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

What this study is about

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.

Who can join (things the study team will check)

✅ You may be able to join if…

• 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.

🚫 You may not be able to join if…

• 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.

Where this trial is running

• Rutgers Cancer Institute of New Jersey, New Brunswick, New Jersey, United States
• Columbia University Medical Center New York Presbyterian, New York, New York, United States
• Seattle Childrens Hospital, Seattle, Washington, United States
• Novartis Investigative Site, Brisbane, Queensland, Australia
• Novartis Investigative Site, North Adelaide, South Australia, Australia
• Novartis Investigative Site, Edmonton, Alberta, Canada
• Novartis Investigative Site, Montreal, Quebec, Canada
• Novartis Investigative Site, Zhengzhou, Henan, China
• Novartis Investigative Site, Tianjin, China
• Novartis Investigative Site, Marseille, France
• Novartis Investigative Site, Paris, France
• Novartis Investigative Site, Poitiers, France

+ 12 more sites.

Who to contact

Novartis Pharmaceuticals · 1-888-669-6682 · novartis.email@novartis.com

It's completely normal to call and ask questions before deciding anything. Mention the study ID: NCT07354074.