Revumenib and Chemotherapy for Acute Myeloid Leukemia with KMT2A or NUP98 Rearrangement
This study aims to evaluate the effectiveness and safety of combining Revumenib with chemotherapy in treating adults with acute myeloid leukemia that involves KMT2A or NUP98 genetic rearrangements.
Revumenib
+ Intensive Chemotherapy Regimen
+ Placebo
Hematologic Diseases+4
+ Hemic and Lymphatic Diseases
+ Leukemia
Treatment Study
Summary
Study start date: December 1, 2025
Actual date on which the first participant was enrolled.This study aims to evaluate whether adding a medication called revumenib to the usual chemotherapy treatments can improve the health outcomes for people recently diagnosed with a type of blood cancer called acute myeloid leukemia (AML). Specifically, it focuses on those whose cancer cells have certain genetic changes, known as KMT2A or NUP98 gene rearrangements. These genetic changes can make the disease harder to treat with standard therapies. By exploring this combination treatment, the study seeks to find better ways to manage this type of leukemia and potentially improve survival rates and quality of life for those affected. Participants in this study will receive either the standard chemotherapy treatment alone or the standard treatment combined with revumenib. The revumenib is given in a manner consistent with the chemotherapy, under careful medical supervision to ensure the safety of all participants. The study will monitor various health markers and outcomes to determine if the addition of revumenib provides any significant benefit. It will also closely watch for any side effects or safety concerns related to the new combination of treatments. This approach helps researchers understand both the effectiveness and the safety of the treatment being tested.
Protocol
This section provides details of the study plan, including how the study is designed and what the study is measuring.235 patients to be enrolled
Total number of participants that the clinical trial aims to recruit.Treatment Study
Eligibility
Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.Any sex
Biological sex of participants that are eligible to enroll.Over 12 Years
Range of ages for which participants are eligible to join.Healthy volunteers not allowed
If individuals who are healthy and do not have the condition being studied can participate.Conditions
Pathology
Criteria
Key Inclusion Criteria: * Participants must weigh ≥ 40 kilograms (kg). * Participants must have newly diagnosed and previously untreated AML and be candidates for intensive induction chemotherapy. * Presence of a KMT2A rearrangement or NUP98 rearrangement. * Performance status: Eastern Cooperative Oncology Group (ECOG) 0-2, or Karnofsky or Lansky ≥ 40. * Have a life expectancy of ≥3 months as judged by the Investigator. * Negative serum pregnancy test. * Adequate liver, kidney, and cardiac function. Key Exclusion Criteria: * Not a candidate for anthracycline based therapy for Induction. * Not a candidate for continuous infusion cytarabine during Induction or intermediate dose cytarabine for Consolidation. * Diagnosis of active acute promyelocytic leukemia. * Active central nervous system (CNS) disease (cytologic, such as any blasts on cytospin, or radiographic). * QTc using Fridericia's correction (QTcF) \> 450 milliseconds (msec) at Screening or history of long QT Syndrome. * Any gastrointestinal (GI) issue of the upper GI tract likely to affect oral drug absorption or ingestion (for example, gastric bypass, gastroparesis, etc). * Any uncontrolled active infection. * Any concurrent malignancy requiring active therapy (except breast or prostate cancer stable on or responding to endocrine therapy). Note: Additional inclusion/exclusion criteria may apply, per protocol.
Study Plan
Find out more about all the medication administered in this study, their detailed description and what they involve.2 intervention groups are designated in this study
50% chance of being blinded to the placebo group
Treatment Groups
Group I
ExperimentalGroup II
PlaceboStudy Objectives
Primary Objectives
Secondary Objectives