Go back to trials list
A Phase 1/2, First-in-Human Study of the Menin-KMT2A (MLL1) Inhibitor Bleximenib in Participants With Acute Leukemia
Description
The purpose of this study is to determine the recommended Phase 2 dose(s) (RP2D\[s\]) of bleximenib in phase 1 (Part 1 \[Dose Escalation\] and to determine the safety and tolerability at RP2D in Phase 1 Part 2 (Dose expansion). The purpose of the Phase 2 part of the study is to evaluate the efficacy of bleximenib at the RP2D.
Trial Eligibility
Inclusion Criteria: Phase 1: * Relapsed or refractory (R/R) acute leukemia and has exhausted, or is ineligible for, available therapeutic options * Participants greater than or equal (\>=)12 and less than (\<) 18 years of age with a body weight of \>= 40 kg are only eligible for the Phase 1 adolescent cohort * Acute leukemia harboring histone-lysine N-methyltransferase 2A (KMT2A), nucleophosmin 1 gene (NPM1) or nucleoporin 98 gene or nucleoporin 214 gene (NUP98 or NUP214) alterations Phase: 2 * Participants greater than 18 years are eligible * Must have had an initial diagnosis of acute myeloid leukemia (AML) per the WHO 2022 classification criteria. * AML harboring KMT2A-r (gene rearrangement/translocation) or NPM1 mutations only For Both Phase 1 and 2: * Pretreatment clinical laboratory values meeting the following criteria: (a) Hematology: white blood cell (WBC) count less than or equal to (\<=) 20\*10\^9/liter (L) and renal function; Estimated or measured glomerular filtration rate greater than or equal to (\>=) 50 milliliter per minute (mL/min) per four variable modified diet in renal disease (MDRD) equation * Eastern Cooperative Oncology Group (ECOG) performance status grade of 0, 1 or 2. Adolescent participants only: Performance status \>=70 by Lansky scale (for participants less than \[\<\]16 years of age) or \>=70 Karnofsky scale (for participants \>=16 years of age) * A participant of childbearing potential must have a negative highly sensitive serum beta-human chorionic gonadotropin at screening and within 48 hours prior to the first dose of study treatment * A participant must agree to all the following during the study and for 90 days after the last dose of study treatment: (a) wear a condom when engaging in any activity that allows for passage of ejaculate to another person; (b) not to donate sperm or freeze for future use for the purpose of reproduction. In addition, the participant should be advised of the benefit for a partner to use a highly effective method of contraception as condom may break or leak Exclusion Criteria: * Acute promyelocytic leukemia, diagnosis of Down syndrome associated leukemia or juvenile myelomonocytic leukemia according to World Health Organization (WHO) 2016 criteria * Active central nervous system (CNS) disease * Prior solid organ transplantation * QTc according to Fridericia's formula (QTcF) for males \>= 450 millisecond (msec) or for females \>= 470 msec. Participants with a family history of Long QT syndrome are excluded * Exclusion criteria related to stem cell transplant: a. Received prior treatment with allogenic bone marrow or stem cell transplant \<=3 months before the first dose of study treatment; b. Has evidence of graft versus host disease; c. Received donor lymphocyte infusion \<=1 month before the first dose of study treatment; d. Requires immunosuppressant therapy (exception: daily doses \<=10 milligrams (mg) prednisone or equivalent are allowed for adrenal replacement) * Prior cancer immunotherapy within 4 weeks prior to enrollment or blinatumomab within 2 weeks prior to enrollment. Additional prior cancer therapies must not be given within 4 weeks prior to enrollment or 5 half-lives of the agent (whichever is shorter)
Study Info
Organization
Janssen Research & Development, LLC
Primary Outcome
Phase 1: Number of Participants with Adverse Events (AEs) as a Measure of Safety and Tolerability
Interventions
Locations Recruiting
City of Hope
United States, California, Duarte
University of California Irvine Medical Center
United States, California, Orange
University of California San Francisco
United States, California, San Francisco
Norton Cancer Institute
United States, Kentucky, Louisville
Massachusetts General Hospital
United States, Massachusetts, Boston
Interested in joining this trial?
Our dedicated patient navigators are here to guide you through the validation and enrollment process with ease.
![newsletter icon](https://static.healthtree.org/icons/newsletter-icon.png)
Get the latest thought leadership on your Chronic Myelomonocytic Leukemia delivered straight to your inbox
Subscribe to the weekly newsletter for news, stories, clinical trial updates, and helpful resources and events with cancer experts.
Thanks to our HealthTree Community for Chronic Myelomonocytic Leukemia Sponsors:
![Abbvie](https://firebasestorage.googleapis.com/v0/b/healthtree-production.appspot.com/o/gallery%2Fpublic%2Fabbvie%20bigger_c5e4f4af-fb3c-45ca-b9f0-831fb4c5f968.jpeg?alt=media)
![Astellas Pharma](https://firebasestorage.googleapis.com/v0/b/healthtree-production.appspot.com/o/university%2Fpublic%2Fshared%2Fastellas%20fo%20realz-100_8fd8c5cb-3421-4cfd-8aa6-07a70f67acf7.jpg?alt=media)
![Servier](https://firebasestorage.googleapis.com/v0/b/healthtree-production.appspot.com/o/gallery%2Fpublic%2Fservier_6f4f6f05-7255-45f8-8f3d-a07651617cbe.png?alt=media)