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Testing asciminib for children with chronic myeloid leukemia

A Multi-center, Open-label Study to Determine the Dose and Safety of Oral Asciminib in Pediatric Patients With Philadelphia Chromosome Positive Chronic Myeloid Leukemia in Chronic Phase (Ph+ CML-CP), Previously Treated With One or More Tyrosine Kinase Inhibitors

PHASE1; PHASE2 · Novartis · NCT04925479

This study is testing a new medication called asciminib to see if it is safe and effective for children with a specific type of leukemia who have already tried other treatments.

Quick facts

PhasePHASE1; PHASE2
Study typeInterventional
Enrollment34 (estimated)
Ages1 Year to 17 Years
SexAll
SponsorNovartis (industry)
Drugs / interventionsasciminib
Locations39 sites (Indianapolis, Indiana and 38 other locations)
Trial IDNCT04925479 on ClinicalTrials.gov

What this trial studies

This study aims to evaluate the safety and appropriate dosing of asciminib in pediatric patients aged 1 to less than 18 years who have Philadelphia chromosome positive chronic myeloid leukemia in chronic phase and have previously been treated with one or more tyrosine kinase inhibitors. The study will involve a dose determination phase to establish a pediatric formulation dose that achieves similar drug exposure as in adults. Participants will be divided into two age groups, and the study will assess pharmacokinetics and safety through a structured regimen of the medication. The goal is to ensure that the pediatric population receives an effective and safe treatment option.

Who should consider this trial

Good fit: Ideal candidates for this study are pediatric patients aged 1 to less than 18 years with Philadelphia chromosome positive chronic myeloid leukemia who have previously been treated with at least one tyrosine kinase inhibitor.

Not a fit: Patients who have not been previously treated with a tyrosine kinase inhibitor or those with advanced stages of leukemia may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide a tailored treatment option for children with chronic myeloid leukemia, improving their outcomes and quality of life.

How similar studies have performed: Other studies have shown promise in using similar approaches for adult populations, but this specific pediatric application is novel.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

\- Male or female participants: Pediatric formulation group: ≥ 1 and less than 18 years of age at study entry. Adult formulation group: ≥ 14 and less than 18 years of age and body weight of ≥ 40 kg at study entry.

* Participants with Ph+ CML-CP must meet all of the following laboratory values at the screening visit. In the case where bone marrow blast and promyelocyte counts are available, these will be accepted if done within 56 days prior to the screening visit, to avoid unnecessary repetition of this test.

  1. \< 15% blasts in peripheral blood and bone marrow
  2. \< 30% combined blasts plus promyelocytes in peripheral blood and bone marrow
  3. \< 20% basophils in the peripheral blood
  4. Neutrophils ≥ 1.5 x 10\^9/L (or WBC ≥ 3 x 10\^9/L if neutrophils are not available) and platelet count ≥ 100 x 10\^9/L
  5. No evidence of extramedullary leukemic involvement, with the exception of hepatosplenomegaly
* Prior treatment with a minimum of one TKI
* Failure (adapted from the 2020 European Leukemia Net (ELN) Guidelines Hochhaus et al 2020 and 2013 ELN Guidelines Baccarani et al 2013) or intolerance to the most recent TKI therapy at the time of screening.
* Performance status: Karnofsky ≥ 50% for patients ≥ 16 years of age, and Lansky ≥ 50 for patients \< 16 years of age at the time of screening
* Participants must have adequate renal, hepatic, pancreatic and cardiac function
* Participants must have electrolyte values within normal limits or corrected to be within normal limits with supplements prior to first dose of study medication:
* Evidence of typical BCR-ABL1 transcript \[e14a2 and/or e13a2\] at the time of screening which are amenable to standardized RQ-PCR quantification.

Exclusion Criteria:

* Known presence of the T315I mutation prior to study entry or a BCR::ABL mutation with known resistance to study treatment any time prior to study entry.
* Known second chronic phase of CML after previous progression to AP/BC.
* Previous treatment with a hematopoietic stem-cell transplantation.
* Patient planning to undergo allogeneic hematopoietic stem cell transplantation.
* Cardiac or cardiac repolarization abnormality
* Severe and/or uncontrolled concurrent medical disease that in the opinion of the Investigator could cause unacceptable safety risks or compromise compliance with the protocol
* History of acute pancreatitis within 1 year of study entry or past medical history of chronic pancreatitis.
* History of acute or chronic liver disease.
* Impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of study drug
* Pregnant or nursing (lactating) females.

Other protocol-defined inclusion/exclusion may apply.

Where this trial is running

Indianapolis, Indiana and 38 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.

View on ClinicalTrials.gov →

Conditions: Myeloid Leukemia, Philadelphia Positive, Asciminib, pediatric patients, Philadelphia chromosome positive chronic myeloid leukemia in chronic phase, Ph+ CML-CP, ABL001

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.