Chronic Myelogenous LeukemiaLeukemia, Myelogenous, Chronic, Philadelphia Chromosome Positive
Investigational drug(s) / intervention(s)
Asciminib single agent
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)
Study 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.
Eligibility
Sex
ALL
Min age
1 Year
Max age
18 Years
Healthy volunteers
No
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 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
2. 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
6\. Evidence of typical BCR::ABL1 transcript \[e14a2 and/or e13a2\] at the time of screening which are amenable to standardized RQ-PCR quantification.
7\. Performance status: Karnofsky ≥ 50% for participants ≥ 16 years of age, and Lansky ≥ 50 for participants \< 16 years of age at the time of screening.
Key Exclusion Criteria:
1. Known second chronic phase (CP) of CML after previous progression to Accelerated Phase (AP)/Blast Phase (BP).
2. Previous treatment with a hematopoietic stem-cell transplantation.
3. Patient planned to undergo allogeneic hematopoietic stem cell transplantation
4. 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.
Primary outcome measure(s)
MMR at Week 48 — 48 weeks 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.
Trial sites (30)
Facility
City
Region
Status
Rutgers Cancer Institute of New Jersey
New Brunswick
New Jersey
Recruiting
Columbia University Medical Center New York Presbyterian
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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