(PIONEER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, Versus Placebo in Patients With Indolent Systemic Mastocytosis
Running, not enrolling · Phase 2 · Has a placebo group
Conditions studied: Indolent Systemic Mastocytosis
In brief
This is a Phase 2, randomized, double-blind, placebo-controlled study comparing the efficacy and safety of avapritinib + best supportive care (BSC) with placebo + BSC in patients with indolent systemic mastocytosis (ISM) whose symptoms are not adequately controlled by BSC. The study will be conducted in 3 parts. All patients will receive treatment with avapritinib during Part 3 including those rolling over from the placebo group.
Key facts
- Study ID
- NCT03731260
- Run by
- Blueprint Medicines Corporation
- People needed
- 251
- Starts
- 2019-04-16
- Expected to finish
- 2027-06-23
- Last updated by the study team
- 2025-09-23
Who can join
Age: 18 and older. Sex: any. Healthy volunteers: not accepted.
Where it is running
- University of Alabama at Birmingham — Birmingham, Alabama, United States
- Mayo Clinic Hospital — Phoenix, Arizona, United States
- Stanford Cancer Institute — Stanford, California, United States
- Mayo Clinic Florida — Jacksonville, Florida, United States
- H. Lee Moffitt Cancer Center — Tampa, Florida, United States
- Winship Cancer Institute, Emory University — Atlanta, Georgia, United States
- Rush University Medical Center — Chicago, Illinois, United States
- University of Kansas Hospital — Kansas City, Kansas, United States
- Brigham & Women's Hospital — Boston, Massachusetts, United States
- Dana Farber Cancer Institute — Boston, Massachusetts, United States
- Michigan Medicine, University of Michigan — Ann Arbor, Michigan, United States
- Mayo Clinic — Rochester, Minnesota, United States
- Washington University School of Medicine — St Louis, Missouri, United States
- Herbert Irving Comprehensive Cancer Center — New York, New York, United States
- Duke University Health System (DUHS) — Durham, North Carolina, United States
- University Hospitals Cleveland Medical Center — Cleveland, Ohio, United States
- University of Texas, MD Anderson Cancer Center — Houston, Texas, United States
- Huntsman Cancer Institute — Salt Lake City, Utah, United States
- Virginia Commonwealth University Medical Center — Richmond, Virginia, United States
- University Hospital Antwerp — Edegem, Belgium
- Tom Baker Cancer Centre — Calgary, Alberta, Canada
- University of Alberta Hospital — Edmonton, Alberta, Canada
- St. Michael's Hospital — Toronto, Ontario, Canada
- Odense Universitetshospital, ORCA/Allergicentret, Hudafdeling I og Allergicenter — Odense, Denmark
- Hôpital de la Timone, Service de dermatologie — Marseille, France
Full record on ClinicalTrials.gov
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