Study of Cannabidiol in Sanfilippo Syndrome

Part of paid clinical trials in Torrance, California.

Sponsor
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Study ID
NCT06333041
Phase
PHASE2/PHASE3
Status
Recruiting
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Conditions

  • Mucopolysaccharidosis III
  • Sanfilippo Syndrome

Eligibility Criteria

Sex
ALL
Age
4 Years - N/A
Healthy Volunteers
Not accepted

Interventions

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

Study Details

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).

Key Dates

Start date
Apr 30, 2026
Status verified
Mar 2026
Primary completion
Apr 30, 2027
Completion
Dec 31, 2028

Study Design

Enrollment
35 participants (estimated)
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT

Arms

  • Experimental: Epidiolex (cannabidiol)
    Oral Epidiolex (cannabidiol) administered twice daily (BID) Week 1: 5 mg/kg/day BID Weeks 2-8: 10 mg/kg/day BID Weeks 9-16: 20 mg/kg/day BID 8-week washout; cross-over to placebo comparator starting on Week 24.
  • Placebo Comparator: Placebo (PBO)
    Week 1: Dose escalate to maximum tolerated placebo dose Weeks 2-16: Maximum tolerated placebo dose 8-week washout; cross-over to Experimental group starting at week 24, following the same dose-escalation as the Experimental arm.

Primary Outcome Measure

Sanfilippo Behavior Rating Scale (SBRS)- mood/anger/aggression score [ Time Frame: Changes over 16 weeks of treatment versus 16 weeks of placebo ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
The Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical CenterTorranceCalifornia90502
Cristi Williams
[email protected]
Lynda Polgreen, MD, MS (PRINCIPAL_INVESTIGATOR)

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By research site
The Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center· Torrance, CA

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