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NCT06333041 · ClinicalTrials.gov registry record · Phase 2
Study of Cannabidiol in Sanfilippo Syndrome
A Phase 2 study, sponsored by Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center.
- Recruiting
- Registry status
- Phase 2
- Development phase
- 35
- Enrollment target
NCT06333041 is a Phase 2 study that is actively recruiting participants, run by Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center. The registered enrollment target is 35 participants.
The verdict
NCT06333041, a Phase 2 study, is actively recruiting participants, sponsored by Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center.
- RECRUITING
- Registry status
- Phase 2
- Development phase
- 35 participants
- Enrollment target
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).
Interventions
- DRUG Placebo
- DRUG Epidiolex
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 35 participants |
| Start Date | 2026-04-01 |
| Est. Completion | 2028-12 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT06333041
The ClinicalTrials.gov registry entry for NCT06333041 describes a study currently listed as recruiting, categorized as Phase 2. The registered enrollment target is 35 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center, which has 55 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 2 interventions - of which Placebo is the first listed.
NCT06333041 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT06333041 about?
NCT06333041 is a clinical study titled "Study of Cannabidiol in Sanfilippo Syndrome". 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 Sanfi...
What is the current status of trial NCT06333041?
This trial is currently recruiting. It is a Phase 2 study. The enrollment target is 35 participants. The study started on 2026-04-01. Estimated completion is 2028-12.
What interventions are being tested in trial NCT06333041?
The interventions under investigation include: Placebo (DRUG), Epidiolex (DRUG).
Who is sponsoring clinical trial NCT06333041?
This trial is sponsored by Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center, which has 55 total clinical trials registered on ClinicalTrials.gov.
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