RecruitingPhase 1Phase 2NCT04925479

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

A Multi-center, Open-label Study to Determine the Dose and Safety of Oral Asciminib in Pediatric Patients With Philadelphia Chromosome Positive Chronic Myeloid Leukemia in Chronic Phase (Ph+ CML-CP), Previously Treated With One or More Tyrosine Kinase Inhibitors


Sponsor

Novartis Pharmaceuticals

Enrollment

34 participants

Start Date

Dec 27, 2021

Study Type

INTERVENTIONAL

Conditions

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

Min Age: 1 YearMax Age: 17 Years

Plain Language Summary

Simplified for easier understanding

This study is testing asciminib, a targeted drug that works differently from other leukemia medications, in children and teenagers with a type of blood cancer called chronic myeloid leukemia (CML) whose cancer has not responded to or cannot tolerate other treatments. **You may be eligible if:** - You are between 1 and under 18 years old (or 14–18 with a weight of at least 40 kg for the adult tablet form) - You have Philadelphia chromosome-positive CML in chronic phase - You have tried at least one prior targeted therapy (TKI) that either failed or caused intolerable side effects - Your blood counts and organ function meet the required thresholds **You may NOT be eligible if:** - Your leukemia has a specific mutation (T315I) known to resist the study drug - You have previously had a stem cell transplant - You are planning to have an allogeneic stem cell transplant - You have significant heart, liver, or pancreatic problems - You are currently taking strong medications that interfere with how the study drug is processed by the body - You are pregnant or breastfeeding Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGAsciminib 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)

DRUGAsciminib Adult formulation group

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


Locations(39)

Indiana UH Riley H for CIU

Indianapolis, Indiana, 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

Novartis Investigative Site

Beijing, China

Novartis Investigative Site

Shanghai, China

Novartis Investigative Site

Tianjin, China

Novartis Investigative Site

Bordeaux, France

Novartis Investigative Site

Lille, France

Novartis Investigative Site

Paris, France

Novartis Investigative Site

Poitiers, France

Novartis Investigative Site

Erlangen, Germany

Novartis Investigative Site

Essen, Germany

Novartis Investigative Site

Hamburg, Germany

Novartis Investigative Site

Athens, Greece

Novartis Investigative Site

Budapest, Hungary

Novartis Investigative Site

Genova, GE, Italy

Novartis Investigative Site

Monza, MB, Italy

Novartis Investigative Site

Roma, RM, Italy

Novartis Investigative Site

Torino, TO, Italy

Novartis Investigative Site

Yokohama, Kanagawa, Japan

Novartis Investigative Site

Shinjuku-ku, Tokyo, Japan

Novartis Investigative Site

Osaka, Japan

Novartis Investigative Site

Utrecht, Netherlands

Novartis Investigative Site

Wroclaw, Poland

Novartis Investigative Site

Moscow, Russia

Novartis Investigative Site

Saint Petersburg, Russia

Novartis Investigative Site

Seoul, South Korea

Novartis Investigative Site

Seoul, South Korea

Novartis Investigative Site

Khon Kaen, THA, Thailand

Novartis Investigative Site

Bangkok, Thailand

Novartis Investigative Site

Chiang Mai, Thailand

Novartis Investigative Site

Istanbul, Fatih, Turkey (Türkiye)

Novartis Investigative Site

Bursa, Nilufer, Turkey (Türkiye)

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NCT04925479