Clinical Trial

(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

Active, Not Recruiting Phase 2
View on ClinicalTrials.gov →
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.
Protocol Amendment History 18 amendments
This ClinicalTrials.gov record has been amended 18 times since 2018-11-02; most recent amendment 2025-09-22.
Status change: Recruiting → Active, Not Recruiting 2022-01-19
Status change: Not Yet Recruiting → Recruiting 2019-01-04
Trial Details
NCT Number NCT03731260
Lead Sponsor Blueprint Medicines Corporation
Conditions Indolent Systemic Mastocytosis
Enrollment 251 participants
Start Date 2019-04-16
Primary Completion 2027-06-23 (estimated)
Study Completion 2027-06-23 (estimated)
Updated on ClinicalTrials.gov 2025-09-23