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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: Recruiting Phase 2 study, sponsored by Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center.
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, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (74% lower). According to ClinicalTrials.gov, the official US trial registry.
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).
Primary Outcome
The Sanfilippo Behavior Rating Scale is a 68-item questionnaire, developed by Shapiro et al to assess the behavioral phenotype of children with Sanfilippo syndrome and its progression over time. The Sanfilippo Behavior Rating Scale mood/anger/aggression cluster is one of four clusters and two domains that make up the Sanfilippo Behavior Rating Scale. A higher score indicates more severe mood/anger/aggression symptoms. Each question within the mood/anger/aggression cluster is scored on a scale of
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 |
What NCT06333041 shows while recruiting
NCT06333041 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 35 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (74% lower).
The record links to 0 conditions, and to 2 interventions - of which Placebo is the first listed.
NCT06333041 does not publish any study locations in the registry export this page uses.
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.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT06333041's enrollment target sits among peer trials
35 1524th of 2000 higher than 462 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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