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Condition (MeSH)

Mastocytosis

ClinicalTrials.gov lists 136 trials for Mastocytosis or a narrower condition beneath it. Blueprint Medicines Corporation has the most among industry and government lead sponsors, with 11.

MeSH D008415 Neoplasms Neoplasm C04 C20 Listed by Orphanet as a rare disease
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Phase 1 Phase 1 9 Phase 1/2 Phase 1/2 6 Phase 2 Phase 2 36 Phase 2/3 Phase 2/3 5 Phase 3 Phase 3 9 Phase 4 Phase 4 2 Not applicable Not applicable 16 Observational Observational 49 Phase 1 Phase 1 9 Phase 1/2 Phase 1/2 6 Phase 2 Phase 2 36 Phase 2/3 Phase 2/3 5 Phase 3 Phase 3 9 Phase 4 Phase 4 2 Not applicable Not applicable 16 Observational Observational 49

Trials per phase (log scale)

2 more sections with rows

49 of the 136 trials are tagged with Mastocytosis itself; the rest are tagged with a narrower condition beneath it.

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The PDF carries up to 50 rows of each section, where the page shows the first few, with the sources listed. Enter a work email and confirm it from the message we send, and the report follows by email.

When did Mastocytosis trials start? (33)

Shown in this order: most recent first, 1990 onwards. 2026 is partial: trials registered with a 2026 start date so far, not a full year. 1 trial is registered to start in 2027 or later, and not listed here.

Trials over time, by phase

  • Phase 1
  • Phase 1/2
  • Phase 2
  • Phase 2/3
  • Phase 3
  • Phase 4
  • Not applicable
  • Observational
  • Other (Unknown)

Top of scale: 10

1991: 1 trials (Observational 1) 1993: 1 trials (Observational 1) 1996: 1 trials (Observational 1) 1997: 1 trials (Observational 1) 1998: 1 trials (Observational 1) 1999: 1 trials (Not applicable 1) 2000: 3 trials (Not applicable 1, Phase 2 1, Phase 1/2 1) 2001: 3 trials (Not applicable 1, Phase 2 1, Phase 1/2 1) 2002: 3 trials (Observational 3) 2003: 2 trials (Observational 1, Phase 2 1) 2004: 6 trials (Observational 3, Phase 2 3) 2005: 7 trials (Observational 1, Phase 3 1, Phase 2 4, Phase 1/2 1) 2006: 4 trials (Observational 1, Not applicable 1, Phase 3 1, Phase 2 1) 2007: 8 trials (Other 1, Observational 1, Not applicable 1, Phase 2 4, Phase 1 1) 2008: 8 trials (Observational 3, Not applicable 1, Phase 3 3, Phase 2 1) 2009: 10 trials (Other 1, Observational 3, Not applicable 1, Phase 3 1, Phase 2 2, Phase 1/2 1, Phase 1 1) 2010: 5 trials (Observational 2, Not applicable 1, Phase 3 1, Phase 2/3 1) 2011: 6 trials (Not applicable 1, Phase 4 1, Phase 2/3 1, Phase 2 2, Phase 1 1) 2012: 2 trials (Phase 2/3 1, Phase 2 1) 2013: 3 trials (Not applicable 1, Phase 2 2) 2014: 1 trials (Observational 1) 2015: 8 trials (Observational 4, Not applicable 1, Phase 2 2, Phase 1 1) 2016: 3 trials (Observational 1, Phase 1 2) 2017: 1 trials (Not applicable 1) 2018: 4 trials (Observational 2, Phase 3 1, Phase 2 1) 2019: 4 trials (Observational 1, Phase 2 3) 2020: 8 trials (Observational 2, Not applicable 1, Phase 3 1, Phase 2 2, Phase 1/2 1, Phase 1 1) 2021: 6 trials (Not applicable 1, Phase 2/3 2, Phase 2 3) 2022: 3 trials (Observational 1, Phase 2 1, Phase 1 1) 2023: 3 trials (Observational 2, Phase 1 1) 2024: 8 trials (Observational 6, Not applicable 1, Phase 4 1) 2025: 4 trials (Observational 4)

Trials registered with a start date in each year, 1990 onward, stacked by phase (complete years only; the current partial year is in the table's notes). The table below lists the same counts, most recent years first.

Trials started per year
Year Trials started
2026 (partial year) 2
2025 4
2024 8
2023 3
2022 3
2021 6
2020 8
2019 4
2018 4
2017 1

No forecast is made for this condition: it averages 4.6 trial starts a year over the five years to September, under the 5 a year the method needs.

Prediction, not a registry value: median of 36 months from start to primary completion across 6 trials still running at the 2026-10-09 snapshot; registered median of 37 months across 86 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.

What phase are Mastocytosis trials in? (10)

Shown in this order: phase order.

Show the trials by phase as a table
Trials by phase
Phase Trials
Phase 1 9
Phase 1/2 6
Phase 2 36
Phase 2/3 5
Phase 3 9
Phase 4 2
Not applicable 16
Observational 49
Expanded access 2
Unknown 2

How many Mastocytosis trials are recruiting? (9)

Shown in this order: the order a trial moves through, from not yet recruiting to unknown.

Trials by recruitment status
Status Trials
Not yet recruiting 2
Recruiting 18
Active, not recruiting 7
Available 1
Completed 73

Status as registered on ClinicalTrials.gov. Each trial is counted once.

Which lead sponsors run the most Mastocytosis trials? (6)

Shown in this order: most trials first. Lead sponsors the registry classes as industry, NIH, US federal, other government or network. A hospital or university the registry files as government can appear here. Sponsors it classes as academic or other are shown on the full page, because that class cannot separate an institution from a named investigator.

Lead sponsors
Sponsor Trials
Blueprint Medicines Corporation 11
National Institute of Allergy and Infectious Diseases (NIAID) 9
Novartis Pharmaceuticals 5
AB Science 4
Cogent Biosciences, Inc. 3

Which drugs are being tested for Mastocytosis? (6)

Shown in this order: most trials first. One row per drug, matched to a single molecule under all its names, ranked by number of trials.

Drugs tested
Drug Trials
Avapritinib 5
Imatinib 4
Midostaurin 4
Bezuclastinib 3
Cytoxan 3

Interventions as named in the registry (9)

Shown in this order: most trials first.

Interventions as named in the registry
Drug Trials
SQ Fludarabine 8
Total Body Irradiation 7
Mofetil Mycophenolate 6
Cyclosporine 0.05% 5
Peripheral blood stem cell transplantation 5

These are the names sponsors entered on ClinicalTrials.gov that we have not matched to a single molecule. Some are drugs under a brand or code name; others are procedures, doses or arm labels.

Which serious adverse events do Mastocytosis trials report? (10)

Shown in this order: most subjects affected first. Subjects affected summed across trials, a cross-trial signal, not a rate.

Serious adverse events in this condition's trials
Adverse event Subjects affected Trials
Pneumonia 31 6
Thrombocytopenia 28 3
Pyrexia 26 3
Anaemia 21 3
Febrile neutropenia 21 7

Which publications cover Mastocytosis? (43)

Shown in this order: major-topic papers first, then newest, then most linked to trials.

Publications
Publication Journal Year Major topic
Mast Cell Diseases in Practice and Research: Issues and Perspectives Raised by Patients and Their Recommendations to the Scientific Community and Beyond. The journal of allergy and clinical immunology. In practice 2022 Yes
Scoring the Risk of Having Systemic Mastocytosis in Adult Patients with Mastocytosis in the Skin. The journal of allergy and clinical immunology. In practice 2021 Yes
Health-Related Quality of Life and Influencing Factors in Adults with Nonadvanced Mastocytosis-A Cross-Sectional Study and Qualitative Approach. The journal of allergy and clinical immunology. In practice 2021 Yes
Clinical relevance of inherited genetic differences in human tryptases: Hereditary alpha-tryptasemia and beyond. Annals of allergy, asthma & immunology : official publication of the American College of Allergy, Asthma, & Immunology 2021 Yes
Mastocytosis and Mast Cell Activation Disorders: Clearing the Air. International journal of molecular sciences 2021 Yes

Prevalence (9)

Shown in this order: jurisdiction, then disease name. Orphanet observation for the mapped ORPHA concept; the mapping relation column says whether that concept is exactly this condition or broader or narrower.

Prevalence
Orphanet disease Type Class Geography Jurisdiction Mapping relation
Mastocytosis Point prevalence 1-5 / 10 000 Worldwide Not stated Exact
Mastocytosis Point prevalence 1-9 / 100 000 Europe Not stated Exact
Mastocytosis Point prevalence 1-9 / 100 000 Europe Not stated Exact
Mastocytosis Point prevalence 1-9 / 100 000 Europe Not stated Unspecified
Mastocytosis Point prevalence 1-9 / 100 000 Denmark Not stated Exact

Rare-disease identity (2)

Shown in this order: exact, broader, narrower, other.

Rare-disease identity
Orphanet disease ORPHA code Relation Source
Mastocytosis 98292 Exact Orphadata Science knowledge files, Product 1
Mastocytosis 98292 Exact Orphanet Rare Disease Ontology (ORDO)

Sub-conditions in MeSH (3)

MeSH descendants one level down with at least one trial, most trials first.

Not on this page

Not measured: delivery record.

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