NCT04925479From ClinicalTrials.govRecruiting

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

A Multi-center, Open-label Study to Determine the Dose and Safety of Oral Asciminib in Pediatric Patients With Philadelphia Chromosome Positive Chronic Myeloid Leukemia in Chronic Phase (Ph+ CML-CP), Previously Treated With One or More Tyrosine Kinase Inhibitors

Myeloid Leukemia, Philadelphia Positive

At a glance

Phase: Phase 1
Study type: Interventional
Sponsor: Novartis Pharmaceuticals
Enrolment target: 34
Started: 27 December 2021
Main results due: 2 September 2027
Study sites: 39
Registry updated: 25 September 2026

Can you take part?

Ages 1 year to 17 years.
Open to any sex.
You need the condition being studied — healthy volunteers are not accepted.

These are the headline rules only. Every study has a longer list, and whether you are eligible is decided by the research team at the site — never by this page.

Read the full eligibility criteria

Inclusion Criteria:

* 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.

1. \< 15% blasts in peripheral blood and bone marrow
2. \< 30% combined blasts plus promyelocytes in peripheral blood and bone marrow
3. \< 20% basophils in the peripheral blood
4. 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
5. No evidence of extramedullary leukemic involvement, with the exception of hepatosplenomegaly
* 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 Criteria:

* 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.

Other protocol-defined inclusion/exclusion may apply.

What this study is about

In the sponsor’s own words, from the registry.

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.

Study sites(39)

Indiana UH Riley H for CIU
Indianapolis, Indiana, United States
Active, not recruiting
Dana Farber Cancer Institute
Boston, Massachusetts, United States
Active, not recruiting
University of Mississippi Medical Center
Jackson, Mississippi, United States
Recruiting
Columbia University Medical Center New York Presbyterian
New York, New York, United States
Active, not recruiting
Cinn Children Hosp Medical Center
Cincinnati, Ohio, United States
Withdrawn
Childrens Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Withdrawn
Uni Of Texas MD Anderson Cancer Ctr
Houston, Texas, United States
Withdrawn
University Of Utah
Salt Lake City, Utah, United States
Withdrawn
Novartis Investigative Site
Hangzhou, Zhejiang, China
Withdrawn
Novartis Investigative Site
Beijing, China
Active, not recruiting
Novartis Investigative Site
Shanghai, China
Withdrawn
Novartis Investigative Site
Tianjin, China
Withdrawn
Novartis Investigative Site
Bordeaux, France
Withdrawn
Novartis Investigative Site
Lille, France
Active, not recruiting
Novartis Investigative Site
Paris, France
Active, not recruiting
Novartis Investigative Site
Poitiers, France
Withdrawn
Novartis Investigative Site
Erlangen, Germany
Active, not recruiting
Novartis Investigative Site
Essen, Germany
Active, not recruiting
Novartis Investigative Site
Hamburg, Germany
Active, not recruiting
Novartis Investigative Site
Athens, Greece
Active, not recruiting
Novartis Investigative Site
Budapest, Hungary
Withdrawn
Novartis Investigative Site
Genova, GE, Italy
Active, not recruiting
Novartis Investigative Site
Monza, MB, Italy
Withdrawn
Novartis Investigative Site
Roma, RM, Italy
Active, not recruiting
Novartis Investigative Site
Torino, TO, Italy
Withdrawn
Novartis Investigative Site
Yokohama, Kanagawa, Japan
Active, not recruiting
Novartis Investigative Site
Shinjuku-ku, Tokyo, Japan
Active, not recruiting
Novartis Investigative Site
Osaka, Japan
Active, not recruiting
Novartis Investigative Site
Utrecht, Netherlands
Active, not recruiting
Novartis Investigative Site
Wroclaw, Poland
Active, not recruiting
Novartis Investigative Site
Moscow, Russia
Withdrawn
Novartis Investigative Site
Saint Petersburg, Russia
Withdrawn
Novartis Investigative Site
Seoul, South Korea
Active, not recruiting
Novartis Investigative Site
Seoul, South Korea
Active, not recruiting
Novartis Investigative Site
Khon Kaen, THA, Thailand
Active, not recruiting
Novartis Investigative Site
Bangkok, Thailand
Withdrawn
Novartis Investigative Site
Chiang Mai, Thailand
Active, not recruiting
Novartis Investigative Site
Istanbul, Fatih, Turkey (Türkiye)
Active, not recruiting
Novartis Investigative Site
Bursa, Nilufer, Turkey (Türkiye)
Active, not recruiting

Showing 39 of 39 sites. 1 of the 39 sites on this study is recruiting right now — a site can stop enrolling while the study as a whole is still open.

Source: ClinicalTrials.gov record NCT04925479. Trialion does not run this study, is not paid to refer anyone to it, and cannot enrol you. Eligibility is always decided by the research team at the site.

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