Enzyme treatment for children with sanfilippo syndrome type B
Part of Genetic & congenital, Hormones & metabolism, Skin clinical trials.
This trial is testing an experimental treatment given directly into the fluid around the brain to replace a missing enzyme in children with Sanfilippo syndrome type B. It aims to see if the treatment can slow or improve some symptoms of the disease.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Confirmed diagnosis of Sanfilippo syndrome type B (MPS IIIB) through a lab test showing low NAGLU enzyme activity.
- The severe form of the disease, not the mild or attenuated type.
- Child is between 1 and 5 years old, with significant developmental delay (low score on a development test).
- No prior treatment with stem cell transplant, gene therapy, or enzyme replacement for MPS IIIB.
- No other brain condition (like head injury, meningitis, or bleeding) that caused developmental problems.
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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