Congenital thrombotic thrombocytopenic purpura
MONDO:0010122Congenital thrombotic thrombocytopenic purpura is the hereditary form of thrombotic thrombocytopenic purpura (TTP) characterized by profound peripheral thrombocytopenia, microangiopathic hemolytic anemia (MAHA) and single or multiple organ failure of variable severity.
Also known as: Upshaw-Schulman syndrome, congenital ADAMTS-13 deficiency, congenital ADAMTS13 deficiency, congenital TTP, congenital thrombotic thrombocytopenic purpura, familial TTP, hereditary thrombotic thrombocytopenic purpura, thrombotic thrombocytopenic purpura, hereditary
22 clinical trials for this condition and its sub-types.
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New enzyme replacement therapy aims to stop dangerous blood clots in rare disease
Disease control OngoingThis study tests a medicine called TAK-755 in people with a rare inherited blood disorder called congenital thrombotic thrombocytopenic purpura (cTTP). The condition causes dangerous blood clots and bleeding. TAK-755 replaces a missing enzyme to help prevent or treat these episod…
Phase: PHASE3 • Sponsor: Takeda • Aim: Disease control
Last updated Jun 27, 2026 12:03 UTC
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AI takes on blood clotting: can ChatGPT guide surgeons on bleeding?
Knowledge-focused TerminatedThis study evaluates whether artificial intelligence models can accurately interpret ROTEM blood clotting tests and recommend treatments for coagulopathy. Researchers will compare AI decisions to those of a panel of clinical experts using data from adults undergoing elective card…
Sponsor: Ondokuz Mayıs University • Aim: Knowledge-focused
Last updated Jul 04, 2026 00:00 UTC