(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 (NCT03731260)
A 3-Part, Randomized, Double-Blind, Placebo-Controlled Phase 2 Study to Evaluate Safety and Efficacy of Avapritinib (BLU-285), a Selective KIT Mutation-Targeted Tyrosine Kinase Inhibitor, in Indolent and Smoldering Systemic Mastocytosis With Symptoms Inadequately Controlled With Standard Therapy
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: University of Alabama at Birmingham — Birmingham, Alabama
Age range: 18 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (49 Sites)
Showing the first 25 of 49 sites.
- University of Alabama at Birmingham — Birmingham, Alabama
- Mayo Clinic Hospital — Phoenix, Arizona
- Stanford Cancer Institute — Stanford, California
- Mayo Clinic Florida — Jacksonville, Florida
- H. Lee Moffitt Cancer Center — Tampa, Florida
- Winship Cancer Institute, Emory University — Atlanta, Georgia
- Rush University Medical Center — Chicago, Illinois
- University of Kansas Hospital — Kansas City, Kansas
- Brigham & Women's Hospital — Boston, Massachusetts
- Dana Farber Cancer Institute — Boston, Massachusetts
- Michigan Medicine, University of Michigan — Ann Arbor, Michigan
- Mayo Clinic — Rochester, Minnesota
- Washington University School of Medicine — St Louis, Missouri
- Herbert Irving Comprehensive Cancer Center — New York, New York
- Duke University Health System (DUHS) — Durham, North Carolina
- University Hospitals Cleveland Medical Center — Cleveland, Ohio
- University of Texas, MD Anderson Cancer Center — Houston, Texas
- Huntsman Cancer Institute — Salt Lake City, Utah
- Virginia Commonwealth University Medical Center — Richmond, Virginia
- University Hospital Antwerp — Edegem,
- Tom Baker Cancer Centre — Calgary, Alberta
- University of Alberta Hospital — Edmonton, Alberta
- St. Michael's Hospital — Toronto, Ontario
- Odense Universitetshospital, ORCA/Allergicentret, Hudafdeling I og Allergicenter — Odense,
- Hôpital de la Timone, Service de dermatologie — Marseille,
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Indolent Systemic Mastocytosis
Explore by condition: All Indolent Systemic Mastocytosis clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.