2023-508129-28-00招募中2 期
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.
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 17
- 试验地点
- 15
- 主要终点
- Primary PK parameters of asciminib: AUClast, AUCtau
研究概览
简要总结
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).
入排标准
- 年龄范围
- 0 years 至 17 years（0-17 Years）
- 接受健康志愿者
- 是
入选标准
- •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.
排除标准
- •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.
结局指标
主要结局
Primary PK parameters of asciminib: AUClast, AUCtau
Primary PK parameters of asciminib: AUClast, AUCtau
Secondary PK parameters of asciminib: Cmax, Tmax, Ctrough.
Secondary PK parameters of asciminib: Cmax, Tmax, Ctrough.
次要结局
- 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.
研究者
Novartis Pharma Arzneimittel GmbH
Scientific
Novartis Pharma AG
研究点 (15)
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