RightFit
RecruitingPhase 3

Bleximenib in Combination With Standard Induction and Consolidation Therapy Followed by Maintenance for Treatment of Patients With Acute Myeloid Leukemia (AML)

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

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
18 Years and older
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

Not stated in the study description — ask the study team about visits and procedures.

Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests whether adding bleximenib to standard chemotherapy improves treatment results for adults with newly diagnosed acute myeloid leukemia (AML), a blood cancer. It is for people with certain changes in the NPM1 or KMT2A genes who can receive strong chemotherapy. Everyone gets chemotherapy plus either bleximenib or a placebo, which looks like the medicine but has no active drug.

The current standard of care treatment for adult patients with acute myeloid leukemia (AML) consists of chemotherapy and, if indicated, donor stem cell transplantation. Bleximenib blocks the interaction between a protein called menin and another protein called KMT2A in the leukemia cells. When this interaction is disrupted in AML with mutations in the NPM1 or KMT2A gene, bleximenib can cause leukemia cells to die. The main objective is to assess if treatment with bleximenib, when added to chemotherapy treatment will improve treatment outcome in adult participants with newly diagnosed AML who present with mutations in the NPM1 or KMT2A genes. This is a randomized, double-blind, placebo-controlled, phase 3 clinical trial. All of the participants will receive standard chemotherapy treatment, combined with either bleximenib or a placebo. A placebo is a substance that looks like the study medicine but has no active ingredients (e.g., a sugar pill). In a double blind trial neither the participant nor the doctor know if placebo or active study drug is given. After the end of the protocol treatment there will be an observational follow-up of 4 years from the time of inclusion of the last patient. The results of the different treatment groups will be compared. 875 previously untreated patients with AML with a specific change in the DNA of the leukemia cells (a KMT2A rearrangement or a NPM1 mutation) will be included. Participants must be 18 years or older and considered eligible for intensive chemotherapy.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Be at least 18 years old, or the legal adult age where the study takes place if that is higher.
  • Have newly diagnosed AML with an NPM1 gene change or a KMT2A gene rearrangement, and at least 10% immature cells called blasts in the blood or bone marrow.
  • Be able to receive strong chemotherapy.
  • Be able to care for yourself and be out of bed or a chair for at least half of your waking hours.
  • Have kidneys and a liver that work well enough for the study.

Who can't join

  • Have already received treatment for AML, including chemotherapy or drugs called hypomethylating agents.
  • Have known active leukemia in the brain or spinal cord.
  • Have received a solid organ transplant.
  • Within the past 6 months, have had a heart attack, uncontrolled or unstable chest pain, severe heart failure, uncontrolled heart rhythms or rhythms causing symptoms, a stroke, or a mini-stroke.
  • Have a heart rhythm test showing a QTcF of 470 milliseconds or more. Some changes related to a pacemaker or a heart electrical block are allowed.
  • Have a heart scan within 28 days before treatment showing that the heart pumps out less than 40% of its blood with each beat.
  • Have received a total dose of at least 500 mg/m² of chemotherapy drugs called anthracyclines or anthracenediones.
  • Have a long-term breathing disease that requires extra oxygen.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility criteria

Inclusion Criteria: 1. ≥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. 2. 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. 3. Considered eligible for intensive chemotherapy. 4. WHO/ECOG performance status ≤2. 5. Adequate renal and hepatic functions prior to randomization. Exclusion 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: 1. 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. 2. QTc interval using Fridericia's formula (QTcF) ≥470 ms. Prolonged QTc interval associated with bundle branch block or pacemaking is permitted. 3. Left ventricular ejection fraction (LVEF) \<40% by ECHO or MUGA scan obtained within 28 days prior to the start of study treatment. 4. Previously received cumulative dose of any combination of anthracyclines or anthracenediones of ≥500 mg/m2. 5. Chronic respiratory disease requiring supplemental oxygen.

Study sites (13)

CA

FL

GA

IL

KS

MD

MO

NC

OH

PA