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Asciminib for Children and Teens with Chronic Myeloid Leukemia

This study is testing a medication called asciminib in children and teenagers (ages 1 to 17) who have Philadelphia chromosome positive chronic myeloid leukemia (Ph+ CML-CP) and have already been treated with other similar medications. The main goal is to find the right dose of asciminib in a special pediatric formulation (granules in a capsule) that gives similar levels of the medicine in the body as the adult dose. The study will also look at how safe asciminib is for young patients. Researchers believe CML works similarly in children and adults, so they are using what they know from adult studies to help children. About 34 participants are planned for this study.

Study design
This study is an interventional study, meaning participants will receive a specific treatment. It plans to enroll 34 participants and will test different doses of asciminib.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
The study will measure how the drug is absorbed and processed in the body for 52 weeks.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT04925479

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

Recruiting
PHASE1Ages 1–17InterventionalTreatment
Novartis Pharmaceuticals
~34 participants
Updated 2026-08-27 on ClinicalTrials.gov
What's tested:Asciminib Pediatric formulation groupAsciminib Adult formulation group

At a glance

Recruiting sites
38 of 39 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Primary Pharmacokinetic (PK) parameter: AUClast
Measured over 52 weeks
+4 more outcomes measured

Conditions

Myeloid Leukemia, Philadelphia Positive

Explore related trials

Chronic Myeloid Leukemia, BCR-ABL1 Positivet(9;22)(q34.1;q11.2)

Where it's being run

39 sites across 29 states
France4
China3
Germany3
Russia2
South Korea2
Thailand2
Indiana1
Massachusetts1

Study leadership

  • Novartis Pharmaceuticals · STUDY_DIRECTOR · Novartis Pharmaceuticals

Who to contact

Novartis Pharmaceuticals
[email protected]
1-888-669-6682
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

Male or female participants:
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.
Prior treatment with a minimum of one TKI
Failure (adapted from the 2020 European Leukemia Net (ELN) Guidelines Hochhaus et al 2020 and 2013 ELN Guidelines Baccarani et al 2013) or intolerance to the most recent TKI therapy at the time of screening.
Performance status: Karnofsky ≥ 50% for patients ≥ 16 years of age, and Lansky ≥ 50 for patients \< 16 years of age at the time of screening
Participants must have adequate renal, hepatic, pancreatic and cardiac function
Participants must have electrolyte values within normal limits or corrected to be within normal limits with supplements prior to first dose of study medication:
Evidence of typical BCR::ABL1 transcript \[e14a2 and/or e13a2\] at the time of screening which are amenable to standardized RQ-PCR quantification.

Exclusion

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.
Cardiac or cardiac repolarization abnormality
Severe and/or uncontrolled concurrent medical disease that in the opinion of the Investigator could cause unacceptable safety risks or compromise compliance with the protocol
History of acute pancreatitis within 1 year of study entry or past medical history of chronic pancreatitis.
History of acute or chronic liver disease.
Impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of study drug
Pregnant or nursing (lactating) females.

What this trial measures

  • Primary Pharmacokinetic (PK) parameter: AUClast52 weeks

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

  • Primary PK parameter: AUCtau52 weeks

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

  • Secondary PK parameter: Cmax52 weeks

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

  • Secondary PK parameter: Tmax52 weeks

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

  • Secondary PK parameter: Ctrough52 weeks

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