TheraRadar
Landscape / Rare Disease
Data updated: Jun 28, 2026

Mastocytosis Clinical Trial Landscape

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, 4 active Phase 2 trials, and 2 active Phase 1 trials.

Top industry sponsors include Blueprint Medicines Corporation, Cogent Biosciences, Inc., MacroGenics.

Trial activity

8 active / 28 total since 2008
Active by phase 1 Ph3 / 5 4 Ph2 / 14 2 Ph1 / 7 1 Ph4 / 2

Sponsor activity

Who is running trials now — green active, blue completed, red failed/terminated.

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

How the field has grown

New-trial starts peaked in 2020 (4 registered); 2025 saw 0. The right-hand chart shows median Phase 3 enrollment by start year — the number in parentheses is that year's Phase 3 trial count (2 in total), so single-trial years (and years with no Phase 3 starts) are obvious. Both are by trial start date; the current year is partial.

New trials started by year

2016
2
2017
0
2018
2
2019
2
2020
4
2021
2
2022
2
2023
1
2024
2
2025
0
2026
0

TheraRadar.com

Median Phase 3 enrollment by start year

2016 (0)
0
2017 (0)
0
2018 (1)
24
2019 (0)
0
2020 (0)
0
2021 (1)
534
2022 (0)
0
2023 (0)
0
2024 (0)
0
2025 (0)
0
2026 (0)
0

TheraRadar.com

Full trial pipeline

Every active and completed trial across Phase 1–4, with enrollment analytics. Sortable, filterable, exportable with Pro.

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
NCT01481909 COMPLETED
Study on Mastocytosis for Rupatadine Treatment
Marcus Maurer n=30
NCT01333293 COMPLETED
The Effect of Xolair ® (Omalizumab) in Mastocytosis Patients
University of Zurich n=17

Full Mastocytosis Pipeline

Every trial across Phase 1–4, plus enrollment analytics. Sortable, filterable, exportable.

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Data: ClinicalTrials.gov · Trials registered 2008 onwards · Industry sponsors only