Study of JR-446 for Sanfilippo syndrome type B
Part of Genetic & congenital, Hormones & metabolism, Skin clinical trials.
This trial tests an experimental drug, JR-446, for children with Sanfilippo syndrome type B (MPS IIIB). The goal is to see if it is safe and can help with symptoms.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your child must be under 18 years old.
- Your child must have a confirmed diagnosis of Sanfilippo syndrome type B (MPS IIIB).
- Your child cannot have had gene therapy or a successful stem cell transplant.
- Your child must not have taken any other experimental drugs in the last 4 to 5 months.
- Your child must not have had a serious allergic reaction to drugs or have a bleeding disorder.
- Your child must be able to have a lumbar puncture (spinal tap) and an MRI.
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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