NIPH Clinical Trials Search

Bleximenib or Placebo in Combination with Standard Induction and Consolidation Therapy followed by Maintenance for the Treatment of Patients with Newly Diagnosed KMT2A-rearranged or NPM1-mutant Acute Myeloid Leukemia Eligible for Intensive Chemotherapy: a double-blind phase 3 study

Basic Information

Recruitment status	Pending
Health condition(s) or Problem(s) studied	Newly diagnosed KMT2A-rearranged or NPM1-mutant Acute Myeloid Leukemia
Date of first enrollment	19/03/2026
Target sample size	40
Countries of recruitment	United States,Japan,Germany,Japan,Netherlands,Japan
Study type	Interventional
Intervention(s)	Experimental: Arm 1: Bleximenib in combination with remission induction and consolidation therapy, followed by bleximenib maintenance therapy. Treatment will continue until PD, unacceptable toxicity or other protocol defined criteria for discontinuation (whichever comes first). Experimental: Arm 2: Bleximenib in combination with remission induction and consolidation therapy, followed by placebo maintenance therapy. Treatment will continue until PD, unacceptable toxicity or other protocol defined criteria for discontinuation (whichever comes first). Placebo Comparator: Arm 3: Placebo comparator in combination with remission induction and consolidation therapy, followed by placebo maintenance therapy . Treatment will continue until PD, unacceptable toxicity or other protocol defined criteria for discontinuation (whichever comes first)

Outcome(s)

Primary Outcome

To assess if treatment with bleximenib, as compared with placebo, in combination with remission induction chemotherapy, prolongs event-free survival (EFS) measured from the time from randomization to failure to achieve CR after remission induction, hematologic relapse after achieving CR, or death, whichever occurs first.

Secondary Outcome

- Overall Survival (OS) in adult patients with newly diagnosed NPM1m or KMT2Ar AML eligible for intensive chemotherapy - Rates of CR, CRh, CRi in adult patients with newly diagnosed NPM1m or KMT2Ar AML eligible for intensive chemotherapy - Prolongation of CR (DoCR) in adult patients with newly diagnosed NPM1m or KMT2Ar AML eligible for intensive chemotherapy - Percentage of participants undergoing an allo-SCT in adult patients with newly diagnosed NPM1m or KMT2Ar AML eligible for intensive chemotherapy

Key inclusion & exclusion criteria

Age minimum	>= 18age old
Age maximum	Not applicable
Gender	Both
Include criteria	>=18 years of age (or the legal age of majority in the jurisdiction in which the study is taking place, whichever is greater) at the time of informed consent. New diagnosis of AML (>=10% blasts in BM or peripheral blood) with mutated NPM1 or with recurring rearrangements involving KMT2A according to ICC 2022 criteria. Considered eligible for intensive chemotherapy. WHO/ECOG performance status =<2. Adequate renal and hepatic functions prior to randomization.
Exclude criteria	1. Prior (chemo-)therapy for AML, including prior treatment with hypomethylating agents. 2. Known active leukemic involvement of the central nervous system (CNS). 3. Recipient of solid organ transplant. 4. Cardiac disease: a. Any of the following within 6 months of randomization: myocardial infarction, uncontrolled/unstable angina, congestive heart failure (NYHA Class III or IV), uncontrolled or symptomatic arrhythmias, stroke, or transient ischemic attack. b. QTc interval using Fridericias formula (QTcF) >= 470 ms. Prolonged QTc interval associated with bundle branch block or pacemaking is permitted. c. Left ventricular ejection fraction (LVEF) <40% by ECHO or MUGA scan (only if ECHO or MUGA was performed for clinical indication within 28 days prior to the start of study treatment). d. Previously received cumulative dose of any combination of anthracyclines or anthracenediones of >= 500 mg/m^2. 5. Chronic respiratory disease requiring supplemental oxygen.