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: 21.0 Level: LLT Classification code 10054352 Term: Chronic phase chronic myeloid leukemia System Organ Class: 100000004864 MedDRA version: 21.0 Level: LLT Classification code 10060498 Term: Juvenile chronic myeloid leukemia System Organ Class: 100000004864 MedDRA version: 24.0 Level: LLT Classification c
Conditions
Interventions
Trade Name: Scemblix
Product Name: Asciminib
Product Code: ABL001
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: ASCIMINIB
Current Sponsor code: ABL001
Other descriptive name: Asciminib
Sponsors
Novartis Pharma AG
Eligibility
Sex/Gender
All
Inclusion criteria
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. =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: 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).;Secondary Objective: •To assess the safety and tolerability of asciminib. •To assess pharmacodynamic markers of asciminib’s anti-leukemic activity. •To assess acceptability and palatability of the pediatric formulation. •To assess long-term safety of asciminib.;Primary end point(s): • Primary PK parameters of asciminib: AUClast, AUCtau • Secondary PK parameters of asciminib: Cmax, Tmax, Ctrough. ;Timepoint(s) of evaluation of this end point: Primary analysis after last patient has completed week 52 visit | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • Number, seriousness, severity, and causality assessments of treatment-emergent adverse events and other safety data as considered appropriate. • Activity: Hematologic and molecular responses. • Questionnaire on acceptability and palatability after first dose, 4 and 52 weeks. • Number, seriousness, severity, and causality assessments of treatment-emergent adverse events and other safety data as considered appropriate including growth and sexual maturation assessments.;Timepoint(s) of evaluation of this end point: Final analysis after last patient completes week 260 visit | — |
Countries
China, France, Germany, Greece, Hungary, Italy, Japan, Korea, Republic of, Netherlands, Poland, Russian Federation, Thailand, Turkey, United States
Contacts
Public ContactEleni Konstantinidou
Novartis (Hellas) S.A.C.I
Outcome results
None listed