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)

A Phase 2, Randomized, Controlled, Open-Label, Clinical Study of the Efficacy and Safety of Pevonedistat Plus Azacitidine Versus Single-Agent Azacitidine in Patients With Higher-Risk Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia and Low-Blast Acute Myelogenous Leukemia

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 120 (actual)
• Sponsor: Millennium Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to evaluate the efficacy and safety of pevonedistat plus azacitidine versus single-agent azacitidine in participants with HR-MDS or CMML, or low-blast AML.

Detailed description

The drug being tested in this study is called pevonedistat. Pevonedistat is being tested to treat people with MDS or CMML, or low-blast 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 120 participants. Once enrolled, participants will be randomly assigned (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 44 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 will enter event-free survival follow-up or response follow-up (study visits every 3 months) if their disease has not transformed to AML (for participants with HR MDS or CMML) or progressed (for participants with low-blast AML), and they have not started subsequent therapy. Participants will also enter overall survival follow-up (contacted every 3 months to document subsequent therapies and survival status).

Conditions

• Myelodysplastic Syndromes
• Leukemia, Myelomonocytic, Chronic
• Leukemia, Myeloid, Acute

Interventions

Type	Name	Description
DRUG	Azacitidine	Azacitidine intravenous or subcutaneous formulation.
DRUG	Pevonedistat	Pevonedistat intravenous infusion.

Timeline

• Start date: 2016-04-14
• Primary completion: 2019-09-04
• Completion: 2021-07-23
• First posted: 2015-11-20
• Last updated: 2022-09-19
• Results posted: 2020-09-23

Locations

59 sites across 12 countries: United States, Belgium, Bulgaria, Canada, Czechia, France, Germany, Ireland, Israel, Italy, Netherlands, Spain

Regulatory

• FDA-regulated drug study