A study on treatment for people with congenital TTP who received ADAMTS13
Part of Blood & lymphatic clinical trials.
This study looks back at medical records of people with a rare blood disorder called congenital thrombotic thrombocytopenic purpura (cTTP) who received a treatment called recombinant ADAMTS13 (rADAMTS13). The goal is to learn more about how this treatment works in real life.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a confirmed diagnosis of severe congenital ADAMTS13 deficiency or cTTP.
- You must have received at least two doses of rADAMTS13 through the early access program.
- You or your legal guardian must agree to let the researchers look at your medical records, or local rules allow your data to be used without your consent.
- You should have been on regular treatment for cTTP or had at least one cTTP event in the past.
- You must not have had any other treatment options available, including other clinical studies.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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