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(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

NCT03731260 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 251

Last updated 2025-09-23

No results posted yet for this study

Summary

This is a Phase 2, randomized, double-blind, placebo-controlled study comparing the efficacy and safety of avapritinib + best supportive care (BSC) with placebo + BSC in patients with indolent systemic mastocytosis (ISM) whose symptoms are not adequately controlled by BSC. The study will be conducted in 3 parts. All patients will receive treatment with avapritinib during Part 3 including those rolling over from the placebo group.

Conditions

  • Indolent Systemic Mastocytosis

Interventions

DRUG

Avapritinib

Avapritinib tablet

DRUG

Placebo

Placebo 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
2019-04-16
Primary Completion
2027-06-23
Completion
2027-06-23
FDA Drug
Yes

Countries

  • United States
  • Belgium
  • Canada
  • Denmark
  • France
  • Germany
  • Italy
  • Netherlands
  • Norway
  • Spain
  • Sweden
  • Switzerland
  • 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 NCT03731260 on ClinicalTrials.gov