Study to Determine the Dose and Safety of Asciminib in Pediatric Patients With Chronic Myeloid Leukemia

Conditions

• Myeloid Leukemia, Philadelphia Positive

Eligibility Criteria

Sex

ALL

Age

1 Year - 17 Years

Healthy Volunteers

Not accepted

Interventions

• Asciminib Pediatric formulation group — DRUG
  Asciminib Pediatric formulation group: 1 mg film-coated granules in a size 0 capsule will be supplied, taken orally (capsules are a container for the granules and are not ingested): 10 mg (10x 1 mg film-coated granules in capsule) 15 mg (15x 1 mg film-coated granules in capsule) 20 mg (20x 1 mg film-coated granules in capsule) 30 mg (30x 1 mg film-coated granules in capsule)
• Asciminib Adult formulation group — DRUG
  Asciminib Adult formulation group: 40 mg tablets BID, taken orally. 20 mg tablets BID, taken orally.

Study Details

The aim of this study is to support development of asciminib in the pediatric population (1 to \<18 years) previously treated with one or more TKIs. Full extrapolation of the efficacy of asciminib from adult to pediatric patients will be conducted. Full extrapolation is based on the concept that CML in the pediatric population has the same pathogenesis, similar clinical characteristics and progression pattern as in adults.

Key Dates

Start date

Dec 27, 2021

Status verified

Apr 2026

Primary completion

Jun 16, 2026

Completion

Nov 1, 2031

Study Design

Enrollment

34 participants (estimated)

Allocation

NA

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Arms

• Experimental: Asciminib
  This arm consists of 2 groups: * The pediatric formulation group where the dose is based on body weight (1.3mg/kg BID or 2.6 mg/kg QD) * The adult formulation group where participants will receive a flat dose of 40mg BID

Primary Outcome Measure

Primary Pharmacokinetic (PK) parameter: AUClast [ Time Frame: 52 weeks ]

Central Contacts

• Novartis Pharmaceuticals
  1-888-669-6682
  [email protected]
• Novartis Pharmaceuticals
  +41613241111

Locations (8)

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