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Study of Cannabidiol in Sanfilippo Syndrome

StatusRecruiting
PhasePhase 2/3
Started
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Record History

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  1. Primary endpoints: details revised at 1 of 1 entry
    • Eligibility criteria expanded, about 29 words
  2. Start date changed from estimate of Apr 2026 to Apr 1, 2026v8
  3. Recruitment openedv7 Show 4 more changesShow less
    • Primary completion pushed from Feb 2027 to Apr 2027
    • Study completion pushed from Jul 2028 to Dec 2028
    • Start date: Mar 15, 2026 to Apr 2026
    • Site details revised
  4. Primary completion date: Jun 2026 to Feb 2027
    • Start date: Jun 2025 to Mar 15, 2026
    • Study completion date: Jan 2027 to Jul 2028
    v6
  5. Start date: Jan 2025 to Jun 2025
    • Primary completion date: Jan 2026 to Jun 2026
    • Site contact updated
    v5
Show 3 earlier versionsHide 3 earlier versions
  1. Start date: May 2024 to Jan 2025
    • Study completion date: May 2027 to Jan 2027
    • Site contact updated
    v4
  2. Site contact updatedv3
  3. Re-verified, no change to tracked fieldsv2
  1. Study First Submitted
  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
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).
Trial Details
NCT Number NCT06333041
Lead Sponsor Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Conditions
(from sponsor)		 Sanfilippo Syndrome, Mucopolysaccharidosis III
Related terms Mucopolysaccharidosis III
Enrollment 35 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov