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)
1868 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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Experimental drug aims to tame rare mast cell disease
Disease control CompletedThis phase 2 trial tests whether the drug RAD001 (everolimus) can help control systemic mastocytosis, a rare condition where too many mast cells build up in organs like the bone marrow. Ten adults with the disease take a daily pill for 30 days. Researchers measure success by chan…
Phase 2 • Sponsor: M.D. Anderson Cancer Center • Aim: Disease control
Last updated Jul 08, 2026 00:00 UTC
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Bone drug shows promise for rare mast cell disorder
Disease control CompletedThis study tested whether denosumab, a drug given as a shot every 6 months, can improve bone density in people with osteoporosis caused by systemic mastocytosis. 24 adults with the condition took part. The goal was to see if the drug strengthens bones and prevents fractures bette…
Phase 3 • Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Disease control
Last updated Jun 27, 2026 12:02 UTC
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New antibody drug tested in patients with relapsed blood cancers
Disease control CompletedThis early-stage trial tested the safety and best dose of flotetuzumab, an antibody drug, in 13 patients with advanced blood cancers that had come back or stopped responding to treatment. The drug works by blocking cancer cell growth. The main goal was to see what side effects oc…
Phase 1 • Sponsor: City of Hope Medical Center • Aim: Disease control
Last updated Jun 27, 2026 09:00 UTC
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Old drug, new hope? thalidomide tested for rare mast cell cancer
Disease control CompletedThis study tested the drug thalidomide in people with systemic mastocytosis, a rare disease where too many mast cells build up in the body. The goal was to see if thalidomide could reduce the amount of disease in the bone marrow or other organs after 6 months. The trial included …
Sponsor: Centre Hospitalier Universitaire, Amiens • Aim: Disease control
Last updated Jun 27, 2026 07:51 UTC