Study to Determine the Dose and Safety of Asciminib in Pediatric Patients With Chronic Myeloid Leukemia
Eligible age
1–17 yrs
Accepts
All genders
Locations
8 states
Healthy volunteers
No
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About this study
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.
Sponsor: Novartis Pharmaceuticals
You may qualify if…
- ✓ 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
You may not qualify if…
- ✕ 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.
Where it's recruiting
Indianapolis
Boston
Jackson
New York
Cincinnati
Philadelphia
Houston
Salt Lake City
Source: ClinicalTrials.gov · NCT04925479 · last updated 2026-09-25