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Condition (MeSH)

Leukemia, Mast-Cell

ClinicalTrials.gov lists 60 trials for Leukemia, Mast-Cell or a narrower condition beneath it. Blueprint Medicines Corporation has the most among industry and government lead sponsors, with 5.

MeSH D007946 Neoplasms Neoplasm C04 C15 C20 Listed by Orphanet as a rare disease
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Phase 1 Phase 1 6 Phase 1/2 Phase 1/2 5 Phase 2 Phase 2 16 Phase 3 Phase 3 5 Not applicable Not applicable 9 Observational Observational 16 Expanded access Expanded access 2 Unknown Unknown 1 Phase 1 Phase 1 6 Phase 1/2 Phase 1/2 5 Phase 2 Phase 2 16 Phase 3 Phase 3 5 Not applicable Not applicable 9 Observational Observational 16 Expanded access Expanded access 2 Unknown Unknown 1

Trials per phase

60 of the 60 trials are tagged with Leukemia, Mast-Cell itself; the rest are tagged with a narrower condition beneath it.

Get the Leukemia, Mast-Cell report as a PDF

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 Leukemia, Mast-Cell trials start? (22)

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 3
  • Not applicable
  • Observational
  • Unknown

Top of scale: 8

1991: 1 trials (Observational 1) 1999: 1 trials (Not applicable 1) 2000: 2 trials (Not applicable 1, Phase 1/2 1) 2001: 2 trials (Phase 2 1, Phase 1/2 1) 2003: 2 trials (Observational 1, Phase 2 1) 2004: 3 trials (Observational 2, Phase 2 1) 2005: 4 trials (Phase 3 1, Phase 2 3) 2006: 3 trials (Not applicable 1, Phase 3 1, Phase 2 1) 2007: 4 trials (Observational 1, Not applicable 1, Phase 2 1, Phase 1 1) 2008: 7 trials (Observational 3, Not applicable 1, Phase 3 2, Phase 2 1) 2009: 8 trials (Unknown 1, Observational 3, Not applicable 1, Phase 3 1, Phase 1/2 1, Phase 1 1) 2010: 3 trials (Observational 2, Not applicable 1) 2011: 2 trials (Phase 2 1, Phase 1 1) 2013: 2 trials (Not applicable 1, Phase 2 1) 2014: 1 trials (Observational 1) 2015: 3 trials (Observational 1, Phase 2 1, Phase 1 1) 2016: 1 trials (Phase 1 1) 2018: 1 trials (Phase 2 1) 2019: 1 trials (Phase 2 1) 2020: 2 trials (Observational 1, Phase 1/2 1) 2021: 1 trials (Phase 2 1) 2023: 1 trials (Phase 1 1)

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
2023 1
2021 1
2020 2
2019 1
2018 1
2016 1
2015 3
2014 1
2013 2
2011 2

Showing 10 of 22 earlier years.

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No forecast is made for this condition: it averages 0.4 trial starts a year over the five years to September, under the 5 a year the method needs.

Recruitment duration, a model prediction: 1 trial still running at the 2026-10-09 snapshot, fewer than the 5 needed for a median. Source: ClinicalTrials.gov / AACT (CTTI), public domain.

What phase are Leukemia, Mast-Cell trials in? (8)

Shown in this order: phase order.

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

How many Leukemia, Mast-Cell 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 1
Recruiting 3
Active, not recruiting 3
Available 1
Completed 37

Showing 5 of 9 statuses.

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

Which lead sponsors run the most Leukemia, Mast-Cell trials? (5)

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 5
National Cancer Institute (NCI) 3
Novartis Pharmaceuticals 3
Children's Oncology Group 2
Cogent Biosciences, Inc. 2

Which drugs are being tested for Leukemia, Mast-Cell? (4)

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
Midostaurin 3
Bezuclastinib 2
Avapritinib 2
Cytoxan 2

Interventions as named in the registry (11)

Shown in this order: most trials first.

Interventions as named in the registry
Drug Trials
SQ Fludarabine 7
Total Body Irradiation 6
Cyclosporine 0.05% 5
Mofetil Mycophenolate 5
Peripheral blood stem cell transplantation 4

Showing 5 of 11 interventions.

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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 Leukemia, Mast-Cell 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 18 5
Febrile neutropenia 12 6
Sepsis 12 2
Anaemia 11 2
Gastrointestinal haemorrhage 11 2

Showing 5 of 10 adverse events.

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Which publications cover Leukemia, Mast-Cell? (4)

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

Publications
Publication Journal Year Major topic
Efficacy and Safety of Midostaurin in Advanced Systemic Mastocytosis. The New England journal of medicine 2016 Yes
Systemic mastocytosis in adults: 2021 Update on diagnosis, risk stratification and management. American journal of hematology 2021 No
Efficacy and safety of midostaurin in patients with advanced systemic mastocytosis: 10-year median follow-up of a phase II trial. Leukemia 2018 No
Diagnostic criteria and classification of mastocytosis: a consensus proposal. Leukemia research 2001 No

Prevalence (6)

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
Mast cell leukemia Point prevalence <1 / 1 000 000 Europe Not stated Unspecified
Mast cell leukemia Point prevalence <1 / 1 000 000 Europe Not stated Exact
Mast cell leukemia Annual incidence <1 / 1 000 000 Europe Not stated Unspecified
Mast cell leukemia Annual incidence <1 / 1 000 000 Europe Not stated Exact
Mast cell leukemia Point prevalence <1 / 1 000 000 Europe Not stated Exact

Showing 5 of 6 prevalence observations.

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Rare-disease identity (2)

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

Rare-disease identity
Orphanet disease ORPHA code Relation Source
Mast cell leukemia 98851 Exact Orphanet Rare Disease Ontology (ORDO)
Mast cell leukemia 98851 Exact Orphadata Science knowledge files, Product 1

Not on this page

Not measured: delivery record.

Checked, with nothing recorded for this condition: sub-conditions in mesh.

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