OncoMatch/Clinical Trials/NCT04925479
Study to Determine the Dose and Safety of Asciminib in Pediatric Patients With Chronic Myeloid Leukemia
Is NCT04925479 recruiting? Yes, currently enrolling (Sep 2026). This Phase 1/2 trial studies multiple treatments including Asciminib Pediatric formulation group and Asciminib Adult formulation group for myeloid leukemia, philadelphia positive.
Treatment: Asciminib Pediatric formulation group · Asciminib Adult formulation group — The aim of this study is to support development of asciminib in the pediatric population (1 to \<18 years) previously treated with one or more TKIs. Full extrapolation of the efficacy of asciminib from adult to pediatric patients will be conducted. Full extrapolation is based on the concept that CML in the pediatric population has the same pathogenesis, similar clinical characteristics and progression pattern as in adults.
Check if I qualifyExtracted eligibility criteria
Treatments studied
Other
Cancer type
Acute Myeloid Leukemia
Biomarker criteria
Required: ABL1 bcr-abl1 fusion
Evidence of typical BCR::ABL1 transcript [e14a2 and/or e13a2] at the time of screening which are amenable to standardized RQ-PCR quantification
Excluded: ABL1 t315i
Known presence of the T315I mutation prior to study entry
Excluded: ABL1 mutation with known resistance to study treatment
BCR::ABL mutation with known resistance to study treatment any time prior to study entry
Disease stage
Required: Stage CHRONIC PHASE
Excluded: Stage SECOND CHRONIC PHASE AFTER PREVIOUS PROGRESSION TO AP/BC
chronic phase; Known second chronic phase of CML after previous progression to AP/BC [excluded]
Demographics
Prior therapy
Must have received: tyrosine kinase inhibitor
Prior treatment with a minimum of one TKI
Cannot have received: hematopoietic stem cell transplantation
Previous treatment with a hematopoietic stem-cell transplantation
Lab requirements
Blood counts
< 15% blasts in peripheral blood and bone marrow; < 30% combined blasts plus promyelocytes in peripheral blood and bone marrow; < 20% basophils in the peripheral blood; 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
Kidney function
adequate renal function
Liver function
adequate hepatic function; history of acute or chronic liver disease [excluded]
Cardiac function
adequate cardiac function; cardiac or cardiac repolarization abnormality [excluded]
Participants must have adequate renal, hepatic, pancreatic and cardiac function; Karnofsky ≥ 50% for patients ≥ 16 years of age, and Lansky ≥ 50 for patients < 16 years of age; laboratory values at the screening visit; cardiac or cardiac repolarization abnormality [excluded]; history of acute or chronic liver disease [excluded]
Structured fields extracted by AI. May contain errors — verify against the official protocol.
US trial sites
- Indiana UH Riley H for CIU · Indianapolis, Indiana
- Dana Farber Cancer Institute · Boston, Massachusetts
- University of Mississippi Medical Center · Jackson, Mississippi
- Columbia University Medical Center New York Presbyterian · New York, New York
- Cinn Children Hosp Medical Center · Cincinnati, Ohio
Showing up to 5 US sites.
See all sites on ClinicalTrials.gov →Frequently asked questions
Is NCT04925479 currently recruiting?
Yes, this trial is currently recruiting patients.
Are there prior therapy exclusions?
Yes. Prior hematopoietic stem cell transplantation disqualifies patients from enrollment.
Does this trial require ABL1?
Yes, ABL1 bcr-abl1 fusion is a required biomarker for enrollment.
Are patients with ABL1 alterations eligible?
No. ABL1 t315i is an exclusion criterion.
Are patients with ABL1 alterations eligible?
No. ABL1 mutation with known resistance to study treatment is an exclusion criterion.
What disease stage is eligible?
Stage CHRONIC PHASE is required.
Is there an age limit?
Yes. Patients must be 17 years or younger and at least 1 years old.
Could you qualify for this trial?
Enter your biomarker results to see how this trial's eligibility criteria match your specific cancer profile.
Check if I qualifyRelated pages