A Study of Avapritinib in Pediatric Patients With Solid Tumors Dependent on KIT or PDGFRA Signaling
- Conditions
- Solid Tumor, Unspecified, ChildRelapsed Solid NeoplasmCNS Tumor
- Interventions
- Registration Number
- NCT04773782
- Lead Sponsor
- Blueprint Medicines Corporation
- Brief Summary
This is a Phase 1/2, multicenter, open-label trial of avapritinib in participants 2 to \< 18 years of age with advanced relapsed/refractory (R/R) solid tumors, including central nervous system (CNS) tumors, that harbor a PDGFRA and/or KIT mutation (including non-synonymous point mutations, insertions, and deletions) or amplification, or DMG-H3K27a who have no available curative treatment options. This is a single-arm trial in which all participants will receive avapritinib. The study consists of 2 parts: dose confirmation, safety, and PK (Part 1) and initial efficacy, safety, and PK at the Part 2 recommended dose (Part 2).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 37
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description avapritinib avapritinib Avapritinib tablets for oral administration. Avapritinib will be dosed daily for 28 day cycles.
- Primary Outcome Measures
Name Time Method Objective Response Rate (Part 2) up to 36 months Rate and severity of adverse events (Part 1) up to 8 months Determination of recommended Part 2 dose (Part 1) up to 8 months
- Secondary Outcome Measures
Name Time Method AUC(0-24) (Part 1 and Part 2) up to 36 months Change from baseline in levels of KIT and PDGFRA mutant allele fractions in peripheral blood (Part 1 and Part 2) up to 42 months Time to response (Part 1 and Part 2) up to 42 months Rate and severity of adverse events (Part 1 and Part 2) up to 42 months Duration of Response (Part 1 and Part 2) up to 42 months Tmax (Part 1) up to 36 months T 1/2 (Part 1) up to 36 months Palatability assessments as measured by the 5-point Hedonic scale (Part 1 and Part 2) up to 42 months Disease control rate (Part 1 and Part 2) up to 42 months Cmax (Part 1 and Part 2) up to 36 months Objective Response Rate (Part 1 and Part 2) up to 42 months Progression-free survival (Part 1 and Part 2) up to 42 months Ctrough (Part 2) up to 36 months
Trial Locations
- Locations (26)
Gustave Roussy
🇫🇷Villejuif, France
Universitaetsmedizin Göttingen
🇩🇪Göttingen, Lower Saxony, Germany
Hopp Children's Cancer Center
🇩🇪Heidelberg, Germany
Dipartimento di Oncologia Medica ed Ematologia - S.C. Pediatria Oncologica
🇮🇹Milan, Italy
Asan Medical Center
🇰🇷Seoul, Korea, Republic of
Samsung Medial Center
🇰🇷Seoul, Korea, Republic of
Great Ormond Street Hospital For Children
🇬🇧London, United Kingdom
Ann and Robert H. Lurie Children's Hospital of Chicago
🇺🇸Chicago, Illinois, United States
Phoenix Children's Hospital
🇺🇸Phoenix, Arizona, United States
University of California San Francisco, Benioff Children's Hospital
🇺🇸San Francisco, California, United States
Children's Hospital Colorado
🇺🇸Aurora, Colorado, United States
The Johns Hopkins Hospital
🇺🇸Baltimore, Maryland, United States
Dana Farber Cancer Institute
🇺🇸Boston, Massachusetts, United States
University of Michigan
🇺🇸Ann Arbor, Michigan, United States
Washington University School of Medicine
🇺🇸Saint Louis, Missouri, United States
Columbia University Medical Center
🇺🇸New York, New York, United States
Duke University Medical Center
🇺🇸Durham, North Carolina, United States
Nationwide Children's Hospital
🇺🇸Columbus, Ohio, United States
Oregon Health and Science University
🇺🇸Portland, Oregon, United States
UPMC Children's Hospital of Pittsburgh
🇺🇸Pittsburgh, Pennsylvania, United States
Children's Medical Center
🇺🇸Dallas, Texas, United States
University of Texas MD Anderson Cancer Center
🇺🇸Houston, Texas, United States
Sydney Children's Hospital, Kids Cancer Center
🇦🇺Sydney, New South Wales, Australia
Royal Children's Hospital
🇦🇺Parkville, Victoria, Australia
Medizinische Universitat Wein
🇦🇹Wien, Vienna, Austria
The Hospital for Sick Children
🇨🇦Toronto, Ontario, Canada