A Study of Pevonedistat and Venetoclax Combined With Azacitidine to Treat Acute Myeloid Leukemia (AML) in Adults Unable to Receive Intensive Chemotherapy

Summary

The main aim is to see how the combination of pevonedistat + venetoclax + azacitidine compares to venetoclax + azacitidine in adults recently diagnosed with AML who are unable to be treated with intensive chemotherapy. Participants will receive either pevonedistat + venetoclax + azacitidine or venetoclax + azacitidine in 28-day treatment cycles. Bone marrow samples (biopsy) will be collected throughout the study. Pevonedistat will be given as an intravenous (IV) infusion and Azacitidine will be given through IV or subcutaneous (under the skin). Study treatments may continue as long as the participant is receiving benefit from it. Participants may choose to stop treatment at any time.

Timeline

Start

2020-10-13

Primary completion

2022-09-06

Completion

2025-10-06

Outcome

Missed primary endpoint

paper There were no differences in event-free survival (primary endpoint; hazard ratio [HR]: 0.99; 95% confidence interval [CI]: 0.61-1.60; p = 0.477) PMID 39606906 ↗

Publications

• Short NJ, Wierzbowska A, Cluzeau T, Laribi K, Recher C, Czyz J, Ochrem B, Ades L, Gallego-Hernanz MP, Heiblig M, Audisio E, Zarzycka E, Li S, Ferenc N, Yeh T, Faller DV, Sedarati F, Papayannidis C. Azacitidine and venetoclax with or without pevonedistat in patients with newly diagnosed acute myeloid leukemia. Leuk Lymphoma. 2025 Mar;66(3):458-468. doi: 10.1080/10428194.2024.2431878. Epub 2024 Nov 28.

Drugs

Indications