Study to Determine the Dose and Safety of Asciminib in Pediatric Patients With Chronic Myeloid Leukemia
NCT ID: NCT04925479
Last Updated: 2025-09-17
Study Results
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Basic Information
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RECRUITING
PHASE1/PHASE2
44 participants
INTERVENTIONAL
2021-12-27
2031-11-01
Brief Summary
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Detailed Description
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The primary objective of this study is to characterize the pharmacokinetic (PK) profile of asciminib in pediatric patients with the goal of identifying the pediatric formulation dose (fed) leading to asciminib exposure comparable to 40 mg BID in adult patients (fasted).
The pediatric formulation group will include at least 15 participants in each of the following two age categories: 1 to \<12 years and 12 to \<18 years; leading to at least 30 participants enrolled treated with the pediatric formulation. It will consist of a dose determination part (Part 1) and a cohort expansion (Part 2 BID regimen and Part 3 QD regimen).
In Part 1, 4-6 participants will be enrolled in order to obtain at least 4 participants evaluable for PK (these participants may be from either of the age categories described above). The initial starting dose will be based on body weight and will be administered BID with food.
Once the body weight adjusted dose has been determined in Part 1 of the study, the patients will be enrolled in Part 2 until at least 20 participants, including those who were included in Part 1, have been enrolled (10 per age group) in the pediatric formulation group. Once the interim safety and PK analysis 2 is completed for one of the age groups, the Part 3 QD regimen will open for the respective age group to enroll 10 patients (5 patients by age group).
Due to the fact that the pediatric formulation was in development and was not available, this study started with the recruitment of adolescent patients. These participants aged 14 to \<18 years, weighing at least 40 kg receive the adult formulation at a flat dose of 40 mg BID under fasted conditions.
The total duration of the treatment period of the study will be 5 years (260 weeks). Participants who, according to Investigator's judgement, are benefiting from study treatment will remain on treatment up to the completion of the treatment period (Week 260/5 years). The primary analysis for the BID regimen is planned after all participants in Part 1 and 2 have completed at least 52 weeks of study treatment or discontinued earlier.
The primary analysis for combined regimen (BID+QD) is planned after all participants in Part 1, 2 and 3 have completed at least 52 weeks of study treatment or discontinued earlier.
Conditions
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Study Design
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NA
SEQUENTIAL
TREATMENT
NONE
Study Groups
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Asciminib
This arm consists of 2 groups:
* The pediatric formulation group where the dose is based on body weight (1.3mg/kg BID or 2.6 mg/kg QD)
* The adult formulation group where participants will receive a flat dose of 40mg BID
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) 20 mg (20x 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.
Interventions
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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) 20 mg (20x 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.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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 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.
1 Year
17 Years
ALL
No
Sponsors
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Novartis Pharmaceuticals
INDUSTRY
Responsible Party
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Principal Investigators
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Novartis Pharmaceuticals
Role: STUDY_DIRECTOR
Novartis Pharmaceuticals
Locations
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Indiana UH Riley H for CIU
Indianapolis, Indiana, United States
Dana Farber Cancer Institute
Boston, Massachusetts, United States
Dana Farber Cancer Institute
Boston, Massachusetts, United States
University of Mississippi Medical Center
Jackson, Mississippi, United States
Columbia University Medical Center New York Presbyterian
New York, New York, United States
Cinn Children Hosp Medical Center
Cincinnati, Ohio, United States
Childrens Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Uni Of Texas MD Anderson Cancer Ctr
Houston, Texas, United States
University Of Utah
Salt Lake City, Utah, United States
Novartis Investigative Site
Hangzhou, Zhejiang, China
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Beijing, , China
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Shanghai, , China
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Tianjin, , China
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Bordeaux, , France
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Lille, , France
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Paris, , France
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Poitiers, , France
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Erlangen, , Germany
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Essen, , Germany
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Hamburg, , Germany
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Athens, , Greece
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Budapest, , Hungary
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Genova, GE, Italy
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Monza, MB, Italy
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Roma, RM, Italy
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Torino, TO, Italy
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Yokohama, Kanagawa, Japan
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Shinjuku-ku, Tokyo, Japan
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Osaka, , Japan
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Utrecht, , Netherlands
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Wroclaw, , Poland
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Moscow, , Russia
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Saint Petersburg, , Russia
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Seoul, , South Korea
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Seoul, , South Korea
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Khon Kaen, THA, Thailand
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Bangkok, , Thailand
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Chiang Mai, , Thailand
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Istanbul, Fatih, Turkey (Türkiye)
Novartis Investigative Site
Bursa, Nilufer, Turkey (Türkiye)
Countries
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Central Contacts
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Novartis Pharmaceuticals
Role: CONTACT
Facility Contacts
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Role: primary
Role: primary
Other Identifiers
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2021-001286-20
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
CABL001I12201
Identifier Type: -
Identifier Source: org_study_id
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