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Study of Cannabidiol in Sanfilippo Syndrome

NCT06333041 · Status: RECRUITING · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 35

Last updated 2026-04-06

No results posted yet for this study

Summary

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

  • Sanfilippo Syndrome
  • Mucopolysaccharidosis III

Interventions

DRUG

Epidiolex

Epidiolex (cannabidiol) oral solution is a clear, colorless to yellow liquid containing cannabidiol at a concentration of 100 mg/mL. Inactive ingredients include dehydrated alcohol (7.9% w/v), sesame seed oil, strawberry flavor, and sucralose.

DRUG

Placebo

Placebo oral solution is a yellow oily solution containing the excipients sesame oil and anhydrous ethanol with added sweetener (sucralose) and strawberry flavoring

Sponsors & Collaborators

  • Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center

    lead OTHER

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
CROSSOVER

Eligibility

Min Age
4 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-04-30
Primary Completion
2027-04-30
Completion
2028-12-31
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06333041 on ClinicalTrials.gov