(HARBOR) Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic Mastocytosis
Condition(s) studied
Investigational drug(s) / intervention(s)
Elenestinib: Elenestinib oral tablet
Placebo: Placebo oral tablet
Study summary
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.
Eligibility
Primary outcome measure(s)
- Part 1: Number of participants with Treatment-emergent Adverse Events (TEAEs) — Up to 12 weeks
- Part 1: Mean change from baseline in ISM-Symptom in Assessment Form (ISM-SAF) Total Symptom Score (TSS) — Baseline, Week 13
- Part 2: Mean change from baseline in ISM-SAF TSS — Baseline, Week 49
- Part 3: Number of participants with Adverse Events (AEs) — Up to 5 years
- Part 3: Change from baseline in ISM-SAF TSS — Baseline up to 5 years
Trial sites (88)
| Facility | City | Region | Status |
|---|---|---|---|
| University of Alabama at Birmingham | Birmingham | Alabama | Recruiting |
| Orso Health, Modena Allergy and Asthma Inc | La Jolla | California | Recruiting |
| David Geffen School of Medicine at UCLA | Los Angeles | California | Recruiting |
| Stanford Cancer Institute | Palo Alto | California | Recruiting |
| UCHealth Blood Disorders and Cell Therapies Center - Anschutz Medical Campus | Aurora | Colorado | Recruiting |
| Winship Cancer Institute, Emory University | Atlanta | Georgia | Recruiting |
| Brigham and Women's Hospital | Boston | Massachusetts | Recruiting |
| Michigan Medicine University of Michigan | Ann Arbor | Michigan | Recruiting |
| Mayo Clinic | Rochester | Minnesota | Recruiting |
| Roswell Park Cancer Institute | Buffalo | New York | Recruiting |
| Columbia University Medical Center | New York | New York | Recruiting |
| Duke Asthma, Allergy and Airway Center | Durham | North Carolina | Recruiting |
| University of Cincinnati Medical Center | Cincinnati | Ohio | Recruiting |
| The University of Texas, MD Anderson Cancer Center | Houston | Texas | Recruiting |
| Huntsman Cancer Institute, University of Utah | Salt Lake City | Utah | Recruiting |
| CHP Centro Hematologico Pavlovsky | Buenos Aires | Argentina | Recruiting |
| Consultorios Medicos Dr. Doreski - Fundacion Respirar | Buenos Aires | Argentina | Recruiting |
| Princess Alexandra Hospital | Woolloongabba | Queensland | Recruiting |
| The Alfred Hospital | Melbourne | Victoria | Recruiting |
| The Canberra Hospital | Garran | Australia | Recruiting |
| Kepler Universitatsklinikum, Med Campus III. Clinic of Internal Medicine 3 - Hematology and Oncology | Linz | Austria | Recruiting |
| Unitversitair Ziekenhuis Antwerpen | Edegem | Antwerpen | Recruiting |
| Universitair Ziekenhuis Gent | Ghent | Belgium | Recruiting |
| CHU Tivoli | La Louvière | Belgium | Recruiting |
| UZ Leuven | Leuven | Belgium | Recruiting |
| Hospital das Clínicas da Faculdade de Medicina da USP - HCFMUSP | São Paulo | Brazil | Recruiting |
| Clinica Alemana de Santiago | Santiago | Chile | Recruiting |
| Fundación Valle del Lili | Cali | Colombia | Recruiting |
| Fakultní nemocnice Brno, Interní hematologická a onkologická klinika | Brno | Czechia | Recruiting |
| Fakultní nemocnice Královské Vinohrady, Hematologická klinika 3. LF UK v Praze a FNKV | Prague | Czechia | Recruiting |
| Odense University Hospital and University of Southern Denmark | Odense | Denmark | Recruiting |
| CHU Amiens-Picardie | Amiens | France | Recruiting |
| CHU de Caen | Caen | France | Recruiting |
| CHU Grenoble | Grenoble | France | Recruiting |
| CHU de Limoges | Limoges | France | Recruiting |
| CHU de Nantes | Nantes | France | Recruiting |
| Hôpital de la Pitié Salpétrière | Paris | France | Recruiting |
| Hôpital Necker - Départementd 'HématologieA dultes | Paris | France | Recruiting |
| CHU de Poitiers | Poitiers | France | Recruiting |
| CHU Toulouse - Hopital Larrey | Toulouse | France | Recruiting |
+ 48 more sites — see the full list on the official registry below.
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Other trials for the same condition
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT04910685 on ClinicalTrials.gov ↗ ← All trials in Italy