(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
Active, not recruiting · Phase 2 · NCT03731260
Conditions: Indolent Systemic Mastocytosis
At a glance
- Research payments reported, 2025
- $1.4M
- U.S. investigators named
- 16
- Sites paid
- 33
- Planned or actual enrollment
- 251
About the trial
- Sponsor
- Blueprint Medicines Corporation
- Tested
- Avapritinib (drug), Placebo (drug)
- Dates
- Started 2019-04-16; completion 2027-06-23
- Results posted
- Not yet
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.
From ClinicalTrials.gov, last updated 2026-10-02. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Blueprint Medicines Corporation reported $1.4M in research payments for this study in 2025 (201 payments).
Products named: Ayvakit
Research payments usually go to hospitals, universities and research sites to run a study, not to the investigators personally. A payment is not evidence of wrongdoing.
U.S. investigators named on this study
- Pankit J. Vachhani, MD Hematology & Oncology · Birmingham, AL
- Cecilia Y. Arana Yi, MD Hematology & Oncology · Scottsdale, AZ
- Jason Gotlib, M.D., M.S. Hematology · Palo Alto, CA
- Andrew T. Kuykendall, M.D. Internal Medicine · Tampa, FL
- Celalettin Ustun, MD Hematology · Chicago, IL
- Maria Castells, MD Allergy & Immunology · Gardner, MA
- Daniel J. Deangelo, M.D.,PH.D. Hematology & Oncology · Boston, MA
- Cem Akin, MD Allergy & Immunology · Northville, MI
- Thanai Pongdee, MD Allergy & Immunology · Rochester, MN
- Stephen T. Oh, MD Hematology · Saint Louis, MO
- Lindsay A. Rein, M.D. Hematology · Durham, NC
- Joseph G. Jurcic, MD Hematology · New York, NY
- Princess Ogbogu, MD Allergy & Immunology · Columbus, OH
- Prithviraj Bose, MD Hematology & Oncology · Houston, TX
- Tsewang Tashi, M.D. Hematology & Oncology · Salt Lake City, UT
- Ruchi J. Desai, MD Hematology · Richmond, VA
Clinicians the company named as principal investigators in its Open Payments reports.
Sites paid to run it
- University of Michigan Health System, MI $373,665
- Brigham and Women's Hospital, MA $236,885
- University of Utah Hospital and Clinics, UT $122,497
- Board of Trustees of the Leland Stanford Junior University $69,980
- University of Alabama Hospital, AL $58,835
- Duke University $53,227
- Uh Cleveland Medical Center, OH $46,984
- The Board of Trustees of the Leland Stanford Junior University $44,199
- Ut MD Anderson Cancer Center $42,869
- Dana Farber/partners Cancer Care Inc $39,959
- University of Utah $39,812
- Mayo Clinic $36,324
- Mayo Clinic School of Continuous Professional Development $35,226
- Stanford University Care Fund Ghghm $26,776
- Virginia Commonwealth University $20,881
- Rush University Medical Center, IL $18,702
- The Trustees of Columbia University in The City of New York $14,318
- Vcu West Hospital Infectious Disease Clinic $13,921
- Mayo Clinic Rochester $12,711
- Moffitt Cancer Center $12,183
- Washington University Division $12,109
- Washington University $12,048
- The Washington University Rollup $10,569
- Dana Farber PArtners Cancer Care Inc $7,788
- Virginia Commonwealth University Health $6,960
- University of Alabama at Birmingham $4,792
- Mayo Clinic Hospital, AZ $4,476
- University Hospitals Cleveland Medical Center $2,982
- 340b Rush University Medical Center $2,225
- Dana Farber Mass General Brigham Ca Care Inc $2,187
- Mayo Clinic Arizona Sc Johnson Research Building $450
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.