Actively Recruiting
Study to Determine the Dose and Safety of Asciminib in Pediatric Patients With Chronic Myeloid Leukemia
Led by Novartis Pharmaceuticals · Updated on 2026-04-30
34
Participants Needed
39
Research Sites
513 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Study to Determine the Dose and Safety of Asciminib in Pediatric Patients With Chronic Myeloid Leukemia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female participants aged 1 to less than 18 years for pediatric formulation group
- Participants aged 14 to less than 18 years and weighing at least 40 kg for adult formulation group
- Diagnosis of Philadelphia chromosome positive chronic myeloid leukemia in chronic phase
- Less than 15% blasts in peripheral blood and bone marrow
- Less than 30% combined blasts plus promyelocytes in peripheral blood and bone marrow
- Less than 20% basophils in peripheral blood
- Neutrophils at least 1.5 x 10^9/L (or WBC at least 3 x 10^9/L if neutrophils not available)
- Platelet count at least 100 x 10^9/L
- No extramedullary leukemic involvement except hepatosplenomegaly
- Prior treatment with at least one tyrosine kinase inhibitor
- Failure or intolerance to most recent TKI therapy at screening
- Karnofsky performance status at least 50% for patients 16 years or older
- Lansky performance status at least 50 for patients under 16 years
- Adequate renal, hepatic, pancreatic, and cardiac function
- Electrolyte values within or corrected to normal limits prior to first dose
- Evidence of typical BCR-ABL1 transcript [e14a2 and/or e13a2] at screening amenable to standardized RQ-PCR quantification
You will not qualify if you...
- Known presence of T315I mutation or BCR-ABL mutation with resistance to study treatment
- Known second chronic phase of CML after progression to accelerated phase or blast crisis
- Previous hematopoietic stem-cell transplantation
- Planned allogeneic hematopoietic stem cell transplantation
- Cardiac or cardiac repolarization abnormalities
- Severe or uncontrolled concurrent medical disease causing safety risks or compliance issues
- History of acute pancreatitis within 1 year or chronic pancreatitis
- History of acute or chronic liver disease
- Gastrointestinal impairment or disease affecting drug absorption
- Pregnant or nursing females
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 39 locations
1
Indiana UH Riley H for CIU
Indianapolis, Indiana, United States, 46202-5167
Actively Recruiting
2
Dana Farber Cancer Institute
Boston, Massachusetts, United States, 02215
Actively Recruiting
3
University of Mississippi Medical Center
Jackson, Mississippi, United States, 39216-4505
Actively Recruiting
4
Columbia University Medical Center New York Presbyterian
New York, New York, United States, 10032
Actively Recruiting
5
Cinn Children Hosp Medical Center
Cincinnati, Ohio, United States, 45206
Actively Recruiting
6
Childrens Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104 4399
Actively Recruiting
7
Uni Of Texas MD Anderson Cancer Ctr
Houston, Texas, United States, 77024
Actively Recruiting
8
University Of Utah
Salt Lake City, Utah, United States, 84132
Actively Recruiting
9
Novartis Investigative Site
Hangzhou, Zhejiang, China, 310052
Actively Recruiting
10
Novartis Investigative Site
Beijing, China, 100044
Actively Recruiting
11
Novartis Investigative Site
Shanghai, China, 200127
Actively Recruiting
12
Novartis Investigative Site
Tianjin, China, 300020
Actively Recruiting
13
Novartis Investigative Site
Bordeaux, France, 33076
Actively Recruiting
14
Novartis Investigative Site
Lille, France, 59000
Actively Recruiting
15
Novartis Investigative Site
Paris, France, 75019
Actively Recruiting
16
Novartis Investigative Site
Poitiers, France, 86021
Actively Recruiting
17
Novartis Investigative Site
Erlangen, Germany, 91054
Actively Recruiting
18
Novartis Investigative Site
Essen, Germany, 45147
Actively Recruiting
19
Novartis Investigative Site
Hamburg, Germany, 20246
Actively Recruiting
20
Novartis Investigative Site
Athens, Greece, 115 27
Actively Recruiting
21
Novartis Investigative Site
Budapest, Hungary, H-1083
Actively Recruiting
22
Novartis Investigative Site
Genova, GE, Italy, 16147
Actively Recruiting
23
Novartis Investigative Site
Monza, MB, Italy, 20900
Actively Recruiting
24
Novartis Investigative Site
Roma, RM, Italy, 00165
Actively Recruiting
25
Novartis Investigative Site
Torino, TO, Italy, 10126
Actively Recruiting
26
Novartis Investigative Site
Yokohama, Kanagawa, Japan, 232-8555
Actively Recruiting
27
Novartis Investigative Site
Shinjuku-ku, Tokyo, Japan, 1608582
Actively Recruiting
28
Novartis Investigative Site
Osaka, Japan, 5340021
Actively Recruiting
29
Novartis Investigative Site
Utrecht, Netherlands, 3584 CS
Actively Recruiting
30
Novartis Investigative Site
Wroclaw, Poland, 50367
Actively Recruiting
31
Novartis Investigative Site
Moscow, Russia, 117198
Actively Recruiting
32
Novartis Investigative Site
Saint Petersburg, Russia, 197022
Actively Recruiting
33
Novartis Investigative Site
Seoul, South Korea, 03080
Actively Recruiting
34
Novartis Investigative Site
Seoul, South Korea, 05505
Actively Recruiting
35
Novartis Investigative Site
Khon Kaen, THA, Thailand, 40002
Actively Recruiting
36
Novartis Investigative Site
Bangkok, Thailand, 10400
Actively Recruiting
37
Novartis Investigative Site
Chiang Mai, Thailand, 50200
Actively Recruiting
38
Novartis Investigative Site
Istanbul, Fatih, Turkey (Türkiye), 34093
Actively Recruiting
39
Novartis Investigative Site
Bursa, Nilufer, Turkey (Türkiye), 16059
Actively Recruiting
Research Team
N
Novartis Pharmaceuticals
CONTACT
N
Novartis Pharmaceuticals
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
1
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