NCT02782468

A Study of Pevonedistat in Adult East Asian Participants

A Phase 1/1b, Open-label Study of Pevonedistat (MLN4924, TAK-924) as Single Agent and in Combination With Azacitidine in Adult East Asian Patients With Acute Myeloid Leukemia (AML) or Myelodysplastic Syndromes (MDS)

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 23 (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 safety and tolerability of pevonedistat administered as a single agent and in combination with azacitidine in adult east Asian participants with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS).

Detailed description

The drug being tested in this study is called Pevonedistat. Pevonedistat is being tested to treat people with myelodysplastic syndromes MDS (including nonproliferative chronic myelomonocytic leukemia \[CMML\]) and AML (acute myeloid leukaemia) as a single-agent and in combination treatment with azacitidine. This study will look at the safety and tolerability, the recommended phase 2/phase 3 dose of pevonedistat administered in combination with azacitidine, pharmacokinetics and response to treatment in participants who take single agent pevonedistat compared to participants who take pevonedistat and azacitidine. The study will enroll approximately 37 participants. Participants will be assigned into one of the four treatment groups which will remain disclosed to the patient and study doctor during the study. Participants will be first enrolled at single-agent low dose level (25 mg/m\^2). If this dose is tolerable, participants will be enrolled in parallel at single-agent higher dose level (44 mg/m\^2) and in combination treatment cohorts. * Pevonedistat 25 mg/m\^2 * Pevonedistat 44 mg/m\^2 * Pevonedistat 10 mg/m\^2 and azacitidine 75 mg/m\^2 combination * Pevonedistat 20 mg/m\^2 and azacitidine 75 mg/m\^2 combination Participants will receive pevonedistat infusion intravenously and azacitidine via intravenous or subcutaneous route. This multi-center trial will be conducted in Japan, Korea and Taiwan. The overall time to participate in this study is approximately 24 months. Participants will attend the End of Study (EOS) visit for safety, 30 days after receiving their last dose of study drug or before the start of subsequent antineoplastic therapy (other than hydroxyurea).

Conditions

• Leukemia, Myeloid, Acute
• Myelodysplastic Syndromes

Interventions

Type	Name	Description
DRUG	Pevonedistat 25 mg/m^2	Pevonedistat 25 mg/m\^2 intravenous infusion.
DRUG	Pevonedistat 44 mg/m^2	Pevonedistat 44 mg/m\^2 intravenous infusion.
DRUG	Pevonedistat 10 mg/m^2	Pevonedistat 10 mg/m\^2 intravenous infusion.
DRUG	Pevonedistat 20 mg/m^2	Pevonedistat 20 mg/m\^2 intravenous infusion.
DRUG	Azacitidine 75 mg/m^2	Azacitidine 75 mg/m\^2 intravenous or subcutaneous formulation.

Timeline

• Start date: 2016-05-16
• Primary completion: 2022-01-25
• Completion: 2022-01-25
• First posted: 2016-05-25
• Last updated: 2023-11-03
• Results posted: 2023-11-03

Locations

9 sites across 3 countries: Japan, South Korea, Taiwan

Regulatory

• FDA-regulated drug study