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Clinical Trials in China / NCT04925479
Recruiting Phase 1/2

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

NCT04925479 · tracked via the Priya Life Science China tracker
Sponsor
Novartis Pharmaceuticals
Phase
Phase 1/2
Started
2021-12-27
Last updated
2026-09-22

Condition(s) studied

Myeloid Leukemia, Philadelphia Positive

Investigational drug(s) / intervention(s)

Asciminib Pediatric formulation groupAsciminib Adult formulation group

Asciminib Pediatric formulation group: 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: Asciminib Adult formulation group: 40 mg tablets BID, taken orally. 20 mg tablets BID, taken orally.

Study 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.

Eligibility

Sex
ALL
Min age
1 Year
Max age
17 Years
Healthy volunteers
No
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.

Primary outcome measure(s)

Trial sites (39)

FacilityCityRegionStatus
Indiana UH Riley H for CIU Indianapolis Indiana Active Not Recruiting
Dana Farber Cancer Institute Boston Massachusetts Active Not Recruiting
University of Mississippi Medical Center Jackson Mississippi Recruiting
Columbia University Medical Center New York Presbyterian New York New York Active Not Recruiting
Cinn Children Hosp Medical Center Cincinnati Ohio Withdrawn
Childrens Hospital of Philadelphia Philadelphia Pennsylvania Withdrawn
Uni Of Texas MD Anderson Cancer Ctr Houston Texas Withdrawn
University Of Utah Salt Lake City Utah Withdrawn
Novartis Investigative Site Hangzhou Zhejiang 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 Active Not Recruiting
Novartis Investigative Site Monza MB Withdrawn
Novartis Investigative Site Roma RM Active Not Recruiting
Novartis Investigative Site Torino TO Withdrawn
Novartis Investigative Site Yokohama Kanagawa Active Not Recruiting
Novartis Investigative Site Shinjuku-ku Tokyo 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 Recruiting
Novartis Investigative Site Seoul South Korea Active Not Recruiting
Novartis Investigative Site Seoul South Korea Active Not Recruiting
Novartis Investigative Site Khon Kaen THA Active Not Recruiting
Novartis Investigative Site Bangkok Thailand Recruiting
Novartis Investigative Site Chiang Mai Thailand Active Not Recruiting
Novartis Investigative Site Istanbul Fatih Active Not Recruiting
Novartis Investigative Site Bursa Nilufer Active Not Recruiting

More Novartis Pharmaceuticals trials in China

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT04925479 on ClinicalTrials.gov ↗ ← All trials in China