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

NCT07354074 | Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: CABL001I122022025-522138-29
• Study Type: interventional
• Target: 50
• Locations: 2 countries
• Sites: 2

Brief 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.

Conditions

Chronic Myelogenous Leukemia; Leukemia, Myelogenous, Chronic, Philadelphia Chromosome Positive

Keywords

Asciminib; ABL001; Pediatric participants; Philadelphia chromosome positive chronic myeloid leukemia in chronic phase; Ph+ CML-CP; tyrosine kinase inhibitor; TKI; Molecular Response; MR; CML; Chronic phase; T3151; Ph+

Outcome Measures

Primary Outcomes (1)

• MMR at Week 48

  MMR is defined as BCR::ABL1 IS ≤0.1%. MMR is defined as a ≥ 3.0 log reduction in BCR::ABL1 transcripts compared to the standardized baseline equivalent to ≤ 0.1 % BCR::ABL1/ABL % by international scale as measured by RQ-PCR.

  48 weeks

Secondary Outcomes (17)

• MMR at Week 96

  96 weeks

• MMR at and by scheduled timepoints

  Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, and 96

• Hematologic response at and by scheduled timepoints

  Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, and 96

• Cytogenetic response at and by scheduled timepoints

  5 years

• Time to response

  240 weeks after the last participant enrolled in the study received the first dose of treatment

Study Arms (1)

Single arm study

EXPERIMENTAL

This study will enroll pediatric patients (≥ 1 and \< 18 years of age) with newly diagnosed or previously treated with Philadelphia positive Chronic Myelogenous Leukemia in Chronic Phase (Ph+ CML-CP) with or without known T315I mutation

Drug: Asciminib single agent

Interventions

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)

Also known as: ABL001

Single arm study

Eligibility Criteria

• Age: 1 Year - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify 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 qualify 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

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (2)

Novartis Investigative Site

Brisbane, Queensland, 4101, Australia

RECRUITING

Novartis Investigative Site

Seoul, 03080, South Korea

RECRUITING

MeSH Terms

Conditions

Leukemia, Myelogenous, Chronic, BCR-ABL Positive; Leukemia; Bronchiolitis Obliterans Syndrome

Interventions

asciminib

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Neoplasms by Histologic Type; Neoplasms; Myeloproliferative Disorders; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Organizing Pneumonia; Bronchiolitis Obliterans; Bronchiolitis; Bronchitis; Bronchial Diseases; Respiratory Tract Diseases; Lung Diseases, Obstructive; Lung Diseases; Graft vs Host Disease; Immune System Diseases

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Central Study Contacts

Novartis Pharmaceuticals

CONTACT

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

Novartis Pharmaceuticals

CONTACT

+41613241111

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR
• Expanded Access: Yes

Model Details: Three cohorts of Ph+ CML-CP pediatric participants receive asciminib: (1) newly-diagnosed without known T315I mutation; (2) resistant or intolerant to previous TKI without known T315I mutation; and (3)with known T315I mutation irrespective of prior TKI treatment. Outcomes are evaluated separately for each cohort.

Study Record Dates

• First Submitted: January 15, 2026
• First Posted: January 21, 2026
• Study Start: May 4, 2026
• Primary Completion (Estimated): February 23, 2033
• Study Completion (Estimated): February 23, 2033
• Last Updated: April 24, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share

Locations

KR (1); AU (1)