Congenital Thrombotic Thrombocytopenic Purpura (TTP) is a condition where blood clots form in small blood vessels, which can block blood flow to organs like the brain and heart. This can lead to serious health issues. TTP also reduces the number of platelets, which help blood clot, causing bleeding problems and purple skin bruises called purpura. Anemia, a condition where red blood cells break down too quickly, can also occur. TTP happens due to a lack of activity in the ADAMTS13 enzyme, which helps control blood clotting. The study is testing a medicine called TAK-755 that replaces ADAMTS13 to prevent or manage TTP flare-ups. Participants can receive two kinds of treatments: to prevent flare-ups or to control them when they happen.
- Commitment: Participation lasts up to 3 years, with clinic visits every 12 weeks.
- Treatment: TAK-755 is given through a vein, and some may receive it at home.
- Eligibility: Participants must be aged 0-70, diagnosed with severe congenital ADAMTS-13 deficiency, and meet other health criteria.
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