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Study of Revumenib, Cytarabine, and Fludarabine in Children with Relapsed or Refractory Acute Myeloid Leukemia

Fast replyRegistered drugNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

This study aims to evaluate the effectiveness of combining certain medications in children with Acute Myeloid Leukemia, which is a type of cancer that affects the blood and bone marrow. The research focuses on specific subtypes of this cancer characterized by certain genetic changes, known as KMT2A-r, NUP98-r, or NPM1-mut. These terms refer to specific mutations, which are permanent changes in the DNA that cause cells to grow abnormally. The study involves patients whose cancer has either returned after treatment or has not responded to initial therapy.

The treatment being investigated consists of revumenib used together with a chemotherapy regimen called FLA. This chemotherapy combination includes the drugs cytarabine and fludarabine, which are administered through an intravenous line, meaning they are delivered directly into a vein. Revumenib is taken orally as either a tablet or an oral solution. The goal is to determine how many patients experience a reduction in cancer cells after receiving up to two cycles of this combined treatment.

The research process

The trial runs in 2 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Start of treatment

    The treatment begins with a combination of revumenib and fludarabine/cytarabine (also known as fla) chemotherapy.

    revumenib is taken orally as either a tablet or an oral solution at a dose of 540 mg.

    fludarabine is administered through an intravenous line (a small tube inserted into a vein) at a dose of 30 mg/m2 (milligrams per square meter of body surface area).

    cytarabine is administered through an intravenous line at a dose of 2000 mg/m2 (milligrams per square meter of body surface area).

  2. Step 2

    Treatment cycles

    The combination of revumenib and fla chemotherapy is administered for up to 2 cycles.

    The goal of these cycles is to evaluate the overall response rate, which is a measure of how well the treatment works against the acute myeloid leukemia (a type of blood cancer).

Who can join the trial?

14 criteria

  • Patients must be older than 30 days but no older than 18 years of age at the time they join the study.
  • Patients must have a performance status of 50% or higher on the Lansky or Karnofsky scale, which are tools used to measure how well a person can perform daily activities and their general health.
  • Patients may have leukemia in the central nervous system (the brain and spinal cord), categorized as CNS1, CNS2, or CNS3, as long as they show no physical signs or symptoms like facial nerve weakness or issues with the eyes or brain.
  • Patients must have completely recovered from the harmful side effects of all previous anti-cancer treatments.
  • Patients must have healthy kidneys (renal function), liver, and heart (cardiac function).
  • Patients must have a specific type of Acute Myeloid Leukemia that is either refractory (meaning the cancer did not respond to treatment) or relapsed (meaning the cancer came back after a period of improvement). This leukemia must involve specific genetic changes known as KMT2A-translocation, NUP98-rearrangement, or NPM1-mutation.
  • At least 21 days must have passed since the last anti-cancer treatment, such as chemotherapy, unless a specific pre-treatment is allowed by the study rules.
  • At least 84 days must have passed since an allogeneic transplant, which is a procedure where a patient receives healthy bone marrow or stem cells from a donor.
  • Patients must not show signs of graft versus host disease (GVHD), which is a condition where the donor's cells attack the patient's body, though mild skin issues being treated with creams are allowed.
  • At least 28 days must have passed since any cellular therapy, such as donor lymphocyte infusion (DLI), which involves giving the patient specific immune cells.
  • A parent or legal guardian must understand and provide written permission for the patient to participate.
  • Patients and their families must be able to follow the scheduled study visits and other rules of the trial.
  • Females who are able to become pregnant must have a negative pregnancy test within 10 days before starting treatment.
  • The white blood cell (WBC) count, which is a measure of certain immune cells in the blood, must be less than 25,000 per microliter before joining.

Who cannot join the trial?

20 criteria

  • Patients whose cancer is only found outside of the bone marrow (the spongy tissue inside bones where blood cells are made), which is called extramedullary disease.
  • Patients who are currently taking another experimental drug (a medicine being tested in a study).
  • Patients taking medications like cyclosporine or tacrolimus to prevent graft-versus-host disease (a condition where transplanted immune cells attack the body) or organ rejection after a transplant.
  • Patients with active graft-versus-host disease, except for very mild skin involvement.
  • Patients taking strong CYP3A4 inhibitors, which are drugs that interfere with how the liver processes certain medicines, unless they are specific types like itraconazole or ketoconazole.
  • Patients taking CYP3A4 inducers, which are drugs that speed up how the liver processes certain medicines, either during the study or within 14 days of starting the study drug.
  • Patients taking medicines that can change the heart rhythm, specifically those that affect the QT interval (the time it takes for the heart muscle to recharge between beats).
  • Patients with certain genetic syndromes that affect how the body functions, such as Bloom syndrome, ataxia-telangiectasia, Fanconi anemia, Kostmann syndrome, or Shwachman syndrome.
  • Female patients who are pregnant or breastfeeding.
  • Female and male patients who are able to have children but are not using highly effective birth control.
  • Patients who have previously used revumenib or any other menin inhibitor (a type of medicine that targets a specific protein).
  • Patients with a baseline QTcF measurement greater than 450ms, which is a way to measure the heart's electrical activity.
  • Patients with an allergy or hypersensitivity (an extreme immune reaction) to the study drug or any of its excipients (the inactive ingredients used to make the medicine).
  • Patients with an active, uncontrolled infection at the time they join the study.
  • Patients with Down Syndrome.
  • Patients whose leukemia developed as a result of previous treatments like cytotoxic chemotherapy (strong drugs used to kill cancer cells).
  • Patients with a specific genetic change called KMT2A partial duplication.
  • Patients with Mixed Lineage Leukemia (a type of blood cancer that has features of more than one type of white blood cell).
  • Patients with a history of congenital prolonged QT syndrome (a heart rhythm problem present from birth), congestive heart failure (a condition where the heart cannot pump blood well), or arrhythmia (an irregular heartbeat) in the last 6 months.
  • Patients with problems in the upper gastrointestinal tract (the throat, esophagus, or stomach) that could prevent the body from absorbing medicine taken by mouth.
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Investigated drugs

  • Revumenib

    is an oral medication that is being studied to see how well it works in treating certain types of acute myeloid leukemia in children. It is given as either a tablet or a liquid solution.

  • Cytarabine

    and Fludarabine are chemotherapy drugs administered through an IV (intravenous) line. In this study, these medications are used together as part of a chemotherapy combination to treat the leukemia.

What is already known about the treatment

  • Cytarabine

    This medication is an anticancer drug administered as an injection directly into a vein. It is currently used in standard medical practice to treat various types of blood cancers, such as leukemia. The drug works by interfering with the DNA production process, which stops cancer cells from growing and dividing. It belongs to a class of medicines known as antimetabolites.

  • Revumenib

    This is an experimental medicine taken by mouth in either tablet or liquid form. It is currently being studied in clinical trials as a specialized treatment for specific types of acute myeloid leukemia. The drug works by targeting and blocking a specific protein that helps cancer cells survive and multiply. It is classified as a menin inhibitor.

  • Fludarabine

    This medication is an anticancer agent that is given through an injection into a vein. It is an established treatment used to manage several types of blood-related cancers and immune system disorders. At a molecular level, it mimics the building blocks of DNA to prevent cancer cells from copying their genetic material, leading to cell death. It is categorized as a purine analog, which is a type of antimetabolite.

Investigated diseases

Acute Myeloid Leukemia - This is a type of cancer that affects the blood and bone marrow. It occurs when the bone marrow begins producing abnormal white blood cells called myeloblasts. These abnormal cells do not function correctly and multiply rapidly. As these cells build up, they crowd out healthy blood cells. The condition can progress quickly as the abnormal cells interfere with the production of red blood cells, normal white blood cells, and platelets.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2025-521390-14-00Protocol codeSTRIVEEstimated enrolment31 patientsSponsorPediatric Research International GmbH

sourced from the EU Clinical Trials Register and site verification

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