Last updated on May 2019

Pevonedistat Plus Azacitidine Versus Single-Agent Azacitidine as First-Line Treatment for Participants With Higher-Risk Myelodysplastic Syndromes (HR MDS) Chronic Myelomonocytic Leukemia (CMML) or Low-Blast Acute Myelogenous Leukemia (AML)


Brief description of study

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).

Detailed Study Description

The drug being tested in this study is called pevonedistat. Pevonedistat is being tested to treat people with higher-risk myelodysplastic syndromes (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:

  • Pevonedistat 20 mg/m^2 and azacitidine 75 mg/m^2 combination
  • Single-agent azacitidine 75 mg/m^2

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 worldwide. 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 or relapse from CR (for participants with low-blast AML at study enrollment).

Clinical Study Identifier: NCT03268954

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Southern Cancer Center

Daphne, AL United States
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Southern Cancer Center

Mobile, AL United States
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Emad Ibrahim, MD, Inc

Redlands, CA United States
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Quest Diagnostics, INC

Denver, CO United States
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Medstar Research Institute

Washington, WA United States
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SCRI Florida Cancer Specialists North

Altamonte Springs, FL United States
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Bonita Springs, FL United States
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Mayo Clinic Jacksonville - PPDS

Jacksonville, FL United States
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SCRI Florida Cancer Specialists North

New Port Richey, FL United States
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SCRI Florida Cancer Specialists South

Port Charlotte, FL United States
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SCRI Florida Cancer Specialists North

Saint Petersburg, FL United States
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Cleveland Clinic Florida

Weston, FL United States
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Menorah Medical Center

Overland Park, KS United States
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Centerpoint Medical Center

Independence, MO United States
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Research Medical Center

Kansas City, MO United States
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SCRI HCA Midwest Health

Kansas City, MO United States
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John Theurer Cancer Center

Hackensack, NJ United States
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Weill Cornell Medical Center

New York, NY United States
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Strong Memorial Hospital

Rochester, NY United States
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Oncology Hematology Care, Inc.

Cincinnati, OH United States
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Fairview Hospital

Cleveland, OH United States
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Cleveland Clinic

Cleveland, OH United States
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Oncology Hematology Care, Inc

Fairfield, OH United States
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St. Luke's Hospital

Bethlehem, PA United States
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Greenville Health System

Easley, SC United States
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Greenville Health System

Greenville, SC United States
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Greenville Health System

Greer, SC United States
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Greenville Health System

Seneca, SC United States
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Greenville Health System

Spartanburg, SC United States
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Texas Oncology (E Common) - USOR

New Braunfels, TX United States
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University of Virginia

Charlottesville, VA United States
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Auchenflower, Australia
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Icon Cancer Care

South Brisbane, Australia
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Liverpool Hospital

Liverpool, Australia
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UZ Leuven

Leuven, Belgium
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CHU Angers

Angers, France
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Asan Medical Center - PPDS

Seoul, Korea, Republic of
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City Clinical Hospital # 40

Moscow, Russian Federation
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Royal Bournemouth Hospital

Bournemouth, United Kingdom
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Maidstone Hospital

Maidstone, United Kingdom
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Singleton Hospital

Swansea, United Kingdom
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Recruitment Status: Open


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