(HARBOR) Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic Mastocytosis (NCT04910685)
A Randomized, Double-Blind, Placebo-Controlled Phase 2/3 Study of BLU-263 in Indolent Systemic Mastocytosis
🩺 Plain-English Summary (8th-Grade Level)
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 with ISM.
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 (75 Sites)
Showing the first 25 of 75 sites.
- University of Alabama at Birmingham — Birmingham, Alabama
- David Geffen School of Medicine at UCLA — Los Angeles, California
- Stanford Cancer Institute — Palo Alto, California
- UCHealth Blood Disorders and Cell Therapies Center - Anschutz Medical Campus — Aurora, Colorado
- Winship Cancer Institute, Emory University — Atlanta, Georgia
- Brigham and Women's Hospital — Boston, Massachusetts
- Michigan Medicine University of Michigan — Ann Arbor, Michigan
- Mayo Clinic — Rochester, Minnesota
- Roswell Park Cancer Institute — Buffalo, New York
- Columbia University Medical Center — New York, New York
- Duke Asthma, Allergy and Airway Center — Durham, North Carolina
- University of Cincinnati Medical Center — Cincinnati, Ohio
- The University of Texas, MD Anderson Cancer Center — Houston, Texas
- Huntsman Cancer Institute, University of Utah — Salt Lake City, Utah
- CHP Centro Hematologico Pavlovsky — Buenos Aires,
- Consultorios Medicos Dr. Doreski - Fundacion Respirar — Buenos Aires,
- Princess Alexandra Hospital — Woolloongabba, Queensland
- The Alfred Hospital — Melbourne, Victoria
- Kepler Universitatsklinikum, Med Campus III. Clinic of Internal Medicine 3 - Hematology and Oncology — Linz,
- Unitversitair Ziekenhuis Antwerpen — Edegem, Antwerpen
- Universitair Ziekenhuis Gent — Ghent,
- CHU Tivoli — La Louvière,
- Hospital das Clínicas da Faculdade de Medicina da USP - HCFMUSP — São Paulo,
- Fundación Valle del Lili — Cali,
- Fakultní nemocnice Brno, Interní hematologická a onkologická klinika — Brno,
📞 Contact the Study Coordinator
Phone: (617) 714-6707
Email: medinfo@blueprintmedicines.com
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
💰 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
- (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 — Birmingham, Alabama
- An Observational Study in Participants With Indolent Systemic Mastocytosis (ISM) — Wheaton, Maryland
Explore by condition: All Indolent Systemic Mastocytosis clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.