A Biomarker-Directed Phase 2 Trial of Tamibarotene (SY-14... | NCT02807558 | Trialant
NCT02807558
Sponsor
Syros Pharmaceuticals
Status
Completed
Last Update Posted
Dec 13, 2024Actual
Enrollment
155Actual
Phase
Phase 2
Conditions
Acute Myeloid Leukemia
Myelodysplastic Syndrome
Interventions
Tamibarotene
Azacitidine
Daratumumab
Countries
United States
France
Protocol Section
Identification Module
NCT ID
NCT02807558
Obsolete or Duplicate NCT IDs
Not provided
Organization Study
SY-1425-201
Secondary IDs
ID
Type
Description
Link
2017-000783-14
EudraCT Number
Brief Title
A Biomarker-Directed Phase 2 Trial of Tamibarotene (SY-1425) in Participants With Acute Myeloid Leukemia or Myelodysplastic Syndrome
Official Title
A Biomarker-Directed Phase 2 Trial of SY-1425, a Selective Retinoic Acid Receptor Alpha Agonist, in Adult Patients With Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS)
Acronym
Not provided
Organization
Syros PharmaceuticalsINDUSTRY
Status Module
Record Verification Date
Nov 2024
Overall Recruitment Status or Expanded Access Status
Completed
Last Known Status
Not provided
Delayed Posting
Not provided
Why Stopped
Not provided
Expanded Access Info
No
Start Date
Sep 20, 2016Actual
Primary Completion Date
Jan 25, 2023Actual
Completion Date
Jan 25, 2023Actual
First Submitted Date
Jun 13, 2016
First Submission Date that Met QC Criteria
Jun 16, 2016
First Posted Date
Jun 21, 2016Estimated
Results Waived
Not provided
Results First Submitted Date
Sep 25, 2024
Results First Submitted that Met QC Criteria
Nov 19, 2024
Results First Posted Date
Dec 13, 2024Actual
Certification/Extension (aka Delayed Results) First Submitted Date
Jan 17, 2024
Certification/Extension First Submitted that Passed QC Review
Not provided
Certification/Extension First Posted Date
Jan 19, 2024Actual
Last Update Submitted Date
Nov 19, 2024
Last Update Posted Date
Dec 13, 2024Actual
Sponsor/Collaborators Module
Responsible Party, by Official Title
Sponsor
Lead Sponsor
Syros PharmaceuticalsINDUSTRY
Collaborators
Not provided
Oversight Module
Has Data Monitoring Committee (DMC)
No
Is FDA Regulated Drug
Yes
Is FDA Regulated Device
Not provided
Is Unapproved Device
Not provided
Pediatric Postmarket Surveillance of a Device Product
Not provided
Product Exported from US
Not provided
FDAAA801 Violation
Not provided
Description Module
Brief Summary
The purpose of this study is to determine the activity of tamibarotene in participants with relapsed/refractory (R/R) AML (administered as a monotherapy or in combination with azacitidine), R/R higher-risk MDS (HR-MDS) (administered as a monotherapy or in combination with daratumumab), newly diagnosed treatment naïve AML participants who are unlikely to tolerate standard intensive chemotherapy (administered as a monotherapy or in combination with azacitidine), or lower-risk MDS (LR-MDS) (administered as a monotherapy).
Detailed Description
Not provided
Conditions Module
Conditions
Acute Myeloid Leukemia
Myelodysplastic Syndrome
Keywords
AML
MDS
non-acute promyelocytic leukemia (non-APL)
lower-risk myelodysplastic syndrome (LR-MDS)
Design Module
Study Type
Interventional
Number of References to an Expanded Access Study
Not provided
Expanded Access Types
Not provided
Patient Registry
Not provided
Target Follow-Up Duration
Not provided
Phases
Phase 2
Interventional Study Design
Allocation
Biospecimen
No data available
No data is available for this block.
Enrollment
155Actual
Arms/Interventions Module
Arm Groups
Label
Type
Description
Intervention Names
R/R Non-APL AML or R/R HR-MDS: Tamibarotene Monotherapy
Experimental
Participants with R/R non-APL AML or R/R HR-MDS will receive tamibarotene at 6 mg/m^2/day in 2 divided doses on Days 1-28 of a 28-day cycle.
Newly diagnosed, treatment-naive participants with non-APL AML who were unlikely to tolerate standard intensive chemotherapy will receive tamibarotene at 6 mg/m^2/day in 2 divided doses on Days 1-28 of a 28-day cycle.
Drug: Tamibarotene
Newly Diagnosed Non-APL AML: Tamibarotene and Azacitidine
Experimental
Newly diagnosed, treatment-naive participants with non-APL AML who are unlikely to tolerate standard intensive chemotherapy will receive tamibarotene at 6 mg/m^2/day in 2 divided doses on Days 8-28 of a 28-day cycle, and azacitidine at 75 mg/m^2 once daily on Days 1-7 of a 28-day cycle.
Drug: Tamibarotene
Drug: Azacitidine
LR-MDS: Tamibarotene Monotherapy
Experimental
Participants with transfusion-dependent LR-MDS without the del 5q abnormality who are refractory to erythropoietin (EPO) treatment or unlikely to respond to EPO treatment will receive tamibarotene at 6 mg/m^2/day in 2 divided doses on Days 1-28 of a 28-day cycle.
Newly Diagnosed Non-APL AML: Tamibarotene and Azacitidine
Outcomes Module
Primary Outcomes
Measure
Description
Time Frame
Overall Response Rate (ORR) in Biomarker Positive AML or HR-MDS Participants Treated With Tamibarotene Monotherapy or in Combination With Azacitidine
ORR was defined as: AML: number of participants with complete remission (CR), CR with incomplete blood count recovery (CRi), CR with partial hematologic recovery (CRh), partial remission(PR), or morphologic leukemia-free state (MLFS) determined by the investigator per revised International Working Group (IWG) AML criteria. HR-MDS: the number of participants with CR, PR, marrow CR (mCR), or HI determined by the investigator per revised IWG MDS criteria.
Up to 48 months
Transfusion Independence Rate (TIR) for LR-MDS Participants Treated With Tamibarotene Monotherapy
TIR was defined as the number of participants who achieved transfusion independence defined as 8 consecutive weeks of red blood cell (RBC) transfusion independence.
Up to 48 months
Number of Participants With Treatment-emergent Adverse Events (TEAEs) Treated With Tamibarotene in Combination With Daratumumab
A TEAE was any untoward medical occurrence associated with use of a study drug/study participation, whether or not considered related to study drug after first dose. A TEAE was any unfavorable/unintended sign (including abnormal laboratory finding), symptom, or disease temporally associated with the study drug. A serious TEAE resulted in death, was life-threatening, required inpatient hospitalization/prolongation of existing hospitalization, persistent or significant disability/incapacity, congenital anomaly/birth defect in the offspring of a participant who received study drug or other important medical events. A summary of serious and all other non-serious adverse events regardless of causality is located in the Reported Adverse Events module.
Up to 48 months
Secondary Outcomes
Measure
Description
Time Frame
ORR in AML Participants Positive for the RARA Super-enhancer Associated Biomarker Treated With Tamibarotene in Combination With Azacitidine
ORR was defined as: AML: the number of participants with CR, CRi, CRh, PR, or MLFS as determined by the investigator per the revised IWG AML criteria.
Per prespecified analysis, data were not collected for this Outcome Measure for the "R/R Non-APL AML or R/R HR-MDS: Tamibarotene Monotherapy" arm, the "Newly Diagnosed Non-APL AML: Tamibarotene Monotherapy" arm, and the "R/R non-APL AML or R/R HR-MDS: Tamibarotene and Daratumumab" arm as there were fewer than 5 responders per arm. Data are presented per specifications in the statistical analysis plan.
Other Outcomes
Not provided
Eligibility Module
Eligibility Criteria
Key Inclusion Criteria:
Must have:
Relapsed and/or refractory non-APL AML that has failed to achieve a complete remission (CR) or partial remission (PR) following standard induction therapy, or has relapsed after any duration of CR or PR i. Must have measurable disease with bone marrow blasts ≥5%at screening
Relapsed and/or refractory HR-MDS (High / Very High Risk, as defined by the Revised International Prognostic Scoring System (IPSS-R)) that has failed to achieve a CR or PR, or any hematologic improvement (HI, per IWG 2006 criteria) after standard therapy with hypomethylating agents (e.g., azacitidine, decitabine), or has relapsed after any duration of CR or PR or HI i. Must have measurable disease with bone marrow blasts >5% at screening
Newly diagnosed, treatment-naïve non-APL AML in participants who, at the time of study entry are unlikely to tolerate standard intensive chemotherapy due to age, performance status, or comorbidities based on at least one of the following criteria:
i. Age ≥ 75 years old
ii. Eastern Cooperative Oncology Group (ECOG) Performance Status (PS) of 3
iii. Cardiac history of congestive heart failure (CHF) or documented ejection fraction (EF) ≤ 50%
iv. Pulmonary disease with diffusing capacity of lung for carbon monoxide ≤ 65% or Forced Expiratory Volume in 1 Second ≤ 65%
v. Creatinine clearance ≥ 30 milliliter (mL)/minute (min) to < 45 mL/min
vi. Hepatic impairment with total bilirubin > 1.5 to ≤ 3.0 x upper limit of normal (ULN)
vii. Any other comorbidity that the Investigator judges to be incompatible with intensive chemotherapy, and reviewed and approved by the Sponsor prior to enrollment
Transfusion-dependent LR-MDS without the del 5q abnormality, in participants refractory to erythropoietin treatment or unlikely to respond to erythropoietin treatment (EPO >500).
i. LR-MDS: Very Low /Low / Intermediate Risk, as defined by IPSS-R.
ii.Red blood cell (RBC) transfusion-dependent anemia defined as no eight consecutive weeks without RBC transfusions within the 16 weeks prior to study entry, or ≥4 RBC transfusions within the 8 weeks prior to study entry.
iii.Refractory to or ineligible for erythropoiesis-stimulating agents (ESAs) is defined as RBC-Transfusion Dependence despite ESA treatment of ≥40,000 units/week recombinant human erythropoietin for 8 weeks or an equivalent dose of darbepoetin (150 µg/week) or serum EPO level >500 milliunits (mU)/mL in participants not previously treated with ESAs.
Participants must be evaluated for the RARA super-enhancer associated biomarker or RARA pathway associated biomarker at the time of study screening.
a. Participants treated with tamibarotene monotherapy and in combination with daratumumab, and R/R AML participants treated with tamibarotene in combination with azacitidine must be positive as defined by a pre-determined cut-off
Must be amenable to serial bone marrow aspirates and peripheral blood sampling during the study.
ECOG Performance Status (PS) of 0, 1 or 2. For newly diagnosed AML participants < 75 years of age, ECOG 0-3; for ≥ 75 years of age, ECOG 0-2.
Adequate organ function as defined by:
Total bilirubin ≤ 1.5 x the upper limit of normal (ULN), unless suspected to have Gilbert's disease. For newly diagnosed AML participants < 75 years of age, total bilirubin ≤ 3 x ULN; for ≥ 75 years of age, total bilirubin ≤ 1.5 x ULN.
Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 3 x ULN or ≤ 5 x ULN if documented liver infiltration with leukemia cells
Serum creatinine ≤ 2.0 x ULN or calculated creatinine clearance ≥ 45 mL/min based on the Cockroft-Gault glomerular filtration rate estimation. For newly diagnosed AML participants < 75 years of age, creatinine clearance ≥ 30 mL/min; for ≥ 75 years of age, creatinine clearance ≥ 45 mL/min.
Discontinued use of chemotherapy, radiation therapy, or growth factors for at least 2 weeks prior to first study treatment, with the exception of hydroxyurea.
No investigational agents within 2 weeks prior to first study treatment.
No strong inducers of CYP3A4 within 2 weeks prior to first study treatment.
Resolved acute effects of any prior AML/MDS therapy to baseline or ≤ Grade 1 CTCAE severity.
Key Exclusion Criteria:
Acute promyelocytic leukemia (APL, M3 subtype of AML) or participants with a t(9:22) cytogenetic translocation.
Hyperleukocytosis (leukocytes ≥25 x 109/L) at study entry. The participants may be treated with hydroxyurea according to routine practice, and enroll in the study when the leukocyte count falls below 25 x 109/L.
Participants known to be refractory to platelet or packed red cell transfusions per Institutional Guidelines, or a participant who refuses blood product support.
Prior treatment with all-trans retinoic acid (ATRA) or systemic retinoid for the treatment of hematologic malignancy.
Tamibarotene and daratumumab combination only - Prior or concurrent exposure to daratumumab or other CD38 therapies.
Tamibarotene and daratumumab combination only - Participant has either of the following:
Known chronic obstructive pulmonary disease (COPD) with a forced expiratory volume in 1 second (FEV1) <50% of predicted normal. Note that FEV1 testing is required for participants suspected of having COPD and participants must be excluded if FEV1 is <50% of predicted normal.
Known moderate or severe persistent asthma within the past 2 years, or uncontrolled asthma of any classification. Note that participants who currently have controlled intermittent asthma or controlled mild persistent asthma are allowed to participate in the study.
Participants with active malignancy (not including basal cell carcinoma, non-melanoma skin cancer, cervical carcinoma in situ, localized prostate cancer treated with hormone therapy). Participants with history of other cancers should be free of disease for at least 2 years.
Participants with hypertriglyceridemia defined as >1000 mg/dL (CTCAE Grade 4).
Participants with clinically significant cardiac disease including one of the following currently or in the previous 6 months: myocardial infarction, unstable cardiac function due to unstable angina or congestive heart failure, congenital long QT syndrome, torsades de pointes or significant ventricular arrhythmias.
Participants with known active uncontrolled central nervous system (CNS) leukemia.
Participants taking Vitamin A supplements (>10,000 IU/d) unless discontinued prior to first dose of study drug, or having hypervitaminosis.
Note: Other protocol defined Inclusion/Exclusion criteria may apply.
de Botton S, Cluzeau T, Vigil C, Cook RJ, Rousselot P, Rizzieri DA, Liesveld JL, Fenaux P, Braun T, Banos A, Jurcic JG, Sekeres MA, Savona MR, Roboz GJ, Bixby D, Madigan K, Volkert A, Stephens K, Kang-Fortner Q, Baker K, Paul S, McKeown M, Carulli J, Eaton M, Hodgson G, Fiore C, Kelly MJ, Roth DA, Stein EM. Targeting RARA overexpression with tamibarotene, a potent and selective RARalpha agonist, is a novel approach in AML. Blood Adv. 2023 May 9;7(9):1858-1870. doi: 10.1182/bloodadvances.2022008806.
See Also Links
Not provided
Available IPD Information
Not provided
IPD Sharing Statement Module
Plan to Share IPD
No
Description
Not provided
Types
Not provided
Time Frame
Not provided
Access Criteria
Not provided
URL
Not provided
Results Section
Participant Flow Module
Pre-assignment Details
Screening assessments were conducted within 30 days of dosing.
Recruitment Details
Not provided
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
FG000
R/R Non-APL AML or R/R HR-MDS: Tamibarotene Monotherapy
Participants with Relapsed/Refractory (R/R) Non-Acute Promyelocytic Leukemia (APL) Acute Myeloid Leukemia (AML) or R/R Higher-risk Myelodysplastic Syndrome (HR-MDS) received tamibarotene at 6 milligrams (mg)/meter (m)^2/day in 2 divided doses on Days 1-28 of a 28-day cycle.
FG001
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
0
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
Aug 31, 2022
Sep 25, 2024
Derived Section
Miscellaneous Info Module
Version Holder
Jul 10, 2026
Removed Countries
Not provided
Submission Tracking
No data available
No data is available for this block.
Condition Browse Module
MeSH Terms
Intervention Browse Module
MeSH Terms
Non-Randomized
Intervention Model
Parallel Assignment
Intervention Model Description
Not provided
Primary Purpose
Treatment
Observational Model
Not provided
Time Perspective
Not provided
Masking Info
Masking
None (Open Label)
Masking Description
Not provided
Who Masked
Not provided
R/R non-APL AML or R/R HR-MDS: Tamibarotene and Daratumumab
Experimental
Participants with R/R non-APL AML or R/R HR-MDS will receive tamibarotene at 6 mg/m^2/day in 2 divided doses during a 7-day lead-in and on Days 1-28 of a 28-day cycle. Participants will also receive daratumumab at 16 mg/kg starting on Cycle 1 Day 1 once weekly for 8 weeks, followed by dosing every 2 weeks for 16 weeks, followed by dosing every 4 weeks.
Drug: Tamibarotene
Drug: Daratumumab
R/R non-APL AML: Tamibarotene and Azacitidine
Experimental
Participants with R/R non-APL AML will receive tamibarotene at 6 mg/m^2/day in 2 divided doses on Days 8-28 of a 28-day cycle, and azacitidine at 75 mg/m^2 once daily on Days 1-7 of a 28-day cycle.
Drug: Tamibarotene
Drug: Azacitidine
R/R Non-APL AML or R/R HR-MDS: Tamibarotene Monotherapy
R/R non-APL AML or R/R HR-MDS: Tamibarotene and Daratumumab
R/R non-APL AML: Tamibarotene and Azacitidine
SY-1425
Azacitidine
Drug
Administered via intravenous (IV) or subcutaneous (SC) infusion
Newly Diagnosed Non-APL AML: Tamibarotene and Azacitidine
R/R non-APL AML: Tamibarotene and Azacitidine
Vidaza
Daratumumab
Drug
Administered via IV infusion
R/R non-APL AML or R/R HR-MDS: Tamibarotene and Daratumumab
Darzalex
Up to 48 months
ORR in AML Participants Positive for the Interferon Regulatory Factor 8 (IRF8) Biomarker and Negative for the RARA Super-enhancer Associated Biomarker Treated With Tamibarotene in Combination With Azacitidine
ORR was defined as: AML: number of participants with CR, CRi, CRh, PR, or MLFS determined by the investigator per the revised IWG AML criteria.
Per prespecified analysis, data were collected for this Outcome Measure only for the arm that enrolled the IRF8-positive participants and that included 5 or more responders. Data are presented per specifications in the statistical analysis plan.
Up to 48 months
ORR in AML Participants Negative for the RARA Super-enhancer Associated Biomarker Treated With Tamibarotene in Combination With Azacitidine
ORR was defined as:
AML: the number of participants with CR, CRi, CRh, PR, or MLFS as determined by the investigator per the revised IWG AML criteria.
Per prespecified analysis, data were collected for this Outcome Measure only for the arm that enrolled the RARA-negative participants and that included 5 or more responders. Data are presented per specifications in the statistical analysis plan.
Up to 48 months
ORR for AML or HR-MDS Participants Treated With Tamibarotene in Combination With Daratumumab
ORR was defined as:
AML: the number of participants with CR, CRi, CRh, PR, or MLFS as determined by the investigator per the revised IWG AML criteria.
HR-MDS: the number of participants with CR, PR, mCR, or HI as determined by the investigator per the revised IWG MDS criteria.
Up to 48 months
Event-Free Survival (EFS) in AML and HR-MDS Participants Treated With Tamibarotene Monotherapy, or in Combination With Azacitidine or Daratumumab
EFS was defined as time from first treatment until date of documentation of disease relapse following CR, CRi, or death, whichever occurred first. If the participant did not achieve a CR, EFS was defined as the point of progression or death, whichever occurred first.
Per prespecified analysis, data were not collected for the Newly Diagnosed Non-APL AML: Tamibarotene Monotherapy arm as there were fewer than 5 participants in the arm. Additionally, per prespecified analysis, data were not collected for this Outcome Measure for the LR-MDS: Tamibarotene Monotherapy arm.
Data are presented per specifications in the statistical analysis plan. All-cause mortality is reported in the Reported Adverse Events module.
Up to 48 months
Duration of Response (DOR) in AML Participants Treated With Tamibarotene in Combination With Azacitidine
DOR was defined as time from first date of response CR, CRi, CRh, MLFS or PR until the date of relapse.
As prespecified, data were not collected for this Outcome Measure for any of the Tamibarotene Monotherapy arms, or R/R non-APL AML or R/R HR-MDS: Tamibarotene and Daratumumab arm.
Data is presented as specified in the statistical analysis plan.
Up to 48 months
Overall Survival (OS) in AML and HR-MDS Participants Treated With Tamibarotene Monotherapy, or in Combination With Azacitidine or Daratumumab
OS was defined as the time from first treatment until death from any cause.
Per planned analysis, data were not collected for this Outcome Measure for the Newly Diagnosed Non-APL AML: Tamibarotene Monotherapy arm as there were fewer than 5 participants in the study arm. Additionally, per prespecified analysis, data were not collected for this Outcome Measure for the LR-MDS: Tamibarotene Monotherapy arm.
Data is presented as specified in the statistical analysis plan. All-cause mortality is reported in the Reported Adverse Events module.
Up to 48 months
Hematologic Improvement (HI) in AML, HR-MDS and LR-MDS Participants Treated With Tamibarotene Monotherapy, or in Combination With Azacitidine or Daratumumab
HI was defined according to the modified IWG response criteria for MDS as the number of participants with a response (lasting at least 8 weeks) after first treatment, including erythroid response, platelet response, or neutrophil response.
Data are presented per specifications in the statistical analysis plan.
Up to 48 months
Number of Participants With TEAEs in AML and MDS Participants Treated With Tamibarotene Monotherapy or in Combination With Azacitidine
A TEAE was any untoward medical occurrence associated with use of a study drug/study participation, whether or not considered related to study drug after first dose. A TEAE was any unfavorable/unintended sign (including abnormal laboratory finding), symptom, or disease temporally associated with the study drug. A serious TEAE resulted in death, was life-threatening, required inpatient hospitalization/prolongation of existing hospitalization, persistent or significant disability/incapacity, congenital anomaly/birth defect in the offspring of a participant who received study drug or other important medical events. A summary of serious and all other non-serious adverse events regardless of causality is located in the Reported Adverse Events module.
Up to 48 months
Miami
Florida
United States
Iowa City
Iowa
United States
Boston
Massachusetts
United States
Ann Arbor
Michigan
United States
New York
New York
United States
Rochester
New York
United States
Durham
North Carolina
United States
Cleveland
Ohio
United States
Portland
Oregon
United States
Allentown
Pennsylvania
United States
Pittsburgh
Pennsylvania
United States
Nashville
Tennessee
United States
Houston
Texas
United States
CHU Amiens
Amiens
France
Centre Hospitalier de la Côte basque
Bayonne
France
Centre Hospitalier Universitiaire Hopital Avicenne
Newly diagnosed, treatment-naive participants with non-APL AML who were unlikely to tolerate standard intensive chemotherapy received tamibarotene at 6 mg/m^2/day in 2 divided doses on Days 1-28 of a 28- day cycle.
FG002
LR-MDS: Tamibarotene Monotherapy
Participants with transfusion-dependent lower-risk myelodysplastic syndrome (LR-MDS) without the del- 5q abnormality who were refractory to erythropoietin (EPO) treatment or unlikely to respond to EPO treatment received tamibarotene at 6 mg/m^2/day in 2 divided doses from Days 1-28 of a 28-day cycle.
FG003
Newly Diagnosed Non-APL AML: Tamibarotene and Azacitidine
Newly diagnosed, treatment-naive participants with non-APL AML who were unlikely to tolerate standard intensive chemotherapy received tamibarotene at 6 mg/m^2/day in 2 divided doses on Days 8-28 of a 28-day cycle, and azacitidine at 75 mg/m^2 once daily on Days 1-7 of a 28-day cycle.
FG004
R/R Non-APL AML: Tamibarotene and Azacitidine
Participants with R/R non-APL AML received tamibarotene at 6 mg/m^2/day in 2 divided doses on Days 8-28 of a 28-day cycle, and azacitidine at 75 mg/m^2 once daily on Days 1-7 of a 28-day cycle.
FG005
R/R Non-APL AML or R/R HR-MDS: Tamibarotene and Daratumumab
Participants with R/R non-APL AML or R/R HR-MDS received tamibarotene at 6 mg/m^2/day in 2 divided doses during a 7-day lead-in and on Days 1-28 of a 28-day cycle. Participants also received daratumumab at 16 mg/kg starting on Cycle 1 Day 1 once weekly for 8 weeks, followed by dosing every 2 weeks for 16 weeks, followed by dosing every 4 weeks.
FG00029 subjects
FG0012 subjects
FG00229 subjects
FG00351 subjects
FG00428 subjects
FG00516 subjects
Received at Least 1 Dose of Study Drug
FG00029 subjects
FG0012 subjects
FG00229 subjects
FG00351 subjects
FG00428 subjects
FG00516 subjects
Response Evaluable Population
FG00022 subjects
FG0012 subjects
FG00227 subjects
FG00346 subjects
FG00421 subjects
FG00512 subjects
Completed Per Study Protocol
FG00029 subjects
FG0012 subjects
FG00229 subjects
FG00351 subjects
FG00427 subjects
FG00516 subjects
Completed Post-Treatment Follow-up
FG0001 subjects
FG0010 subjects
FG0020 subjects
FG0036 subjects
FG0041 subjects
FG0050 subjects
Evaluable for Hematological Improvement (HI)
FG00016 subjects
FG0012 subjects
FG00225 subjects
FG00316 subjects
FG00417 subjects
FG0059 subjects
COMPLETED
FG0001 subjects
FG0010 subjects
FG0020 subjects
FG0036 subjects
FG0042 subjects
FG0050 subjects
NOT COMPLETED
FG00028 subjects
FG0012 subjects
FG00229 subjects
FG00345 subjects
FG00426 subjects
FG00516 subjects
Type
Comment
Reasons
Consent withdrawn by participant
FG0004 subjects
FG0010 subjects
FG0024 subjects
FG0032 subjects
FG0041 subjects
FG0050 subjects
Non-compliance with Study Drug
FG0000 subjects
FG0010 subjects
FG0021 subjects
FG0030 subjects
FG004
Adverse Event
FG0000 subjects
FG0010 subjects
FG0029 subjects
FG0030 subjects
FG004
Death
FG00023 subjects
FG0012 subjects
FG0023 subjects
FG00343 subjects
FG004
Progressive Disease
FG0000 subjects
FG0010 subjects
FG0021 subjects
FG0030 subjects
FG004
Other than specified
FG0001 subjects
FG0010 subjects
FG0022 subjects
FG0030 subjects
FG004
Lack of Efficacy
FG0000 subjects
FG0010 subjects
FG0029 subjects
FG0030 subjects
FG004
Safety population included all screened participants who received any amount of study drug (tamibarotene or combination treatment).
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
BG000
R/R Non-APL AML or R/R HR-MDS: Tamibarotene Monotherapy
Participants with R/R non-APL AML or R/R HR-MDS received tamibarotene at 6 mg/m^2/day in 2 divided doses on Days 1-28 of a 28-day cycle.
Newly diagnosed, treatment-naive participants with non-APL AML who were unlikely to tolerate standard intensive chemotherapy received tamibarotene at 6 mg/m^2/day in 2 divided doses on Days 1-28 of a 28- day cycle.
BG002
LR-MDS: Tamibarotene Monotherapy
Participants with transfusion-dependent LR-MDS without the del- 5q abnormality who were refractory to EPO treatment or unlikely to respond to EPO treatment received tamibarotene at 6 mg/m^2/day in 2 divided doses from Days 1-28 of a 28-day cycle.
BG003
Newly Diagnosed Non-APL AML: Tamibarotene and Azacitidine
Newly diagnosed, treatment-naive participants with non-APL AML who were unlikely to tolerate standard intensive chemotherapy received tamibarotene at 6 mg/m^2/day in 2 divided doses on Days 8-28 of a 28-day cycle, and azacitidine at 75 mg/m^2 once daily on Days 1-7 of a 28-day cycle.
BG004
R/R Non-APL AML: Tamibarotene and Azacitidine
Participants with R/R non-APL AML received tamibarotene at 6 mg/m^2/day in 2 divided doses on Days 8-28 of a 28-day cycle, and azacitidine at 75 mg/m^2 once daily on Days 1-7 of a 28-day cycle.
BG005
R/R Non-APL AML or R/R HR-MDS: Tamibarotene and Daratumumab
Participants with R/R non-APL AML or R/R HR-MDS received tamibarotene at 6 mg/m^2/day in 2 divided doses during a 7-day lead-in and on Days 1-28 of a 28-day cycle. Participants also received daratumumab at 16 mg/kg starting on Cycle 1 Day 1 once weekly for 8 weeks, followed by dosing every 2 weeks for 16 weeks, followed by dosing every 4 weeks.
BG006
Total
Total of all reporting groups
Denominators
Units
Counts
Participants
BG00029
BG0012
BG00229
BG00351
BG00428
BG00516
BG006155
Baseline Measures
Title
Description
Population Description
Parameter Type
Dispersion Type
Unit of Measure
Calculate Percentage
Denominator Units Selected
Denominators
Classes
Age, Customized
Count of Participants
Participants
Title
Denominators
Categories
Adults (18-64 years)
Title
Measurements
BG0005
BG0010
BG0022
BG003
Sex: Female, Male
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
Female
BG00012
BG0010
BG002
Ethnicity (NIH/OMB)
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
Hispanic or Latino
BG0003
BG0012
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
Overall Response Rate (ORR) in Biomarker Positive AML or HR-MDS Participants Treated With Tamibarotene Monotherapy or in Combination With Azacitidine
ORR was defined as: AML: number of participants with complete remission (CR), CR with incomplete blood count recovery (CRi), CR with partial hematologic recovery (CRh), partial remission(PR), or morphologic leukemia-free state (MLFS) determined by the investigator per revised International Working Group (IWG) AML criteria. HR-MDS: the number of participants with CR, PR, marrow CR (mCR), or HI determined by the investigator per revised IWG MDS criteria.
Response Evaluable Population included participants who completed 1 cycle of study treatment, and had a follow-up assessment of disease status, and did not have any major protocol violations, or withdrew from the study before completion of Cycle 1 because of documented disease progression. Overall Number of Participants analyzed (N) = participants evaluable for this Outcome Measure.
Posted
Count of Participants
Participants
Up to 48 months
ID
Title
Description
OG000
R/R Non-APL AML or R/R HR-MDS: Tamibarotene Monotherapy
Participants with R/R non-APL AML or R/R HR-MDS received tamibarotene at 6 mg/m^2/day in 2 divided doses on Days 1-28 of a 28-day cycle.
Newly diagnosed, treatment-naive participants with non-APL AML who were unlikely to tolerate standard intensive chemotherapy received tamibarotene at 6 mg/m^2/day in 2 divided doses on Days 1-28 of a 28- day cycle.
OG002
R/R Non-APL AML: Tamibarotene and Azacitidine
Participants with R/R non-APL AML received tamibarotene at 6 mg/m^2/day in 2 divided doses on Days 8-28 of a 28-day cycle, and azacitidine at 75 mg/m^2 once daily on Days 1-7 of a 28-day cycle.
OG003
ND Non-APL AML: Tamibarotene and Azacitidine: RARA-Positive
Participants from the "Newly Diagnosed Non-APL AML: Tamibarotene and Azacitidine" arm who were positive for the retinoic acid receptor alpha (RARA) super-enhancer associated biomarker (RARA-positive) were included in this subgroup analysis.
Participants who were unlikely to tolerate standard intensive chemotherapy received tamibarotene at 6 mg/m^2/day in 2 divided doses on Days 8-28 of a 28-day cycle, and azacitidine at 75 mg/m^2 once daily on Days 1-7 of a 28-day cycle.
Units
Counts
Participants
OG00022
OG0012
OG00221
OG003
Title
Denominators
Categories
Title
Measurements
OG0002
OG0010
OG0024
OG003
Primary
Transfusion Independence Rate (TIR) for LR-MDS Participants Treated With Tamibarotene Monotherapy
TIR was defined as the number of participants who achieved transfusion independence defined as 8 consecutive weeks of red blood cell (RBC) transfusion independence.
Population evaluable for TIR, which included all participants who received at least 8 weeks of study treatment.
Here, Overall Number of Participants analyzed (N) = participants evaluable for this Outcome Measure.
Posted
Count of Participants
Participants
Up to 48 months
ID
Title
Description
OG000
LR-MDS: Tamibarotene Monotherapy
Participants with transfusion-dependent LR-MDS without the del-5q abnormality who were refractory to EPO treatment or unlikely to respond to EPO treatment received tamibarotene at 6 mg/m^2/day in 2 divided doses from Days 1-28 of a 28-day cycle.
Units
Counts
Participants
OG000
Primary
Number of Participants With Treatment-emergent Adverse Events (TEAEs) Treated With Tamibarotene in Combination With Daratumumab
A TEAE was any untoward medical occurrence associated with use of a study drug/study participation, whether or not considered related to study drug after first dose. A TEAE was any unfavorable/unintended sign (including abnormal laboratory finding), symptom, or disease temporally associated with the study drug. A serious TEAE resulted in death, was life-threatening, required inpatient hospitalization/prolongation of existing hospitalization, persistent or significant disability/incapacity, congenital anomaly/birth defect in the offspring of a participant who received study drug or other important medical events. A summary of serious and all other non-serious adverse events regardless of causality is located in the Reported Adverse Events module.
Safety population included all screened participants who received any amount of study drug (tamibarotene or combination treatment).
Posted
Count of Participants
Participants
Up to 48 months
ID
Title
Description
OG000
R/R Non-APL AML or R/R HR-MDS: Tamibarotene and Daratumumab
Participants with R/R non-APL AML or R/R HR-MDS received tamibarotene at 6 mg/m^2/day in 2 divided doses during a 7-day lead-in and on Days 1-28 of a 28-day cycle. Participants also received daratumumab at 16 mg/kg starting on Cycle 1 Day 1 once weekly for 8 weeks, followed by dosing every 2 weeks for 16 weeks, followed by dosing every 4 weeks.
Secondary
ORR in AML Participants Positive for the RARA Super-enhancer Associated Biomarker Treated With Tamibarotene in Combination With Azacitidine
ORR was defined as: AML: the number of participants with CR, CRi, CRh, PR, or MLFS as determined by the investigator per the revised IWG AML criteria.
Per prespecified analysis, data were not collected for this Outcome Measure for the "R/R Non-APL AML or R/R HR-MDS: Tamibarotene Monotherapy" arm, the "Newly Diagnosed Non-APL AML: Tamibarotene Monotherapy" arm, and the "R/R non-APL AML or R/R HR-MDS: Tamibarotene and Daratumumab" arm as there were fewer than 5 responders per arm. Data are presented per specifications in the statistical analysis plan.
Response evaluable population (which included participants who completed 1 cycle of study treatment, and had a follow-up assessment of disease status, and did not have any major protocol violations, or withdrew from the study before completion of Cycle 1 because of documented disease progression) in participants who were RARA-positive. Overall Number of Participants analyzed (N) = participants evaluable for this Outcome Measure.
Posted
Count of Participants
Participants
Up to 48 months
ID
Title
Description
OG000
R/R Non-APL AML: Tamibarotene and Azacitidine
Participants with R/R non-APL AML received tamibarotene at 6 mg/m^2/day in 2 divided doses on Days 8-28 of a 28-day cycle, and azacitidine at 75 mg/m^2 once daily on Days 1-7 of a 28-day cycle.
OG001
ND Non-APL AML: Tamibarotene and Azacitidine: RARA-Positive
Secondary
ORR in AML Participants Positive for the Interferon Regulatory Factor 8 (IRF8) Biomarker and Negative for the RARA Super-enhancer Associated Biomarker Treated With Tamibarotene in Combination With Azacitidine
ORR was defined as: AML: number of participants with CR, CRi, CRh, PR, or MLFS determined by the investigator per the revised IWG AML criteria.
Per prespecified analysis, data were collected for this Outcome Measure only for the arm that enrolled the IRF8-positive participants and that included 5 or more responders. Data are presented per specifications in the statistical analysis plan.
Response Evaluable Population (which included participants who completed 1 cycle of study treatment, had a follow-up assessment of disease status, did not have any major protocol violations or withdrew from study before completion of Cycle 1 because of documented disease progression) in participants who were positive for the IRF8 biomarker and negative for the RARA superenhancer associated biomarker. Overall Number of Participants analyzed (N) = participants evaluable for this Outcome Measure.
Posted
Count of Participants
Participants
Up to 48 months
ID
Title
Description
OG000
Newly Diagnosed Non-APL AML: Tamibarotene and Azacitidine
Newly diagnosed, treatment-naive participants with non-APL AML who were unlikely to tolerate standard intensive chemotherapy received tamibarotene at 6 mg/m^2/day in 2 divided doses on Days 8-28 of a 28-day cycle, and azacitidine at 75 mg/m^2 once daily on Days 1-7 of a 28-day cycle.
Secondary
ORR in AML Participants Negative for the RARA Super-enhancer Associated Biomarker Treated With Tamibarotene in Combination With Azacitidine
ORR was defined as:
AML: the number of participants with CR, CRi, CRh, PR, or MLFS as determined by the investigator per the revised IWG AML criteria.
Per prespecified analysis, data were collected for this Outcome Measure only for the arm that enrolled the RARA-negative participants and that included 5 or more responders. Data are presented per specifications in the statistical analysis plan.
Response evaluable population included participants who completed 1 cycle of study treatment, and had a follow-up assessment of disease status, and did not have any major protocol violations, or withdrew from the study before completion of Cycle 1 because of documented disease progression. Overall Number of Participants analyzed (N) = participants evaluable for this Outcome Measure.
Posted
Count of Participants
Participants
Up to 48 months
ID
Title
Description
OG000
ND Non-APL AML: Tamibarotene and Azacitidine: RARA-Negative
Participants from the "Newly Diagnosed Non-APL AML:Tamibarotene and Azacitidine" arm who were negative for the RARA super-enhancer associated biomarker (RARA-negative) were included in this subgroup analysis.
Participants who were unlikely to tolerate standard intensive chemotherapy received tamibarotene at 6 mg/m^2/day in 2 divided doses on Days 8-28 of a 28-day cycle, and azacitidine at 75 mg/m^2 once daily on Days 1-7 of a 28-day cycle.
Secondary
ORR for AML or HR-MDS Participants Treated With Tamibarotene in Combination With Daratumumab
ORR was defined as:
AML: the number of participants with CR, CRi, CRh, PR, or MLFS as determined by the investigator per the revised IWG AML criteria.
HR-MDS: the number of participants with CR, PR, mCR, or HI as determined by the investigator per the revised IWG MDS criteria.
Response evaluable population (which included participants who completed 1 cycle of study treatment, and had a follow-up assessment of disease status, and did not have any major protocol violations, or withdrew from the study before completion of Cycle 1 because of documented disease progression) who had evaluable data for the Outcome Measure.
Posted
Count of Participants
Participants
Up to 48 months
ID
Title
Description
OG000
R/R Non-APL AML or R/R HR-MDS: Tamibarotene and Daratumumab
Participants with R/R non-APL AML or R/R HR-MDS received tamibarotene at 6 mg/m^2/day in 2 divided doses during a 7-day lead-in and on Days 1-28 of a 28-day cycle. Participants also received daratumumab at 16 mg/kg starting on Cycle 1 Day 1 once weekly for 8 weeks, followed by dosing every 2 weeks for 16 weeks, followed by dosing every 4 weeks.
Units
Counts
Participants
Secondary
Event-Free Survival (EFS) in AML and HR-MDS Participants Treated With Tamibarotene Monotherapy, or in Combination With Azacitidine or Daratumumab
EFS was defined as time from first treatment until date of documentation of disease relapse following CR, CRi, or death, whichever occurred first. If the participant did not achieve a CR, EFS was defined as the point of progression or death, whichever occurred first.
Per prespecified analysis, data were not collected for the Newly Diagnosed Non-APL AML: Tamibarotene Monotherapy arm as there were fewer than 5 participants in the arm. Additionally, per prespecified analysis, data were not collected for this Outcome Measure for the LR-MDS: Tamibarotene Monotherapy arm.
Data are presented per specifications in the statistical analysis plan. All-cause mortality is reported in the Reported Adverse Events module.
Safety population included all screened participants who received any amount of study drug (tamibarotene or combination treatment). Overall Number of Participants analyzed (N) = participants evaluable for this Outcome Measure.
Posted
Median
95% Confidence Interval
months
Up to 48 months
ID
Title
Description
OG000
R/R Non-APL AML or R/R HR-MDS: Tamibarotene Monotherapy
Participants with R/R non-APL AML or R/R HR-MDS received tamibarotene at 6 mg/m^2/day in 2 divided doses on Days 1-28 of a 28-day cycle.
OG001
R/R Non-APL AML: Tamibarotene and Azacitidine
Participants with R/R non-APL AML received tamibarotene at 6 mg/m^2/day in 2 divided doses on Days 8-28 of a 28-day cycle, and azacitidine at 75 mg/m^2 once daily on Days 1-7 of a 28-day cycle.
Secondary
Duration of Response (DOR) in AML Participants Treated With Tamibarotene in Combination With Azacitidine
DOR was defined as time from first date of response CR, CRi, CRh, MLFS or PR until the date of relapse.
As prespecified, data were not collected for this Outcome Measure for any of the Tamibarotene Monotherapy arms, or R/R non-APL AML or R/R HR-MDS: Tamibarotene and Daratumumab arm.
Data is presented as specified in the statistical analysis plan.
Response evaluable population included participants who completed 1 cycle of study treatment, and had a follow-up assessment of disease status, and did not have any major protocol violations, or withdrew from the study before completion of Cycle 1 because of documented disease progression. Overall Number of Participants analyzed (N) = participants evaluable for this Outcome Measure.
Posted
Median
95% Confidence Interval
months
Up to 48 months
ID
Title
Description
OG000
R/R Non-APL AML: Tamibarotene and Azacitidine
Participants with R/R non-APL AML received tamibarotene at 6 mg/m^2/day in 2 divided doses on Days 8-28 of a 28-day cycle, and azacitidine at 75 mg/m^2 once daily on Days 1-7 of a 28-day cycle.
OG001
ND Non-APL AML: Tamibarotene and Azacitidine: RARA-Positive
Participants from the "Newly Diagnosed Non-APL AML: Tamibarotene and Azacitidine" arm who were positive for the RARA super-enhancer associated biomarker (RARA-positive) were included in this subgroup analysis.
Participants who were unlikely to tolerate standard intensive chemotherapy received tamibarotene at 6 mg/m^2/day in 2 divided doses on Days 8-28 of a 28-day cycle, and azacitidine at 75 mg/m^2 once daily on Days 1-7 of a 28-day cycle.
Secondary
Overall Survival (OS) in AML and HR-MDS Participants Treated With Tamibarotene Monotherapy, or in Combination With Azacitidine or Daratumumab
OS was defined as the time from first treatment until death from any cause.
Per planned analysis, data were not collected for this Outcome Measure for the Newly Diagnosed Non-APL AML: Tamibarotene Monotherapy arm as there were fewer than 5 participants in the study arm. Additionally, per prespecified analysis, data were not collected for this Outcome Measure for the LR-MDS: Tamibarotene Monotherapy arm.
Data is presented as specified in the statistical analysis plan. All-cause mortality is reported in the Reported Adverse Events module.
Safety population included all screened participants who received any amount of study drug (tamibarotene or combination treatment).
Overall Number of Participants analyzed (N) = participants evaluable for this Outcome Measure.
Posted
Median
95% Confidence Interval
months
Up to 48 months
ID
Title
Description
OG000
R/R Non-APL AML or R/R HR-MDS: Tamibarotene Monotherapy
Participants with R/R non-APL AML or R/R HR-MDS received tamibarotene at 6 mg/m^2/day in 2 divided doses on Days 1-28 of a 28-day cycle.
OG001
R/R Non-APL AML: Tamibarotene and Azacitidine
Participants with R/R non-APL AML received tamibarotene at 6 mg/m^2/day in 2 divided doses on Days 8-28 of a 28-day cycle, and azacitidine at 75 mg/m^2 once daily on Days 1-7 of a 28-day cycle.
Secondary
Hematologic Improvement (HI) in AML, HR-MDS and LR-MDS Participants Treated With Tamibarotene Monotherapy, or in Combination With Azacitidine or Daratumumab
HI was defined according to the modified IWG response criteria for MDS as the number of participants with a response (lasting at least 8 weeks) after first treatment, including erythroid response, platelet response, or neutrophil response.
Data are presented per specifications in the statistical analysis plan.
Measured in the population evaluable for HI, which included all participants who received at least 8 weeks of study treatment.
Overall Number of Participants analyzed (N) = participants evaluable for this Outcome Measure.
Posted
Count of Participants
Participants
Up to 48 months
ID
Title
Description
OG000
R/R Non-APL AML or R/R HR-MDS: Tamibarotene Monotherapy
Participants with R/R non-APL AML or R/R HR-MDS received tamibarotene at 6 mg/m^2/day in 2 divided doses on Days 1-28 of a 28-day cycle.
Newly diagnosed, treatment-naive participants with non-APL AML who were unlikely to tolerate standard intensive chemotherapy received tamibarotene at 6 mg/m^2/day in 2 divided doses on Days 1-28 of a 28- day cycle.
OG002
LR-MDS: Tamibarotene Monotherapy
Secondary
Number of Participants With TEAEs in AML and MDS Participants Treated With Tamibarotene Monotherapy or in Combination With Azacitidine
A TEAE was any untoward medical occurrence associated with use of a study drug/study participation, whether or not considered related to study drug after first dose. A TEAE was any unfavorable/unintended sign (including abnormal laboratory finding), symptom, or disease temporally associated with the study drug. A serious TEAE resulted in death, was life-threatening, required inpatient hospitalization/prolongation of existing hospitalization, persistent or significant disability/incapacity, congenital anomaly/birth defect in the offspring of a participant who received study drug or other important medical events. A summary of serious and all other non-serious adverse events regardless of causality is located in the Reported Adverse Events module.
Safety population included all screened participants who received any amount of study drug (tamibarotene or combination treatment).
Posted
Count of Participants
Participants
Up to 48 months
ID
Title
Description
OG000
R/R Non-APL AML or R/R HR-MDS: Tamibarotene Monotherapy
Participants with R/R non-APL AML or R/R HR-MDS received tamibarotene at 6 mg/m^2/day in 2 divided doses on Days 1-28 of a 28-day cycle.
Newly diagnosed, treatment-naive participants with non-APL AML who were unlikely to tolerate standard intensive chemotherapy received tamibarotene at 6 mg/m^2/day in 2 divided doses on Days 1-28 of a 28- day cycle.
Time Frame
Up to 48 months
Description
Safety population included all screened participants who received any amount of study drug (tamibarotene or combination treatment).
All-Cause Mortality Comment
Not provided
Arm/Groups
ID
Title
Description
Deaths (Affected)
Deaths (At Risk)
Serious Events (Affected)
Serious Events (At Risk)
Other Events (Affected)
Other Events (At Risk)
EG000
R/R Non-APL AML or R/R HR-MDS: Tamibarotene Monotherapy
Participants with R/R non-APL AML or R/R HR-MDS received tamibarotene at 6 mg/m^2/day in 2 divided doses on Days 1-28 of a 28-day cycle.
Newly diagnosed, treatment-naive participants with non-APL AML who were unlikely to tolerate standard intensive chemotherapy received tamibarotene at 6 mg/m^2/day in 2 divided doses on Days 1-28 of a 28- day cycle.
2
2
2
2
2
2
EG002
LR-MDS: Tamibarotene Monotherapy
Participants with transfusion-dependent LR-MDS without the del- 5q abnormality who were refractory to EPO treatment or unlikely to respond to EPO treatment received tamibarotene at 6 mg/m^2/day in 2 divided doses from Days 1-28 of a 28-day cycle.
3
29
15
29
29
29
EG003
Newly Diagnosed Non-APL AML: Tamibarotene and Azacitidine
Newly diagnosed, treatment-naive participants with non-APL AML who were unlikely to tolerate standard intensive chemotherapy received tamibarotene at 6 mg/m^2/day in 2 divided doses on Days 8-28 of a 28-day cycle, and azacitidine at 75 mg/m^2 once daily on Days 1-7 of a 28-day cycle.
43
51
43
51
50
51
EG004
R/R Non-APL AML: Tamibarotene and Azacitidine
Participants with R/R non-APL AML received tamibarotene at 6 mg/m^2/day in 2 divided doses on Days 8-28 of a 28-day cycle, and azacitidine at 75 mg/m^2 once daily on Days 1-7 of a 28-day cycle.
24
28
20
28
27
28
EG005
R/R Non-APL AML or R/R HR-MDS: Tamibarotene and Daratumumab
Participants with R/R non-APL AML or R/R HR-MDS received tamibarotene at 6 mg/m^2/day in 2 divided doses during a 7-day lead-in and on Days 1-28 of a 28-day cycle. Participants also received daratumumab at 16 mg/kg starting on Cycle 1 Day 1 once weekly for 8 weeks, followed by dosing every 2 weeks for 16 weeks, followed by dosing every 4 weeks.
15
16
12
16
15
16
Serious Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Orthostatic hypotension
Vascular disorders
MedDRA 19.0
Systematic Assessment
EG0001 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG0030 affected51 at risk
EG0040 affected28 at risk
EG0050 affected16 at risk
Deep vein thrombosis
Vascular disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Vasculitis
Vascular disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Pyrexia
General disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0021 affected29 at risk
EG003
Multiple organ dysfunction syndrome
General disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Fatigue
General disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
General physical health deterioration
General disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Postmenopausal haemorrhage
Reproductive system and breast disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Hypoxia
Respiratory, thoracic and mediastinal disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0021 affected29 at risk
EG003
Pulmonary embolism
Respiratory, thoracic and mediastinal disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0022 affected29 at risk
EG003
Pulmonary granuloma
Respiratory, thoracic and mediastinal disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0021 affected29 at risk
EG003
Respiratory failure
Respiratory, thoracic and mediastinal disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Acute respiratory distress syndrome
Respiratory, thoracic and mediastinal disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Dyspnoea
Respiratory, thoracic and mediastinal disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Haemoptysis
Respiratory, thoracic and mediastinal disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Lung disorder
Respiratory, thoracic and mediastinal disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Pleural effusion
Respiratory, thoracic and mediastinal disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Pneumonitis
Respiratory, thoracic and mediastinal disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Respiratory distress
Respiratory, thoracic and mediastinal disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Delirium
Psychiatric disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0021 affected29 at risk
EG003
Mental status changes
Psychiatric disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Blood bilirubin increased
Investigations
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Fall
Injury, poisoning and procedural complications
MedDRA 19.0
Systematic Assessment
EG0001 affected29 at risk
EG0010 affected2 at risk
EG0023 affected29 at risk
EG003
Overdose
Injury, poisoning and procedural complications
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0021 affected29 at risk
EG003
Subdural haematoma
Injury, poisoning and procedural complications
MedDRA 19.0
Systematic Assessment
EG0001 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Femur fracture
Injury, poisoning and procedural complications
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Cardiac failure congestive
Cardiac disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0021 affected29 at risk
EG003
Cardiac arrest
Cardiac disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Acute myocardial infarction
Cardiac disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Angina pectoris
Cardiac disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Angina unstable
Cardiac disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Atrial fibrillation
Cardiac disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Cardiomyopathy
Cardiac disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Myocardial infarction
Cardiac disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Cranial nerve disorder
Nervous system disorders
MedDRA 19.0
Systematic Assessment
EG0001 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Dizziness
Nervous system disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0021 affected29 at risk
EG003
Hydrocephalus
Nervous system disorders
MedDRA 19.0
Systematic Assessment
EG0001 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Neuralgia
Nervous system disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0011 affected2 at risk
EG0020 affected29 at risk
EG003
Syncope
Nervous system disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Cerebral haemorrhage
Nervous system disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Cerebrovascular accident
Nervous system disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Headache
Nervous system disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Lacunar stroke
Nervous system disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Partial seizures
Nervous system disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Sciatica
Nervous system disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Anaemia
Blood and lymphatic system disorders
MedDRA 19.0
Systematic Assessment
EG0001 affected29 at risk
EG0010 affected2 at risk
EG0021 affected29 at risk
EG003
Febrile neutropenia
Blood and lymphatic system disorders
MedDRA 19.0
Systematic Assessment
EG0006 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Pancytopenia
Blood and lymphatic system disorders
MedDRA 19.0
Systematic Assessment
EG0001 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Thrombocytopenia
Blood and lymphatic system disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Leukocytosis
Blood and lymphatic system disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Leukostasis syndrome
Blood and lymphatic system disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Lymphopenia
Blood and lymphatic system disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Constipation
Gastrointestinal disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0021 affected29 at risk
EG003
Dysphagia
Gastrointestinal disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0021 affected29 at risk
EG003
Nausea
Gastrointestinal disorders
MedDRA 19.0
Systematic Assessment
EG0001 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Small intestinal obstruction
Gastrointestinal disorders
MedDRA 19.0
Systematic Assessment
EG0001 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Vomiting
Gastrointestinal disorders
MedDRA 19.0
Systematic Assessment
EG0001 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Abdominal pain
Gastrointestinal disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Colitis
Gastrointestinal disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Diarrhoea
Gastrointestinal disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Enteritis
Gastrointestinal disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Neutropenic colitis
Gastrointestinal disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Pancreatitis
Gastrointestinal disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Rectal haemorrhage
Gastrointestinal disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Stomatitis
Gastrointestinal disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Cholecystitis acute
Hepatobiliary disorders
MedDRA 19.0
Systematic Assessment
EG0001 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Cholelithiasis
Hepatobiliary disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0021 affected29 at risk
EG003
Dermatitis allergic
Skin and subcutaneous tissue disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0021 affected29 at risk
EG003
Acute kidney injury
Renal and urinary disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Renal failure
Renal and urinary disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Renal injury
Renal and urinary disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Urethral stenosis
Renal and urinary disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Back pain
Musculoskeletal and connective tissue disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0022 affected29 at risk
EG003
Bone pain
Musculoskeletal and connective tissue disorders
MedDRA 19.0
Systematic Assessment
EG0001 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Muscle haemorrhage
Musculoskeletal and connective tissue disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0021 affected29 at risk
EG003
Pain in extremity
Musculoskeletal and connective tissue disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0011 affected2 at risk
EG0020 affected29 at risk
EG003
Arthralgia
Musculoskeletal and connective tissue disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Flank pain
Musculoskeletal and connective tissue disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Groin pain
Musculoskeletal and connective tissue disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Musculoskeletal pain
Musculoskeletal and connective tissue disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Infection
Infections and infestations
MedDRA 19.0
Systematic Assessment
EG0001 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Lung infection
Infections and infestations
MedDRA 19.0
Systematic Assessment
EG0001 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Pharyngeal abscess
Infections and infestations
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0021 affected29 at risk
EG003
Pneumonia
Infections and infestations
MedDRA 19.0
Systematic Assessment
EG0001 affected29 at risk
EG0010 affected2 at risk
EG0024 affected29 at risk
EG003
Pneumonia fungal
Infections and infestations
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0011 affected2 at risk
EG0020 affected29 at risk
EG003
Pneumonia parainfluenzae viral
Infections and infestations
MedDRA 19.0
Systematic Assessment
EG0001 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Pneumonia staphylococcal
Infections and infestations
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0021 affected29 at risk
EG003
Pseudomonal sepsis
Infections and infestations
MedDRA 19.0
Systematic Assessment
EG0001 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Sepsis
Infections and infestations
MedDRA 19.0
Systematic Assessment
EG0002 affected29 at risk
EG0010 affected2 at risk
EG0023 affected29 at risk
EG003
Septic shock
Infections and infestations
MedDRA 19.0
Systematic Assessment
EG0001 affected29 at risk
EG0010 affected2 at risk
EG0021 affected29 at risk
EG003
Urinary tract infection
Infections and infestations
MedDRA 19.0
Systematic Assessment
EG0001 affected29 at risk
EG0010 affected2 at risk
EG0021 affected29 at risk
EG003
Wound infection
Infections and infestations
MedDRA 19.0
Systematic Assessment
EG0001 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Bacteraemia
Infections and infestations
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Cellulitis
Infections and infestations
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Device related infection
Infections and infestations
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Influenza
Infections and infestations
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Klebsiella bacteraemia
Infections and infestations
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Pseudomonas infection
Infections and infestations
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Pulmonary mycosis
Infections and infestations
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Arthritis infective
Infections and infestations
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Atypical pneumonia
Infections and infestations
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Diverticulitis
Infections and infestations
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Enterobacter pneumonia
Infections and infestations
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Erysipelas
Infections and infestations
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Escherichia bacteraemia
Infections and infestations
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Lung abscess
Infections and infestations
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Malaria
Infections and infestations
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Nocardiosis
Infections and infestations
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Oral candidiasis
Infections and infestations
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Proteus infection
Infections and infestations
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Pyelonephritis
Infections and infestations
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Serratia sepsis
Infections and infestations
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Skin infection
Infections and infestations
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Staphylococcal infection
Infections and infestations
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Streptococcal bacteraemia
Infections and infestations
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Dehydration
Metabolism and nutrition disorders
MedDRA 19.0
Systematic Assessment
EG0002 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Hyperkalaemia
Metabolism and nutrition disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0021 affected29 at risk
EG003
Lactic acidosis
Metabolism and nutrition disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0021 affected29 at risk
EG003
Failure to thrive
Metabolism and nutrition disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Decreased appetite
Metabolism and nutrition disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Gout
Metabolism and nutrition disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Hypercalcaemia
Metabolism and nutrition disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Hypertriglyceridaemia
Metabolism and nutrition disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Cardiac Failure
Cardiac disorders
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Other Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Fibrous histiocytoma
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA 19.0
Systematic Assessment
EG0001 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG0030 affected51 at risk
EG004
Superficial spreading melanoma stage unspecified
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0021 affected29 at risk
EG003
Anal cancer
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Endometrial cancer
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA 19.0
Systematic Assessment
EG0000 affected29 at risk
EG0010 affected2 at risk
EG0020 affected29 at risk
EG003
Squamous cell carcinoma of skin
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Note that the agreements are between the clinical sites and the Sponsor (or its agents), which restrict the clinical trial sites' rights to discuss or publish trial results after the trial is completed.
Participants from the "Newly Diagnosed Non-APL AML: Tamibarotene and Azacitidine" arm who were positive for the RARA super-enhancer associated biomarker (RARA-positive) were included in this subgroup analysis.
Participants who were unlikely to tolerate standard intensive chemotherapy received tamibarotene at 6 mg/m^2/day in 2 divided doses on Days 8-28 of a 28-day cycle, and azacitidine at 75 mg/m^2 once daily on Days 1-7 of a 28-day cycle.
Units
Counts
Participants
OG00021
OG00118
Title
Denominators
Categories
Title
Measurements
OG0004
OG00112
Units
Counts
Participants
OG0004
Title
Denominators
Categories
Title
Measurements
OG0002
Units
Counts
Participants
OG00028
Title
Denominators
Categories
Title
Measurements
OG00012
OG00012
Title
Denominators
Categories
Title
Measurements
OG0002
OG002
R/R Non-APL AML or R/R HR-MDS: Tamibarotene and Daratumumab
Participants with R/R non-APL AML or R/R HR-MDS received tamibarotene at 6 mg/m^2/day in 2 divided doses during a 7-day lead-in and on Days 1-28 of a 28-day cycle. Participants also received daratumumab at 16 mg/kg starting on Cycle 1 Day 1 once weekly for 8 weeks, followed by dosing every 2 weeks for 16 weeks, followed by dosing every 4 weeks.
OG003
ND Non-APL AML: Tamibarotene and Azacitidine: RARA-Positive
Participants from the "Newly Diagnosed Non-APL AML: Tamibarotene and Azacitidine" arm who were positive for the RARA super-enhancer associated biomarker (RARA-positive) were included in this subgroup analysis.
Participants who were unlikely to tolerate standard intensive chemotherapy received tamibarotene at 6 mg/m^2/day in 2 divided doses on Days 8-28 of a 28-day cycle, and azacitidine at 75 mg/m^2 once daily on Days 1-7 of a 28-day cycle.
OG004
ND Non-APL AML: Tamibarotene and Azacitidine: RARA-Negative
Participants from the "Newly Diagnosed Non-APL AML:Tamibarotene and Azacitidine" arm who were negative for the RARA super-enhancer associated biomarker (RARA-negative) were included in this subgroup analysis.
Participants who were unlikely to tolerate standard intensive chemotherapy received tamibarotene at 6 mg/m^2/day in 2 divided doses on Days 8-28 of a 28-day cycle, and azacitidine at 75 mg/m^2 once daily on Days 1-7 of a 28-day cycle.
Units
Counts
Participants
OG00029
OG00128
OG00216
OG00322
OG00429
Title
Denominators
Categories
Title
Measurements
OG0002.6(1.2 to 2.8)
OG0013.3(1.9 to 5.5)
OG0021.2(1.1 to 2.1)
OG0038.3(3.1 to 11.8)
OG0046.6(2.4 to 9.1)
OG002
ND Non-APL AML: Tamibarotene and Azacitidine: RARA-Negative
Participants from the "Newly Diagnosed Non-APL AML:Tamibarotene and Azacitidine" arm who were negative for the RARA super-enhancer associated biomarker (RARA-negative) were included in this subgroup analysis.
Participants who were unlikely to tolerate standard intensive chemotherapy received tamibarotene at 6 mg/m^2/day in 2 divided doses on Days 8-28 of a 28-day cycle, and azacitidine at 75 mg/m^2 once daily on Days 1-7 of a 28-day cycle.
Units
Counts
Participants
OG00021
OG00118
OG00228
Title
Denominators
Categories
Title
Measurements
OG0005.9(1.0 to NA)Upper confidence interval was not estimable by Brookmeyer and Crowley method due to insufficient observed events.
OG00110.8(2.9 to NA)Upper confidence interval was not estimable by Brookmeyer and Crowley method due to insufficient observed events.
OG0024.7(2.5 to 32.3)
OG002
R/R Non-APL AML or R/R HR-MDS: Tamibarotene and Daratumumab
Participants with R/R non-APL AML or R/R HR-MDS received tamibarotene at 6 mg/m^2/day in 2 divided doses during a 7-day lead-in and on Days 1-28 of a 28-day cycle. Participants also received daratumumab at 16 mg/kg starting on Cycle 1 Day 1 once weekly for 8 weeks, followed by dosing every 2 weeks for 16 weeks, followed by dosing every 4 weeks.
OG003
ND Non-APL AML: Tamibarotene and Azacitidine: RARA-Positive
Participants from the "Newly Diagnosed Non-APL AML: Tamibarotene and Azacitidine" arm who were positive for the RARA super-enhancer associated biomarker (RARA-positive) were included in this subgroup analysis.
Participants who were unlikely to tolerate standard intensive chemotherapy received tamibarotene at 6 mg/m^2/day in 2 divided doses on Days 8-28 of a 28-day cycle, and azacitidine at 75 mg/m^2 once daily on Days 1-7 of a 28-day cycle.
OG004
ND Non-APL AML: Tamibarotene and Azacitidine: RARA-Negative
Participants from the "Newly Diagnosed Non-APL AML:Tamibarotene and Azacitidine" arm who were negative for the RARA super-enhancer associated biomarker (RARA-negative) were included in this subgroup analysis.
Participants who were unlikely to tolerate standard intensive chemotherapy received tamibarotene at 6 mg/m^2/day in 2 divided doses on Days 8-28 of a 28-day cycle, and azacitidine at 75 mg/m^2 once daily on Days 1-7 of a 28-day cycle.
Units
Counts
Participants
OG00029
OG00128
OG00216
OG00322
OG00429
Title
Denominators
Categories
Title
Measurements
OG0005.7(2.7 to 7.1)
OG0015.9(2.7 to 11.0)
OG0023.6(1.8 to 4.6)
OG0038.4(5.2 to 15.6)
OG00411.7(6.6 to 15.8)
Participants with transfusion-dependent LR-MDS without the del-5q abnormality who were refractory to EPO treatment or unlikely to respond to EPO treatment received tamibarotene at 6 mg/m^2/day in 2 divided doses from Days 1-28 of a 28-day cycle.
OG003
R/R Non-APL AML: Tamibarotene and Azacitidine
Participants with R/R non-APL AML received tamibarotene at 6 mg/m^2/day in 2 divided doses on Days 8-28 of a 28-day cycle, and azacitidine at 75 mg/m^2 once daily on Days 1-7 of a 28-day cycle.
OG004
R/R Non-APL AML or R/R HR-MDS: Tamibarotene and Daratumumab
Participants with R/R non-APL AML or R/R HR-MDS received tamibarotene at 6 mg/m^2/day in 2 divided doses during a 7-day lead-in and on Days 1-28 of a 28-day cycle. Participants also received daratumumab at 16 mg/kg starting on Cycle 1 Day 1 once weekly for 8 weeks, followed by dosing every 2 weeks for 16 weeks, followed by dosing every 4 weeks.
OG005
ND Non-APL AML: Tamibarotene and Azacitidine: RARA-Positive
Participants from the "Newly Diagnosed Non-APL AML: Tamibarotene and Azacitidine" arm who were positive for the RARA super-enhancer associated biomarker (RARA-positive) were included in this subgroup analysis.
Participants who were unlikely to tolerate standard intensive chemotherapy received tamibarotene at 6 mg/m^2/day in 2 divided doses on Days 8-28 of a 28-day cycle, and azacitidine at 75 mg/m^2 once daily on Days 1-7 of a 28-day cycle.
OG006
ND Non-APL AML: Tamibarotene and Azacitidine: RARA-Negative
Participants from the "Newly Diagnosed Non-APL AML: Tamibarotene and Azacitidine" arm who were positive for the RARA super-enhancer associated biomarker (RARA-positive) were included in this subgroup analysis.
Participants who were unlikely to tolerate standard intensive chemotherapy received tamibarotene at 6 mg/m^2/day in 2 divided doses on Days 8-28 of a 28-day cycle, and azacitidine at 75 mg/m^2 once daily on Days 1-7 of a 28-day cycle.
Units
Counts
Participants
OG00016
OG0012
OG00225
OG00317
OG0049
OG00516
OG00619
Title
Denominators
Categories
Title
Measurements
OG0003
OG0010
OG0021
OG0031
OG0040
OG0054
OG0063
OG002
LR-MDS: Tamibarotene Monotherapy
Participants with transfusion-dependent lower-risk myelodysplastic syndrome (LR-MDS) without the del- 5q abnormality who were refractory to erythropoietin (EPO) treatment or unlikely to respond to EPO treatment received tamibarotene at 6 mg/m^2/day in 2 divided doses from Days 1-28 of a 28-day cycle.
OG003
Newly Diagnosed Non-APL AML: Tamibarotene and Azacitidine
Newly diagnosed, treatment-naive participants with non-APL AML who were unlikely to tolerate standard intensive chemotherapy received tamibarotene at 6 mg/m^2/day in 2 divided doses on Days 8-28 of a 28-day cycle, and azacitidine at 75 mg/m^2 once daily on Days 1-7 of a 28-day cycle.
OG004
R/R Non-APL AML: Tamibarotene and Azacitidine
Participants with R/R non-APL AML received tamibarotene at 6 mg/m^2/day in 2 divided doses on Days 8-28 of a 28-day cycle, and azacitidine at 75 mg/m^2 once daily on Days 1-7 of a 28-day cycle.