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| ID | Type | Description | Link |
|---|---|---|---|
| 2017-000318-40 | EudraCT Number | ||
| U1111-1189-8055 | Other Identifier | World Health Organization | |
| MOH_2018-02-04_002154 | Other Identifier | CRS | |
| JapicCTI-183848 | Registry Identifier | JapicCTI | |
| NCI-2017-02059 | Other Identifier | National Cancer Institute |
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| Name | Class |
|---|---|
| Takeda Development Center Americas, Inc. | INDUSTRY |
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The purpose of this study is to determine whether the combination of pevonedistat and azacitidine improves event-free survival (EFS) when compared with single-agent azacitidine. (An event is defined as death or transformation to AML in participants with MDS or CMML, whichever occurs first, and is defined as death in participants with low-blast AML).
The drug being tested in this study is called pevonedistat. Pevonedistat is being tested to treat people with higher-risk myelodysplastic syndromes (HR MDS), chronic myelomonocytic leukemia (CMML) and low-blast acute myelogenous leukemia (AML) as a combination treatment with azacitidine. This study will look at the overall survival, event-free survival and response to treatment in people who take pevonedistat and azacitidine when compared to people who take single-agent azacitidine.
The study will enroll approximately 450 participants. Once enrolled, participants will be randomly assigned in 1:1 ratio (by chance, like flipping a coin) to one of the two treatment groups in 28-day treatment cycles:
All participants will receive azacitidine via intravenous or subcutaneous route. Participants randomized to the combination arm will also receive pevonedistat intravenous infusion.
This multi-center trial will be conducted Spain, Belgium, Brazil, Canada, Czech Republic, France, Germany, Israel, Italy, the United States, Australia, Greece, Japan, Mexico, Poland, Russia, Korea, Turkey, China and United Kingdom. The overall time to participate in this study is approximately 63 months. Participants will attend the end-of-treatment visit 30 days after the last dose of study drug or before the start of subsequent anti-neoplastic therapy if that occurs sooner.
Participants with HR MDS or CMML will have EFS follow-up study visits every month if their disease has not transformed to AML and they have not started subsequent therapy. Participants with low-blast AML will have response follow-up study visits every month until they relapse from CR or meet the criteria for PD. All participants will enter OS follow-up (contacted every 3 months) when they have confirmed transformation to AML (for participants with HR MDS or CMML at enrollment) or experienced PD (for participants with low-blast AML at study enrollment).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Azacitidine 75 mg/m^2 | Experimental | Participants were administered azacitidine 75 milligram per square meter (mg/m^2) intravenous (IV) or subcutaneous (SC) injection on Days 1 to 5, Days 8 and 9, in 28-day treatment cycles until disease progression or unacceptable toxicity or up to a maximum of 63 cycles. |
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| Pevonedistat 20 mg/m^2 + Azacitidine 75 mg/m^2 | Experimental | Participants were administered azacitidine 75 mg/m^2 IV or SC injection on Days 1 to 5, Days 8 and 9 and pevonedistat 20 mg/m^2 IV infusion, on Days 1, 3, and 5 in 28-day treatment cycles until disease progression or unacceptable toxicity or up to a maximum of 63 cycles. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Azacitidine | Drug | Azacitidine intravenous or subcutaneous formulation. |
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| Measure | Description | Time Frame |
|---|---|---|
| Event-Free Survival (EFS) | EFS was defined as the time from randomization to the date of an EFS event. An EFS event was defined as death or transformation to acute myelogenous leukemia (AML) (World Health Organization [WHO] classification as a participant having greater than 20 % blasts in the blood or marrow and an increase of blast count by 50%), whichever event occurred first, in participants with myelodysplastic syndromes (MDS) or chronic myelomonocytic leukemias (CMML). An EFS event was defined as death in participants with low-blast AML. | From randomization until transformation to acute myeloid leukemia, or death due to any cause: up to approximately 42 months |
| Measure | Description | Time Frame |
|---|---|---|
| Overall Survival (OS) | Overall survival was defined as the time from randomization to death from any cause. | Up to approximately 6.9 years |
| Kaplan-Meier Estimates of Six-Month Survival Rate | Kaplan-Meier estimates for the probability (expressed as a percentage) of participants that survived at the end of Month 6 from randomization are presented. |
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Inclusion Criteria:
Has morphologically confirmed diagnosis of myelodysplastic syndromes (MDS) or CMML (i.e., with white blood cell [WBC] <13,000/microliter [mcL]) or low-blast acute myelogenous leukemia (AML).
Has MDS or CMML and must also have one of the following Prognostic Risk Categories, based on the Revised International Prognostic Scoring System (IPSS-R):
Eastern Cooperative Oncology Group (ECOG) status of 0, 1, or 2.
Participants with AML (20%-30% blasts) must have a treatment-related mortality (TRM) score >=4 for intensive, induction chemotherapy as calculated using the simplified model described by Walter and coworkers.
Calculation of TRM score:
Exclusion Criteria:
Has previous treatment for HR MDS or CMML or low-blast AML with chemotherapy or other antineoplastic agents including hypomethylating agent (HMAs) such as decitabine or azacitidine. Previous treatment is permitted with hydroxyurea and with lenalidomide, except that lenalidomide may not be given within 8 weeks before the first dose of study drug.
Has acute promyelocytic leukemia as diagnosed by morphologic examination of bone marrow, by fluorescent in situ hybridization or cytogenetics of peripheral blood or bone marrow, or by other accepted analysis.
Participants with AML with a WBC count >50,000/mcL. Participants who are cytoreduced with leukapheresis or with hydroxyurea may be enrolled if they meet the eligibility criteria.
Is eligible for intensive chemotherapy and/or allogeneic stem cell transplantation. The reason a participant is not eligible for intensive chemotherapy and/or allogeneic stem cell transplantation may consist of one or more of the following factors:
Has either clinical evidence of or history of central nervous system involvement by AML.
Has active uncontrolled infection or severe infectious disease, such as severe pneumonia, meningitis, or septicemia.
Is diagnosed or treated for another malignancy within 2 years before randomization or previously diagnosed with another malignancy and have any evidence of residual disease.
Has nonmelanoma skin cancer or carcinoma in situ of any type are not excluded if they have undergone resection.
Has prothrombin time (PT) or aPTT >1.5× upper limit of normal (ULN) or active uncontrolled coagulopathy or bleeding disorder. Participants therapeutically anticoagulated with warfarin, direct thrombin inhibitors, direct factor Xa inhibitors, or heparin are excluded from enrollment.
Has known human immunodeficiency virus (HIV) seropositive.
Has known hepatitis B surface antigen seropositive, or known or suspected active hepatitis C infection. Note: Participants who have isolated positive hepatitis B core antibody (i.e., in the setting of negative hepatitis B surface antigen and negative hepatitis B surface antibody) must have an undetectable hepatitis B viral load.
Has known hepatic cirrhosis or severe preexisting hepatic impairment.
Has known cardiopulmonary disease defined as unstable angina, clinically significant arrhythmia, congestive heart failure (New York Heart Association Class III or IV), and/or myocardial infarction within 6 months before first dose, or severe pulmonary hypertension.
Has treatment with strong cytochrome P 3A (CYP3A) inducers within 14 days before the first dose of pevonedistat.
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Takeda | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Southern Cancer Center - USOR | Daphne | Alabama | 36526 | United States | ||
| Southern Cancer Center - USOR |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 35728048 | Derived | Ades L, Girshova L, Doronin VA, Diez-Campelo M, Valcarcel D, Kambhampati S, Viniou NA, Woszczyk D, De Paz Arias R, Symeonidis A, Anagnostopoulos A, Munhoz EC, Platzbecker U, Santini V, Fram RJ, Yuan Y, Friedlander S, Faller DV, Sekeres MA. Pevonedistat plus azacitidine vs azacitidine alone in higher-risk MDS/chronic myelomonocytic leukemia or low-blast-percentage AML. Blood Adv. 2022 Sep 13;6(17):5132-5145. doi: 10.1182/bloodadvances.2022007334. |
| Label | URL |
|---|---|
| Related Info | View source |
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Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
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IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
Participants diagnosed with myelomonocytic, and myelogenous leukemia were randomized into two groups in 1:1 ratio to receive single-agent azacitidine or azacitidine + pevonedistat.
Participants took part in the study at 130 investigative sites globally from 28 November 2017 to 14 October 2024.
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| ID | Title | Description |
|---|---|---|
| FG000 | Azacitidine 75 mg/m^2 | Participants were administered azacitidine 75 milligram per square meter (mg/m^2) intravenous (IV) or subcutaneous (SC) injection on Days 1 to 5, Days 8 and 9, in 28-day treatment cycles until disease progression or unacceptable toxicity or up to a maximum of 63 cycles. |
| FG001 |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Sep 21, 2021 | Oct 10, 2025 |
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| Pevonedistat | Drug | Pevonedistat intravenous infusion. |
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| Month 6 |
| Kaplan-Meier Estimates of One-Year Survival Rate | Kaplan-Meier estimates for the probability (expressed as a percentage) of participants that survived at the end of the first year from randomization are presented. | Year 1 |
| Thirty-Day Mortality Reported as Number of Participants Who Died Up to Day 30 | 30-day mortality was defined as number of participants who died within 30 days from the first dose of study drug. | Up to Day 30 |
| Sixty-Day Mortality Reported as Number of Participants Who Died Up to Day 60 | 60-day mortality was defined as number of participants who died within 60 days from the first dose of study drug. | Up to Day 60 |
| Time to Acute Myelogenous Leukemia (AML) Transformation in Higher-Risk Myelodysplastic Syndromes (HR MDS), Higher-Risk Chronic Myelomonocytic Leukemias (HR CMML) and HR MDS/CMML Participants | Time to AML transformation in HR MDS and CMML participants was defined as time from randomization to documented AML transformation as determined by the independent review committee (IRC) assessment. Participants who died before progression to AML were censored. Transformation to AML was defined, according to WHO classification, as a participant having 20% blasts in the blood or marrow and increase of blast count by 50%. | From randomization until transformation to AML (up to approximately 42 months) |
| Number of Participants With Complete Remission (CR) and CR+ Complete Remission With Incomplete Blood Count Recovery (CRi) | CR for HR MDS or CMML is defined as <=5% myeloblasts with normal maturation of all cell lines in the bone marrow, and greater than or equal to >=11 gram per deciliter (g/dL) hemoglobin (Hgb),>=100*10^9/liter (/L) platelets (pl),>=1.0*10^9/L neutrophils and 0% blasts in peripheral blood. CR for low-blast AML: morphologic leukemia-free state, neutrophils of more than 1.0*10^9/L and pl of >=100*10^9/L, transfusion independence, and no residual evidence of extramedullary leukemia. CR with incomplete blood count recovery (CRi) for low-blast AML: participants fulfill all of the criteria for CR except for residual neutropenia (<1.0*10^9/L) or thrombocytopenia (pl<100*10^9/L). | From randomization until CR (up to approximately 42 months) |
| Number of Participants With CR and Marrow CR | Disease responses for HR MDS or CMML are based on the International Working Group (IWG) Response Criteria for MDS. CR for HR MDS or CMML is defined as <=5% myeloblasts with normal maturation of all cell lines in the bone marrow, and >=11 g/dL Hgb, >=100*10^9/L platelets (pl), >=1.0*10^9/L neutrophils and 0% blasts in peripheral blood. Marrow CR: Bone marrow: <=5% myeloblasts and decrease by >=50% over pretreatment. | From randomization until CR or marrow CR (up to approximately 42 months) |
| Number of Participants With CR, Partial Remission (PR) and Hematologic Improvement (HI) | Disease responses for HR MDS/CMML based on Modified IWG Response Criteria for MDS. CR: <=5% myeloblasts with normal maturation of all bone marrow cell lines, >=11 g/dL Hgb, >=100*10^9/L pl, >=1.0*10^9/L neutrophils,0% blasts in peripheral blood. Marrow CR: Bone marrow: <=5% myeloblasts and decrease by >=50% over pretreatment. PR: all CR criteria met except bone marrow blasts >=50% decrease over pretreatment but still >5%. HI: Hgb increase >=1.5 g/dL if <11 g/dL; pl increase >=30*10^9/L if baseline>20*10^9/L or increase from <20*10^9/L to >20*10^9/L and by at least 100%; neutrophil increase by 100% and absolute increase of >0.5*10^9/L if baseline <1.0*10^9/L. | From randomization until, CR, PR or HI (up to approximately 42 months) |
| Number of Participants With CR and Marrow CR and PR | Disease responses for HR MDS/CMML based on Modified IWG Response Criteria for MDS. CR: <=5% myeloblasts with normal maturation of all bone marrow cell lines, >=11 g/dL Hgb, >=100*10^9/L pl, >=1.0*10^9/L neutrophils,0% blasts in peripheral blood. Marrow CR: Bone marrow: <=5% myeloblasts and decrease by >=50% over pretreatment. PR: all CR criteria met except bone marrow blasts >=50% decrease over pretreatment but still >5%. | From randomization until CR or Marrow CR and PR (up to approximately 42 months) |
| Number of Participants With CR and Marrow CR, PR and Hematologic Improvement (HI) | Disease responses for HR MDS/CMML based on Modified IWG Response Criteria for MDS. CR: <=5% myeloblasts with normal maturation of all bone marrow cell lines, >=11 g/dL Hgb, >=100*10^9/L pl, >=1.0*10^9/L neutrophils,0% blasts in peripheral blood. Marrow CR: Bone marrow: <=5% myeloblasts and decrease by >=50% over pretreatment. PR: all CR criteria met except bone marrow blasts >=50% decrease over pretreatment but still>5%. HI: Hgb increase >=1.5 g/dL if baseline <11 g/dL; pl increase >=30*10^9/L if baseline>20*10^9/L or increases from <20*10^9/L to >20*10^9/L and by at least 100%; neutrophil increases by 100% and absolute increases of >0.5*10^9/L if baseline <1.0*10^9/L. | From randomization until CR, marrow CR, PR or HI (up to approximately 42 months) |
| Number of Participants With Overall Response (OR) | Disease responses for HR MDS/CMML based on Modified IWG Response Criteria for MDS; for low-blast AML on Revised IWG Response Criteria for AML. Overall response=CR or PR for HR MDS/CMML and CR + CRi + PR for low-blast AML. CR for HR MDS/CMML: <=5% myeloblasts with normal maturation of all bone marrow cell lines, >=11 g/dL Hgb, >=100*10^9/L pl, >=1.0*10^9/L neutrophils, 0% blasts in peripheral blood and PR: all CR criteria met except bone marrow blasts >=50% decrease over pretreatment but still>5%. For low-blast AML-CR:morphologic leukemia-free state>1.0*10^9 neutrophils, >=100*10^9/L pl, transfusion independence, no residual evidence of extramedullary leukemia; CR with incomplete blood count recovery (CRi): fulfill CR criteria except residual neutropenia <1.0*10^9/L or pl <100*10^9/L; PR: all CR hematological values but >=50% decrease in bone marrow aspirate. | From randomization until CR and PR or CR, CRi and PR (up to approximately 42 months) |
| Number of Participants With Overall Response 2 (OR2) | OR2=participant with best overall response of CR+PR+HI in HR MDS/CMML participants,or of CR+CRi+PR in low-blast AML participants.CR:≤5% myeloblasts with normal maturation of all bone marrow(BM)cell lines,≥11g/dL Hgb,≥100*10^9/L pl,≥1.0*10^9/L neutrophils,0% blasts in peripheral blood;PR:all CR criteria met except BM blasts ≥50% decrease over pretreatment but still >5%;HI:Hgb increase(inc) ≥1.5g/dL if baseline(BL)<11 g/dL;pl inc≥30*10^9/L if BL>20*10^9/L or inc from <20*10^9/L to >20*10^9/L and by 100%;neutrophil inc by 100%;absolute inc of >0.5*10^9/L if BL<100*10^9/L.For low-blast AML-CR:morphologic leukemia-free state >1.0*10^9 neutrophils,≥100*10^9/L pl,transfusion independence,no residual evidence of extramedullary leukemia;CR with incomplete blood count recovery:fulfill CR criteria except residual neutropenia <1.0*10^9/L or pl<100*10^9/L;PR:all CR hematological values but ≥50% decrease in BM aspirate.Number of responders determined by independent review committee(IRC) assessment. | From randomization until, CR, PR or HI or CR, CRi or PR (up to approximately 42 months) |
| Duration of Complete Remission (CR) | Duration of CR is first documented CR to the first documentation of PD or relapse from CR (participants with low-blast AML) or relapse after CR or PR (participants with HR MDS/CMML). Disease responses for HR MDS or CMML are based on the Modified IWG Response Criteria for MDS and for low-blast AML on the Revised IWG Response Criteria for AML. CR for HR MDS or CMML is defined as <=5% myeloblasts with normal maturation of all cell lines in the bone marrow, and greater than or equal to >=11 g/dL Hgb,>=100*10^9/L pl,>=1.0*10^9/L neutrophils and 0% blasts in peripheral blood. CR for low-blast AML: morphologic leukemia-free state, neutrophils of more than 1.0*10^9/L and pl of >=100*10^9/L, transfusion independence, and no residual evidence of extramedullary leukemia. CR with incomplete blood count recovery (CRi) for low-blast AML: participants fulfill all of the criteria for CR except for residual neutropenia (<1.0*10^9/L) or thrombocytopenia (pl<100*10^9/L). | From CR until first documentation of PD or relapse from CR or relapse after CR or PR (up to approximately 42 months) |
| Duration of Complete Remission + Complete Remission With Incomplete Blood Count Recovery (CRi) | Duration of CR is first documented CR to the first documentation of PD or relapse from CR (participants with low-blast AML). Disease responses for low-blast AML were based on the Revised IWG Response Criteria for AML. CR is defined as <=5% myeloblasts with normal maturation of all cell lines in the bone marrow, and greater than or equal to >=11 g/dL hemoglobin (Hgb),>=100*10^9/liter (/L) platelets (pl),>=1.0*10^9/L neutrophils and 0% blasts in peripheral blood. CR for low-blast AML: morphologic leukemia-free state, neutrophils of more than 1.0*10^9/L and pl of >=100*10^9/L, transfusion independence, and no residual evidence of extramedullary leukemia. CR with incomplete blood count recovery (CRi) for low-blast AML: participants fulfill all of the criteria for CR except for residual neutropenia (<1.0*10^9/L) or thrombocytopenia (pl<100*10^9/L). | From CR until first documentation of PD or relapse from CR or relapse after CR or PR (up to approximately 42 months) |
| Duration of Overall Response (OR) | Duration of OR: response to first documentation of PD or relapse from CR for low-blast AML or relapse after CR or PR for HR MDS/CMML. Disease responses for HR MDS/CMML based on Modified IWG Response Criteria for MDS; for low-blast AML on Revised IWG Response Criteria for AML. Overall response=CR+PR for HR MDS/CMML and CR+Cri+ PR for low-blast AML.CR for HR MDS/CMML:<=5% myeloblasts with normal maturation of all bone marrow cell lines,>=11 g/dL Hgb,>=100*10^9/L pl,>=1.0*10^9/L neutrophils,0% blasts in peripheral blood and PR:all CR criteria met except bone marrow blasts>=50% decrease over pretreatment but still >5%. For low-blast AML-CR: morphologic leukemia-free state >1.0*10^9 neutrophils,>=100*10^9/L pl, transfusion independence, no residual evidence of extramedullary leukemia; CR with CRi: fulfill CR criteria except residual neutropenia <1.0*10^9/L or pl <100*10^9/L; PR: all CR hematological values but >=50% decrease in % of blasts in bone marrow aspirate. | Up to approximately 42 months |
| Duration of Overall Response 2 (OR2) | Duration of OR2: from date of first documentation of CR+PR+HI to first documentation of PD/relapse after CR/PR for responders of CR+PR+HI for HR MDS/CMML and CR,CRi,PR for low-blast AML. For HR MDS/CMML-CR:<=5% myeloblasts with normal maturation of all bone marrow cell lines,>=11 g/dL Hgb, >=100*10^9/L pl,>=1.0*10^9/L neutrophils, 0% blasts in peripheral blood; PR: all CR criteria met except bone marrow blasts>=50% decrease over pretreatment, still >5%; HI:Hgb inc >=1.5 g/dL if baseline <11 g/dL; pl inc>=30*10^9/L if baseline>20*10^9/L or inc from<20*10^9/L to>20*10^9/L by at least 100%; neutrophil inc by 100% and absolute inc of >0.5*10^9/L if baseline <1.0*10^9/L.For low-blast AML-CR: morphologic leukemia-free state,>1.0*10^9 neutrophils,>=100*10^9/L pl, transfusion independence, no residual evidence of extramedullary leukemia;CRi: fulfill CR criteria except residual neutropenia<1.0*10^9/L or pl <100*10^9/L;PR:all CR hematological values but>=50% decrease in bone marrow aspirate. | Up to approximately 42 months |
| Percentage of Participants With Red Blood Cells (RBCs) and Platelet-transfusion Independence | A participant was defined as RBC or platelet-transfusion independent if he/she received no RBC or platelet transfusions for a period of at least 8 weeks before the first dose of study drug through 30 days after the last dose of any study drug. Rate of transfusion independence was defined as number of participants who became transfusion independent divided by the number of participants who were transfusion dependent at Baseline. | Up to approximately 42 months |
| Duration of Red Blood Cells (RBCs) and Duration of Platelet-transfusion Independence and Duration of Red Blood Cells (RBCs) and Platelet-transfusion Independence | Duration of RBC and platelet transfusion independence was defined as the longest time between the last RBC and/or platelet transfusion before the start of the RBC and/or platelet transfusion-independent period and the first RBC and/or platelet transfusion after the start of the transfusion-independent period, which occurs >= 8 weeks later. | Up to approximately 42 months |
| Time to First Complete Remission (CR) or Partial Remission (PR) or Complete Remission With Incomplete Blood Count Recovery (CRi) | Time to first CR or PR is defined as the time from randomization to first documented CR or PR, whichever occurs first. Disease responses for HR MDS or CMML or low-blast AML cycle 6 are based on Modified IWG Response Criteria for MDS and for low-blast AML on Revised IWG Response Criteria for AML. For HR MDS or CMML-CR:<=5% myeloblasts with normal maturation of all bone marrow cell lines,>=11 g/dL Hgb,>=100*10^9/L pl,>=1.0*10^9/L neutrophils, 0% blasts in peripheral blood; PR: all CR criteria met except bone marrow blasts>=50% decrease over pretreatment but still>5%; For low-blast AML-CR: morphologic leukemia-free state,>1.0*10^9/L neutrophils, pl>=100*10^9/L, transfusion independence, no residual evidence of extramedullary leukemia; CR with incomplete blood count recovery: fulfill CR criteria except residual neutropenia<1.0*10^9/L or pl <100*10^9/L; PR: all CR hematological values but with a decrease of at least 50% in the percentage of blasts to 5% to 25% in the bone marrow aspirate. | From randomization until CR or PR (up to approximately 42 months) |
| Number of Participants With Hematologic Improvement (HI) | Disease responses for HR MDS/CMML based on Modified IWG Response Criteria for MDS. HI: Hgb increase >=1.5 g/dL if baseline <11 g/dL; pl increase >=30*10^9/L if baseline >20*10^9/L or increase from <20*10^9/L to >20*10^9/L by at least 100%; neutrophil increase by 100% and absolute increase of >0.5*10^9/L if baseline <1.0*10^9/L. | From randomization until HI (up to approximately 42 months) |
| Number of Participants With at Least 1 Inpatient Hospital Admissions Related to HR MDS, CMML or Low-blast AML | Inpatient hospital admission data was collected through transformation to AML (HR MDS/CMML participants) or disease progression (low-blast AML participants) or until initiation of subsequent therapy (all participants), whichever occurred first. Transformation to AML is defined, according to WHO Classification, as a participant having 20% blasts in the blood or marrow and increase of blast count by 50%. | From randomization until transformation to AML or until initiation of subsequent therapy (up to approximately 42 months) |
| Time to Progressive Disease (PD), Relapse After CR (Low-blast AML), Relapse After CR or PR (HR MDS/CMML), or Death | Time to PD, relapse after CR(low-blast AML), relapse after CR or PR(HR MDS/CMML), or death,defined as time from date of randomization until date of first documentation of PD,relapse after CR(low-blast AML),relapse after CR or PR(HR MDS/CMML),or death due to any cause, whichever occurs first. In HR MDS/CMML,PD: Participants with<5% blasts:>=50% inc >5% blasts, with 5%-9% blasts:>=50% inc>10% blasts, with 10%-19% blasts:>=50% inc >20% blasts,with 20%-30% blasts, at least 50% decrement from maximum remission/response in granulocytes or pl or reduction in Hgb by>=2 g/dL/new transfusion dependence.Relapse after CR or PR: return to pretreatment bone marrow blast %/Decrement of >=50% from maximum remission/response levels in granulocytes/pl/reduction in Hgb conc.>=1.5 g/dL/transfusion dependence. In AML,PD:>50% inc in bone marrow blasts to >30% blasts,>50% inc in circulating blasts to>30% blasts in peripheral blood, Development of extramedullary disease/new sites of extramedullary leukemia. | From randomization until PD, relapse after CR, or relapse after CR or PR, or death due to any cause, whichever occurs first (up to approximately 42 months) |
| Change From Baseline in Health-Related Quality of Life (HRQOL) Using European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire- Core 30 | The EORTC QLQ-C30 contains 30 items across 5 functional scales (physical, role, cognitive, emotional, and social), 9 symptom scales (fatigue, nausea and vomiting, pain, dyspnea, sleep disturbance, appetite loss, constipation, diarrhea, and financial difficulties) and a global health status/QOL scale. Most of the 30 items have 4 response levels (not at all, a little, quite a bit, and very much), with 2 questions relying on a 7-point numeric rating scale. Raw scores are converted into scale scores ranging from 0 to 100. For the functional scales and the global health status/QOL scale, higher scores represent better QOL; for the symptom scales, lower scores represent better QOL. The change from baseline at end of treatment is reported. | Baseline, at approximately 58 months |
| Number of Participants With Overall Response in Participants Who Have TP53 Mutations, 17p Deletions, and/or Are Determined to be in an Adverse Cytogenetic Risk Group | Disease responses for HR MDS/CMML based on Modified IWG Response Criteria for MDS; for low-blast AML on Revised IWG Response Criteria for AML. Overall response=CR + PR for HR MDS/CMML and CR + CRi + PR for low-blast AML. CR for HR MDS/CMML: <=5% myeloblasts with normal maturation of all bone marrow cell lines, >=11 g/dL Hgb, >=100*10^9/L pl, >=1.0*10^9/L neutrophils,0% blasts in peripheral blood and PR: all CR criteria met except bone marrow blasts>=50% decrease over pretreatment but still>5%. For low-blast AML-CR: morphologic leukemia-free state, >1.0*10^9 neutrophils,>=100*10^9/L pl, transfusion independence, no residual evidence of extramedullary leukemia; CR with incomplete blood count recovery (CRi): fulfill CR criteria except residual neutropenia<1.0*10^9/L or pl<100*10^9/L; PR: all CR hematological values but>=50% decrease in the percentage of blasts to 5% to 25% in bone marrow aspirate. | From randomization until CR, CRi and PR (up to approximately 42 months) |
| Event-Free Survival in Participants Who Have TP53 Mutations, 17p Deletions, and/or Are Determined to be in an Adverse Cytogenetic Risk Group | Event was defined as death or transformation to AML in participants with MDS or CMML, whichever occurred first. Transformation to AML was defined, according to World Health Organization (WHO) Classification as a participant having >20% blasts in the blood or marrow and increase of blast count by 50%. Event was defined as death in participants with low-blast AML. | From randomization until transformation to AML if eligible or death (up to approximately 42 months) |
| Overall Survival in Participants Who Have TP53 Mutations, 17p Deletions, and/or Are Determined to be in an Adverse Cytogenetic Risk Group | OS was calculated from date of randomization to the date of death due to any cause. Participants without documented death at the time of the analysis were censored as of the date the participant was last known to be alive. | From randomization until death (up to approximately 42 months) |
| Number of Participants With Overall Response by Cycle 6 | Responses for HR MDS/CMML are based on Modified International Working Group (IWG) Response Criteria for MDS and for low-blast AML on Revised IWG Response Criteria for AML. Overall response=CR and PR for HR MDS/CMML and CR+CR with incomplete blood count recovery(CRi)+PR for low-blast AML. CR for HR MDS/CMML:<=5% myeloblasts with normal maturation of all bone marrow cell lines,>=11g/dL hemoglobin (Hgb),>=100*10^9/L platelet (pl),>=1.0*10^9/L neutrophils, 0% blasts in peripheral blood, and PR:all CR criteria met except bone marrow blasts >=50% decrease over pretreatment but still >5%. For low-blast AML-CR:morphologic leukemia-free state,>1.0*10^9 neutrophils, >=100*10^9/L pl, transfusion independence, no residual evidence of extramedullary leukemia;CRi:fulfill CR criteria except residual neutropenia <1.0*10^9/L/thrombocytopenia (pl<100*10^9/L); PR:all CR hematological values but>=50% decrease in percentage of blasts to 5%-25% in bone marrow aspirate. | Up to Cycle 6 (up to approximately Day 168) |
| Mobile |
| Alabama |
| 36607 |
| United States |
| Southern Cancer Center - USOR | Mobile | Alabama | 36608 | United States |
| Southern Cancer Center- USOR | Mobile | Alabama | 36608 | United States |
| Southeastern Regional Medical Center - CTCA - PPDS | Goodyear | Arizona | 85338 | United States |
| Arizona Oncology Associates (Orange HOPE) - USOR | Tucson | Arizona | 85704 | United States |
| Arizona Oncology Associates (Rudasill HOPE) - USOR | Tucson | Arizona | 85704 | United States |
| Arizona Oncology Associates (Wilmot HOPE) - USOR | Tucson | Arizona | 85711 | United States |
| Compassionate Care Research Group Inc. at Compassionate Cancer Care Medical Group, Inc | Corona | California | 92879 | United States |
| Compassionate Cancer Care Medical Group Inc | Fountain Valley | California | 92708 | United States |
| UC San Diego Moores Cancer Center | La Jolla | California | 92093 | United States |
| Emad Ibrahim, MD, Inc | Redlands | California | 92373 | United States |
| Compassionate Care Research Group Inc. at Compassionate Cancer Care Medical Group, Inc | Riverside | California | 92501 | United States |
| Rocky Mountain Cancer Centers (Aurora) - USOR | Aurora | Colorado | 80012 | United States |
| Rocky Mountain Cancer Centers (Boulder) - USOR | Boulder | Colorado | 80303 | United States |
| Rocky Mountain Cancer Centers (Colorado Springs) - USOR | Colorado Springs | Colorado | 80907 | United States |
| Quest Diagnostics, INC | Denver | Colorado | 80209 | United States |
| Colorado Blood Cancer Institute - PPDS | Denver | Colorado | 80218 | United States |
| Presbyterian Saint Lukes Medical Center Laboratory | Denver | Colorado | 80218 | United States |
| Presbyterian/St. Luke's Medical Center | Denver | Colorado | 80218 | United States |
| Rocky Mountain Cancer Centers (Williams) - USOR | Denver | Colorado | 80218 | United States |
| Rocky Mountain Cancer Centers (Denver) - USOR | Denver | Colorado | 80220 | United States |
| Kaiser Foundation Health Plan | Denver | Colorado | 80239 | United States |
| Laboratory Corporation of America | Englewood | Colorado | 80112 | United States |
| Rocky Mountain Cancer Centers (Lakewood) - USOR | Lakewood | Colorado | 80228 | United States |
| Rocky Mountain Cancer Centers (Littleton) - USOR | Littleton | Colorado | 80120 | United States |
| Rocky Mountain Cancer Centers (Lone Tree) - USOR | Lone Tree | Colorado | 80124 | United States |
| Rocky Mountain Cancer Centers (Longmont) - USOR | Longmont | Colorado | 80501 | United States |
| Rocky Mountain Cancer Centers (Parker) - USOR | Parker | Colorado | 80138 | United States |
| Rocky Mountain Cancer Centers (Pueblo) - USOR | Pueblo | Colorado | 81008 | United States |
| Rocky Mountain Cancer Centers (Thornton) - USOR | Thornton | Colorado | 80260 | United States |
| Medstar Research Institute | Washington D.C. | District of Columbia | 20010 | United States |
| Florida Cancer Specialists - NORTH - SCRI - PPDS | Altamonte Springs | Florida | 32701 | United States |
| SCRI Florida Cancer Specialists South | Bonita Springs | Florida | 34135 | United States |
| SCRI Florida Cancer Specialists South | Bradenton | Florida | 34209 | United States |
| Florida Cancer Specialists - NORTH - SCRI - PPDS | Brandon | Florida | 33511 | United States |
| SCRI Florida Cancer Specialists South | Cape Coral | Florida | 33914 | United States |
| Florida Cancer Specialists - NORTH - SCRI - PPDS | Clearwater | Florida | 33761 | United States |
| SCRI Florida Cancer Specialists South | Fort Myers | Florida | 33905 | United States |
| SCRI Florida Cancer Specialists South | Fort Myers | Florida | 33908 | United States |
| SCRI Florida Cancer Specialists South | Fort Myers | Florida | 33916 | United States |
| Florida Cancer Specialists - NORTH - SCRI - PPDS | Gainesville | Florida | 32605 | United States |
| Mayo Clinic Jacksonville - PPDS | Jacksonville | Florida | 32224 | United States |
| Florida Cancer Specialists - NORTH - SCRI - PPDS | Largo | Florida | 33770 | United States |
| Florida Cancer Specialists - NORTH - SCRI - PPDS | Lecanto | Florida | 34461 | United States |
| University of Miami Miller School of Medicine | Miami | Florida | 33136 | United States |
| Baptist Health System (N Kendall) - USOR | Miami | Florida | 33176 | United States |
| SCRI Florida Cancer Specialists South | Naples | Florida | 34102 | United States |
| Florida Cancer Specialists - NORTH - SCRI - PPDS | New Port Richey | Florida | 34655 | United States |
| Florida Cancer Specialists - NORTH - SCRI - PPDS | Ocala | Florida | 34471 | United States |
| Florida Cancer Specialists - NORTH - SCRI - PPDS | Orange City | Florida | 32763 | United States |
| Florida Cancer Specialists - NORTH - SCRI - PPDS | Orlando | Florida | 32806 | United States |
| SCRI Florida Cancer Specialists South | Port Charlotte | Florida | 33980 | United States |
| SCRI Florida Cancer Specialists South | Sarasota | Florida | 34232 | United States |
| SCRI Florida Cancer Specialists South | Sarasota | Florida | 34236 | United States |
| Florida Cancer Specialists - NORTH - SCRI - PPDS | Spring Hill | Florida | 34608 | United States |
| Florida Cancer Specialists - NORTH - SCRI - PPDS | St. Petersburg | Florida | 33705 | United States |
| Florida Cancer Specialists - NORTH - SCRI - PPDS | Tampa | Florida | 33607 | United States |
| Florida Cancer Specialists - NORTH - SCRI - PPDS | Tavares | Florida | 32778 | United States |
| Florida Cancer Specialists - NORTH - SCRI - PPDS | The Villages | Florida | 32159 | United States |
| SCRI Florida Cancer Specialists South | Venice | Florida | 34285 | United States |
| SCRI Florida Cancer Specialists South | Venice | Florida | 34292 | United States |
| Cleveland Clinic Florida | Weston | Florida | 33331 | United States |
| Florida Cancer Specialists - NORTH - SCRI - PPDS | Winter Park | Florida | 32792 | United States |
| Winship Cancer Institute, Emory University | Atlanta | Georgia | 30322 | United States |
| Saint Alphonsus Regional Medical Center | Boise | Idaho | 83706 | United States |
| Saint Alphonsus Caldwell Cancer Care Center | Caldwell | Idaho | 83605 | United States |
| Saint Alphonsus Medical Center | Nampa | Idaho | 83687 | United States |
| Rush University Medical Center | Chicago | Illinois | 60612 | United States |
| Menorah Medical Center | Overland Park | Kansas | 66209 | United States |
| Centerpoint Medical Center | Independence | Missouri | 64057 | United States |
| HCA Midwest Health - SCRI - PPDS | Kansas City | Missouri | 64132 | United States |
| Research Medical Center | Kansas City | Missouri | 64132 | United States |
| Nebraska Cancer Specialists | Omaha | Nebraska | 68130 | United States |
| New Jersey Hematology Oncology Associates LLC | Brick | New Jersey | 08724 | United States |
| John Theurer Cancer Center | Hackensack | New Jersey | 07601 | United States |
| New Jersey Hematology and Oncology | Toms River | New Jersey | 08755 | United States |
| Weill Cornell Medical Center - Monitoring Location | New York | New York | 10021 | United States |
| Weill Cornell Medical Center | New York | New York | 10021 | United States |
| Strong Memorial Hospital | Rochester | New York | 14642 | United States |
| University of North Carolina at Chapel Hill | Chapel Hill | North Carolina | 27599 | United States |
| Oncology Hematology Care, Inc. | Cincinnati | Ohio | 45211 | United States |
| Oncology Hematology Care, Inc. | Cincinnati | Ohio | 45230 | United States |
| Oncology Hematology Care Inc - USOR | Cincinnati | Ohio | 45236 | United States |
| Oncology Hematology Care Inc - USOR | Cincinnati | Ohio | 45242 | United States |
| Fairview Hospital | Cleveland | Ohio | 44111 | United States |
| The Cleveland Clinic Foundation | Cleveland | Ohio | 44195 | United States |
| Oncology Hematology Care, Inc - Fairfield | Fairfield | Ohio | 45014 | United States |
| Hillcrest Hospital Cancer Care Center | Mayfield | Ohio | 44124 | United States |
| St. Luke's Hospital | Bethlehem | Pennsylvania | 18015 | United States |
| St. Luke's University Health Network | Easton | Pennsylvania | 18045 | United States |
| Greenville Health System | Easley | South Carolina | 29640 | United States |
| Greenville Health System | Greenville | South Carolina | 29605 | United States |
| Greenville Health System Cancer Institute | Greenville | South Carolina | 29615 | United States |
| Greenville Health System | Greer | South Carolina | 29650 | United States |
| Greenville Health System | Seneca | South Carolina | 29672 | United States |
| Greenville Health System | Spartanburg | South Carolina | 29307 | United States |
| Tennessee Oncology - DICKSON - SCRI - PPDS | Dickson | Tennessee | 37055 | United States |
| Tennessee Oncology - FRANKLIN - SCRI - PPDS | Franklin | Tennessee | 37067 | United States |
| Tennessee Oncology - GALLATIN - SCRI - PPDS | Gallatin | Tennessee | 37066 | United States |
| Tennessee Oncology - SUMMIT - SCRI - PPDS | Hermitage | Tennessee | 37076 | United States |
| Tennessee Oncology - LEBANON - SCRI - PPDS | Lebanon | Tennessee | 37090 | United States |
| Tennessee Oncology - MURFREESBORO - SCRI - PPDS | Murfreesboro | Tennessee | 37129 | United States |
| Sarah Cannon Center for Blood Centers - SCRI - PPDS | Nashville | Tennessee | 37203 | United States |
| Tennessee Oncolgy - BAPTIST - SCRI - PPDS | Nashville | Tennessee | 37203 | United States |
| Tennessee Oncology NASH - SCRI - PPDS | Nashville | Tennessee | 37203 | United States |
| Tennessee Oncology - ST THOMAS WEST - SCRI - PPDS | Nashville | Tennessee | 37205 | United States |
| Tennessee Oncology SKYLINE - SCRI - PPDS | Nashville | Tennessee | 37207 | United States |
| Tennessee Oncology - SOUTHERN HILLS - SCRI - PPDS | Nashville | Tennessee | 37211 | United States |
| Tennessee Oncology - SHELBYVILLE - SCRI - PPDS | Shelbyville | Tennessee | 37160 | United States |
| Tennessee Oncology - SMYRNA - SCRI - PPDS | Smyrna | Tennessee | 37167 | United States |
| Texas Oncology (West 38) - USOR | Austin | Texas | 78705 | United States |
| Texas Oncology (Balcones) - USOR | Austin | Texas | 78731 | United States |
| Texas Oncology (James Casey) - USOR | Austin | Texas | 78745 | United States |
| Texas Oncology (Medical City) - USOR | Dallas | Texas | 75230 | United States |
| Baylor Sammons Cancer Center | Dallas | Texas | 75246 | United States |
| University of Texas Southwestern Medical Center | Dallas | Texas | 75390 | United States |
| Texas Oncology (Tyler) - USOR | Longview | Texas | 75601 | United States |
| Texas Oncology (E Common) - USOR | New Braunfels | Texas | 78130 | United States |
| Texas Oncology (Round Rock) - USOR | Round Rock | Texas | 78681 | United States |
| Texas Oncology - San Antonio Medical Center - USOR | San Antonio | Texas | 78240 | United States |
| Texas Oncology (Tyler) - USOR | Tyler | Texas | 75702 | United States |
| Oncology and Hematology Associates of Southwest Virginia (Blacksburg) - USOR | Blacksburg | Virginia | 24060 | United States |
| University of Virginia | Charlottesville | Virginia | 22903 | United States |
| Oncology and Hematology Associates of Southwest Virginia (Low Moor) - USOR | Low Moor | Virginia | 24457 | United States |
| Oncology and Hematology Associates of Southwest Virginia (Roanoke) - USOR | Roanoke | Virginia | 24014 | United States |
| Oncology and Hematology Associates of Southwest Virginia Inc | Salem | Virginia | 24153 | United States |
| Oncology and Hematology Associates of Southwest Virginia | Wytheville | Virginia | 24382 | United States |
| Icon Cancer Care Wesley | Auchenflower | Queensland | 4066 | Australia |
| Icon Cancer Care Chermside | Chermside | Queensland | 4032 | Australia |
| Icon Cancer Care South Brisbane | South Brisbane | Queensland | 4101 | Australia |
| Icon Cancer Care | South Brisbane | Queensland | 4101 | Australia |
| Icon Cancer Care Southport | Southport | Queensland | 4215 | Australia |
| Royal Hobart Hospital | Hobart | Tasmania | 7000 | Australia |
| Liverpool Hospital | Liverpool | 1871 | Australia |
| Algemeen Ziekenhuis Klina | Brasschaat | Antwerpen | 2930 | Belgium |
| CHU UCL Namur asbl - Site Godinne | Yvoir | Namur | 5530 | Belgium |
| AZ Sint-Jan AV | Bruges | West-Vlaanderen | 8000 | Belgium |
| Cliniques Universitaires Saint-Luc | Brussels | 1200 | Belgium |
| UZ Leuven | Leuven | 3000 | Belgium |
| Liga Paranaense de Combate Ao Cancer - Hospital Erasto Gaertner | Curitiba | Paraná | 81520-060 | Brazil |
| Liga Norte Riograndense Contra O Cancer | Natal | Rio Grande do Norte | 59075-740 | Brazil |
| Centro de Pesquisas Oncologicas | Florianópolis | Santa Catarina | 88034-000 | Brazil |
| Hospital de Clinicas de Porto Alegre (HCPA) - PPDS | Porto Alegre | 90035-903 | Brazil |
| Universidade Federal do Rio de Janeiro - UFRJ | Rio de Janeiro | 21941-913 | Brazil |
| Hospital Santa Marcelina | São Paulo | 08270-120 | Brazil |
| Hospital Santa Marcelina | São Paulo | 08270-270 | Brazil |
| Tom Baker Cancer Centre | Calgary | Alberta | T2N 4 | Canada |
| University of Alberta | Edmonton | Alberta | T6G 2B7 | Canada |
| Kaye Edmonton Clinic | Edmonton | Alberta | T6G1Z1 | Canada |
| Saint John Regional Hospital | Saint John | New Brunswick | E2L 4L2 | Canada |
| Sunnybrook Health Sciences Centre | Toronto | Ontario | M4N3M5 | Canada |
| Princess Margaret Hospital | Toronto | Ontario | M5G 2M9 | Canada |
| Princess Margaret Hospital | Toronto | Ontario | M5G2M9 | Canada |
| Xuanwu Hospital Capital Medical University | Beijing | Beijing Municipality | 100053 | China |
| Institute of Hematology and Blood Diseases Hospital Chinese Academy of Medical Sciences | Tianjin | Tianjin Municipality | 300020 | China |
| Fakultni nemocnice Hradec Kralove | Hradec Králové | Kralovehradeck Kraj | 500 05 | Czechia |
| Fakultni nemocnice Kralovske Vinohrady | Prague | 100 34 | Czechia |
| Vseobecna Fakultni Nemocnice V Praze | Prague | 128 08 | Czechia |
| Hopital Cote de Nacre | Caen | Calvados | 14033 | France |
| CHU Angers | Angers | Maine-et-Loire | 49100 | France |
| Centre Hospitalier Le Mans | Le Mans | 74000 | France |
| Hopital Saint Louis | Paris | 75010 | France |
| Hopital Saint Louis | Paris | 75475 | France |
| Universitatsklinikum Tubingen | Tübingen | Baden-Wurttemberg | 72076 | Germany |
| Universitatsklinikum Leipzig | Leipzig | Saxony | 4103 | Germany |
| Universitatsklinikum Carl Gustav Carus an der TU Dresden | Dresden | 1307 | Germany |
| Marien Hospital Akademisches Lehrkrankenhaus | Düsseldorf | 40479 | Germany |
| Laiko General Hospital of Athens | Athens | Attica | 115 27 | Greece |
| Athens General Hospital 'G Gennimatas' | Athens | Attica | 11527 | Greece |
| Attikon University General Hospital | Athens | Attica | 12462 | Greece |
| University Hospital of Alexandroupolis | Alexandroupoli | 68100 | Greece |
| Laiko General Hospital of Athens | Athens | 11527 | Greece |
| University General Hospital of Ioannina | Ioannina | 45500 | Greece |
| University General Hospital of Larissa | Larissa | 41110 | Greece |
| University General Hospital of Patras | Pátrai | 26504 | Greece |
| Georgios Papanikolaou General Hospital of Thessaloniki | Thessaloniki | 57010 | Greece |
| Edith Wolfson Medical Center | Holon | 58100 | Israel |
| Shaare Zedek Medical Center | Jerusalem | 91031 | Israel |
| Hadassah Medical Center PPDS - | Jerusalem | 91120 | Israel |
| Galilee Medical Center | Nahariya | Israel |
| ZIV Medical Center | Safed | 13100 | Israel |
| Tel Aviv Sourasky Medical Center PPDS | Tel Aviv | 64239 | Israel |
| Azienda Ospedaliero Universitaria Di Bologna - Policlinico S Orsola Malpighi | Bologna | Emilia-Romagna | 40138 | Italy |
| ASST degli Spedali Civili di Brescia - Spedali Civili di Brescia - INCIPIT - PIN | Brescia | Lombardy | 25123 | Italy |
| Azienda Ospedaliera Universitaria Careggi | Florence | 50134 | Italy |
| IRCCS Centro Di Riferimento Oncologico Della Basilicata | Rionero in Vulture | 85028 | Italy |
| Istituto Clinico Humanitas | Rozzano | 20089 | Italy |
| Azienda Ospedaliero Universitaria San Giovanni Battista Di Torino | Torino | 10126 | Italy |
| Japan Mutual Aid Association of Public School Teachers Chugoku Central Hospital | Fukuyama | Hiroshima | 720-0001 | Japan |
| Hokkaido University Hospital | Sapporo | Hokkaido | 060 8638 | Japan |
| Fukushima Medical University Hospital | Fukushima | Hukusima | 960-1295 | Japan |
| Kobe City Medical Center General Hospital | Kobe | Hyōgo | 650-0047 | Japan |
| University Hospital, Kyoto Prefectural University of Medicine | Kyoto | Kyoto | 602-8566 | Japan |
| Kindai University Hospital | Sayama | Osaka | 589-8511 | Japan |
| Saitama Medical Center | Kawagoe | Saitama | 3508550 | Japan |
| Juntendo University Hospital | Bunkyo | Tokyo | 113-8431 | Japan |
| NTT Medical Center Tokyo | Shinagawa-ku | Tokyo | 141-8625 | Japan |
| Kyushu University Hospital | Fukuoka | 812-8582 | Japan |
| Dokkyo Medical University Hospital | Mibu | 321-0293 | Japan |
| Nagasaki University Hospital | Nagasaki | 8528102 | Japan |
| Osaka Metropolitan University Hospital | Osaka | 545-8586 | Japan |
| Yokohama City University Hospital | Yokohama | 232-0024 | Japan |
| University of Fukui Hospital | Yoshida-gun | 910-1193 | Japan |
| Hematologica Alta Especialidad S.C. | Huixquilucan | 52787 | Mexico |
| Capital Humano para Investigacion Clinica SC | México | Mexico |
| Instytut Hematologii i Transfuzjologii | Warsaw | Masovian Voivodeship | 02-776 | Poland |
| Uniwersyteckie Centrum Kliniczne, Klinika Hematologii I Transplantologii, Budynek Centrum Medycyny N | Gdansk | Pomeranian Voivodeship | 80-214 | Poland |
| Uniwersyteckie Centrum Kliniczne | Gdansk | Pomeranian Voivodeship | 80-952 | Poland |
| Centrum Onkologii Ziemi Lubelskiej | Lublin | 20-090 | Poland |
| Zaklad Diagnostyki Obrazowej SOR | Opole | 45-061 | Poland |
| Szpital Wojewodzki w Opolu | Opole | 45-372 | Poland |
| Swietokrzyskie Centrum Onkologii | Kielce | Świętokrzyskie Voivodeship | 25-734 | Poland |
| City Clinical Hospital # 40 | Moscow | 129301 | Russia |
| Russian Research Institute of Hematology and Blood Transfusion | Saint Petersburg | 191024 | Russia |
| North-West Federal Medical Research Center n.a. V.A. Almazov | Saint Petersburg | 197341 | Russia |
| Pusan National University Hospital | Busan | 49241 | South Korea |
| Kyungpook National University Hospital | Daegu | 41944 | South Korea |
| Chonnam National University Hwasun Hospital | Jeongnam | 58128 | South Korea |
| Asan Medical Center - PPDS | Seoul | 5505 | South Korea |
| Samsung Medical Center - PPDS | Seoul | 6351 | South Korea |
| The Catholic University of Korea, Seoul St. Mary's Hospital | Seoul | 6591 | South Korea |
| ICO lHospitalet Hospital Duran i Reynals | LHospitalet de Llobregat | Barcelona | 8907 | Spain |
| Complejo Asistencial Universitario de Leon | León | Castille and León | 24071 | Spain |
| Hospital Universitario Vall d'Hebron - PPDS | Barcelona | 8035 | Spain |
| Hospital Universitario de La Princesa | Madrid | 28006 | Spain |
| Hospital General Universitario Gregorio Maranon | Madrid | 28009 | Spain |
| Hospital Universitario Ramon y Cajal | Madrid | 28034 | Spain |
| Hospital Universitario La Paz - PPDS | Madrid | 28046 | Spain |
| Complejo Asistencial Universitario de Salamanca - H. Clinico | Salamanca | 37007 | Spain |
| Hospital Clinico Universitario de Valencia | Valencia | 46010 | Spain |
| Hospital Universitari i Politecnic La Fe de Valencia | Valencia | 46026 | Spain |
| Gazi University Medical Faculty Gazi Hospital | Ankara | 6500 | Turkey (Türkiye) |
| Ege University Medical Faculty | Izmir | 35100 | Turkey (Türkiye) |
| Mersin University Medical Faculty | Mersin | 33343 | Turkey (Türkiye) |
| Ondokuz Mayis Universitesi Tip Fakultesi Hastanesi | Samsun | 55139 | Turkey (Türkiye) |
| Namik Kemal University | Tekirdağ | 59100 | Turkey (Türkiye) |
| Karadeniz Technical University Faculty of Medicine | Trabzon | 61080 | Turkey (Türkiye) |
| Royal Bournemouth Hospital | Bournemouth | Dorset | BH7 7DW | United Kingdom |
| Maidstone Hospital | Maidstone | Kent | ME16 9QQ | United Kingdom |
| St Bartholomew's Hospital | London | EC1A 7BE | United Kingdom |
| Singleton Hospital - PPDS | Swansea | SA2 8QA | United Kingdom |
| Pevonedistat 20 mg/m^2 + Azacitidine 75 mg/m^2 |
Participants were administered azacitidine 75 mg/m^2 IV or SC injection on Days 1 to 5, Days 8 and 9 and pevonedistat 20 mg/m^2 IV infusion, on Days 1, 3, and 5 in 28-day treatment cycles until disease progression or unacceptable toxicity or up to a maximum of 63 cycles. |
| Safety Population | Safety Population included all enrolled participants who received at least 1 dose of azacitidine alone or pevonedistat + azacitidine. |
|
| COMPLETED | Participants were considered to have completed the study if they were followed until death or until the sponsor terminated the study. |
|
| NOT COMPLETED |
|
|
ITT Population included all participants who were randomized.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Azacitidine 75 mg/m^2 | Participants were administered azacitidine 75 mg/m^2 IV or SC injection on Days 1 to 5, Days 8 and 9, in 28-day treatment cycles until disease progression or unacceptable toxicity or up to a maximum of 63 cycles. |
| BG001 | Pevonedistat 20 mg/m^2 + Azacitidine 75 mg/m^2 | Participants were administered azacitidine 75 mg/m^2 IV or SC injection on Days 1 to 5, Days 8 and 9 and pevonedistat 20 mg/m^2 IV infusion, on Days 1, 3, and 5 in 28-day treatment cycles until disease progression or unacceptable toxicity or up to a maximum of 63 cycles. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Region of Enrollment | Count of Participants | Participants |
| ||||||||||||||||
| Height | ITT Population included all participants who were randomized. Number analyzed is the number of participants with data available for height at Baseline. | Mean | Standard Deviation | centimeter (cm) |
| ||||||||||||||
| Weight | ITT Population included all participants who were randomized. Number analyzed is the number of participants with data available for weight at Baseline. | Mean | Standard Deviation | kilogram (kg) |
| ||||||||||||||
| Body Surface Area | Body surface area is defined as [height (cm) × weight (kg) / 3600]^1/2 | ITT Population included all participants who were randomized. Number analyzed is the number of participants with data available for body surface area at Baseline. | Mean | Standard Deviation | meter square (m^2) |
|
| 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 | Event-Free Survival (EFS) | EFS was defined as the time from randomization to the date of an EFS event. An EFS event was defined as death or transformation to acute myelogenous leukemia (AML) (World Health Organization [WHO] classification as a participant having greater than 20 % blasts in the blood or marrow and an increase of blast count by 50%), whichever event occurred first, in participants with myelodysplastic syndromes (MDS) or chronic myelomonocytic leukemias (CMML). An EFS event was defined as death in participants with low-blast AML. | ITT Population included all participants who were randomized. | Posted | Median | 95% Confidence Interval | months | From randomization until transformation to acute myeloid leukemia, or death due to any cause: up to approximately 42 months |
|
|
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Overall Survival (OS) | Overall survival was defined as the time from randomization to death from any cause. | ITT Population included all participants who were randomized. | Posted | Median | 95% Confidence Interval | months | Up to approximately 6.9 years |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Kaplan-Meier Estimates of Six-Month Survival Rate | Kaplan-Meier estimates for the probability (expressed as a percentage) of participants that survived at the end of Month 6 from randomization are presented. | ITT Population included all participants who were randomized. Overall number analyzed is the number of participants with data available for analyses. | Posted | Number | 95% Confidence Interval | percentage probability | Month 6 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Kaplan-Meier Estimates of One-Year Survival Rate | Kaplan-Meier estimates for the probability (expressed as a percentage) of participants that survived at the end of the first year from randomization are presented. | ITT Population included all participants who were randomized. Overall number analyzed is the number of participants with data available for analyses. | Posted | Number | 95% Confidence Interval | percentage probability | Year 1 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Thirty-Day Mortality Reported as Number of Participants Who Died Up to Day 30 | 30-day mortality was defined as number of participants who died within 30 days from the first dose of study drug. | Safety Population included all enrolled participants who received at least 1 dose of azacitidine alone or pevonedistat + azacitidine. | Posted | Count of Participants | Participants | Up to Day 30 |
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| Secondary | Sixty-Day Mortality Reported as Number of Participants Who Died Up to Day 60 | 60-day mortality was defined as number of participants who died within 60 days from the first dose of study drug. | Safety Population included all enrolled participants who received at least 1 dose of azacitidine alone or pevonedistat + azacitidine. | Posted | Count of Participants | Participants | Up to Day 60 |
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| Secondary | Time to Acute Myelogenous Leukemia (AML) Transformation in Higher-Risk Myelodysplastic Syndromes (HR MDS), Higher-Risk Chronic Myelomonocytic Leukemias (HR CMML) and HR MDS/CMML Participants | Time to AML transformation in HR MDS and CMML participants was defined as time from randomization to documented AML transformation as determined by the independent review committee (IRC) assessment. Participants who died before progression to AML were censored. Transformation to AML was defined, according to WHO classification, as a participant having 20% blasts in the blood or marrow and increase of blast count by 50%. | ITT Population included all participants who were randomized. Overall number analyzed is the number of participants with data available for analyses. Number analyzed is the number of participants with data available for analysis for the given category. | Posted | Median | 95% Confidence Interval | months | From randomization until transformation to AML (up to approximately 42 months) |
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| Secondary | Number of Participants With Complete Remission (CR) and CR+ Complete Remission With Incomplete Blood Count Recovery (CRi) | CR for HR MDS or CMML is defined as <=5% myeloblasts with normal maturation of all cell lines in the bone marrow, and greater than or equal to >=11 gram per deciliter (g/dL) hemoglobin (Hgb),>=100*10^9/liter (/L) platelets (pl),>=1.0*10^9/L neutrophils and 0% blasts in peripheral blood. CR for low-blast AML: morphologic leukemia-free state, neutrophils of more than 1.0*10^9/L and pl of >=100*10^9/L, transfusion independence, and no residual evidence of extramedullary leukemia. CR with incomplete blood count recovery (CRi) for low-blast AML: participants fulfill all of the criteria for CR except for residual neutropenia (<1.0*10^9/L) or thrombocytopenia (pl<100*10^9/L). | Response-Evaluable Population (REP) included all participants who received at least 1 dose of study drug and had a Baseline and at least 1 postbaseline disease assessment. | Posted | Count of Participants | Participants | From randomization until CR (up to approximately 42 months) |
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| Secondary | Number of Participants With CR and Marrow CR | Disease responses for HR MDS or CMML are based on the International Working Group (IWG) Response Criteria for MDS. CR for HR MDS or CMML is defined as <=5% myeloblasts with normal maturation of all cell lines in the bone marrow, and >=11 g/dL Hgb, >=100*10^9/L platelets (pl), >=1.0*10^9/L neutrophils and 0% blasts in peripheral blood. Marrow CR: Bone marrow: <=5% myeloblasts and decrease by >=50% over pretreatment. | REP included all participants who received at least 1 dose of study drug and had a Baseline and at least 1 postbaseline disease assessment. | Posted | Count of Participants | Participants | From randomization until CR or marrow CR (up to approximately 42 months) |
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| Secondary | Number of Participants With CR, Partial Remission (PR) and Hematologic Improvement (HI) | Disease responses for HR MDS/CMML based on Modified IWG Response Criteria for MDS. CR: <=5% myeloblasts with normal maturation of all bone marrow cell lines, >=11 g/dL Hgb, >=100*10^9/L pl, >=1.0*10^9/L neutrophils,0% blasts in peripheral blood. Marrow CR: Bone marrow: <=5% myeloblasts and decrease by >=50% over pretreatment. PR: all CR criteria met except bone marrow blasts >=50% decrease over pretreatment but still >5%. HI: Hgb increase >=1.5 g/dL if <11 g/dL; pl increase >=30*10^9/L if baseline>20*10^9/L or increase from <20*10^9/L to >20*10^9/L and by at least 100%; neutrophil increase by 100% and absolute increase of >0.5*10^9/L if baseline <1.0*10^9/L. | REP included all participants who received at least 1 dose of study drug and had a Baseline and at least 1 postbaseline disease assessment. | Posted | Count of Participants | Participants | From randomization until, CR, PR or HI (up to approximately 42 months) |
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| Secondary | Number of Participants With CR and Marrow CR and PR | Disease responses for HR MDS/CMML based on Modified IWG Response Criteria for MDS. CR: <=5% myeloblasts with normal maturation of all bone marrow cell lines, >=11 g/dL Hgb, >=100*10^9/L pl, >=1.0*10^9/L neutrophils,0% blasts in peripheral blood. Marrow CR: Bone marrow: <=5% myeloblasts and decrease by >=50% over pretreatment. PR: all CR criteria met except bone marrow blasts >=50% decrease over pretreatment but still >5%. | REP included all participants who received at least 1 dose of study drug and had a Baseline and at least 1 postbaseline disease assessment. | Posted | Count of Participants | Participants | From randomization until CR or Marrow CR and PR (up to approximately 42 months) |
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| Secondary | Number of Participants With CR and Marrow CR, PR and Hematologic Improvement (HI) | Disease responses for HR MDS/CMML based on Modified IWG Response Criteria for MDS. CR: <=5% myeloblasts with normal maturation of all bone marrow cell lines, >=11 g/dL Hgb, >=100*10^9/L pl, >=1.0*10^9/L neutrophils,0% blasts in peripheral blood. Marrow CR: Bone marrow: <=5% myeloblasts and decrease by >=50% over pretreatment. PR: all CR criteria met except bone marrow blasts >=50% decrease over pretreatment but still>5%. HI: Hgb increase >=1.5 g/dL if baseline <11 g/dL; pl increase >=30*10^9/L if baseline>20*10^9/L or increases from <20*10^9/L to >20*10^9/L and by at least 100%; neutrophil increases by 100% and absolute increases of >0.5*10^9/L if baseline <1.0*10^9/L. | REP included all participants who received at least 1 dose of study drug and had a Baseline and at least 1 postbaseline disease assessment. | Posted | Count of Participants | Participants | From randomization until CR, marrow CR, PR or HI (up to approximately 42 months) |
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| Secondary | Number of Participants With Overall Response (OR) | Disease responses for HR MDS/CMML based on Modified IWG Response Criteria for MDS; for low-blast AML on Revised IWG Response Criteria for AML. Overall response=CR or PR for HR MDS/CMML and CR + CRi + PR for low-blast AML. CR for HR MDS/CMML: <=5% myeloblasts with normal maturation of all bone marrow cell lines, >=11 g/dL Hgb, >=100*10^9/L pl, >=1.0*10^9/L neutrophils, 0% blasts in peripheral blood and PR: all CR criteria met except bone marrow blasts >=50% decrease over pretreatment but still>5%. For low-blast AML-CR:morphologic leukemia-free state>1.0*10^9 neutrophils, >=100*10^9/L pl, transfusion independence, no residual evidence of extramedullary leukemia; CR with incomplete blood count recovery (CRi): fulfill CR criteria except residual neutropenia <1.0*10^9/L or pl <100*10^9/L; PR: all CR hematological values but >=50% decrease in bone marrow aspirate. | REP included all participants who received at least 1 dose of study drug and had a Baseline and at least 1 postbaseline disease assessment. | Posted | Count of Participants | Participants | From randomization until CR and PR or CR, CRi and PR (up to approximately 42 months) |
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| Secondary | Number of Participants With Overall Response 2 (OR2) | OR2=participant with best overall response of CR+PR+HI in HR MDS/CMML participants,or of CR+CRi+PR in low-blast AML participants.CR:≤5% myeloblasts with normal maturation of all bone marrow(BM)cell lines,≥11g/dL Hgb,≥100*10^9/L pl,≥1.0*10^9/L neutrophils,0% blasts in peripheral blood;PR:all CR criteria met except BM blasts ≥50% decrease over pretreatment but still >5%;HI:Hgb increase(inc) ≥1.5g/dL if baseline(BL)<11 g/dL;pl inc≥30*10^9/L if BL>20*10^9/L or inc from <20*10^9/L to >20*10^9/L and by 100%;neutrophil inc by 100%;absolute inc of >0.5*10^9/L if BL<100*10^9/L.For low-blast AML-CR:morphologic leukemia-free state >1.0*10^9 neutrophils,≥100*10^9/L pl,transfusion independence,no residual evidence of extramedullary leukemia;CR with incomplete blood count recovery:fulfill CR criteria except residual neutropenia <1.0*10^9/L or pl<100*10^9/L;PR:all CR hematological values but ≥50% decrease in BM aspirate.Number of responders determined by independent review committee(IRC) assessment. | REP: all participants who received at least 1 dose of study drug and had a Baseline and at least 1 postbaseline disease assessment. Data is reported for responders. Three participants were not counted as responders for OR2 as the IRC assessed these participants as non-responders. | Posted | Count of Participants | Participants | From randomization until, CR, PR or HI or CR, CRi or PR (up to approximately 42 months) |
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| Secondary | Duration of Complete Remission (CR) | Duration of CR is first documented CR to the first documentation of PD or relapse from CR (participants with low-blast AML) or relapse after CR or PR (participants with HR MDS/CMML). Disease responses for HR MDS or CMML are based on the Modified IWG Response Criteria for MDS and for low-blast AML on the Revised IWG Response Criteria for AML. CR for HR MDS or CMML is defined as <=5% myeloblasts with normal maturation of all cell lines in the bone marrow, and greater than or equal to >=11 g/dL Hgb,>=100*10^9/L pl,>=1.0*10^9/L neutrophils and 0% blasts in peripheral blood. CR for low-blast AML: morphologic leukemia-free state, neutrophils of more than 1.0*10^9/L and pl of >=100*10^9/L, transfusion independence, and no residual evidence of extramedullary leukemia. CR with incomplete blood count recovery (CRi) for low-blast AML: participants fulfill all of the criteria for CR except for residual neutropenia (<1.0*10^9/L) or thrombocytopenia (pl<100*10^9/L). | REP included all participants who received at least 1 dose of study drug and had a Baseline and at least 1 postbaseline disease assessment. Data is reported for participants with complete remission. | Posted | Median | 95% Confidence Interval | months | From CR until first documentation of PD or relapse from CR or relapse after CR or PR (up to approximately 42 months) |
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| Secondary | Duration of Complete Remission + Complete Remission With Incomplete Blood Count Recovery (CRi) | Duration of CR is first documented CR to the first documentation of PD or relapse from CR (participants with low-blast AML). Disease responses for low-blast AML were based on the Revised IWG Response Criteria for AML. CR is defined as <=5% myeloblasts with normal maturation of all cell lines in the bone marrow, and greater than or equal to >=11 g/dL hemoglobin (Hgb),>=100*10^9/liter (/L) platelets (pl),>=1.0*10^9/L neutrophils and 0% blasts in peripheral blood. CR for low-blast AML: morphologic leukemia-free state, neutrophils of more than 1.0*10^9/L and pl of >=100*10^9/L, transfusion independence, and no residual evidence of extramedullary leukemia. CR with incomplete blood count recovery (CRi) for low-blast AML: participants fulfill all of the criteria for CR except for residual neutropenia (<1.0*10^9/L) or thrombocytopenia (pl<100*10^9/L). | REP included all participants who received at least 1 dose of study drug and had a Baseline and at least 1 postbaseline disease assessment. Data is reported for participants with CR and CRi. | Posted | Median | 95% Confidence Interval | months | From CR until first documentation of PD or relapse from CR or relapse after CR or PR (up to approximately 42 months) |
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| Secondary | Duration of Overall Response (OR) | Duration of OR: response to first documentation of PD or relapse from CR for low-blast AML or relapse after CR or PR for HR MDS/CMML. Disease responses for HR MDS/CMML based on Modified IWG Response Criteria for MDS; for low-blast AML on Revised IWG Response Criteria for AML. Overall response=CR+PR for HR MDS/CMML and CR+Cri+ PR for low-blast AML.CR for HR MDS/CMML:<=5% myeloblasts with normal maturation of all bone marrow cell lines,>=11 g/dL Hgb,>=100*10^9/L pl,>=1.0*10^9/L neutrophils,0% blasts in peripheral blood and PR:all CR criteria met except bone marrow blasts>=50% decrease over pretreatment but still >5%. For low-blast AML-CR: morphologic leukemia-free state >1.0*10^9 neutrophils,>=100*10^9/L pl, transfusion independence, no residual evidence of extramedullary leukemia; CR with CRi: fulfill CR criteria except residual neutropenia <1.0*10^9/L or pl <100*10^9/L; PR: all CR hematological values but >=50% decrease in % of blasts in bone marrow aspirate. | REP included all participants who received at least 1 dose of study drug and had a Baseline and at least 1 postbaseline disease assessment. Data is reported for responders. | Posted | Median | 95% Confidence Interval | months | Up to approximately 42 months |
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| Secondary | Duration of Overall Response 2 (OR2) | Duration of OR2: from date of first documentation of CR+PR+HI to first documentation of PD/relapse after CR/PR for responders of CR+PR+HI for HR MDS/CMML and CR,CRi,PR for low-blast AML. For HR MDS/CMML-CR:<=5% myeloblasts with normal maturation of all bone marrow cell lines,>=11 g/dL Hgb, >=100*10^9/L pl,>=1.0*10^9/L neutrophils, 0% blasts in peripheral blood; PR: all CR criteria met except bone marrow blasts>=50% decrease over pretreatment, still >5%; HI:Hgb inc >=1.5 g/dL if baseline <11 g/dL; pl inc>=30*10^9/L if baseline>20*10^9/L or inc from<20*10^9/L to>20*10^9/L by at least 100%; neutrophil inc by 100% and absolute inc of >0.5*10^9/L if baseline <1.0*10^9/L.For low-blast AML-CR: morphologic leukemia-free state,>1.0*10^9 neutrophils,>=100*10^9/L pl, transfusion independence, no residual evidence of extramedullary leukemia;CRi: fulfill CR criteria except residual neutropenia<1.0*10^9/L or pl <100*10^9/L;PR:all CR hematological values but>=50% decrease in bone marrow aspirate. | REP included all participants who received at least 1 dose of study drug and had a Baseline and at least 1 postbaseline disease assessment. Three participants who were non-responders (for OR2) as per the IRC were included in this outcome measure as they had assessment of HI and duration of OR2 for these participants could still be derived based on the pre-specified criteria for HR MDS/CMML participants. | Posted | Median | 95% Confidence Interval | months | Up to approximately 42 months |
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| Secondary | Percentage of Participants With Red Blood Cells (RBCs) and Platelet-transfusion Independence | A participant was defined as RBC or platelet-transfusion independent if he/she received no RBC or platelet transfusions for a period of at least 8 weeks before the first dose of study drug through 30 days after the last dose of any study drug. Rate of transfusion independence was defined as number of participants who became transfusion independent divided by the number of participants who were transfusion dependent at Baseline. | ITT Population included all participants who were randomized. Overall number analyzed is the number of participants from a subset of the ITT Population who were transfusion dependent at Baseline. Number analyzed is the number of participants who were transfusion dependent at Baseline for the specified category. | Posted | Number | percentage of participants | Up to approximately 42 months |
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| Secondary | Duration of Red Blood Cells (RBCs) and Duration of Platelet-transfusion Independence and Duration of Red Blood Cells (RBCs) and Platelet-transfusion Independence | Duration of RBC and platelet transfusion independence was defined as the longest time between the last RBC and/or platelet transfusion before the start of the RBC and/or platelet transfusion-independent period and the first RBC and/or platelet transfusion after the start of the transfusion-independent period, which occurs >= 8 weeks later. | ITT Population included all participants who were randomized. Number analyzed is the number of participants who achieved transfusion independence for the specified category, with data available for analysis. | Posted | Median | 95% Confidence Interval | months | Up to approximately 42 months |
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| Secondary | Time to First Complete Remission (CR) or Partial Remission (PR) or Complete Remission With Incomplete Blood Count Recovery (CRi) | Time to first CR or PR is defined as the time from randomization to first documented CR or PR, whichever occurs first. Disease responses for HR MDS or CMML or low-blast AML cycle 6 are based on Modified IWG Response Criteria for MDS and for low-blast AML on Revised IWG Response Criteria for AML. For HR MDS or CMML-CR:<=5% myeloblasts with normal maturation of all bone marrow cell lines,>=11 g/dL Hgb,>=100*10^9/L pl,>=1.0*10^9/L neutrophils, 0% blasts in peripheral blood; PR: all CR criteria met except bone marrow blasts>=50% decrease over pretreatment but still>5%; For low-blast AML-CR: morphologic leukemia-free state,>1.0*10^9/L neutrophils, pl>=100*10^9/L, transfusion independence, no residual evidence of extramedullary leukemia; CR with incomplete blood count recovery: fulfill CR criteria except residual neutropenia<1.0*10^9/L or pl <100*10^9/L; PR: all CR hematological values but with a decrease of at least 50% in the percentage of blasts to 5% to 25% in the bone marrow aspirate. | REP included all participants who received at least 1 dose of study drug and had a Baseline and at least 1 postbaseline disease assessment. | Posted | Median | 95% Confidence Interval | months | From randomization until CR or PR (up to approximately 42 months) |
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| Secondary | Number of Participants With Hematologic Improvement (HI) | Disease responses for HR MDS/CMML based on Modified IWG Response Criteria for MDS. HI: Hgb increase >=1.5 g/dL if baseline <11 g/dL; pl increase >=30*10^9/L if baseline >20*10^9/L or increase from <20*10^9/L to >20*10^9/L by at least 100%; neutrophil increase by 100% and absolute increase of >0.5*10^9/L if baseline <1.0*10^9/L. | REP included all participants who received at least 1 dose of study drug and had a baseline and at least 1 postbaseline disease assessment. Overall number analyzed is the number of participants with data available for analyses. | Posted | Count of Participants | Participants | From randomization until HI (up to approximately 42 months) |
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| Secondary | Number of Participants With at Least 1 Inpatient Hospital Admissions Related to HR MDS, CMML or Low-blast AML | Inpatient hospital admission data was collected through transformation to AML (HR MDS/CMML participants) or disease progression (low-blast AML participants) or until initiation of subsequent therapy (all participants), whichever occurred first. Transformation to AML is defined, according to WHO Classification, as a participant having 20% blasts in the blood or marrow and increase of blast count by 50%. | ITT Population included all participants who were randomized. | Posted | Count of Participants | Participants | From randomization until transformation to AML or until initiation of subsequent therapy (up to approximately 42 months) |
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| Secondary | Time to Progressive Disease (PD), Relapse After CR (Low-blast AML), Relapse After CR or PR (HR MDS/CMML), or Death | Time to PD, relapse after CR(low-blast AML), relapse after CR or PR(HR MDS/CMML), or death,defined as time from date of randomization until date of first documentation of PD,relapse after CR(low-blast AML),relapse after CR or PR(HR MDS/CMML),or death due to any cause, whichever occurs first. In HR MDS/CMML,PD: Participants with<5% blasts:>=50% inc >5% blasts, with 5%-9% blasts:>=50% inc>10% blasts, with 10%-19% blasts:>=50% inc >20% blasts,with 20%-30% blasts, at least 50% decrement from maximum remission/response in granulocytes or pl or reduction in Hgb by>=2 g/dL/new transfusion dependence.Relapse after CR or PR: return to pretreatment bone marrow blast %/Decrement of >=50% from maximum remission/response levels in granulocytes/pl/reduction in Hgb conc.>=1.5 g/dL/transfusion dependence. In AML,PD:>50% inc in bone marrow blasts to >30% blasts,>50% inc in circulating blasts to>30% blasts in peripheral blood, Development of extramedullary disease/new sites of extramedullary leukemia. | ITT Population included all participants who were randomized. | Posted | Median | 95% Confidence Interval | months | From randomization until PD, relapse after CR, or relapse after CR or PR, or death due to any cause, whichever occurs first (up to approximately 42 months) |
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| Secondary | Change From Baseline in Health-Related Quality of Life (HRQOL) Using European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire- Core 30 | The EORTC QLQ-C30 contains 30 items across 5 functional scales (physical, role, cognitive, emotional, and social), 9 symptom scales (fatigue, nausea and vomiting, pain, dyspnea, sleep disturbance, appetite loss, constipation, diarrhea, and financial difficulties) and a global health status/QOL scale. Most of the 30 items have 4 response levels (not at all, a little, quite a bit, and very much), with 2 questions relying on a 7-point numeric rating scale. Raw scores are converted into scale scores ranging from 0 to 100. For the functional scales and the global health status/QOL scale, higher scores represent better QOL; for the symptom scales, lower scores represent better QOL. The change from baseline at end of treatment is reported. | ITT Population included all participants who were randomized. All participants with PRO measurements at baseline and with data available at end of treatment were analyzed for this outcome measure. | Posted | Mean | Standard Deviation | score on a scale | Baseline, at approximately 58 months |
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| Secondary | Number of Participants With Overall Response in Participants Who Have TP53 Mutations, 17p Deletions, and/or Are Determined to be in an Adverse Cytogenetic Risk Group | Disease responses for HR MDS/CMML based on Modified IWG Response Criteria for MDS; for low-blast AML on Revised IWG Response Criteria for AML. Overall response=CR + PR for HR MDS/CMML and CR + CRi + PR for low-blast AML. CR for HR MDS/CMML: <=5% myeloblasts with normal maturation of all bone marrow cell lines, >=11 g/dL Hgb, >=100*10^9/L pl, >=1.0*10^9/L neutrophils,0% blasts in peripheral blood and PR: all CR criteria met except bone marrow blasts>=50% decrease over pretreatment but still>5%. For low-blast AML-CR: morphologic leukemia-free state, >1.0*10^9 neutrophils,>=100*10^9/L pl, transfusion independence, no residual evidence of extramedullary leukemia; CR with incomplete blood count recovery (CRi): fulfill CR criteria except residual neutropenia<1.0*10^9/L or pl<100*10^9/L; PR: all CR hematological values but>=50% decrease in the percentage of blasts to 5% to 25% in bone marrow aspirate. | ITT Population included all participants who were randomized. Overall number analyzed is the number of participants with data available for analyses. Number analyzed is the number of participants with data available for analysis for the specified category. | Posted | Count of Participants | Participants | From randomization until CR, CRi and PR (up to approximately 42 months) |
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| Secondary | Event-Free Survival in Participants Who Have TP53 Mutations, 17p Deletions, and/or Are Determined to be in an Adverse Cytogenetic Risk Group | Event was defined as death or transformation to AML in participants with MDS or CMML, whichever occurred first. Transformation to AML was defined, according to World Health Organization (WHO) Classification as a participant having >20% blasts in the blood or marrow and increase of blast count by 50%. Event was defined as death in participants with low-blast AML. | ITT Population included all participants who were randomized. | Posted | Median | 95% Confidence Interval | months | From randomization until transformation to AML if eligible or death (up to approximately 42 months) |
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| Secondary | Overall Survival in Participants Who Have TP53 Mutations, 17p Deletions, and/or Are Determined to be in an Adverse Cytogenetic Risk Group | OS was calculated from date of randomization to the date of death due to any cause. Participants without documented death at the time of the analysis were censored as of the date the participant was last known to be alive. | ITT Population included all participants who were randomized. | Posted | Median | 95% Confidence Interval | months | From randomization until death (up to approximately 42 months) |
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| Secondary | Number of Participants With Overall Response by Cycle 6 | Responses for HR MDS/CMML are based on Modified International Working Group (IWG) Response Criteria for MDS and for low-blast AML on Revised IWG Response Criteria for AML. Overall response=CR and PR for HR MDS/CMML and CR+CR with incomplete blood count recovery(CRi)+PR for low-blast AML. CR for HR MDS/CMML:<=5% myeloblasts with normal maturation of all bone marrow cell lines,>=11g/dL hemoglobin (Hgb),>=100*10^9/L platelet (pl),>=1.0*10^9/L neutrophils, 0% blasts in peripheral blood, and PR:all CR criteria met except bone marrow blasts >=50% decrease over pretreatment but still >5%. For low-blast AML-CR:morphologic leukemia-free state,>1.0*10^9 neutrophils, >=100*10^9/L pl, transfusion independence, no residual evidence of extramedullary leukemia;CRi:fulfill CR criteria except residual neutropenia <1.0*10^9/L/thrombocytopenia (pl<100*10^9/L); PR:all CR hematological values but>=50% decrease in percentage of blasts to 5%-25% in bone marrow aspirate. | REP included all participants who received at least 1 dose of study drug and had a Baseline and at least 1 post-baseline disease assessment. Number analyzed is the number of participants with data available for analysis for the specified category. | Posted | Count of Participants | Participants | Up to Cycle 6 (up to approximately Day 168) |
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From first dose of study drug up to end of study (up to approximately 6.9 years)
The Safety Population included all participants who received at least 1 dose of study drug (i.e., pevonedistat + azacitidine or single-agent azacitidine).
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Azacitidine 75 mg/m^2 | Participants were administered azacitidine 75 mg/m^2 IV or SC injection on Days 1 to 5, Days 8 and 9, in 28-day treatment cycles until disease progression or unacceptable toxicity or up to a maximum of 63 cycles. | 153 | 220 | 145 | 220 | 207 | 220 |
| EG001 | Pevonedistat 20 mg/m^2 + Azacitidine 75 mg/m^2 | Participants were administered azacitidine 75 mg/m^2 IV or SC injection on Days 1 to 5, Days 8 and 9 and pevonedistat 20 mg/m^2 IV infusion, on Days 1, 3, and 5 in 28-day treatment cycles until disease progression or unacceptable toxicity or up to a maximum of 63 cycles. | 150 | 223 | 156 | 223 | 210 | 223 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal infection | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
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| Abdominal sepsis | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
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| Abscess limb | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
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| Acute febrile neutrophilic dermatosis | Skin and subcutaneous tissue disorders | MedDRA 26.0 | Systematic Assessment |
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| Acute myeloid leukaemia | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 26.0 | Systematic Assessment |
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| Acute myocardial infarction | Cardiac disorders | MedDRA 26.0 | Systematic Assessment |
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| Acute pulmonary oedema | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
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| Acute respiratory distress syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
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| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Adenocarcinoma of colon | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 26.0 | Systematic Assessment |
| |
| Altered state of consciousness | Nervous system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Anaemia | Blood and lymphatic system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Anal abscess | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Angina pectoris | Cardiac disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Arrhythmia | Cardiac disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Arthritis infective | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Atrial fibrillation | Cardiac disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Atrial flutter | Cardiac disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Atrioventricular block complete | Cardiac disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Bacteraemia | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Basal cell carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 26.0 | Systematic Assessment |
| |
| Blood loss anaemia | Blood and lymphatic system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Bronchiectasis | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Bronchopulmonary aspergillosis | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| C-reactive protein increased | Investigations | MedDRA 26.0 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| COVID-19 pneumonia | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Cardiac failure | Cardiac disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Cardiac failure acute | Cardiac disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Cardiac failure congestive | Cardiac disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Cardio-respiratory arrest | Cardiac disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Cardiopulmonary failure | Cardiac disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Cardiovascular insufficiency | Cardiac disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Cataract | Eye disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Cellulitis staphylococcal | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Central nervous system haemorrhage | Nervous system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Central nervous system injury | Injury, poisoning and procedural complications | MedDRA 26.0 | Systematic Assessment |
| |
| Cerebral haemorrhage | Nervous system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Cerebral infarction | Nervous system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Cerebral ischaemia | Nervous system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Cerebrovascular accident | Nervous system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Cervical vertebral fracture | Injury, poisoning and procedural complications | MedDRA 26.0 | Systematic Assessment |
| |
| Chest wall haematoma | Musculoskeletal and connective tissue disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Cholecystitis | Hepatobiliary disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Cholecystitis acute | Hepatobiliary disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Chronic obstructive pulmonary disease | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Clostridium difficile colitis | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Clostridium difficile infection | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Colitis | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Colonic abscess | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Concussion | Injury, poisoning and procedural complications | MedDRA 26.0 | Systematic Assessment |
| |
| Confusional state | Psychiatric disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Conjunctival haemorrhage | Eye disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Craniocerebral injury | Injury, poisoning and procedural complications | MedDRA 26.0 | Systematic Assessment |
| |
| Deafness unilateral | Ear and labyrinth disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Death | General disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Dementia with Lewy bodies | Nervous system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Device related sepsis | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Disseminated intravascular coagulation | Blood and lymphatic system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Diverticulitis | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Duodenal ulcer | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Electrolyte imbalance | Metabolism and nutrition disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Encephalopathy | Nervous system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Enteritis | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Epididymitis | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Escherichia bacteraemia | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Escherichia infection | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Escherichia sepsis | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 26.0 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Febrile neutropenia | Blood and lymphatic system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Femur fracture | Injury, poisoning and procedural complications | MedDRA 26.0 | Systematic Assessment |
| |
| Fungal infection | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Gastric disorder | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Gastric haemorrhage | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Gastric ulcer haemorrhage | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Gastrointestinal angiodysplasia | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Gastrointestinal vascular malformation haemorrhagic | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| General physical health deterioration | General disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Gingival bleeding | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Glaucoma | Eye disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Guillain-Barre syndrome | Nervous system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Haemarthrosis | Musculoskeletal and connective tissue disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Haematemesis | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Haemoglobin decreased | Investigations | MedDRA 26.0 | Systematic Assessment |
| |
| Haemorrhage intracranial | Nervous system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Haemorrhagic stroke | Nervous system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Haemothorax | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Hand fracture | Injury, poisoning and procedural complications | MedDRA 26.0 | Systematic Assessment |
| |
| Head injury | Injury, poisoning and procedural complications | MedDRA 26.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Hepatic cyst infection | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Hepatic encephalopathy | Nervous system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Herpes zoster | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Hip fracture | Injury, poisoning and procedural complications | MedDRA 26.0 | Systematic Assessment |
| |
| Humerus fracture | Injury, poisoning and procedural complications | MedDRA 26.0 | Systematic Assessment |
| |
| Hydronephrosis | Renal and urinary disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Hydrothorax | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Hyperferritinaemia | Metabolism and nutrition disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Hypervolaemia | Metabolism and nutrition disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Hyphaema | Injury, poisoning and procedural complications | MedDRA 26.0 | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Hyponatraemia | Metabolism and nutrition disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Impaired gastric emptying | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Incarcerated inguinal hernia | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Infection | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Interstitial lung disease | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Intestinal haemorrhage | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Intestinal obstruction | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Intracranial haematoma | Nervous system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Ischaemic stroke | Nervous system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Klebsiella bacteraemia | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Leukocytosis | Blood and lymphatic system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Leukostasis syndrome | Blood and lymphatic system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Listeriosis | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Liver abscess | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Loss of consciousness | Nervous system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Lower gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Lower limb fracture | Injury, poisoning and procedural complications | MedDRA 26.0 | Systematic Assessment |
| |
| Lower respiratory tract infection | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Lower respiratory tract infection fungal | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Lung infiltration | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Lymphadenitis | Blood and lymphatic system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Malaise | General disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Malignant melanoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 26.0 | Systematic Assessment |
| |
| Malignant neoplasm progression | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 26.0 | Systematic Assessment |
| |
| Medical device site inflammation | General disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Melaena | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Mental status changes | Psychiatric disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Mouth haemorrhage | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Mucormycosis | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Multiple organ dysfunction syndrome | General disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Musculoskeletal chest pain | Musculoskeletal and connective tissue disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Myelodysplastic syndrome | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 26.0 | Systematic Assessment |
| |
| Myocardial infarction | Cardiac disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Nervous system disorder | Nervous system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Neutropenia | Blood and lymphatic system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Neutropenic colitis | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Neutropenic sepsis | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Neutrophil count decreased | Investigations | MedDRA 26.0 | Systematic Assessment |
| |
| Non-cardiac chest pain | General disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Occult blood | Investigations | MedDRA 26.0 | Systematic Assessment |
| |
| Organising pneumonia | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Overdose | Injury, poisoning and procedural complications | MedDRA 26.0 | Systematic Assessment |
| |
| Pancytopenia | Blood and lymphatic system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Paraparesis | Nervous system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Paronychia | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Perichondritis | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Peripheral arterial occlusive disease | Vascular disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Pharyngeal stenosis | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Phlebitis | Vascular disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Pneumocystis jirovecii pneumonia | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Pneumonia aspiration | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Pneumonia fungal | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Pneumonia legionella | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Pneumonia pseudomonal | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Pneumonia viral | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Pneumonitis | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Poisoning | Injury, poisoning and procedural complications | MedDRA 26.0 | Systematic Assessment |
| |
| Polypoid cystitis | Renal and urinary disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Postoperative wound infection | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Procedural haemorrhage | Injury, poisoning and procedural complications | MedDRA 26.0 | Systematic Assessment |
| |
| Procedural pain | Injury, poisoning and procedural complications | MedDRA 26.0 | Systematic Assessment |
| |
| Proctalgia | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Product contamination microbial | Product Issues | MedDRA 26.0 | Systematic Assessment |
| |
| Prostate cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 26.0 | Systematic Assessment |
| |
| Pseudomonal bacteraemia | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Pulmonary congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Pulmonary oedema | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Pulmonary sepsis | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Pure white cell aplasia | Blood and lymphatic system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Purpura | Skin and subcutaneous tissue disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Pyelonephritis acute | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Rectal abscess | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Rectal haemorrhage | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Refractoriness to platelet transfusion | Injury, poisoning and procedural complications | MedDRA 26.0 | Systematic Assessment |
| |
| Renal failure | Renal and urinary disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Renal impairment | Renal and urinary disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Septic shock | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Serratia bacteraemia | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Shock haemorrhagic | Vascular disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Silent myocardial infarction | Cardiac disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Skin infection | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Small intestinal ulcer haemorrhage | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Soft tissue infection | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Soft tissue injury | Injury, poisoning and procedural complications | MedDRA 26.0 | Systematic Assessment |
| |
| Soft tissue necrosis | Musculoskeletal and connective tissue disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Somnolence | Nervous system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Spinal fracture | Injury, poisoning and procedural complications | MedDRA 26.0 | Systematic Assessment |
| |
| Splenic infarction | Blood and lymphatic system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Spondylitis | Musculoskeletal and connective tissue disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Squamous cell carcinoma of skin | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 26.0 | Systematic Assessment |
| |
| Staphylococcal bacteraemia | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Staphylococcal sepsis | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Subarachnoid haemorrhage | Nervous system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Subdural haematoma | Injury, poisoning and procedural complications | MedDRA 26.0 | Systematic Assessment |
| |
| Subdural haemorrhage | Injury, poisoning and procedural complications | MedDRA 26.0 | Systematic Assessment |
| |
| Sudden death | General disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Thermal burn | Injury, poisoning and procedural complications | MedDRA 26.0 | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Thrombocytopenic purpura | Blood and lymphatic system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Thrombophlebitis septic | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Tooth abscess | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Tooth infection | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Transient ischaemic attack | Nervous system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Traumatic haemorrhage | Injury, poisoning and procedural complications | MedDRA 26.0 | Systematic Assessment |
| |
| Tumour associated fever | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 26.0 | Systematic Assessment |
| |
| Tumour lysis syndrome | Metabolism and nutrition disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Upper gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Upper limb fracture | Injury, poisoning and procedural complications | MedDRA 26.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Ureterolithiasis | Renal and urinary disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Urethral stenosis | Renal and urinary disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Visceral pain | General disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Vitreous haemorrhage | Eye disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Vulvitis | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| White blood cell count increased | Investigations | MedDRA 26.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal pain | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Abnormal loss of weight | Metabolism and nutrition disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 26.0 | Systematic Assessment |
| |
| Anaemia | Blood and lymphatic system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA 26.0 | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Blood bilirubin increased | Investigations | MedDRA 26.0 | Systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA 26.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA 26.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 26.0 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Febrile neutropenia | Blood and lymphatic system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Haemorrhoids | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Hyperuricaemia | Metabolism and nutrition disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Hypomagnesaemia | Metabolism and nutrition disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Hypophosphataemia | Metabolism and nutrition disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Injection site erythema | General disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Injection site pain | General disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Leukopenia | Blood and lymphatic system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Neutropenia | Blood and lymphatic system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Neutrophil count decreased | Investigations | MedDRA 26.0 | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Platelet count decreased | Investigations | MedDRA 26.0 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 26.0 | Systematic Assessment |
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| Pyrexia | General disorders | MedDRA 26.0 | Systematic Assessment |
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| Stomatitis | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
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| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA 26.0 | Systematic Assessment |
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| Upper respiratory tract infection | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
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| Urinary tract infection | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
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| White blood cell count decreased | Investigations | MedDRA 26.0 | Systematic Assessment |
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The first study related publication will be a multi-center publication submitted within 24 months after conclusion or termination of a study at all sites. After such multi site publication, all proposed site publications and presentations will be submitted to sponsor for review 60 days in advance of publication. Site will remove Sponsor confidential information unrelated to study results. Sponsor can delay a proposed publication for another 60 days to preserve intellectual property.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Takeda | +1-877-825-3327 | TrialDisclosures@takeda.com |
| Prot_002.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jun 16, 2021 | Oct 10, 2025 | SAP_003.pdf |
Not provided
| ID | Term |
|---|---|
| D009190 | Myelodysplastic Syndromes |
| D015477 | Leukemia, Myelomonocytic, Chronic |
| D015470 | Leukemia, Myeloid, Acute |
| ID | Term |
|---|---|
| D001855 | Bone Marrow Diseases |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D007951 | Leukemia, Myeloid |
| D007938 | Leukemia |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D054437 | Myelodysplastic-Myeloproliferative Diseases |
| D002908 | Chronic Disease |
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
Not provided
Not provided
| ID | Term |
|---|---|
| D001374 | Azacitidine |
| C539933 | pevonedistat |
| ID | Term |
|---|---|
| D001372 | Aza Compounds |
| D009930 | Organic Chemicals |
| D003562 | Cytidine |
| D011741 | Pyrimidine Nucleosides |
| D011743 | Pyrimidines |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
| D009705 | Nucleosides |
| D009706 | Nucleic Acids, Nucleotides, and Nucleosides |
| D012263 | Ribonucleosides |
Not provided
Not provided
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| Belgium |
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| Brazil |
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| China |
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| Czech Republic |
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| Germany |
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| Spain |
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| France |
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| United Kingdom |
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| Greece |
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| Israel |
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| Italy |
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| Japan |
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| Korea, Republic of |
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| Poland |
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| Russia |
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| Turkey |
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| Canada |
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| Mexico |
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| United States of America |
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| Units | Counts |
|---|---|
| Participants |
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| Units | Counts |
|---|---|
| Participants |
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Participants were administered azacitidine 75 mg/m^2 IV or SC injection on Days 1 to 5, Days 8 and 9 and pevonedistat 20 mg/m^2 IV infusion, on Days 1, 3, and 5 in 28-day treatment cycles until disease progression or unacceptable toxicity or up to a maximum of 63 cycles. |
|
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| OG001 | Pevonedistat 20 mg/m^2 + Azacitidine 75 mg/m^2 | Participants were administered azacitidine 75 mg/m^2 IV or SC injection on Days 1 to 5, Days 8 and 9 and pevonedistat 20 mg/m^2 IV infusion, on Days 1, 3, and 5 in 28-day treatment cycles until disease progression or unacceptable toxicity or up to a maximum of 63 cycles. |
|
|
|
| OG001 | Pevonedistat 20 mg/m^2 + Azacitidine 75 mg/m^2 | Participants were administered azacitidine 75 mg/m^2 IV or SC injection on Days 1 to 5, Days 8 and 9 and pevonedistat 20 mg/m^2 IV infusion, on Days 1, 3, and 5 in 28-day treatment cycles until disease progression or unacceptable toxicity or up to a maximum of 63 cycles. |
|
|
|
| Pevonedistat 20 mg/m^2 + Azacitidine 75 mg/m^2 |
Participants were administered azacitidine 75 mg/m^2 IV or SC injection on Days 1 to 5, Days 8 and 9 and pevonedistat 20 mg/m^2 IV infusion, on Days 1, 3, and 5 in 28-day treatment cycles until disease progression or unacceptable toxicity or up to a maximum of 63 cycles. |
|
|
|
Participants were administered azacitidine 75 mg/m^2 IV or SC injection on Days 1 to 5, Days 8 and 9 and pevonedistat 20 mg/m^2 IV infusion, on Days 1, 3, and 5 in 28-day treatment cycles until disease progression or unacceptable toxicity or up to a maximum of 63 cycles. |
|
|
|
| OG001 | Pevonedistat 20 mg/m^2 + Azacitidine 75 mg/m^2 | Participants were administered azacitidine 75 mg/m^2 IV or SC injection on Days 1 to 5, Days 8 and 9 and pevonedistat 20 mg/m^2 IV infusion, on Days 1, 3, and 5 in 28-day treatment cycles until disease progression or unacceptable toxicity or up to a maximum of 63 cycles. |
|
|
|
|
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
|
| OG001 | Pevonedistat 20 mg/m^2 + Azacitidine 75 mg/m^2 | Participants were administered azacitidine 75 mg/m^2 IV or SC injection on Days 1 to 5, Days 8 and 9 and pevonedistat 20 mg/m^2 IV infusion, on Days 1, 3, and 5 in 28-day treatment cycles until disease progression or unacceptable toxicity or up to a maximum of 63 cycles. |
|
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
|
| OG001 | Pevonedistat 20 mg/m^2 + Azacitidine 75 mg/m^2 | Participants were administered azacitidine 75 mg/m^2 IV or SC injection on Days 1 to 5, Days 8 and 9 and pevonedistat 20 mg/m^2 IV infusion, on Days 1, 3, and 5 in 28-day treatment cycles until disease progression or unacceptable toxicity or up to a maximum of 63 cycles. |
|
|
|
Participants were administered azacitidine 75 mg/m^2 IV or SC injection on Days 1 to 5, Days 8 and 9 and pevonedistat 20 mg/m^2 IV infusion, on Days 1, 3, and 5 in 28-day treatment cycles until disease progression or unacceptable toxicity or up to a maximum of 63 cycles. |
|
|
| OG001 | Pevonedistat 20 mg/m^2 + Azacitidine 75 mg/m^2 | Participants were administered azacitidine 75 mg/m^2 IV or SC injection on Days 1 to 5, Days 8 and 9 and pevonedistat 20 mg/m^2 IV infusion, on Days 1, 3, and 5 in 28-day treatment cycles until disease progression or unacceptable toxicity or up to a maximum of 63 cycles. |
|
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
|
| Participants |
|
|
|
| OG001 |
| Pevonedistat 20 mg/m^2 + Azacitidine 75 mg/m^2 |
Participants were administered azacitidine 75 mg/m^2 IV or SC injection on Days 1 to 5, Days 8 and 9 and pevonedistat 20 mg/m^2 IV infusion, on Days 1, 3, and 5 in 28-day treatment cycles until disease progression or unacceptable toxicity or up to a maximum of 63 cycles. |
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