Trial · 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
NCT03731260, Phase 2, Active not recruiting, Blueprint Medicines Corporation
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. (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. Trials, record NCT03731260. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03731260
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- (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
- NCT number
- NCT03731260
- Status
- Active not recruiting
- Phases
- Phase 2
- Enrollment
- 251 participants, actual
- Lead sponsor
- Blueprint Medicines Corporation
- Conditions
- Indolent Systemic Mastocytosis
- Interventions
- Avapritinib, Placebo
- First posted
- 2018-11-06
- Last updated
- 2026-10-02
- Other registries' IDs
- eudract:2018-000588-99
Source ClinicalTrials.gov
Registered outcomes19
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Part 1: Recommended Phase 2 dose (RP2D) in patients with ISM | 9 months | |
| Primary | Part 2: Mean change in ISM Symptom Assessment Form (ISM-SAF) total symptom score (TSS) as compared to placebo | 6 months | 0 - 110 points (higher value represents worse symptom outcomes) |
| Primary | Part 3: Number of Participants with Adverse Events | Up to 5 years | |
| Secondary | Part 2: Proportion of patients with a ≥50% reduction in serum tryptase | 6 months | |
| Secondary | Part 2: Proportion of patients with a ≥50% reduction in peripheral blood V-kit Hardy-Zuckerman 4 feline sarcoma viral oncogene homolog aspartate 816 valine (KIT D816V) allele fraction or undetectable for patients with detectable mutation at Baseline | 6 months | |
| Secondary | Part 2: Proportion of patients with ≥50% reduction in ISM-SAF TSS | 6 months | |
| Secondary | Part 2: Proportion of patients with ≥30% reduction in ISM-SAF TSS | 6 months | |
| Secondary | Part 2: Proportion of patients with a ≥50% reduction in bone marrow mast cells or no aggregates for patients with aggregates at Baseline | 6 months | |
| Secondary | Parts 1, 2, and 3: Change in serum tryptase | Up to 5 years | |
| Secondary | Parts 1, 2, and 3: Change in KIT D816V allele burden in blood | Up to 5 years |
1 to 10 of 19Source ClinicalTrials.gov
See all 19 registered outcomesOutcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers4
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT03731260
GET https://conductscience.org/api/trials/v1/NCT03731260/registered