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.
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
At three months after the initiation of therapy: BCR::ABL1 ratio > 10% IS (if confirmed within 1-3 months)
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
Single group
Asciminib single agent
DrugAsciminib (labelled as ABL001) administered as 40 mg tablet (adult formulation) or as 1 mg film-coated granules mini-tablets (pediatric formulation)
MMR at Week 48
MMR is defined as BCR::ABL1 IS ≤0.1%. MMR is defined as a ≥ 3.0 log reduction in BCR::ABL1 transcripts compared to the standardized baseline equivalent to ≤ 0.1 % BCR::ABL1/ABL % by international scale as measured by RQ-PCR.
MMR at Week 96
MMR is defined as BCR::ABL1 IS ≤0.1%. MMR is defined as a ≥ 3.0 log reduction in BCR::ABL1 transcripts compared to the standardized baseline equivalent to ≤ 0.1 % BCR::ABL1/ABL % by international scale as measured by RQ-PCR.
MMR at and by scheduled timepoints
MMR is defined as BCR::ABL1 IS ≤0.1%. MMR is defined as a ≥ 3.0 log reduction in BCR::ABL1 transcripts compared to the standardized baseline equivalent to ≤ 0.1 % BCR::ABL1/ABL % by international scale as measured by RQ-PCR.
Hematologic response at and by scheduled timepoints
Complete hematologic response (CHR) is defined as all of the following present for ≥ 4 weeks (which means present at least at 2 visits 4-week apart, with no intermediate visit showing no CHR): * White blood cell(s) (WBC) count \< 10 x 10\^9/L * Platelet count \< 450 x 10\^9/L * Basophils \< 5% * No blasts and promyelocytes in peripheral blood * Myelocytes + metamyelocytes \< 5% in peripheral blood * No evidence of extramedullary disease, including spleen and liver
Cytogenetic response at and by scheduled timepoints
Cytogenetic response is defined as follows: * Complete (CCyR) - 0% Ph+ metaphases * Partial (PCyR) - \>0 to 35% Ph+ metaphases * Major\* (MCyR) - 0 to 35% Ph+ metaphases * Minor (mCyR) - \>35 to 65% Ph+ metaphases * Minimal - \>65 to 95% Ph+ metaphases * None - \>95 to 100% Ph+ metaphases Bone marrow aspirate for cytogenetic analyses will be performed at screening, and upon unfavorable response to treatment at the physician's discretion.
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