Study to Determine the Efficacy and Safety of Asciminib in Pediatric Patients With Ph+ CML-CP

A vizsgálat adatai

Ki végzi

Novartis Pharmaceuticals

Azonosító

NCT07354074

A regiszteradat frissítve

2026. november 10.

Eredeti leírás

The aim of this study is to support development of asciminib in the pediatric population (1 to < 18 years) with Ph+ CML-CP. The study will evaluate the efficacy and safety of asciminib in pediatric formulation (weigh-based dose, fed state) or adult formulation (fasted) in newly diagnosed and resistant or intolerant Ph+ CML-CP with or without T315I mutation.A teljes eredeti leírásKevesebb

A Phase II, Multicenter, Open-label, Single Arm Study to Evaluate the Safety and Efficacy of Asciminib in Pediatric Participants Newly Diagnosed or Previously Treated With Philadelphia Positive Chronic Myelogenous Leukemia in Chronic Phase (Ph+ CML-CP) With or Without Known T315I Mutation

The aim of this study is to support development of asciminib in the pediatric population (1 to < 18 years) with Ph+ CML-CP. The study will evaluate the efficacy and safety of asciminib in pediatric formulation (weigh-based dose, fed state) or adult formulation (fasted) in newly diagnosed and resistant or intolerant Ph+ CML-CP with or without T315I mutation.

Bevonási feltételek: Participants eligible for inclusion in this study must meet all of the following criteria:; Signed informed consent must be obtained prior to participation in the study; Male or female participants 1 and < 18 years of age at study enrollment; Diagnosis of CML-CP (Apperley et al 2025) with cytogenetic confirmation of Philadelphia positive (Ph+) chromosome; For participants with CML-CP newly diagnosed within 3 months of screening OR 5 For participants with CML - CP with high risk of developing resistance or intolerance to previous TKI:; Unfavourable response to TKI is defined following the Apperley et al 2025 guidelines as:; At three months after the initiation of therapy: BCR::ABL1 ratio > 10% IS (if confirmed within 1-3 months); At six months after the initiation of therapy: BCR::ABL1 ratio > 10% IS; At twelve months after initiation of therapy: BCR::ABL1 ratio > 1% IS; At any time loss of previous response; At any time emergent resistant BCR::ABL1 mutations or high-risk ACA from prior TKI treatment as per local test results; Intolerance to TKI is defined as:; Non-hematologic intolerance: participants with grade 3 or 4 toxicity while on therapy (in which case the patient is eligible whether or not there was a dose reduction); or with persistent grade 2 toxicity unresponsive to optimal management including dose adjustments (unless dose reduction is not considered in the best interest of the patient if response is already suboptimal); Hematologic intolerance: participants with grade 3 or 4 toxicity (absolute neutrophil count [ANC] or platelets) while on therapy that is recurrent after dose reduction to the lowest doses of the TKI; Evidence of typical BCR::ABL1 transcript [e14a2 and/or e13a2] at the time of screening which are amenable to standardized RQ-PCR quantification; Performance status: Karnofsky ≥ 50% for participants ≥ 16 years of age, and Lansky ≥ 50 for participants < 16 years of age at the time of screening.

Kizáró feltételek: Known second chronic phase (CP) of CML after previous progression to Accelerated Phase (AP)/Blast Phase (BP); Previous treatment with a hematopoietic stem-cell transplantation; Patient planned to undergo allogeneic hematopoietic stem cell transplantation; Known presence of a BCR::ABL1 mutation with known resistance to study treatment in accordance with the most recent public version of international CML clinical guidelines (e.g. NCCN CML treatment guidelines v 1.2026 and Apperley et al 2025) any time prior to study entry; Other inclusion/exclusion criteria may apply.

ClinicalTrials.gov NCT07354074