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Recruiting Phase 2Phase 3 Interventional

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

ClinicalTrials.gov ID: NCT04910685

Public ClinicalTrials.gov record NCT04910685. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

ClinicalTrials.gov public records Last synced Sep 5, 2026, 4:15 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

A Randomized, Double-Blind, Placebo-Controlled Phase 2/3 Study of BLU-263 in Indolent Systemic Mastocytosis

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

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.

Study identification

NCT ID
NCT04910685
Recruitment status
Recruiting
Study type
Interventional
Phase
Phase 2, Phase 3
Lead sponsor
Blueprint Medicines Corporation
Industry
Enrollment
534 participants

Conditions and interventions

Eligibility (public fields only)

Age range
18 Years and older
Sex
All
Healthy volunteers
Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date
Nov 29, 2021
Primary completion
Sep 29, 2032
Completion
Sep 29, 2032
Last update posted
Aug 3, 2026

2021 – 2032

United States locations

U.S. sites
14
U.S. states
12
U.S. cities
14
Facility City State ZIP Site status
University of Alabama at Birmingham Birmingham Alabama 35294 Recruiting
David Geffen School of Medicine at UCLA Los Angeles California 90095 Recruiting
Stanford Cancer Institute Palo Alto California 94305 Recruiting
UCHealth Blood Disorders and Cell Therapies Center - Anschutz Medical Campus Aurora Colorado 80045 Recruiting
Winship Cancer Institute, Emory University Atlanta Georgia 30322 Recruiting
Brigham and Women's Hospital Boston Massachusetts 02115 Recruiting
Michigan Medicine University of Michigan Ann Arbor Michigan 48109 Recruiting
Mayo Clinic Rochester Minnesota 55902 Recruiting
Roswell Park Cancer Institute Buffalo New York 14263 Recruiting
Columbia University Medical Center New York New York 10032 Recruiting
Duke Asthma, Allergy and Airway Center Durham North Carolina 27705 Recruiting
University of Cincinnati Medical Center Cincinnati Ohio 45219 Recruiting
The University of Texas, MD Anderson Cancer Center Houston Texas 77030 Recruiting
Huntsman Cancer Institute, University of Utah Salt Lake City Utah 84112 Recruiting

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

Non-U.S. locations

This page focuses on the U.S. directory. The official record also lists 61 non-U.S. sites.

About this trial record page

What this page shows
Public field values for ClinicalTrials.gov record NCT04910685, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do
No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from
Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh
Last update posted Aug 3, 2026 · Synced Sep 5, 2026

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Open the official record

The complete protocol, eligibility criteria, and contact information for NCT04910685 live on ClinicalTrials.gov.

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