NCT06333041 Study of Cannabidiol in Sanfilippo Syndrome
| NCT ID | NCT06333041 |
| Status | Recruiting |
| Phase | Phase 2, Phase 3 |
| Sponsor | Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center |
| Condition | Sanfilippo Syndrome |
| Study Type | INTERVENTIONAL |
| Enrollment | 35 participants |
| Start Date | 2026-04-01 |
| Primary Completion | 2027-04 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
In Phase 2, researchers evaluate early signs of effectiveness. You may be randomized to receive the active treatment or a comparator. Monitoring continues closely.
This trial targets 35 participants in total. It began in 2026-04-01 with a primary completion date of 2027-04.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
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).
Eligibility Criteria
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
Contact & Investigator
Frequently Asked Questions
Who can join the NCT06333041 clinical trial?
This trial is open to participants of all sexes, aged 4 Years or older, studying Sanfilippo Syndrome. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT06333041 trial and what does that mean for participants?
Phase 2 trials evaluate whether the treatment shows signs of effectiveness while continuing to monitor safety. More participants are enrolled than in Phase 1 to help refine the treatment protocol.
Is NCT06333041 currently recruiting?
Yes, NCT06333041 is actively recruiting participants. Contact the research team at jonathan.acevedo@lundquist.org for enrollment information.
Where is the NCT06333041 trial being conducted?
This trial is being conducted at Torrance, United States.
Who is sponsoring the NCT06333041 clinical trial?
NCT06333041 is sponsored by Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center. The trial plans to enroll 35 participants.