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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 (May 2026). This Phase 1/2 trial studies multiple treatments including Asciminib Pediatric formulation group and Asciminib Adult formulation group for myeloid leukemia, philadelphia positive.

Phase 1/2RecruitingNovartis PharmaceuticalsNCT04925479Data as of May 2026

Treatment: Asciminib Pediatric formulation group · Asciminib Adult formulation groupThe 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.

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Extracted eligibility criteria

Cancer type

Acute Myeloid Leukemia

Biomarker criteria

Required: BCR e14a2, e13a2

Evidence of typical BCR-ABL1 transcript [e14a2 and/or e13a2] at the time of screening which are amenable to standardized RQ-PCR quantification.

Required: ABL1 e14a2, e13a2

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 any 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

Participants with Ph+ CML-CP

Prior therapy

Min 1 prior line

Must have received: BCR-ABL 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 required

Liver function

adequate hepatic function required; history of acute or chronic liver disease excluded

Cardiac function

adequate cardiac function required; cardiac or cardiac repolarization abnormality excluded

Participants must have adequate renal, hepatic, pancreatic and cardiac function; see laboratory values at screening

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
  • Dana Farber Cancer Institute · Boston, Massachusetts
  • University of Mississippi Medical Center · Jackson, Mississippi
  • Columbia University Medical Center New York Presbyterian · New York, New York

Showing up to 5 US sites. See all sites on ClinicalTrials.gov →

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