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 drug ADZYNMA tracked for safety in rare blood disorder
Disease control Recruiting nowThis study monitors 40 people in Japan with congenital thrombotic thrombocytopenic purpura (cTTP) who are receiving ADZYNMA (recombinant ADAMTS13) as part of their routine care. Researchers will track side effects and check if the drug improves platelet counts and ADAMTS13 activi…
Sponsor: Takeda • Aim: Disease control
Last updated Jun 27, 2026 07:53 UTC
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New study tracks safety of adzynma for rare blood clot disorder
Knowledge-focused Recruiting nowThis study looks at the real-world safety of Adzynma, a lab-made protein, in people with congenital thrombotic thrombocytopenic purpura (cTTP), a rare inherited blood disorder. Researchers will review medical records of about 50 children and adults to see how many develop allergi…
Sponsor: Takeda • Aim: Knowledge-focused
Last updated Jun 27, 2026 13:06 UTC