New hope for kids with tough leukemia: targeted drug combo enters first human trial

NCT ID NCT06376162

Recruiting now Disease control Sponsor: PedAL BCU, LLC Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-phase trial tests a new drug called ziftomenib, taken as a capsule, together with standard chemotherapy (cytarabine and fludarabine) for children and young adults up to age 21 who have a relapsed or refractory acute leukemia with specific genetic changes (KMT2A-r, NUP98-r, or NPM1-m). The main goal is to find the safest dose and understand how the drug behaves in the body. Only 20 participants will be enrolled, and the study is currently recruiting.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: ziftomenib (a menin inhibitor) combined with chemotherapy (cytarabine and fludarabine)
What this could lead to: If successful, this could point toward a new treatment option for children with hard-to-treat leukemia that has come back or not responded to standard therapy.
What could go wrong: This is a very early phase 1 trial with only 20 participants, so safety and dosing are still being figured out. It may not work or could cause serious side effects.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

acute myeloid leukemia with mutated NPM1 acute myeloid leukemia, MLL gene rearrangement Recurrence

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

RELAPSED/REFRACTORY KMT2A-R ACUTE LEUKEMIA RELAPSED/REFRACTORY NPM1-M ACUTE LEUKEMIA RELAPSED/REFRACTORY NUP98-R ACUTE LEUKEMIA

Contacts and locations

Locations

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

Chicago, Illinois, 60611, United States
CHU de Nantes - Hôpital Femme-Enfant-Adolescent
RECRUITING

Nantes, Loire-Atlantique, 44093, France
CHU de Reims - Hôpital Robert Debré
RECRUITING

Paris, Île-de-France Region, 75019, France
Children's Healthcare of Atlanta
RECRUITING

Atlanta, Georgia, 30329, United States
Children's Hospital Colorado
RECRUITING

Aurora, Colorado, 80045, United States
Children's Hospital Los Angeles
RECRUITING

Los Angeles, California, 90027, United States
Children's Hospital of Philadelphia
RECRUITING

Philadelphia, Pennsylvania, 19104, United States
Cincinnati Children's Hospital Medical Center
RECRUITING

Cincinnati, Ohio, 45229-3026, United States
Dana-Farber Cancer Institute
RECRUITING

Boston, Massachusetts, 02215, United States
Fondazione IRCCS San Gerardo dei Tintori (Ospedale San Gerardo)
RECRUITING

Monza, 20900, Italy
Hospital Infantil Universitario Niño Jesús
RECRUITING

Madrid, 28009, Spain
Hospital Universitari Vall d'Hebrón
RECRUITING

Barcelona, 08035, Spain
Memorial Sloan Kettering Cancer Center - New York
RECRUITING

New York, New York, 10065, United States
Ospedale Pediatrico Bambino Gesù
RECRUITING

Roma, Rome, 00165, Italy
Prinses Maxima Centrum Kinderoncologie
RECRUITING

Utrecht, 3584 CS, Netherlands
Sankt Anna-Kinderspital
RECRUITING

Vienna, State of Vienna, 1090, Austria
Seattle Children's Hospital
RECRUITING

Seattle, Washington, 98105, United States
SickKids - The Hospital for Sick Children
RECRUITING

Toronto, Ontario, M5G 1X8, Canada
St. Jude Children's Research Hospital
RECRUITING

Memphis, Tennessee, 38105-3678, United States
Texas Children's Hospital
RECRUITING

Houston, Texas, 77030, United States