Early access treatment for Sanfilippo syndrome type B
Part of Genetic & congenital, Hormones & metabolism, Skin clinical trials.
This program provides early access to an experimental enzyme therapy given directly into the brain for children with Sanfilippo syndrome type B, a genetic condition that affects the nervous system. The treatment aims to replace a missing enzyme to slow or stabilize symptoms.
Summary written for real people, not researchers, by Clin2.
Who can take part
- The child must be diagnosed with Sanfilippo syndrome type B, confirmed by a blood test showing low NAGLU enzyme activity.
- Children must be at least 12 months old. For those under 5 years, there is a minimum developmental score requirement; older children can join regardless of development level.
- A parent or legal guardian must agree to the treatment and sign a consent form.
- The child must not have medical conditions that make brain surgery or infusions risky, such as severe heart, lung, or bleeding problems, uncontrolled seizures, hydrocephalus, or ventricular shunts.
- The child must not have taken any other investigational drug within 30 days before the start of treatment.
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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