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.
- Conditions
- 10024324chronic leukemiablood cancer
- Registration Number
- NL-OMON54333
- Lead Sponsor
- ovartis
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Pending
- Sex
- Not specified
- Target Recruitment
- 1
* 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 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 (RQPCR) quantification.
* Known presence of the T315I mutation 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.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>The primary objective of this study is to characterize the pharmacokinetic (PK)<br /><br>profile of asciminib in pediatric patients, with the goal of identifying the<br /><br>pediatric formulation dose (fed) leading to asciminib exposure comparable to 40<br /><br>mg BID in adult patients (fasted).</p><br>
- Secondary Outcome Measures
Name Time Method <p>The secondary objectives of this study are:<br /><br>* To assess the safety and tolerability of asciminib.<br /><br>* To assess pharmacodynamic markers of asciminib*s anti-leukemic activity.<br /><br>* To assess acceptability and palatability of the pediatric formulation.<br /><br>* To assess long-term safety of asciminib.</p><br>