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

NCT04910685 · Status: RECRUITING · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 534

Last updated 2026-05-22

No results posted yet for this 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.

Conditions

  • Indolent Systemic Mastocytosis
  • Smoldering Systemic Mastocytosis

Interventions

DRUG

Elenestinib

Elenestinib oral tablet

DRUG

Placebo

Placebo oral tablet

Sponsors & Collaborators

  • Blueprint Medicines Corporation

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-11-30
Primary Completion
2032-09-30
Completion
2032-09-30
FDA Drug
Yes

Countries

  • United States
  • Argentina
  • Australia
  • Austria
  • Belgium
  • Czechia
  • France
  • Germany
  • Greece
  • Italy
  • Netherlands
  • Norway
  • Poland
  • Portugal
  • Spain
  • Sweden
  • Switzerland
  • Turkey (Türkiye)
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04910685 on ClinicalTrials.gov