Early access treatment for Sanfilippo syndrome type B
Part of Genetic & congenital, Hormones & metabolism, Skin clinical trials.
This treatment program gives an early access enzyme therapy called tralesinidase alfa directly into the fluid around the brain (via a small pump) to help children with Sanfilippo syndrome type B. It aims to slow or stop the worsening of symptoms.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your child must have a confirmed diagnosis of Sanfilippo syndrome type B (MPS IIIB) through a blood test showing low NAGLU enzyme activity.
- Your child must be either between 12 and 60 months old with a certain developmental score, or older than 60 months regardless of development level.
- Your child cannot have conditions that make brain surgery unsafe, like serious heart, lung, or bleeding problems.
- Your child cannot have poorly controlled seizures, hydrocephalus, or a shunt (a tube that drains fluid from the brain).
- Your child must not have taken any other experimental drugs in the last 30 days.
- You must agree to follow birth control rules if your child is a female who could become pregnant.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
This trial tests an experimental treatment (tralesinidase alfa) delivered directly into the brain for children with MPS IIIB, a rare genetic disorder that affects thinking and development. The goal is to slow or stop cognitive decline by replacing a missing enzyme.
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.
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 early-stage trial tests JR-446, a new treatment for MPS IIIB, a rare genetic disorder that affects the brain and body. The treatment is delivered directly into the fluid around the spinal cord and aims to slow or improve symptoms in young children.
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.
This study follows people with MPS IIIC (Sanfilippo syndrome type C) over time to understand how the disease changes. It does not test a new treatment, but helps researchers learn more about the condition, which could lead to better care in the future.
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