Novartis Pharmaceuticals
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.
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…
Key Inclusion Criteria: Participants eligible for inclusion in this study must meet all of the following criteria: 1. Signed informed consent must be obtained prior to participation in the study. 2. Male or female participants 1 and \< 18 years of age at study enrollment 3. Diagnosis of CML-CP (Apperley et al 2025) with cytogenetic confirmation of Philadelphia positive (Ph+) chromosome 4. 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: 1. Unfavourable resp…
Asciminib (labelled as ABL001) administered as 40 mg tablet (adult formulation) or as 1 mg film-coated granules mini-tablets (pediatric formulation)