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NCT03580655 · ClinicalTrials.gov registry record · Phase 2
(PATHFINDER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, in Patients With Advanced Systemic Mastocytosis
A Phase 2 study of Systemic Mastocytosis With an Associated Hematologic Neoplasm and Aggressive Systemic Mastocytosis, sponsored by Blueprint Medicines Corporation.
- Completed
- Registry status
- Phase 2
- Development phase
- 107
- Enrollment target
- 20
- Study locations
NCT03580655: Completed Phase 2 study of Systemic Mastocytosis With an Associated Hematologic Neoplasm and Aggressive Systemic Mastocytosis, sponsored by Blueprint Medicines Corporation.
NCT03580655 is a Phase 2 study of Systemic Mastocytosis With an Associated Hematologic Neoplasm and Aggressive Systemic Mastocytosis that has completed, run by Blueprint Medicines Corporation. The registered enrollment target is 107 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (20% lower). The trial reports 20 study locations across 12 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03580655, a Phase 2 study of Systemic Mastocytosis With an Associated Hematologic Neoplasm and Aggressive Systemic Mastocytosis, has completed, sponsored by Blueprint Medicines Corporation.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 107 participants
- Enrollment target
- 20
- Study locations
Study Summary
This is an open-label, single arm, Phase 2 study evaluating the efficacy and safety of avapritinib (BLU-285) in patients with advanced systemic mastocytosis (AdvSM), including patients with aggressive SM (ASM), SM with associated hematologic neoplasm (SM-AHN), and mast cell leukemia (MCL)
Primary Outcome
ORR was defined as the percentage of participants with a confirmed best response of complete remission (CR), complete remission with partial recovery of peripheral blood counts (CRh), partial remission (PR), or clinical improvement (CI) by mIWG-MRT-ECNM criteria. The mIWG-MRT-ECNM criteria were based on findings from bone marrow biopsy and aspirate, and peripheral blood smear; bone marrow cytogenetics; other extracutaneous tissue biopsies (when available); liver and spleen imaging; and other cli
Conditions Studied
Interventions
- DRUG Avapritinib
Study Locations (20)
Other
- Medizinische Universität Wien, Universitätsklinik für Innere Medizin I, Klinische Abteilung für Hämatologie und Hämostaseologie - Vienna
- Odense University Hospital, Department of Haematology - Odense
- Hôpital Necker-Enfants Malades - Paris
- CHU Toulouse - Hôpital Larrey - Toulouse
- Uniklinik RWTH Aachen, Klinik für Hämatologie, Onkologie, Hämostaseologie und Stammzelltranslplantation - Aachen
- Universitätsklinikum Hamburg-Eppendorf, Zentrum für Onkologie - Hamburg
- Universitätsklinikum Leipzig, Medizinische Klinik und Poliklinik I - Hämatologie und Zelltherapie, lnternistische Onkologie, Hämostaseologie - Leipzig
New York
- Roswell Park Comprehensive Cancer Center - Buffalo
- Herbert Irving Comprehensive Cancer Center - New York
Texas
- University of Texas, MD Anderson Cancer Center - Houston
- Mays Cancer Center - San Antonio
California
- Stanford Cancer Institute - Stanford
Illinois
- Rush University Medical Center - Chicago
Kansas
- The University of Kansas Cancer Center - Westwood
Massachusetts
- Dana Farber Cancer Institute - Boston
Michigan
- University of Michigan - Ann Arbor
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 107 participants |
| Start Date | 2018-11-21 |
| Est. Completion | 2024-12-18 |
| Phase | Phase 2 |
What the finished NCT03580655 record still lists
NCT03580655 is an interventional study that assigns participants to a tested intervention. The registered 107 participants enrollment target is mid-sized for trials with a published cap, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (20% lower).
The record links to 4 conditions, with Systemic Mastocytosis With an Associated Hematologic Neoplasm appearing as the primary indexed condition, and to 1 intervention - of which Avapritinib is the first listed.
NCT03580655 names 20 study sites across 12 states, led by Other, New York, Texas.
Frequently Asked Questions
What is clinical trial NCT03580655 about?
NCT03580655 is a clinical study titled "(PATHFINDER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, in Patients With Advanced Systemic Mastocytosis". This is an open-label, single arm, Phase 2 study evaluating the efficacy and safety of avapritinib (BLU-285) in patients with advanced systemic mastocytosis (AdvSM), including patients with aggressive SM (ASM), SM with associated hematologic neoplasm (SM-AHN), and mast cell leukemia (MCL)
What is the current status of trial NCT03580655?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 107 participants. The study started on 2018-11-21. Estimated completion is 2024-12-18.
What conditions does trial NCT03580655 study?
This clinical trial studies the following conditions: Systemic Mastocytosis With an Associated Hematologic Neoplasm, Aggressive Systemic Mastocytosis, Mast Cell Leukemia, Advanced Systemic Mastocytosis.
What interventions are being tested in trial NCT03580655?
The interventions under investigation include: Avapritinib (DRUG).
Who is sponsoring clinical trial NCT03580655?
This trial is sponsored by Blueprint Medicines Corporation, which has 24 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03580655 being conducted?
This trial has 20 study locations across California, Illinois, Kansas, Massachusetts, Michigan. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT03580655's enrollment target sits among peer trials
107 646th of 2000 higher than 1,353 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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