(PATHFINDER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, in Patients With Advanced Systemic Mastocytosis
Completed · Phase 2 · NCT03580655
Conditions: Advanced Systemic Mastocytosis, Aggressive Systemic Mastocytosis, Systemic Mastocytosis With an Associated Hematologic Neoplasm, Mast Cell Leukemia
At a glance
- Research payments reported, 2025
- $523,025
- U.S. investigators named
- 11
- Sites paid
- 12
- Planned or actual enrollment
- 107
About the trial
- Sponsor
- Blueprint Medicines Corporation
- Tested
- Avapritinib (drug)
- Dates
- Started 2018-11-21; completion 2024-12-18
- Results posted
- Yes
This is an open-label, single arm, Phase 2 study evaluating the efficacy and safety of avapritinib (BLU-285) in patients with advanced systemic mastocytosis (AdvSM), including patients with aggressive SM (ASM), SM with associated hematologic neoplasm (SM-AHN), and mast cell leukemia (MCL)
From ClinicalTrials.gov, last updated 2026-10-20. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Blueprint Medicines Corporation reported $523,025 in research payments for this study in 2025 (46 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
- Jason Gotlib, M.D., M.S. Hematology · Palo Alto, CA
- Celalettin Ustun, MD Hematology · Chicago, IL
- Abdulraheem Yacoub, MD Student in an Organized Health Care Education/Training Program · Westwood, KS
- Daniel J. Deangelo, M.D.,PH.D. Hematology & Oncology · Boston, MA
- Kristen M. Pettit, M.D. Hematology · Ann Arbor, MI
- Stephen T. Oh, MD Hematology · Saint Louis, MO
- Elizabeth A. Griffiths, MD Hematology & Oncology · Buffalo, NY
- Joseph G. Jurcic, MD Hematology · New York, NY
- Elizabeth O. Hexner, MD Hematology · Philadelphia, PA
- Matthew J. Butler, M.D. Hematology & Oncology · San Antonio, TX
- Tsewang Tashi, M.D. Hematology & Oncology · Salt Lake City, UT
Clinicians the company named as principal investigators in its Open Payments reports.
Sites paid to run it
- Dana Farber Mass General Brigham Ca Care Inc $110,361
- The Trustees of Columbia University in The City of New York $77,777
- Board of Trustees of the Leland Stanford Junior University $63,617
- University of Utah Hospital and Clinics, UT $59,875
- Health Research Inc $57,191
- Dana Farber/partners Cancer Care Inc $50,488
- University of Michigan Health System, MI $38,637
- Rush University Medical Center, IL $31,368
- Hospital of Univ of Pennsylvania, PA $10,503
- Washington University $10,398
- University of Kansas Medical Center Research Institute Inc $9,532
- University of Texas Health Science Ctr $3,278
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.