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Study of Epidiolex for Sanfilippo Syndrome

This study is testing Epidiolex (cannabidiol), an oral solution, in people with Sanfilippo Syndrome (Mucopolysaccharidosis III). Researchers want to see if Epidiolex is safe and if it can help with the behavioral problems and daily function often seen in Sanfilippo Syndrome. You may be able to join if you are at least 4 years old and have a confirmed diagnosis of Sanfilippo Syndrome through genetic testing. The study will look at changes in mood, anger, and aggression using a special rating scale over 16 weeks of treatment compared to a placebo (an inactive substance). The study aims to enroll about 35 participants, but its current status is unclear.

Study design
This interventional study plans to enroll about 35 participants. The first 5 participants will receive Epidiolex, while others will be assigned to either Epidiolex or a placebo.
What's involved
Caregivers will complete surveys about the participant's behavior, mood, sleep, pain, and their own stress at different times throughout the study.
Compensation
Not stated in the trial record.
Follow-up
The primary endpoint is measured at changes over 16 weeks of treatment versus 16 weeks of placebo.

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NCT06333041

Study of Cannabidiol in Sanfilippo Syndrome

Recruiting
PHASE2Ages 4+InterventionalTreatment
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
~35 participants
Updated 2026-06-25 on ClinicalTrials.gov
What's tested:EpidiolexPlacebo

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Sanfilippo Behavior Rating Scale (SBRS)- mood/anger/aggression score
Measured over Changes over 16 weeks of treatment versus 16 weeks of placebo

Conditions

Sanfilippo Syndrome
Mucopolysaccharidosis III

Explore related trials

Sanfilippo Syndrome

Where it's being run

1 sites across 1 states
California1

Who to contact

Jonathan Acevedo, BS
[email protected]
310-357-9023
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

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:
One of the following criteria are met:

Exclusion

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

What this trial measures

  • Sanfilippo Behavior Rating Scale (SBRS)- mood/anger/aggression scoreChanges over 16 weeks of treatment versus 16 weeks of placebo

    The Sanfilippo Behavior Rating Scale is a 68-item questionnaire, developed by Shapiro et al to assess the behavioral phenotype of children with Sanfilippo syndrome and its progression over time. The Sanfilippo Behavior Rating Scale mood/anger/aggression cluster is one of four clusters and two domains that make up the Sanfilippo Behavior Rating Scale. A higher score indicates more severe mood/anger/aggression symptoms. Each question within the mood/anger/aggression cluster is scored on a scale of 0-6 and a total mean score is calculated. The mean mood/anger/aggression score is standardized using the mean and standard deviation from a cohort of Sanfilippo patients, categorized as "slow progressors," ages 81-220 months. This reference cohort was chosen to best match the age distribution of our participants.