Recruiting
Phase 2
Phase 3

Cannabidiol

Sponsor:

UCLA

Code:

NCT06333041

Conditions

Sanfilippo Syndrome

Mucopolysaccharidosis III

Eligibility Criteria

Sex: All

Age: 4+

Healthy Volunteers: Not accepted

Interventions

Epidiolex

Placebo

Study Details

Brief 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

Study ID

NCT06333041

Start date

Apr 1, 2026

Status verified date

Jun, 2026

Completion date

Dec, 2028

Anticipated

Primary completion date

Apr, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 4+

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • 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:

1. Anakinra
2. Fluoxetine
3. Probiotic supplement
  • One of the following criteria are met:

1. Previous participation in a gene/cell therapy or enzyme restorative clinical trial.
2. Previously ineligible to participate in a gene/cell therapy or enzyme restorative clinical trial due to advanced disease state.
3. Functional age, as measured by the Vineland, is ≤ 0.5 chronological age

Exclusion Criteria:

Equitable selection will be used when choosing participants. An individual who meets any of the following criteria will be excluded from participation in this study:

  • 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

Study Design

Enrollment

35 participants

Anticipated

Allocation

Randomized

Intervention Model

Crossover

Primary purpose

Treatment

Interventions and Outcome Measures

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.

Interventions

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.

Placebo

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

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 and Locations

Central contacts

Jonathan Acevedo, BS

310-357-9023jonathan.acevedo@lundquist.org

Adolfo Morales, BA

310-357-9023adolfo.morales@lundquist.org

Locations

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

Recruiting

Torrance, California, United States, 90502

Contacts

Cristi Williams

cristi.williams@lundquist.org

Principal Investigator:

Lynda Polgreen, MD, MS

More Information

Sponsor

Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center

Last update posted

Jun 25, 2026

Last verified

Jun, 2026

Keywords

  • Cannabidiol
  • Epidiolex

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-11. This information was provided to ClinicalTrials.gov by Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center on 2026-06-25.