Mastocytosis Clinical Trial Landscape
Data updated: May 20, 2026
28 clinical trials tracked since 2008
Mastocytosis is being studied across 28 clinical trials registered since 2008, with 8 programs currently active. The competitive pipeline includes 1 active Phase 3 trials, 5 active Phase 2 trials, and 2 active Phase 1 trials. Top industry sponsors include Blueprint Medicines Corporation, Cogent Biosciences, Inc., MacroGenics.
8 active
/
28 total (since 2008)
2
Phase 1 Active
8 total
5
Phase 2 Active
17 total
1
Phase 3 Active
5 total
1
Phase 4 Active
2 total
Top Industry Sponsors
Sorted by active Active Done Failed
Blueprint Medicines Corporation 3 2 1
Cogent Biosciences, Inc. 2 0 0
MacroGenics 1 0 0
Telios Pharma, Inc. 1 0 0
Featured Phase 3 Trials
NCT04910685 RECRUITING
(HARBOR) Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic Mastocytosis
Blueprint Medicines Corporation n=534
NCT03401060 COMPLETED
Interest of Denosumab Treatment in Osteoporosis Associated to Systemic Mastocytosis
Assistance Publique - Hôpitaux de Paris n=24
NCT00814073 COMPLETED
Masitinib in Severe Indolent or Smoldering Systemic Mastocytosis
AB Science n=135
NCT01333293 COMPLETED
The Effect of Xolair ® (Omalizumab) in Mastocytosis Patients
University of Zurich n=17
Mastocytosis — Trials Started by Year (last 10 years)
2016 2
2017 0
2018 2
2019 2
2020 4
2021 2
2022 2
2023 1
2024 2
2025 0
2026 0
TheraRadar.com
Full Trial Database
Complete sponsor table, every trial across all phases, plus competitive analytics. Sortable, filterable, exportable for Pro users.
Top Sponsors (all-time)
Blueprint Medicines Corporation 6
Cogent Biosciences, Inc. 2
MacroGenics 1
Telios Pharma, Inc. 1
LEO Pharma 1
Deciphera Pharmaceuticals, LLC 1
GT Biopharma, Inc. 1
AB Science 1
Allakos Inc. 1
Patara Pharma 1
Active Trials by Phase
Phase 1 2
Phase 2 5
Phase 3 1
Phase 4 1
Enrollment Size Distribution
1-50 16
51-200 7
201-500 3
501-1k 1
1k+ 0
All Industry Sponsors
Sorted by active Active Done Failed
Blueprint Medicines Corporation 3 2 1
Cogent Biosciences, Inc. 2 0 0
MacroGenics 1 0 0
Telios Pharma, Inc. 1 0 0
NCT04910685 RECRUITING
(HARBOR) Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic Mastocytosis
Blueprint Medicines Corporation n=534
NCT03401060 COMPLETED
Interest of Denosumab Treatment in Osteoporosis Associated to Systemic Mastocytosis
Assistance Publique - Hôpitaux de Paris n=24
NCT00814073 COMPLETED
Masitinib in Severe Indolent or Smoldering Systemic Mastocytosis
AB Science n=135
NCT01333293 COMPLETED
The Effect of Xolair ® (Omalizumab) in Mastocytosis Patients
University of Zurich n=17
Data: ClinicalTrials.gov · Trials registered 2008 onwards · Industry sponsors only