Asciminib for children and teens with Philadelphia chromosome–positive chronic myeloid leukemia in chronic phase

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a multi-center, open-label, single-arm Phase 2 trial enrolling pediatric participants aged 1 to under 18 with Ph+ CML in the chronic phase into three cohorts (newly diagnosed without T315I, resistant/intolerant without T315I, and any with known T315I). Participants will receive asciminib as a single agent using a pediatric weight‑based fed formulation or the adult fasted formulation depending on age/weight. There is no fixed treatment duration; the study will continue until five years after the last enrolled participant starts dosing to allow extended safety monitoring including growth and development. The primary focus is on clinical efficacy and safety outcomes in the pediatric population, with regular laboratory and molecular monitoring (BCR::ABL1) per protocol.

Why it matters

Who can join: the full rules

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.

Where it's running

• Ann and Robert H Lurie Childs Hosp
  Chicago, Illinois, United States Recruiting
• Rutgers Cancer Institute of New Jersey
  New Brunswick, New Jersey, United States Recruiting
• Columbia University Medical Center New York Presbyterian
  New York, New York, United States Recruiting
• Cinn Children Hosp Medical Center
  Cincinnati, Ohio, United States Recruiting
• Seattle Childrens Hospital
  Seattle, Washington, United States Recruiting
• Novartis Investigative Site
  Brisbane, Queensland, Australia Recruiting

Study contacts

• Study coordinator: Novartis Pharmaceuticals
• Email: novartis.email@novartis.com
• Phone: 1-888-669-6682

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Long-term safety treatment for chronic myelogenous leukemia patients

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• Asciminib for chronic phase CML with T315I mutation

  This study tests asciminib 200 mg twice daily in adults with chronic phase CML and a T315I mutation after stem cell transplant to see if it helps control leukemia.

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• Asciminib for Ph+ CML-CP in Germany: real-world patient experiences

  This real-world study tests how asciminib performs in adults with Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase, including those newly diagnosed or after one prior TKI.

  Recruiting at 74 sites Novartis · Baden-Baden, Baden-Wurttemberg and 73 other locations · NCT07549516

• Asciminib in children and young adults with relapsed or refractory Ph+ or ABL-class Ph-like ALL

  This study tests asciminib in kids and young adults with relapsed or refractory Philadelphia-positive or ABL-class Ph-like ALL to see if it is safe and helps treat the leukemia.

  Recruiting at 4 sites Phase 1/2 Novartis · Sydney, New South Wales and 3 other locations · NCT07387926

• Scemblix (asciminib) for newly diagnosed chronic-phase CML in China

  This project will try asciminib (Scemblix) in adults newly diagnosed with chronic-phase CML to see if it controls the disease and is safe.

  Recruiting Novartis · Wuhan, Hubei · NCT07375355

• Asciminib maintenance after alloHCT or CAR T for adults with Ph+ ALL

  Doctors will try asciminib in adults with Ph+ ALL who are in remission after an allogeneic stem cell transplant or CAR T therapy to check safety and find the best dose to help prevent relapse.

  Recruiting Phase 1 H. Lee Moffitt Cancer Center and Research Institute · Tampa, Florida · NCT07250087

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.