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

The film gives the main points. The full list below is copied from the registry; the study team makes the final decision.

• Age1 Year to 18 Years
• SexAll
• Healthy volunteersNo

You may be able to join if

• Key Inclusion Criteria:
• 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.
• Key Exclusion Criteria:
• 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.

This is how the treatment is meant to work. Whether it helps, and how safe it is, is what this trial is testing.

asciminib hydrochloride. The hydrochloride salt form of asciminib, an orally bioavailable, allosteric Bcr-Abl1 tyrosine kinase inhibitor, with antineoplastic activity. Upon administration, asciminib targets and binds to the myristoyl pocket of the Bcr-Abl1 fusion protein at a location that is distinct from the ATP-binding domain, thereby inhibiting the activity of both wild-type Bcr-Abl and certain mutation forms, including the T315I mutation. This binding results in the inhibition of Bcr-Abl1-mediated proliferation and enhanced apoptosis of Philadelphia chromosome-positive (Ph+) hematological malignancies. The Bcr-Abl1 fusion protein tyrosine kinase is an abnormal enzyme produced by leukemia cells that contain the Philadelphia chromosome. NCI Drug Dictionary

Treatments listed in the registry

• Asciminib single agent (Drug): Asciminib (labelled as ABL001) administered as 40 mg tablet (adult formulation) or as 1 mg film-coated granules mini-tablets (pediatric formulation)

Na · Single Group · masking: None

Main goals

• MMR at Week 48 (48 weeks)

Other goals

• MMR at Week 96
• MMR at and by scheduled timepoints
• Hematologic response at and by scheduled timepoints
• Cytogenetic response at and by scheduled timepoints
• Time to response
• Duration of response (limited to the binary response endpoints)
• Time to treatment failure (TTF)
• Time to disease progression
• Event-free survival (EFS)
• Overall survival (OS)
• Growth: Height/length, weight, bone age measured by X-Ray
• Sexual maturation: Height/length, weight, bone age measured by Tanner staging
• PK parameters of asciminib: AUClast
• PK parameters of asciminib: AUCtau
• PK parameter of asciminib: Cmax
• PK parameter of asciminib: Tmax
• PK parameter of asciminib: Ctrough

Planned enrollment: 50 (estimated).

• Rutgers Cancer Institute of New Jersey, New Brunswick, New Jersey
• Columbia University Medical Center New York Presbyterian, New York, New York
• Cinn Children Hosp Medical Center, Cincinnati, Ohio
• Seattle Childrens Hospital, Seattle, Washington

30 recruiting sites outside the US. Site status as of Oct 30, 2026. Check the live record before contacting a site.

• ClinicalTrials.gov NCT07354074
• NCI Drug Dictionary: C171722

Registry record reviewed as fetched Oct 30, 2026, last updated by the sponsor Oct 14, 2026. Public record; not medical advice.