Study to Determine the Dose and Safety of Asciminib in Pediatric Patients With Chronic Myeloid Leukemia
- Conditions
- Myeloid Leukemia, Philadelphia Positive
- Registration Number
- NCT04925479
- Lead Sponsor
- Novartis Pharmaceuticals
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- Not specified
Inclusion Criteria:<br><br> - Male or female participants: Pediatric formulation group: = 1 and less than 18 years<br> of age at study entry. Adult formulation group: = 14 and less than 18 years of age<br> and body weight of = 40 kg at study entry.<br><br> - Participants with Ph+ CML-CP must meet all of the following laboratory values at the<br> screening visit. In the case where bone marrow blast and promyelocyte counts are<br> available, these will be accepted if done within 56 days prior to the screening<br> visit, to avoid unnecessary repetition of this test.<br><br> 1. < 15% blasts in peripheral blood and bone marrow<br><br> 2. < 30% combined blasts plus promyelocytes in peripheral blood and bone marrow<br><br> 3. < 20% basophils in the peripheral blood<br><br> 4. Neutrophils = 1.5 x 10^9/L (or WBC = 3 x 10^9/L if neutrophils are not<br> available) and platelet count = 100 x 10^9/L<br><br> 5. No evidence of extramedullary leukemic involvement, with the exception of<br> hepatosplenomegaly<br><br> - Prior treatment with a minimum of one TKI<br><br> - Failure (adapted from the 2020 European Leukemia Net (ELN) Guidelines Hochhaus et al<br> 2020 and 2013 ELN Guidelines Baccarani et al 2013) or intolerance to the most recent<br> TKI therapy at the time of screening.<br><br> - Performance status: Karnofsky = 50% for patients = 16 years of age, and Lansky = 50<br> for patients < 16 years of age at the time of screening<br><br> - Participants must have adequate renal, hepatic, pancreatic and cardiac function<br><br> - Participants must have electrolyte values within normal limits or corrected to be<br> within normal limits with supplements prior to first dose of study medication:<br><br> - Evidence of typical BCR-ABL1 transcript [e14a2 and/or e13a2] at the time of<br> screening which are amenable to standardized RQ-PCR quantification.<br><br>Exclusion Criteria:<br><br> - Known presence of the T315I mutation prior to study entry.<br><br> - Known second chronic phase of CML after previous progression to AP/BC.<br><br> - Previous treatment with a hematopoietic stem-cell transplantation.<br><br> - Patient planning to undergo allogeneic hematopoietic stem cell transplantation.<br><br> - Cardiac or cardiac repolarization abnormality<br><br> - Severe and/or uncontrolled concurrent medical disease that in the opinion of the<br> Investigator could cause unacceptable safety risks or compromise compliance with the<br> protocol<br><br> - History of acute pancreatitis within 1 year of study entry or past medical history<br> of chronic pancreatitis.<br><br> - History of acute or chronic liver disease.<br><br> - Impairment of gastrointestinal (GI) function or GI disease that may significantly<br> alter the absorption of study drug<br><br> - Pregnant or nursing (lactating) females.<br><br>Other protocol-defined inclusion/exclusion may apply.
Not provided
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Primary Pharmacokinetic (PK) parameter: AUClast;Primary PK parameter: AUCtau;Secondary PK parameter: Cmax;Secondary PK parameter: Tmax;Secondary PK parameter: Ctrough
- Secondary Outcome Measures
Name Time Method Hematologic responses;Molecular responses;Questionnaire on acceptability and palatability after first dose, 4 and 52 weeks