OncRef

Clinical Trial: NCT03731260

(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

Status:

ACTIVE NOT RECRUITING

Phase:

Phase 2

Lead Sponsor:Blueprint Medicines Corporation
Information Provided By:Blueprint Medicines Corporation
Last Update Posted:2026-10-02

Study Overview

Brief 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.

Official Title

A 3-Part, Randomized, Double-Blind, Placebo-Controlled Phase 2 Study to Evaluate Safety and Efficacy of Avapritinib (BLU-285), a Selective KIT Mutation-Targeted Tyrosine Kinase Inhibitor, in Indolent and Smoldering Systemic Mastocytosis With Symptoms Inadequately Controlled With Standard Therapy

Conditions

Indolent Systemic Mastocytosis

Intervention / Treatment

  • Drug: Avapritinib
  • Drug: Placebo

Other Study ID Numbers

  • BLU-285-2203
  • 2018-000588-99
  • 2018

Start Date (Actual)

2019-04-16

Primary Completion Date (Estimated)

2027-06-23

Study Completion Date (Estimated)

2027-06-23

Enrollment (Actual)

251

Study Type

Interventional

Phase

Phase 2