A study of GC1130A for Sanfilippo syndrome type A
Part of Genetic & congenital, Hormones & metabolism, Skin clinical trials.
This trial tests a new medicine called GC1130A for children with Sanfilippo syndrome type A (MPS IIIA). The goal is to see if it is safe and if it might help with symptoms.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have been diagnosed with Sanfilippo syndrome type A (MPS IIIA).
- You must be between 1 and 18 years old.
- You cannot have any major brain problems that are not caused by Sanfilippo syndrome.
- You cannot have had problems before from receiving medicine through a port in your head.
- You cannot have had a stem cell or bone marrow transplant or gene therapy for this condition.
- You cannot have taken another experimental treatment for Sanfilippo in the last 30 days (or 5 half-lives).
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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