Systemic mastocytosis
MONDO:0016586Systemic mastocytosis (SM) comprises a heterogeneous group of rare acquired and chronic hematological malignancies that are related to an abnormal proliferation of mast cells in tissue, including bone marrow, with or without skin involvement.
Also known as: Mast cell disease, systemic mastocytosis, systemic tissue Mast cell disease, systemic tissue mast cell disease, Aggressive systemic mastocytosis, SM, systemic mast cell disease, systemic mastocytosis with associated hemotologic non-mast cell lineage disease (SM-AHNMD)
1875 clinical trials for this condition and its sub-types, 11 tagged with Systemic mastocytosis itself.
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Sub-types of Systemic mastocytosis
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Mast cell leukemia 9 trials
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Indolent systemic mastocytosis 8 trials
2 sub-types
- Smouldering systemic mastocytosis 3 trials
- Isolated bone marrow mastocytosis 2 trials
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Aggressive systemic mastocytosis 4 trials
1 sub-type
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Extracutaneous mastocytoma 0 trials
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New hope for rare blood cancer: experimental drug targets mast cells
Disease control Recruiting nowThis study tests an experimental drug called CGT9486 (bezuclastinib) in people with advanced systemic mastocytosis, a rare and serious blood disorder. The drug aims to control the disease by blocking a key protein that drives abnormal mast cell growth. About 140 adults with aggre…
Phase 2 • Sponsor: Cogent Biosciences, Inc. • Aim: Disease control
Last updated Sep 17, 2026 00:00 UTC
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Avapritinib rollover trial aims to confirm Long-Term safety for mastocytosis patients
Disease control Recruiting nowThis study looks at the long-term safety of the drug avapritinib (AYVAKIT®) in people with systemic mastocytosis who have already been taking it and benefiting from it in a previous study. About 60 participants will continue receiving the drug and be monitored for serious side ef…
Phase 4 • Sponsor: Blueprint Medicines Corporation • Aim: Disease control
Last updated Jun 27, 2026 09:04 UTC
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Blood test revolution: pinpointing the cause of swelling attacks
Diagnosis Recruiting nowThis study evaluates whether new blood tests can more accurately diagnose the cause of recurrent angioedema—a condition causing severe swelling—compared to current tests. Researchers will compare novel diagnostic assays with traditional ones in 600 people aged 12 and older who ha…
Sponsor: Foundation For Rare Disease Research • Aim: Diagnosis
Last updated Aug 26, 2026 00:00 UTC
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Blood test may spare patients painful bone marrow biopsy
Diagnosis Recruiting nowThis study is testing whether a simple blood test can accurately diagnose systemic mastocytosis, a rare disease where too many mast cells build up in the body. Currently, diagnosis requires an invasive bone marrow biopsy. The blood test looks for a specific genetic mutation (KIT-…
Sponsor: The First Affiliated Hospital of Soochow University • Aim: Diagnosis
Last updated Jun 27, 2026 12:28 UTC