RecruitingPhase 2

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

Australia, South Korea50 participantsStarted 2026-05-04

Plain-language 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.

Who can participate

Age range1 Year – 18 Years

SexALL

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Inclusion 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:
✓. Intolerance to TKI is defined as:

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

What they're measuring

MMR at Week 48

Timeframe: 48 weeks

Trial details

NCT IDNCT07354074

SponsorNovartis Pharmaceuticals

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2033-02-23

Contact for this trial

Novartis Pharmaceuticals

1-888-669-6682 novartis.email@novartis.com

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