Enzyme replacement for classical homocystinuria in children
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism, Skin clinical trials.
This study tests a new enzyme replacement therapy called pegtibatinase for children with classical homocystinuria (HCU). The enzyme helps break down homocysteine, which may lower its levels in the blood and reduce symptoms.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are between 5 and 12 years old
- You have been diagnosed with classical homocystinuria (HCU)
- Your blood homocysteine level is at least 50 micromol/L
- You have not taken pegtibatinase before
- You can keep a stable treatment routine, including diet and other HCU medications
- You weigh at least 15 kg
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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