RecruitingPhase 2NCT07354074
Official study title

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

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.

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Key things to know

Main goalTreatment

This describes the study’s primary purpose.

What is being studiedAsciminib single agent

The study team can explain what participation involves.

Who it may be forChronic Myelogenous Leukemia · Leukemia, Myelogenous, Chronic, Philadelphia Chromosome Positive

These are broad labels—not an eligibility decision.

Checked against the public recordLast updated Oct 15, 2026 · Source: ClinicalTrials.gov

Is enrollment open?Recruiting

The study is currently accepting participants.

What kind of study?Phase 2

Interventional

Where is it offered?34 U.S. locations

Locations may change over time.

Who runs it?Novartis Pharmaceuticals

Study sponsor

01
Study overview

What this study is about

Purpose
Treatment
Study type
Interventional
Phase
Phase 2
Sponsor
Novartis Pharmaceuticals
Interventions being studied
Drug: Asciminib single agent
02
Explore related studies

How this study is categorized

These labels come from structured fields and exact terms in the public record.

03
Public criteria

Who may be able to participate

The sponsor separates these requirements into two groups. You do not need to interpret them alone—use them to guide a conversation with the study team.

Requirements people may need to meetInclusion criteria
  • Key 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
Reasons someone may not be able to joinExclusion 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/

Important: This is the sponsor’s public criteria, not a determination of eligibility. The study team must review your individual situation.

04
Study sites

U.S. locations

  • Rutgers Cancer Institute of New JerseyNew Brunswick, New Jersey
  • Columbia University Medical Center New York PresbyterianNew York, New York
  • Cinn Children Hosp Medical CenterCincinnati, Ohio
  • Seattle Childrens HospitalSeattle, Washington
  • Novartis Investigative SiteBrisbane, Queensland
  • Novartis Investigative SiteNorth Adelaide, South Australia
  • Novartis Investigative SiteRio de Janeiro, Rio de Janeiro
  • Novartis Investigative SiteBarretos, São Paulo
  • Novartis Investigative SiteSão Paulo, São Paulo
  • Novartis Investigative SiteEdmonton, Alberta
  • Novartis Investigative SiteMontreal, Quebec
  • Novartis Investigative SiteZhengzhou, Henan

Source and freshness
Processed from ClinicalTrials.gov. Last public update: Oct 15, 2026. Always confirm current availability with the study team.

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