(HARBOR) Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic Mastocytosis
Recruiting · Phase 2, Phase 3 · NCT04910685
Conditions: Indolent Systemic Mastocytosis, Smoldering Systemic Mastocytosis
At a glance
- Research payments reported, 2025
- $399,273
- U.S. investigators named
- 9
- Sites paid
- 10
- Planned or actual enrollment
- 534
About the trial
- Sponsor
- Blueprint Medicines Corporation
- Tested
- Elenestinib (drug), Placebo (drug)
- Dates
- Started 2021-11-30; completion 2032-09-30
- Results posted
- Not yet
This is a randomized, double-blind, placebo-controlled, Phase 2/3 study comparing the efficacy and safety of elenestinib (BLU-263) + symptom directed therapy (SDT) with placebo + SDT in participants with indolent systemic mastocytosis (ISM) whose symptoms are not adequately controlled by SDT. Parts 1 and 2 will enroll participants with ISM. Participants enrolled in Part 2 will roll over onto Part 3 to receive treatment with elenestinib in an open-label fashion following completion of the earlier Part. Part K will enroll participants with ISM who have previously received an approved selective KIT inhibitor. The study also includes pharmacokinetic (PK) groups that will enroll participants w…
From ClinicalTrials.gov, last updated 2026-10-07. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Sanofi US Services Inc. reported $399,273 in research payments for this study in 2025 (73 payments).
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
- Brian D. Modena, M.D., MSC Allergy & Immunology · La Jolla, CA
- William E. Shomali, M.D Hematology · Stanford, CA
- Maria Castells, MD Allergy & Immunology · Gardner, MA
- Cem Akin, MD Allergy & Immunology · Northville, MI
- Elizabeth A. Griffiths, MD Hematology & Oncology · Buffalo, NY
- Joseph G. Jurcic, MD Hematology · New York, NY
- Jonathan A. Bernstein, MD Allergy & Immunology · Cincinnati, OH
- 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
- University of Utah Hospital and Clinics, UT $132,049
- Brian D Modena Orso Health Inc $58,504
- University of Michigan Health System, MI $48,495
- Brigham and Women's Hospital, MA $38,354
- The Trustees of Columbia University $32,047
- Stanford University Care Fund Ghghm $31,214
- Health Research,inc $20,176
- University of Alabama Hospital, AL $14,741
- University of Cincinnati Physicians $13,798
- The Trustees of Columbia University in The City of Newyork $9,895
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.