NCT07354074

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

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

• Status: Not Yet Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 50 (estimated)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 1 Year – 18 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

This is a multi-center, open-label, single arm study of asciminib in pediatric participants aged 1 to \<18 years old with Ph+ CML-CP newly diagnosed and previously treated with TKI treatment, with or without T315I mutation. The study population will consist of three cohorts of Ph+ CML-CP pediatric participants: * Newly-diagnosed Ph+ CML-CP participants without known T315I mutation * Ph+ CML-CP participants resistant or intolerant to previous TKI without known T315I mutation * Ph+ CML-CP participants with known T315I mutation irrespective of prior TKI treatment There is no fixed duration of study treatment for the participants. The study will end 5 years (240 weeks) after the last enrolled participants received their first dose of treatment in the study. The objective is to have enough follow up for safety, including growth and development and efficacy.

Conditions

• Chronic Myelogenous Leukemia
• Leukemia, Myelogenous, Chronic, Philadelphia Chromosome Positive

Interventions

Type	Name	Description
DRUG	Asciminib single agent	Asciminib (labelled as ABL001) administered as 40 mg tablet (adult formulation) or as 1 mg film-coated granules mini-tablets (pediatric formulation)

Timeline

• Start date: 2026-05-04
• Primary completion: 2033-02-23
• Completion: 2033-02-23
• First posted: 2026-01-21
• Last updated: 2026-03-23

Regulatory

• FDA-regulated drug study