Inherited thrombocytopenia
MONDO:0100241An instance of thrombocytopenia that is inherited.
Also known as: hereditary thrombocytopenia
30 clinical trials for this condition and its sub-types.
Follow this condition — get notified about new trialsSub-types
Broader categories
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New drug UX016 targets rare muscle disease
Disease control Not yet recruitingThis early-stage trial tests UX016, a drug designed to help people with GNE myopathy, a rare genetic muscle-weakening disease. Researchers will give the drug or a placebo to 24 adults to see if it is safe and improves muscle strength. The study is not yet recruiting.
Phase: PHASE1, PHASE2 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated Jun 27, 2026 12:04 UTC
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New hope for rare blood disorder: study reviews real-world use of enzyme therapy
Knowledge-focused Not yet recruitingThis study looks back at medical records of people with congenital thrombotic thrombocytopenic purpura (cTTP), a rare inherited blood disorder that causes dangerous blood clots. Participants received a manufactured version of the missing ADAMTS13 enzyme through an early access pr…
Sponsor: Takeda • Aim: Knowledge-focused
Last updated Aug 09, 2026 00:00 UTC
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Does an enlarged spleen change the outlook for low platelet patients?
Knowledge-focused Not yet recruitingThis study will look at 100 adults with low platelet counts to see how many have an enlarged spleen and whether that affects their symptoms, complications, or response to treatment. The goal is to better understand the role of the spleen in these blood disorders. No new treatment…
Sponsor: Assiut University • Aim: Knowledge-focused
Last updated Jun 26, 2026 18:59 UTC