Study to Determine the Efficacy and Safety of Asciminib in Pediatric Patients With Ph+ CML-CP
Start Date
4/28/2026
Completion Date
2/23/2033
Summary
The aim of this study is to support development of asciminib in the pediatric population (1 to \< 18 years) with Ph+ CML-CP. The study will evaluate the efficacy and safety of asciminib in pediatric formulation (weigh-based dose, fed state) or adult formulation (fasted) in newly diagnosed and resistant or intolerant Ph+ CML-CP with or without T315I mutation.
Detailed Description
This is a multi-center, open-label, single arm study of asciminib in pediatric participants aged 1 to \<18 years old with Ph+ CML-CP newly diagnosed and previously treated with TKI treatment, with or without T315I mutation. The study population will consist of three cohorts of Ph+ CML-CP pediatric participants: * Newly-diagnosed Ph+ CML-CP participants without known T315I mutation * Ph+ CML-CP participants resistant or intolerant to previous TKI without known T315I mutation * Ph+ CML-CP participants with known T315I mutation irrespective of prior TKI treatment There is no fixed duration of study treatment for the participants. The study will end 5 years (240 weeks) after the last enrolled participants received their first dose of treatment in the study. The objective is to have enough follow up for safety, including growth and development and efficacy.
Eligibility Criteria
Age Range: 1 years to 18 years
Interventions
Asciminib single agent
Conditions
Locations
Rutgers Cancer Institute of New Jersey
New Brunswick, New Jersey 08901
United States
Columbia University Medical Center New York Presbyterian
New York, New York 10032
United States
Seattle Childrens Hospital
Seattle, Washington 98105
United States
Novartis Investigative Site
Brisbane, Queensland 4101
Australia
Novartis Investigative Site
North Adelaide, South Australia 5066
Australia
Novartis Investigative Site
Edmonton, Alberta T6G 1C9
Canada
Novartis Investigative Site
Montreal, Quebec H3T 1C5
Canada
Novartis Investigative Site
Zhengzhou, Henan 450003
China
Novartis Investigative Site
Tianjin, 300020
China
Novartis Investigative Site
Marseille, 13885
France
Novartis Investigative Site
Paris, 75019
France
Novartis Investigative Site
Poitiers, 86021
France
Novartis Investigative Site
Strasbourg, 67000
France
Novartis Investigative Site
Roma, RM 00165
Italy
Novartis Investigative Site
Yokohama, Kanagawa 232-8555
Japan
Novartis Investigative Site
Fukuoka, 8128582
Japan
Novartis Investigative Site
Saitama, 330 8777
Japan
Novartis Investigative Site
Utrecht, 3584 CS
Netherlands
Novartis Investigative Site
Gdansk, 80-214
Poland
Novartis Investigative Site
Wroclaw, 50-556
Poland
Novartis Investigative Site
Seoul, 03080
South Korea
Novartis Investigative Site
Seoul, 06591
South Korea
Novartis Investigative Site
Barcelona, 08035
Spain
Novartis Investigative Site
Madrid, 28009
Spain