Study to determine the dose and safety of asciminib in pediatric patients with chronic myeloid leukemia.
Key facts
- Where
- Budapest, MagyarországSee the site
- Phase
- 1/2. fázis
Where in Magyarország
- Semmelweis UniversityBudapest, MagyarországNo longer taking part
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Novartis Pharma AG
- Trial code
- EU 2023-508129-28-00
- Record updated
- 2026. október 2.
- Original description
The primary objective of this study is to characterize the pharmacokinetic (PK) profile of asciminib in pediatric patients, with the goal of identifying the pediatric formulation dose (fed) leading to asciminib exposure comparable to 40 mg BID in adult patients (fasted).Read full original descriptionShow less
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.
The primary objective of this study is to characterize the pharmacokinetic (PK) profile of asciminib in pediatric patients, with the goal of identifying the pediatric formulation dose (fed) leading to asciminib exposure comparable to 40 mg BID in adult patients (fasted).
Inclusion: Male or female participants: a. Pediatric formulation group: ≥1 and less than 18 years of age at study entry. b. 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 locally within 56 days prior to the screening visit, to avoid unnecessary repetition of this test. a. <15% blasts in peripheral blood and bone marrow b. < 30% combined blasts plus promyelocytes in peripheral blood and bone marrow c. < 20% basophils in the peripheral blood d. Neutrophils ≥ 1.5 x 10^9/L (or white blood cell (WBC) ≥ 3 x 10^9/L if neutrophils are not available) and platelet count ≥ 100 x 10^9/L e. No evidence of extramedullary leukemic involvement, with the exception of hepatosplenomegaly; Prior treatment with a minimum of one TKI; Failure or intolerance to the most recent TKI therapy at the time of screening; Evidence of typical BCR-ABL fusion gene (BCR-ABL1) transcript [e14a2 and/or e13a2] at the time of screening which are amenable to standardized real time quantitative polymerase chain reaction (RQ-PCR) quantification.
Exclusion: Known presence of the T315I mutation prior to study entry or of 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.