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(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 · View on ClinicalTrials.gov ↗
Study 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 Studied
Interventions
- DRUG Placebo
- DRUG Avapritinib
Study Locations (20)
Florida
- Mayo Clinic Florida — Jacksonville
- H. Lee Moffitt Cancer Center — Tampa
Massachusetts
- Brigham & Women's Hospital — Boston
- Dana Farber Cancer Institute — Boston
Alabama
- University of Alabama at Birmingham — Birmingham
Arizona
- Mayo Clinic Hospital — Phoenix
California
- Stanford Cancer Institute — Stanford
Georgia
- Winship Cancer Institute, Emory University — Atlanta
Illinois
- Rush University Medical Center — Chicago
Kansas
- University of Kansas Hospital — Kansas City
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 251 participants |
| Start Date | 2019-04-16 |
| Est. Completion | 2027-06-23 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03731260
The ClinicalTrials.gov registry entry for NCT03731260 describes a study currently listed as active not recruiting. It is categorized as Phase 2, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 251 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Blueprint Medicines Corporation, which has 9 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Indolent Systemic Mastocytosis appearing as the primary indexed condition, and to 2 interventions — of which Placebo is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT03731260 reports 20 study locations spanning 18 distinct geographic areas — top geographies include Florida, Massachusetts, Alabama. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT03731260 about?
NCT03731260 is a clinical study titled "(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". 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 conducte...
What is the current status of trial NCT03731260?
This trial is currently active not recruiting. It is a Phase 2 study. The enrollment target is 251 participants. The study started on 2019-04-16. Estimated completion is 2027-06-23.
What conditions does trial NCT03731260 study?
This clinical trial studies the following conditions: Indolent Systemic Mastocytosis. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT03731260?
The interventions under investigation include: Placebo (DRUG), Avapritinib (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT03731260?
This trial is sponsored by Blueprint Medicines Corporation, which has 9 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT03731260 being conducted?
This trial has 20 study locations across Alabama, Arizona, California, Florida, Georgia. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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