0001
OG0011
Title
Denominators
Categories
Percentage of participants with CR
Title
Measurements
OG0000
OG0010
Percentage of participants with PR
Title
Measurements
OG0000
OG0010
Percentage of participants with mCR
Title
Measurements
OG0000
OG0010
Units
Counts
Participants
OG00057
OG001118
OG00260
Title
Denominators
Categories
Title
Measurements
OG00055.3(39.16 to NA)Upper limit of 95% CI was not estimable due to low number of participants with events.
OG00140.4(37.06 to NA)Upper limit of 95% CI was not estimable due to low number of participants with events.
OG002NA(32.16 to NA)Median and upper limit of 95% CI were not estimable due to low number of participants with events.
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG001
OG002
Log Rank
0.949
P value (two-sided) for superiority of imetelstat sodium versus placebo in hazard ratio was calculated using stratified log-rank test.
Hazard Ratio (HR)
0.98
2-Sided
95
0.526
1.823
Hazard ratio and 95% CI were calculated from the Cox proportional hazard model, stratified by prior RBC transfusion burden (≤6 vs. >6 units RBC) and IPSS risk group (low vs. intermediate-1), with treatment as the only covariate.
Superiority
Phase 3: Placebo
Imetelstat sodium-matching placebo administered IV, every 4 weeks (on a 28-day cycle), until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
Units
Counts
Participants
OG00057
OG001118
OG00260
Title
Denominators
Categories
Title
Measurements
OG00034.2(25.10 to 39.29)
OG001NA(29.24 to NA)Median and upper limit of 95% CI were not estimable due to low number of participants with events.
OG002NA(16.72 to NA)Median and upper limit of 95% CI were not estimable due to low number of participants with events.
Imetelstat sodium-matching placebo administered IV, every 4 weeks (on a 28-day cycle), until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
Units
Counts
Participants
OG00057
OG001118
OG00260
Title
Denominators
Categories
Title
Measurements
OG000NA(41.36 to NA)Median and upper limit of 95% CI were not estimable by Kaplan-Meir method due to low number of participants with events.
OG001NA(NA to NA)Median, lower limit and upper limit of 95% CI were not estimable by Kaplan-Meir method due to low number of participants with events.
OG002NA(NA to NA)Median, lower limit and upper limit of 95% CI were not estimable by Kaplan-Meir method due to low number of participants with events.
OG002
Phase 3: Placebo
Imetelstat sodium-matching placebo administered IV, every 4 weeks (on a 28-day cycle), until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
Units
Counts
Participants
OG00057
OG001118
OG00260
Title
Denominators
Categories
Title
Measurements
OG0003.0± 3.48
OG0013.1± 3.62
OG0023.4± 2.13
Imetelstat sodium-matching placebo administered IV, every 4 weeks (on a 28-day cycle), until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.