Actively Recruiting

Phase 2
Phase 3
Age: 4Years +
All Genders
NCT06333041

Study of Cannabidiol 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

139 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

The goal of this clinical trial is to test cannabidiol in Sanfilippo syndrome. The main questions it aims to answer are: 1) determine the safety of cannabidiol in Sanfilippo syndrome, and 2) explore the efficacy of cannabidiol in treating the neurobehavioral symptoms and functional outcomes of Sanfilippo syndrome. Each participant's caregiver will be asked to complete surveys related to the participant's behavior, mood, sleep, stooling, pain, and caregiver stress intermittently throughout the study. All participants will be enrolled into one of two cohorts based on enrollment order: 1. Sentinel Safety Cohort (first 5 participants) - all participants treated with Epidiolex (cannabidiol) 2. Controlled Cohort (next 30 participants) - participants randomized 1:1 (equal chance) to start treatment with Epidiolex (cannabidiol) or placebo for 16 weeks, followed by an 8-week washout period (no treatment). Participants then switch to the opposite treatment group for 16 weeks followed by all participants treated for 52 weeks with Epidiolex (cannabidiol).

CONDITIONS

Official Title

Study of Cannabidiol in Sanfilippo Syndrome

Who Can Participate

Age: 4Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Diagnosis of Mucopolysaccharidosis type III (Sanfilippo syndrome) confirmed by genetic testing
  • 4 years of age or older
  • Patient or parent/legal guardian able and willing to provide informed consent; assent required for patients aged 7 to 16 when cognitively possible
  • No dose changes in the last 8 weeks if taking Anakinra, Fluoxetine, or probiotic supplements
  • Meets at least one of the following: previous participation in gene/cell therapy or enzyme restorative trial; previously ineligible for such trials due to advanced disease; or functional age at or below 0.5 of chronological age measured by Vineland
Not Eligible

You will not qualify if you...

  • Mutation known to cause slowly progressive disease
  • Use of any cannabis products, including cannabidiol, within the last 8 weeks
  • Currently enrolled in another ongoing clinical trial
  • Use of the following medications: Rifampin, Diazepam (except intermittent use for prolonged seizures), Clobazam, Stiripentol, Everolimus, Sirolimus, Tacrolimus, Digoxin, Valproate, recreational or medical THC or synthetic cannabinoids within 3 months, and Felbamate if taken less than one year
  • Non-pharmacological therapies (e.g., ketogenic diet) not stable for at least 4 weeks prior to enrollment
  • Clinical evidence of liver disease or liver injury (AST or ALT > 2 x ULN; Bilirubin > 2 x ULN)
  • Known allergy to any component of Epidiolex (cannabidiol)
  • Pregnant or lactating women
  • Any other social or medical condition posing significant risk or detriment to study participation

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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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

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Research Team

J

Jonathan Acevedo, BS

CONTACT

A

Adolfo Morales, BA

CONTACT

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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