(HARBOR) Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic Mastocytosis

Recruiting now · Phase 2/Phase 3 · Has a placebo group

Conditions studied: Indolent Systemic Mastocytosis, Smoldering Systemic Mastocytosis

In brief

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.

Key facts

Study ID

NCT04910685

Run by

Blueprint Medicines Corporation

People needed

534

Starts

2021-11-30

Expected to finish

2032-09-30

Last updated by the study team

2026-08-04

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

Where it is running

• David Geffen School of Medicine at UCLA — Los Angeles, California, United States (enrolling)
• Stanford Cancer Institute — Palo Alto, California, United States (enrolling)
• UCHealth Blood Disorders and Cell Therapies Center - Anschutz Medical Campus — Aurora, Colorado, United States (enrolling)
• Winship Cancer Institute, Emory University — Atlanta, Georgia, United States (enrolling)
• Brigham and Women's Hospital — Boston, Massachusetts, United States (enrolling)
• Michigan Medicine University of Michigan — Ann Arbor, Michigan, United States (enrolling)
• Mayo Clinic — Rochester, Minnesota, United States (enrolling)
• Roswell Park Cancer Institute — Buffalo, New York, United States (enrolling)
• Columbia University Medical Center — New York, New York, United States (enrolling)
• Duke Asthma, Allergy and Airway Center — Durham, North Carolina, United States (enrolling)
• University of Cincinnati Medical Center — Cincinnati, Ohio, United States (enrolling)
• The University of Texas, MD Anderson Cancer Center — Houston, Texas, United States (enrolling)
• Huntsman Cancer Institute, University of Utah — Salt Lake City, Utah, United States (enrolling)
• CHP Centro Hematologico Pavlovsky — Buenos Aires, Argentina (enrolling)
• Consultorios Medicos Dr. Doreski - Fundacion Respirar — Buenos Aires, Argentina (enrolling)
• Princess Alexandra Hospital — Woolloongabba, Queensland, Australia (enrolling)
• The Alfred Hospital — Melbourne, Victoria, Australia (enrolling)
• Kepler Universitatsklinikum, Med Campus III. Clinic of Internal Medicine 3 - Hematology and Oncology — Linz, Austria (enrolling)
• Unitversitair Ziekenhuis Antwerpen — Edegem, Antwerpen, Belgium (enrolling)
• Universitair Ziekenhuis Gent — Ghent, Belgium (enrolling)
• CHU Tivoli — La Louvière, Belgium (enrolling)
• Hospital das Clínicas da Faculdade de Medicina da USP - HCFMUSP — São Paulo, Brazil (enrolling)
• Fundación Valle del Lili — Cali, Colombia (enrolling)
• Fakultní nemocnice Brno, Interní hematologická a onkologická klinika — Brno, Czechia (enrolling)
• University of Alabama at Birmingham — Birmingham, Alabama, United States (enrolling)

Full record on ClinicalTrials.gov

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