CBD trial aims to ease sanfilippo syndrome symptoms
NCT ID NCT06333041
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests whether cannabidiol (CBD) can safely improve behavior, mood, sleep, and daily function in people with Sanfilippo syndrome, a rare genetic disorder. Thirty-five participants will receive either CBD or a placebo, then switch after a break. Caregivers will report on symptoms and stress levels.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- cannabidiol (CBD) oral solution
- What this could lead to
- If it works, this could point toward a way to manage difficult behaviors, mood, and sleep problems in Sanfilippo syndrome.
- What could go wrong
- This is a small, early-stage trial with only 35 people. It may not show clear benefits, and CBD might not work for everyone or could cause side effects.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 2/3
Runs two stages together: whether the treatment works, then large-scale confirmation.
- Participants
-
About 35 people
The number the study aims to enrol. It can still change while the study runs.
- Started
-
Apr 2026
- Expected to finish
-
Dec 2028
An estimate. End dates often move.
- Lead sponsor
-
Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
4 years and older
- Sex
-
Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * MPS III diagnosis confirmed by genetic testing * ≥ 4 years of age * Patient or parent/legal guardian is able and willing to provide informed consent. For patients 7 to 16 years of age, assent must also be provided when cognitively possible. * If taking any of the following, no dose changes for the last 8 weeks: 1. Anakinra 2. Fluoxetine 3. Probiotic supplement * One of the following criteria are met: 1. Previous participation in a gene/cell therapy or enzyme restorative clinical trial. 2. Previously ineligible to participate in a gene/cell therapy or enzyme restorative clinical trial due to advanced disease state. 3. Functional age, as measured by the Vineland, is ≤ 0.5 chronological age Exclusion Criteria: Equitable selection will be used when choosing participants. An individual who meets any of the following criteria will be excluded from participation in this study: * Mutation known to cause slowly progressive disease * Taken any form of cannabis, including cannabidiol, in the last 8 weeks * Currently enrolled in another ongoing clinical trial * Concomitant use of any of the following therapies: * Rifampin * Diazepam (except for intermittent use as needed for treatment of a prolonged seizure episode) * Clobazam * Stiripentol * Everolimus, sirolimus, tacrolimus * Digoxin * Valproate * Recreational or medical Tetrahydrocannabinol (THC) or synthetic cannabinoid medications (including Sativex) within the last three months * Felbamate (if taking for less than one year) * Non-pharmacological therapies (e.g. ketogenic diet) must be stable for up to four weeks prior to enrollment * Clinical evidence of liver disease or liver injury as indicated by the presence of abnormal tests (AST or ALT \> 2 x ULN; Bilirubin \> 2 x ULN) * Known hypersensitivity to any components of Epidiolex (cannabidiol) * Pregnant or lactating women * Any other social or medical condition that the investigator believes would pose a significant hazard to the subject if the investigational therapy were initiated or be detrimental to the study
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for Mucopolysaccharidosis iii are added.
By submitting, you agree to our Terms of use
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
-
The places running it
1 site. The list below names each one and where it is.
-
The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
-
A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
-
The Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
RECRUITINGTorrance, California, 90502, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.