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 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 tests a high dose of a medicine called ambroxol in adults with MPS III (Sanfilippo syndrome). It aims to see if the drug is safe and can help symptoms.
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 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 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 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.
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