cAMeLot-2
Research type
Research Study
Full title
A Phase 3 Randomized, Double-blind, Placebo-controlled, Study of Bleximenib, Venetoclax and Azacitidine for the Treatment of Participants with Newly Diagnosed Acute Myeloid Leukemia Harboring KMT2A Rearrangements or NPM1Mutations who are Ineligible for Intensive Chemotherapy
IRAS ID
1011871
Contact name
Elena Cascales
Contact email
Sponsor organisation
Janssen-Cilag International NV
Clinicaltrials.gov Identifier
Research summary
Background and study aim: Acute myeloid leukemia is a highly aggressive blood cancer typically characterized by large numbers of immature white blood cells in the bone marrow, and it affects blood cells that fight bacterial infections. Treatment options for AML are limited, survival rates are poor, and many patients are ineligible for standard chemotherapy treatments due to toxicity.
The study drug, bleximenib, specifically targets and blocks the interaction between the proteins histone-lysine N-methyltransferase 2A (KMT2A) and menin. In AML with KMT2A gene rearrangements (KMT2Ar) or NPM1 mutations (NPM1m), blocking this protein-protein interaction kills leukemia cells and helps stop the disease from worsening.
The purpose of this study is to find out how well bleximenib and Venetoclax (VEN)+ Azacitidine (AZA) works as compared to placebo and VEN+AZA for the treatment of participants with KMT2Ar or NPM1m AML.
Study will be conducted in 3 phases:
1. Screening (Up to 28 days): Confirm if the participants can take part in the study.
2. Treatment Phase: Participants will be randomly (by chance) assigned in the following arms:
o Arm A: Bleximenib and Venetoclax (VEN) + Azacitidine (AZA)
o Arm B: Placebo and VEN + AZA
Participants will receive treatment until disease progression, unacceptable toxicity, or if any of the discontinuation criteria defined in the protocol are met.
3. Follow-up Phase: Participants will be followed-up for their overall health throughout the study until death, withdrawal of consent, loss to follow-up, or end of the study, whichever occurs first.During the study, some tests such as blood and urine tests, and physical examination will be performed. Information on side effects will be collected while participants are receiving study treatment and for a period of time after study treatment is discontinued. The overall duration of the study will be approximately 4.5 years.
REC name
London - Brighton & Sussex Research Ethics Committee
REC reference
25/LO/0274
Date of REC Opinion
14 May 2025
REC opinion
Further Information Favourable Opinion