Trial · NCT02610777
An Efficacy and Safety Study of Pevonedistat Plus Azacitidine Versus Single-Agent Azacitidine in Participants With Higher-Risk Myelodysplastic Syndromes (HR MDS), Chronic Myelomonocytic Leukemia (CMML) and Low-Blast Acute Myelogenous Leukemia (AML)
NCT02610777, Phase 2, Completed, Millennium Pharmaceuticals, Inc.; results first posted 2020-09-23
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. An Efficacy and Safety Study of Pevonedistat Plus Azacitidine Versus Single-Agent Azacitidine in Participants With Higher-Risk Myelodysplastic Syndromes (HR MDS), Chronic Myelomonocytic Leukemia (CMML) and Low-Blast Acute Myelogenous Leukemia (AML). Trials, record NCT02610777. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02610777
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
An Efficacy and Safety Study of Pevonedistat Plus Azacitidine Versus Single-Agent Azacitidine in Participants With Higher-Risk Myelodysplastic Syndromes (HR MDS), Chronic Myelomonocytic Leukemia (CMML) and Low-Blast Acute Myelogenous Leukemia (AML)
- NCT number
- NCT02610777
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 120 participants, actual
- Lead sponsor
- Millennium Pharmaceuticals, Inc.
- Conditions
- Myelodysplastic Syndromes, Leukemia, Myelomonocytic, Chronic, Leukemia, Myeloid, Acute
- Interventions
- Azacitidine, Pevonedistat
- First posted
- 2015-11-20
- Last updated
- 2022-09-19
- Other registries' IDs
- eudract:2015-000221-37
- Results first posted
- 2020-09-23
Source ClinicalTrials.gov
Registered outcomes27
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Overall Survival (OS) | From date of randomization until death (up to 3 years and 5 months) | OS was defined as the time from the date of randomization to the date of death due to any cause. Participants without documented death at the time of the analysis were censored at the date the participant was last known to be alive. The Kaplan Meier estimates was used for the analysis. |
| Secondary | Event-Free Survival (EFS) | From date of randomization until transformation to AML, or death due to any cause (up to approximately 5 years) | EFS is defined as the time from the date of randomization to the date of the occurrence of an event. An event is defined as death or transformation to AML for HR MDS/CMML participants, whichever occurs first, or defined as death for low-blast AML participants. HR MDS/CMML participants without documented EFS event will be censored at the date of the last response assessment. HR MDS/CMML participants with no response assessment and no death will be censored at the date of randomization. Low-blast AML participants without documentation of death will be censored at the date the participant was last known to be alive. HR MDS/CMML participants who received alternative antineoplastic therapy before death or transformation to AML will be censored at the date of last adequate assessment prior to starting alternate antineoplastic therapy. The Kaplan-Meier estimate was used for the analysis. |
| Secondary | Six-month Survival Rate | Month 6 | Kaplan-Meier estimate of probability of OS at the end of the month 6 from randomization. |
| Secondary | One-year Survival Rate | Month 12 | Kaplan-Meier estimate of probability of OS at the end of the first year from randomization. |
| Secondary | Time to AML Transformation in HR MDS or CMML Participants | From date of randomization until transformation to AML (up to approximately 5 years) | Time to AML transformation in HR MDS and CMML participants is defined as time from randomization to documented AML transformation. Participants without documented AML transformation at the time of the analysis are censored at the date of the last assessment. Participants who died before progression to AML are censored at the date of death. Transformation to AML is 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%. |
| Secondary | Percentage of Participants With Complete Remission (CR) | From date of randomization until CR (up to approximately 5 years) | Disease responses for HR MDS or CMML is based on the modified International Working Group (IWG) response criteria for MDS and for low-blast AML on the revised IWG response criteria for AML. CR for HR MDS or CMML: \<= 5% myeloblasts with normal maturation of all cell lines in the bone marrow, and \>= 11 gram/deciliter (g/dL) hemoglobin (Hb), \>=100\*10\^9/liter (/L) platelets (plt), \>=1.0\*10\^9/L absolute neutrophil count (ANC) and 0% blasts in peripheral blood. CR for low-blast AML: morphologic leukemia-free state, ANC of more than 1.0\*10\^9/L and plt of \>=1.0\*10\^9/L, transfusion independence, and no residual evidence of extramedullary leukemia. CR with incomplete blood count recovery for low-blast AML: some participants fulfill all of the criteria for CR except for residual neutropenia (\<1.0\*10\^9/L) or thrombocytopenia (TTP) (\<100\*10\^9/L). The percentages are rounded off to report the nearest whole numbers. |
| Secondary | Percentage of Participants With CR and Partial Remission (PR) | From date of randomization until CR and PR (up to approximately 5 years) | Disease responses for HR MDS/CMML per modified IWG response criteria for MDS and for low-blast AML on revised IWG response criteria for AML.CR for HR MDS/CMML: \<=5% myeloblasts with normal maturation of all cell lines in bone marrow ,\>=11 g/dL Hb;\>=100\*10\^9/L plt; \>=1.0\*10\^9/L ANC and 0% blasts in peripheral blood. PR for HR MDS/CMML:considered achieved if all CR criteria is met except for bone marrow blasts decreased by \>=50%over pretreatment but still \>5%. CR for low-blast AML: morphologic leukemia-free state, ANC of \>1.0\*10\^9/L and plt of \>=100\*10\^9/L, transfusion independence, no residual evidence of extramedullary leukemia. CR with incomplete blood count recovery for low-blast AML:fulfill all criteria for CR except for residual neutropenia (\<100\*10\^9/L)/TTP(\<100\*10\^9/L). PR for low-blast AML:all hematological values for CR but with decrease of \>=50% in percentage of blasts to 5%-25% in bone marrow aspirate. The percentages are rounded off to report the nearest whole numbers. |
| Secondary | Percentage of Participants With Overall Response | From date of randomization until CR, PR, or hematologic improvement (HI) (up to approximately 5 years) | Disease responses (HR MDS/CMML): modified IWG criteria for MDS; low-blast (LB) AML: revised IWG criteria for AML. Overall response(HR MDS/CMML)=CR,PR/HI,LB AML=CR+ CR with incomplete blood count recovery(Cri)+PR.HR MDS/CMML-CR:\<=5%myeloblasts with normal maturation of bone marrow (BM) cell lines,\>=11g/dL Hb,\>=100\*10\^9/L plt,\>=1.0\*10\^9/L ANC,0% blasts in peripheral blood; PR:CR criteria met except BM blasts\>=50%less over pretreatment but still\>5%; HI:hb increase (inc)\>=1.5g/dL if baseline\<11g/dL;plt inc\>=30\*10\^9/L if baseline\>20\*10\^9/L/Inc. from \<20\*10\^9/L-\>20\*10\^9/L, ANC inc. by 100%;absolute inc. of\>0.5\*10\^9/L if baseline\<100\*10\^9/L. LB AML-CR:morphologic leukemia-freestate\>1.0\*10\^9ANC,\>=100\*10\^9/L plt, transfusion independence, no residual evidence of extramedullary leukemia;CRi:fulfill CR criteria except residual neutropenia\<1.0\*10\^9/L/TTP\<100\*10\^9/L;PR:all CR hematological values but\>=50%less in BM aspirate. The percentages are rounded off to report the nearest whole numbers. |
| Secondary | Percentage of Participants With CR in Low-blast AML | From date of randomization until CR (up to approximately 5 years) | Disease response for low-blast AML is based on the revised IWG response criteria for AML. CR for low-blast AML: morphologic leukemia-free state, ANC of more than 1.0\*10\^9/L and plt of \>=1.0\*10\^9/L, transfusion independence, and no residual evidence of extramedullary leukemia. CR with incomplete blood count recovery for low-blast AML: some participants fulfill all of the criteria for CR except for residual neutropenia (\<1.0\*10\^9/L) or thrombocytopenia (TTP) (\<100\*10\^9/L). The percentages are rounded off to report the nearest whole numbers. |
| Secondary | Percentage of Participants With CR by Cycle 4 | From date of randomization until CR by Cycle 4 (cycle length is equal to [=] 28 days) | Disease responses for HR MDS or CMML were based on modified IWG response criteria for MDS and for low-blast AML on revised IWG response criteria for AML. CR for HR MDS or CMML: \<=5% myeloblasts with normal maturation of all cell lines in bone marrow, and \>=11 g/dL Hb, \>=100\*10\^9/L plt, ANC \>=1.0\*10\^9/L and 0% blasts in peripheral blood. CR for low-blast AML: morphologic leukemia-free state, ANC of more than 1.0\*10\^9/L and plt of \>=100\*10\^9/L, transfusion independence, no residual evidence of extramedullary leukemia. CR with incomplete blood count recovery for low-blast AML: some participants fulfill all criteria for CR except for residual neutropenia (\<1.0\*10\^9/L)/TTP (\<100\*10\^9/L). The percentages are rounded off to report the nearest whole numbers. |
1 to 10 of 27Source ClinicalTrials.gov
See all 27 registered outcomesOutcomes27
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Overall Survival (OS) | From date of randomization until death (up to 3 years and 5 months) | ITT Population was defined as all participants who were randomized. | months | MEDIAN |
| SECONDARY | Event-Free Survival (EFS) | From date of randomization until transformation to AML, or death due to any cause (up to approximately 5 years) | ITT Population was defined as all participants who were randomized. | months | MEDIAN |
| SECONDARY | Six-month Survival Rate | Month 6 | ITT Population was defined as all participants who were randomized. Overall number analyzed are the number of participants with data available for analyses. | survival probability | NUMBER |
| SECONDARY | One-year Survival Rate | Month 12 | ITT Population was defined as all participants who were randomized. Overall number analyzed are the number of participants with data available for analyses. | survival probability | NUMBER |
| SECONDARY | Time to AML Transformation in HR MDS or CMML Participants | From date of randomization until transformation to AML (up to approximately 5 years) | ITT Population was defined as all participants who were randomized. Overall number of participants analyzed is the number of participants with data available for analyses. | months | MEDIAN |
| SECONDARY | Percentage of Participants With Complete Remission (CR) | From date of randomization until CR (up to approximately 5 years) | 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. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With CR and Partial Remission (PR) | From date of randomization until CR and PR (up to approximately 5 years) | REP included all participants who received at least 1 dose of study drug and had a Baseline and at least 1 postbaseline disease assessment. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Overall Response | From date of randomization until CR, PR, or hematologic improvement (HI) (up to approximately 5 years) | REP included all participants who received at least 1 dose of study drug and had a Baseline and at least 1 postbaseline disease assessment. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With CR in Low-blast AML | From date of randomization until CR (up to approximately 5 years) | 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 are the number of participants with data available for analyses. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With CR by Cycle 4 | From date of randomization until CR by Cycle 4 (cycle length is equal to [=] 28 days) | 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. | percentage of participants | NUMBER |
1 to 10 of 27Source ClinicalTrials.gov
See all 27 outcomesResults by group84
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Overall Survival (OS) | Azacitidine 75 mg/m^2 | 19.0 | 95% Confidence Interval 13.57 to 27.73 | months | |
| Overall Survival (OS) | Azacitidine 75 mg/m^2 + Pevonedistat 20 mg/m^2 | 21.8 | 95% Confidence Interval 18.53 to 30.88 | months | |
| Event-Free Survival (EFS) | Azacitidine 75 mg/m^2 | 17.6 | 95% Confidence Interval 11.96 to 20.50 | months | |
| Event-Free Survival (EFS) | Azacitidine 75 mg/m^2 + Pevonedistat 20 mg/m^2 | 21.0 | 95% Confidence Interval 17.41 to 28.02 | months | |
| Six-month Survival Rate | Azacitidine 75 mg/m^2 | 0.806 | 95% Confidence Interval 0.684 to 0.885 | survival probability | |
| Six-month Survival Rate | Azacitidine 75 mg/m^2 + Pevonedistat 20 mg/m^2 | 0.914 | 95% Confidence Interval 0.805 to 0.963 | survival probability | |
| One-year Survival Rate | Azacitidine 75 mg/m^2 | 0.677 | 95% Confidence Interval 0.546 to 0.778 | survival probability | |
| One-year Survival Rate | Azacitidine 75 mg/m^2 + Pevonedistat 20 mg/m^2 | 0.845 | 95% Confidence Interval 0.723 to 0.916 | survival probability | |
| Time to AML Transformation in HR MDS or CMML Participants | Azacitidine 75 mg/m^2 | NA | 95% Confidence Interval NA to NA | months | |
| Time to AML Transformation in HR MDS or CMML Participants | Azacitidine 75 mg/m^2 + Pevonedistat 20 mg/m^2 | NA | 95% Confidence Interval NA to NA | months |
1 to 10 of 84Source ClinicalTrials.gov
See all 84 results by groupParticipant flow8
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Azacitidine 75 mg/m^2 | 62 | |
| Overall Study | STARTED | Azacitidine 75 mg/m^2 + Pevonedistat 20 mg/m^2 | 58 | |
| Overall Study | Response Evaluable Population (REP) | Azacitidine 75 mg/m^2 | 53 | |
| Overall Study | Response Evaluable Population (REP) | Azacitidine 75 mg/m^2 + Pevonedistat 20 mg/m^2 | 55 | |
| Overall Study | COMPLETED | Azacitidine 75 mg/m^2 | 0 | |
| Overall Study | COMPLETED | Azacitidine 75 mg/m^2 + Pevonedistat 20 mg/m^2 | 0 | |
| Overall Study | NOT COMPLETED | Azacitidine 75 mg/m^2 | 62 | |
| Overall Study | NOT COMPLETED | Azacitidine 75 mg/m^2 + Pevonedistat 20 mg/m^2 | 58 |
Source ClinicalTrials.gov
Adverse events402
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Abdominal pain | Gastrointestinal disorders | Azacitidine 75 mg/m^2 | 10 | 62 | |
| other | Abdominal pain | Gastrointestinal disorders | Azacitidine 75 mg/m^2 + Pevonedistat 20 mg/m^2 | 3 | 58 | |
| other | Acute kidney injury | Renal and urinary disorders | Azacitidine 75 mg/m^2 | 0 | 62 | |
| other | Acute kidney injury | Renal and urinary disorders | Azacitidine 75 mg/m^2 + Pevonedistat 20 mg/m^2 | 4 | 58 | |
| other | Alanine aminotransferase increased | Investigations | Azacitidine 75 mg/m^2 | 4 | 62 | |
| other | Alanine aminotransferase increased | Investigations | Azacitidine 75 mg/m^2 + Pevonedistat 20 mg/m^2 | 4 | 58 | |
| other | Anaemia | Blood and lymphatic system disorders | Azacitidine 75 mg/m^2 | 27 | 62 | |
| other | Anaemia | Blood and lymphatic system disorders | Azacitidine 75 mg/m^2 + Pevonedistat 20 mg/m^2 | 19 | 58 | |
| other | Arthralgia | Musculoskeletal and connective tissue disorders | Azacitidine 75 mg/m^2 | 12 | 62 | |
| other | Arthralgia | Musculoskeletal and connective tissue disorders | Azacitidine 75 mg/m^2 + Pevonedistat 20 mg/m^2 | 6 | 58 |
1 to 10 of 402Source ClinicalTrials.gov
See all 402 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Azacitidine 75 mg/m^2 | 50 | 62 |
| Total, all-cause mortality | Azacitidine 75 mg/m^2 + Pevonedistat 20 mg/m^2 | 47 | 58 |
| Total, other adverse events | Azacitidine 75 mg/m^2 | 58 | 62 |
| Total, other adverse events | Azacitidine 75 mg/m^2 + Pevonedistat 20 mg/m^2 | 51 | 58 |
| Total, serious adverse events | Azacitidine 75 mg/m^2 | 40 | 62 |
| Total, serious adverse events | Azacitidine 75 mg/m^2 + Pevonedistat 20 mg/m^2 | 40 | 58 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 33483617 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Azacitidine 75 mg/m^2 vs Azacitidine 75 mg/m^2 + Pevonedistat 20 mg/m^2: deaths from any cause | Trial comparisons |
| Azacitidine 75 mg/m^2 vs Azacitidine 75 mg/m^2 + Pevonedistat 20 mg/m^2: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
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