Purpose

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

Eligibility

Eligible Ages
Between 1 Year and 18 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

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.

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.

Study Design

Phase
Phase 2
Study Type
Interventional
Allocation
N/A
Intervention Model
Single Group Assignment
Intervention Model Description
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.
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Single arm study
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
    Asciminib (labelled as ABL001) administered as 40 mg tablet (adult formulation) or as 1 mg film-coated granules mini-tablets (pediatric formulation)
    Other names:
    • ABL001

Recruiting Locations

Rutgers Cancer Institute of New Jersey
New Brunswick, New Jersey 08901
Contact:
Jesmin Habeeb Mohamed
jsh216@cinj.rutgers.edu

Columbia University Medical Center New York Presbyterian
New York, New York 10032
Contact:
Karishma Sookraj
212-305-6953
kss2158@cumc.columbia.edu

More Details

Status
Recruiting
Sponsor
Novartis Pharmaceuticals

Study Contact

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

Detailed Description

This is a multi-center, open-label, single arm study of asciminib in pediatric participants aged 1 to <18 years old with Ph+ CML-CP newly diagnosed and previously treated with TKI treatment, with or without T315I mutation. The study population will consist of three cohorts of Ph+ CML-CP pediatric participants: - Newly-diagnosed Ph+ CML-CP participants without known T315I mutation - Ph+ CML-CP participants resistant or intolerant to previous TKI without known T315I mutation - Ph+ CML-CP participants with known T315I mutation irrespective of prior TKI treatment There is no fixed duration of study treatment for the participants. The study will end 5 years (240 weeks) after the last enrolled participants received their first dose of treatment in the study. The objective is to have enough follow up for safety, including growth and development and efficacy.

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.