A study testing pegtibatinase for classical homocystinuria
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism, Skin clinical trials.
This trial tests an injectable drug called pegtibatinase for people with a rare condition called homocystinuria. It aims to lower high levels of homocysteine in the blood compared to a placebo, while you continue your usual treatments.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be between 12 and 65 years old.
- You must have a confirmed diagnosis of classical homocystinuria (a rare genetic condition that affects how your body processes an amino acid called methionine).
- Your blood level of homocysteine must be at least 50 micromoles per liter at the screening visit (most people need a level of 80 or higher).
- You must be willing to keep your current diet and medications stable throughout the study (unless changes are needed for safety).
- If you could become pregnant, you must use highly effective birth control and have a negative pregnancy test before starting.
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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