Study to determine the dose and safety of asciminib in pediatric patients with chronic myeloid leukemia

Phase 1

• Conditions: Pediatric patients with Philadelphia chromosome positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP), previously treated with one or more tyrosine kinase inhibitors; MedDRA version: 21.0Level: LLTClassification code 10054352Term: Chronic phase chronic myeloid leukemiaSystem Organ Class: 100000004864; MedDRA version: 21.0Level: LLTClassification code 10060498Term: Juvenile chronic myeloid leukemiaSystem Organ Class: 100000004864; MedDRA version: 24.0Level: LLTClassification code 10082178Term: Philadelphia positive chronic myeloid leukemiaSystem Organ Class: 100000004864; Therapeutic area: Diseases [C] - Cancer [C04]

• Registration Number: EUCTR2021-001286-20-DE
• Lead Sponsor: ovartis Pharma AG

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2021-09-03
• Last Update Posted: 3 June 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 35

Inclusion Criteria

• 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 (RQ-PCR) quantification.
Are the trial subjects under 18? yes
Number of subjects for this age range: 35
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

• 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 clinical trial of medicinal product
• Study Design: Not specified