# (PIONEER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, Versus Placebo 

Source: https://onco.cc/trials/nct03731260/  
OnCo record `nct03731260` (Trial). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

A phase 2 trial of Avapritinib in systemic mastocytosis, run by Blueprint Medicines Corporation, active and no longer recruiting.

## Summary

(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 is a phase 2 interventional study registered as NCT03731260 by Blueprint Medicines Corporation, with 251 participants enrolled, started 2019-04-16 and due to reach its primary completion in 2027-06-23. Interventions recorded: Avapritinib, Placebo. Conditions listed: Indolent Systemic Mastocytosis. No results are recorded here; the registry entry is the source.

## Fields

- Kind: Trial
- Status: active
- Last checked: 2026-09-16
- Tags: ctgov-ingest
- Registry id: NCT03731260
- Phase: 2
- Setting: 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
- Sponsor: Blueprint Medicines Corporation
- Enrolled: 251

## Sources

- ClinicalTrials.gov NCT03731260: https://clinicaltrials.gov/study/NCT03731260

## Connected records

- cancers: [Systemic mastocytosis](https://onco.cc/cancers/systemic-mastocytosis/)
- drugs: [Avapritinib](https://onco.cc/drugs/avapritinib/)
- companies: [Sanofi](https://onco.cc/companies/sanofi/)

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JSON: https://onco.cc/api/v1/entities/nct03731260.json