NCT06333041View on ClinicalTrials.gov

Study of Cannabidiol in Sanfilippo Syndrome

PHASE2/PHASE3Not yet recruitingINTERVENTIONAL
Enrollment

35

Participants

Timeline

Start Date

June 30, 2025

Primary Completion Date

June 30, 2026

Study Completion Date

January 31, 2027

Conditions
Sanfilippo SyndromeMucopolysaccharidosis 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

Trial Locations (1)

90502

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

All Listed Sponsors
lead

Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center

OTHER