RecruitingPhase 1NCT06376162

Ziftomenib in Combination With Chemotherapy for Children With Relapsed/Refractory Acute Leukemia

A Phase 1 Trial of Menin-inhibitor Ziftomenib in Combination With Chemotherapy for Children With Relapsed/Refractory KMT2A-r/NUP98-r/NPM1-m Acute Leukemia

Sponsor

PedAL BCU, LLC

Enrollment

20 participants

Start Date

Mar 18, 2025

Study Type

INTERVENTIONAL

Conditions

Relapsed/Refractory KMT2A-r Acute Leukemia Relapsed/Refractory NUP98-r Acute Leukemia Relapsed/Refractory NPM1-m Acute Leukemia

Summary

The primary objective of the study is to determine the recommended phase 2 dose (RP2D) of ziftomenib in combination with chemotherapy (FLA) in children with relapsed or refractory KMT2A-r, NUP98-r, or NPM1-m acute leukemia based on safety and pharmacokinetics (PK).

Eligibility

Min Age: 0 YearsMax Age: 21 Years

Plain Language Summary

Simplified for easier understanding

This study is testing a drug called ziftomenib combined with chemotherapy in children and young adults with relapsed or treatment-resistant acute leukemia that has specific genetic changes (KMT2A, NPM1, or NUP98 mutations). **You may be eligible if...** - You are between 0 and 21 years old (at least 5 kg body weight), and at least 80% of participants will be under 18 - You have been diagnosed with acute leukemia with a KMT2A rearrangement, NPM1 mutation, or NUP98 rearrangement - Your leukemia has relapsed (come back) or has not responded to standard treatment, including stem cell transplant - Your genetic alteration must be confirmed by the central study laboratory **You may NOT be eligible if...** - Your leukemia does not have one of the required genetic changes - Your genetic alteration cannot be confirmed by the central lab - You have other serious health conditions that would make treatment unsafe Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGZiftomenib

Oral capsule

DRUGCytarabine

Intravenous (IV) infusion

DRUGFludarabine

IV infusion

Locations(20)

Children's Hospital Los Angeles

Los Angeles, California, United States

Children's Hospital Colorado

Aurora, Colorado, United States

Children's Healthcare of Atlanta

Atlanta, Georgia, United States

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

Dana-Farber Cancer Institute

Boston, Massachusetts, United States

Memorial Sloan Kettering Cancer Center - New York

New York, New York, United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

St. Jude Children's Research Hospital

Memphis, Tennessee, United States

Texas Children's Hospital

Houston, Texas, United States

Seattle Children's Hospital

Seattle, Washington, United States

Sankt Anna-Kinderspital

Vienna, State of Vienna, Austria

SickKids - The Hospital for Sick Children

Toronto, Ontario, Canada

CHU de Nantes - Hôpital Femme-Enfant-Adolescent

Nantes, Loire-Atlantique, France

CHU de Reims - Hôpital Robert Debré

Paris, Île-de-France Region, France

Ospedale Pediatrico Bambino Gesù

Roma, Rome, Italy

Fondazione IRCCS San Gerardo dei Tintori (Ospedale San Gerardo)

Monza, Italy

Prinses Maxima Centrum Kinderoncologie

Utrecht, Netherlands

Hospital Universitari Vall d'Hebrón

Barcelona, Spain

Hospital Infantil Universitario Niño Jesús

Madrid, Spain

View Full Details on ClinicalTrials.gov

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NCT06376162