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, 5 tagged with Congenital thrombotic thrombocytopenic purpura itself.
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New hope for rare blood disorder: experimental drug TGD001 enters human trials
Disease control Not yet recruitingThis early-phase trial tests a new drug called TGD001 in about 70 adults with acute episodes of immune-mediated thrombotic thrombocytopenic purpura (iTTP) or related blood clotting conditions. The main goal is to check the drug's safety and how the body processes it, while also l…
Phase 1/2 • Sponsor: TargED Biopharmaceuticals B.V. • Aim: Disease control
Last updated Jun 27, 2026 11:00 UTC
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Ginger vs. heavy periods: a natural approach for women with bleeding disorders
Symptom relief Not yet recruitingThis study tests whether taking ginger powder for the first three days of each period can reduce heavy menstrual bleeding in women with coagulation disorders (bleeding problems). 74 women will be randomly assigned to receive either ginger or a placebo for six cycles. The goal is …
Early phase 1 • Sponsor: University of Valladolid • Aim: Symptom relief
Last updated Jun 27, 2026 12:35 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 30, 2026 00:00 UTC