CBD study for Sanfilippo syndrome
Treatments studied
Part of Genetic & congenital, Hormones & metabolism, Skin clinical trials.
This study tests whether a cannabis-derived medicine called cannabidiol (CBD) can help children with Sanfilippo syndrome, a rare genetic disorder. It may be a good fit for children who have already tried other treatments or whose disease is more advanced.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your child must have a confirmed diagnosis of Sanfilippo syndrome (MPS III) through genetic testing.
- Your child must be at least 4 years old.
- Your child cannot have used any cannabis or CBD products (including Epidiolex) in the last 8 weeks.
- Your child must have either been in a previous gene/cell therapy or enzyme trial, or been too advanced for such a trial, or have a functional age (Vineland score) that is half or less than their actual age.
- Your child cannot be taking certain medications like rifampin, diazepam (except for seizures), clobazam, or others listed.
- Your child cannot have liver problems or be pregnant or breastfeeding.
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 study follows people with MPS IIIA (Sanfilippo syndrome type A) who have received or plan to receive the gene therapy rebisufligene etisparvovec, to monitor their health and treatment effects over time.
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 follow-up study looks at how a past gene therapy treatment (UX111) is affecting you over time. It mainly involves continued visits and safety checks, and it helps researchers understand long-term benefits and risks.
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.
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 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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