# (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** · PHASE2 · ACTIVE_NOT_RECRUITING · sponsor: **Blueprint Medicines Corporation** · enrollment: 251 (actual)

## Conditions studied

- Indolent Systemic Mastocytosis

## Interventions

- **DRUG:** Avapritinib
- **DRUG:** Placebo

## Key facts

- **NCT ID:** NCT03731260
- **Lead sponsor:** Blueprint Medicines Corporation
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** ACTIVE_NOT_RECRUITING
- **Start date:** 2019-04-16
- **Primary completion:** 2027-06-23
- **Final completion:** 2027-06-23
- **Target enrollment:** 251 (ACTUAL)
- **Last updated:** 2025-09-23


## Primary source

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

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration 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". Retrieved via AI Analytics 2026-07-10 from https://api.ai-analytics.org/clinical/NCT03731260. Licensed CC0.

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*[Clinical trials dataset](/datasets/clinical-trials) · CC0 1.0*