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

Recruiting now · Phase 1/Phase 2

Conditions studied: Myeloid Leukemia, Philadelphia Positive

In brief

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 facts

Study ID

NCT04925479

Run by

Novartis Pharmaceuticals

People needed

34

Starts

2021-12-27

Expected to finish

2031-11-01

Last updated by the study team

2026-06-30

Who can join

Age: 1 and older, up to 17. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.
• < 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
• 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.

You may not qualify if…

• 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.

Where it is running

• Indiana UH Riley H for CIU — Indianapolis, Indiana, United States (enrolling)
• Dana Farber Cancer Institute — Boston, Massachusetts, United States (enrolling)
• University of Mississippi Medical Center — Jackson, Mississippi, United States (enrolling)
• Columbia University Medical Center New York Presbyterian — New York, New York, United States (enrolling)
• Cinn Children Hosp Medical Center — Cincinnati, Ohio, United States (enrolling)
• Childrens Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (enrolling)
• Uni Of Texas MD Anderson Cancer Ctr — Houston, Texas, United States (enrolling)
• University Of Utah — Salt Lake City, Utah, United States (enrolling)
• Novartis Investigative Site — Hangzhou, Zhejiang, China (enrolling)
• Novartis Investigative Site — Beijing, China (enrolling)
• Novartis Investigative Site — Shanghai, China (enrolling)
• Novartis Investigative Site — Tianjin, China (enrolling)
• Novartis Investigative Site — Bordeaux, France (enrolling)
• Novartis Investigative Site — Lille, France (enrolling)
• Novartis Investigative Site — Paris, France (enrolling)
• Novartis Investigative Site — Poitiers, France (enrolling)
• Novartis Investigative Site — Erlangen, Germany (enrolling)
• Novartis Investigative Site — Essen, Germany (enrolling)
• Novartis Investigative Site — Hamburg, Germany (enrolling)
• Novartis Investigative Site — Athens, Greece (enrolling)
• Novartis Investigative Site — Budapest, Hungary (enrolling)
• Novartis Investigative Site — Genova, GE, Italy (enrolling)
• Novartis Investigative Site — Monza, MB, Italy (enrolling)
• Novartis Investigative Site — Roma, RM, Italy (enrolling)
• Novartis Investigative Site — Torino, TO, Italy (enrolling)

Full record on ClinicalTrials.gov

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