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Cannabinoid
Cannabidiol for Sanfilippo Syndrome
Phase 2 & 3
Waitlist Available
Research Sponsored by Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up changes over 16 weeks of treatment versus 16 weeks of placebo
Summary
This trial aims to test the use of cannabidiol in treating Sanfilippo syndrome. They want to see if it is safe and effective in improving the behavior and daily functioning of patients with this
Who is the study for?
This trial is for individuals with Sanfilippo syndrome, a rare genetic condition. Participants will be selected based on their order of enrollment and must have caregivers able to complete various surveys about the patient's behavior and health throughout the study.
What is being tested?
The study tests cannabidiol (Epidiolex) against a placebo to assess its safety and effectiveness in treating neurobehavioral symptoms of Sanfilippo syndrome. It includes an initial safety cohort followed by a controlled group that switches between Epidiolex and placebo.
What are the potential side effects?
While specific side effects are not listed here, cannabidiol may commonly cause drowsiness, changes in appetite, diarrhea, fatigue, malaise, weakness or sleeping problems among others.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ changes over 16 weeks of treatment versus 16 weeks of placebo
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~changes over 16 weeks of treatment versus 16 weeks of placebo
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
Sanfilippo Behavior Rating Scale (SBRS)- mood/anger/aggression score
Side effects data
From 2022 Phase 1 & 2 trial • 3 Patients • NCT0423875467%
Abdominal Pain
33%
Drowsiness
33%
Headache
33%
Nausea
100%
80%
60%
40%
20%
0%
Study treatment Arm
Epidiolex
Placebo
Trial Design
2Treatment groups
Experimental Treatment
Placebo Group
Group I: Epidiolex (cannabidiol)Experimental Treatment1 Intervention
Oral Epidiolex (cannabidiol) administered twice daily (BID) Week 1: 5 mg/kg/day BID Weeks 2-8: 10 mg/kg/day BID Weeks 9-16: 20 mg/kg/day BID
8-week washout; cross-over to placebo comparator starting on Week 24.
Group II: Placebo (PBO)Placebo Group1 Intervention
Week 1: Dose escalate to maximum tolerated placebo dose Weeks 2-16: Maximum tolerated placebo dose
8-week washout; cross-over to Experimental group starting at week 24, following the same dose-escalation as the Experimental arm.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Epidiolex
2023
Completed Phase 2
~530
Find a Location
Who is running the clinical trial?
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical CenterLead Sponsor
104 Previous Clinical Trials
46,548 Total Patients Enrolled