Long-term study of pegtibatinase for classical homocystinuria
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism, Skin clinical trials.
This trial is a long-term follow-up study examining the safety and effectiveness of pegtibatinase for people with classical homocystinuria (HCU). It is open to those who have recently taken part in or finished the HARMONY or COMPOSE studies.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are currently taking part in the COMPOSE study for HCU.
- Or you fully completed the 24-week treatment period (pegtibatinase or placebo) in the HARMONY study.
- You did not stop taking pegtibatinase in an earlier study due to serious side effects or intolerance.
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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