Actively Recruiting
Randomized, Double-Blind, Placebo-Controlled Cross-Over Study to Evaluate the Effects of Cannabidiol on Neurobehavioral and Functional Outcomes in Sanfilippo Syndrome
Led by Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center · Updated on 2026-04-06
35
Participants Needed
1
Research Sites
87 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating cannabidiol (CBD) in children with Sanfilippo syndrome, a rare genetic disorder causing progressive brain degeneration and severe neurobehavioral problems. This trial aims to determine the safety of cannabidiol and explore its effects on behavior, mood, sleep, pain, and overall function in affected children. The study uses Epidiolex, an FDA-approved pharmaceutical-grade cannabidiol solution, to address the lack of current treatments for these symptoms. Participants will be divided into two groups: the first group of 5 participants will receive Epidiolex only, while the next 30 participants will be randomly assigned to receive either Epidiolex or a placebo for 16 weeks, then switch after an 8-week break. After this blinded, crossover phase, all participants will receive Epidiolex openly for 52 weeks to assess long-term safety. Epidiolex dosing starts low and increases over the first eight weeks, with treatments given twice daily. Caregivers will regularly complete surveys about the participant's behavior, mood, sleep patterns, pain, stooling, and caregiver stress throughout the study. Safety labs and questionnaires will be collected during all treatment periods to monitor adverse events and treatment effects. The main outcome measured is the change in mood, anger, and aggression over 16 weeks of treatment compared to placebo. The study is expected to last several years, with careful monitoring and follow-up for all participants.
CONDITIONS
Brief Title
Study of Cannabidiol in Sanfilippo Syndrome
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Confirmed diagnosis of Mucopolysaccharidosis type III (Sanfilippo syndrome) by genetic testing
- At least 4 years old
- Patient or legal guardian able and willing to provide informed consent; assent required for patients aged 7 to 16 when possible
- No dose changes in anakinra, fluoxetine, or probiotic supplements for at least 8 weeks prior
- Meets one of the following: previous gene/cell therapy or enzyme restorative trial participation, ineligibility for such trials due to advanced disease, or functional age less than or equal to half of chronological age as measured by Vineland
You will not qualify if you...
- Mutation causing slowly progressive disease
- Use of any cannabis, including cannabidiol, within the last 8 weeks
- Current enrollment in another clinical trial
- Use of rifampin, clobazam, stiripentol, everolimus, sirolimus, tacrolimus, digoxin, valproate, felbamate (less than one year), or recent use of THC or synthetic cannabinoids within last 3 months
- Unstable non-pharmacological therapies within 4 weeks prior
- Liver disease or injury indicated by abnormal liver tests
- Known allergy to Epidiolex components
- Pregnancy or lactation
- Any other condition deemed a significant risk by the investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - Approximately 40 weeks including both treatment periods and washout
Participants receive either Epidiolex or placebo for 16 weeks, followed by an 8-week washout, then cross over to the opposite treatment for another 16 weeks. Safety labs and questionnaires are collected throughout these treatment periods to assess safety and efficacy.
Visits occur regularly during the 16-week treatment periods and the 8-week washout to collect safety labs and questionnaires
Duration - 52 weeks
After the blinded crossover treatment periods, all participants receive open-label Epidiolex for 52 weeks to measure long-term safety and efficacy through behavioral and functional outcomes and adverse event monitoring.
Regular visits throughout the 52-week open-label treatment period to assess safety and efficacy
Trial Site Locations
Total: 1 location
1
The Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Torrance, California, United States, 90502
Actively Recruiting
Research Team
J
Jonathan Acevedo, BS
A
Adolfo Morales, BA
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
CROSSOVER
Primary Purpose
TREATMENT
Number of Arms
2
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