Gene therapy for Sanfilippo syndrome type B
Part of Genetic & congenital, Hormones & metabolism, Skin clinical trials.
This trial tests a one-time gene therapy injection for children with Sanfilippo syndrome type B (MPS IIIB), a condition that affects the brain and development. The goal is to see if adding a healthy gene can slow or stop the disease.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your child must be older than 6 months.
- A confirmed diagnosis of Sanfilippo syndrome type B (MPS IIIB) is required.
- Your child should have a history of developmental delays, such as delayed speech, learning, or memory skills.
- Your child must not have received any other experimental drug or procedure in the last 30 days.
- Your child cannot have serious heart, liver, or blood problems, or severe seizure disorders that are not well controlled.
- Your child must be clear of infections or vaccines within the last 6 weeks.
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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This trial tests a gene-transfer treatment (delivered into the spinal fluid) for children with MPS IIIA, a rare genetic condition. It aims to improve or slow down brain and developmental changes caused by a missing enzyme.
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.
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.
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This study follows people with Sanfilippo syndrome type C to better understand how the disease changes over time. It does not appear to test a new medicine; instead, it collects health and development information that can help future treatments.
This trial tests a gene therapy given as a nose spray for people with cerebral palsy or HIE. It aims to see if the treatment is safe and helps improve movement over two years.
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