RecruitingPhase 1/2

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

United States, China, France34 participantsStarted 2021-12-27

Plain-language summary

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.

Who can participate

Age range

1 Year – 17 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. \< 15% blasts in peripheral blood and bone marrow
. \< 30% combined blasts plus promyelocytes in peripheral blood and bone marrow
. \< 20% basophils in the peripheral blood
. Neutrophils ≥ 1.5 x 10\^9/L (or WBC ≥ 3 x 10\^9/L if neutrophils are not available) and platelet count ≥ 100 x 10\^9/L
. No evidence of extramedullary leukemic involvement, with the exception of hepatosplenomegaly

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Primary Pharmacokinetic (PK) parameter: AUClast

Timeframe: 52 weeks

Primary PK parameter: AUCtau

Timeframe: 52 weeks

Secondary PK parameter: Cmax

Timeframe: 52 weeks

Secondary PK parameter: Tmax

Timeframe: 52 weeks

Secondary PK parameter: Ctrough

Timeframe: 52 weeks

Trial details

NCT IDNCT04925479

SponsorNovartis Pharmaceuticals

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2026-06-16

Contact for this trial

Novartis Pharmaceuticals

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

View on ClinicalTrials.gov More Myeloid Leukemia, Philadelphia Positive trials

Plain-language summary

Who can participate

Age range

1 Year – 17 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. \< 15% blasts in peripheral blood and bone marrow
. \< 30% combined blasts plus promyelocytes in peripheral blood and bone marrow
. \< 20% basophils in the peripheral blood
. Neutrophils ≥ 1.5 x 10\^9/L (or WBC ≥ 3 x 10\^9/L if neutrophils are not available) and platelet count ≥ 100 x 10\^9/L
. No evidence of extramedullary leukemic involvement, with the exception of hepatosplenomegaly