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

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

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 251 (actual)
• Sponsor: Blueprint Medicines Corporation · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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

Type	Name	Description
DRUG	Avapritinib	Avapritinib tablet
DRUG	Placebo	Placebo tablet

Timeline

• Start date: 2019-04-16
• Primary completion: 2027-06-23
• Completion: 2027-06-23
• First posted: 2018-11-06
• Last updated: 2025-09-23

Locations

49 sites across 13 countries: United States, Belgium, Canada, Denmark, France, Germany, Italy, Netherlands, Norway, Spain, Sweden, Switzerland, United Kingdom

Regulatory

• FDA-regulated drug study