Health Trials

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

ConditionMyeloid Leukemia, Philadelphia Positive
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age1-17
SponsorNovartis Pharmaceuticals

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Pediatric formulation group: ≥ 1 and less than 18 years of age at study entry.

Adult formulation group: ≥ 14 and less than 18 years of age and body weight of ≥ 40 kg at study entry.

Participants with Ph+ CML-CP must meet all of the following laboratory values at the screening visit. In the case where bone marrow blast and promyelocyte counts are available, these will be accepted if done within 56 days prior to the screening visit, to avoid unnecessary repetition of this test.

< 15% blasts in peripheral blood and bone marrow

Disqualifiers

Known presence of the T315I mutation prior to study entry or a BCR::ABL mutation with known resistance to study treatment any time prior to study entry.

Known second chronic phase of CML after previous progression to AP/BC.

Previous treatment with a hematopoietic stem-cell transplantation.

Patient planning to undergo allogeneic hematopoietic stem cell transplantation.

Trial design

Design model

Sequential

Treatments tested in this trial

  • 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) 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.

Treatment groups

34 Participants
are divided into 1 treatment group
Group A: AsciminibExperimental treatment 2 interventions

Trial outcomes

Primary outcomes

1

Primary Pharmacokinetic (PK) parameter: AUClast

Goal: identifying the pediatric formulation dose (fed) leading to asciminib exposure comparable to 40 mg BID in adult patients (fasted).

Time frame
52 weeks
2

Primary PK parameter: AUCtau

Goal: identifying the pediatric formulation dose (fed) leading to asciminib exposure comparable to 40 mg BID in adult patients (fasted).

Time frame
52 weeks
3

Secondary PK parameter: Cmax

Goal: identifying the pediatric formulation dose (fed) leading to asciminib exposure comparable to 40 mg BID in adult patients (fasted).

Time frame
52 weeks
4

Secondary PK parameter: Tmax

Goal: identifying the pediatric formulation dose (fed) leading to asciminib exposure comparable to 40 mg BID in adult patients (fasted).

Time frame
52 weeks

Secondary outcomes

1

Hematologic responses

Complete hematological response will be defined as all of the following present for ≥ 4 weeks: * WBC count \< 10 x 10\^9/L * Platelet count \< 450 x 10\^9/L * Basophils \< 5% * No blasts and promyelocytes in peripheral blood * Myelocytes + metamyelocytes \< 5% in peripheral blood * No evidence of extramedullary disease, including spleen and liver

Time frame
52 weeks
2

Molecular responses

To assess pharmacodynamic markers of asciminib's anti-leukemic activity. Molecular response will be assessed by Breakpoint Cluster Region gene-Abelson proto-oncogene (BCR-ABL) 1 level.

Time frame
52 weeks
3

Questionnaire on acceptability and palatability after first dose, 4 and 52 weeks

To assess acceptability and palatability of the pediatric formulation

Time frame
after first dose at Week 1 Day 1, 4 weeks, 52 weeks

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Novartis Pharmaceuticals

Lead sponsor

Source ClinicalTrials.gov
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