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Cannabinoid
Epidiolex (cannabidiol) for Mucopolysaccharidosis
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
Awards & highlights
Study 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.Check my eligibility
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.See study design
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
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
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%
Nausea
33%
Headache
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
2020
Completed Phase 2
~380
Find a Location
Who is running the clinical trial?
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical CenterLead Sponsor
101 Previous Clinical Trials
46,266 Total Patients Enrolled
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Are there any available positions for patients in this ongoing clinical trial?
"According to clinicaltrials.gov, this study is currently not accepting new participants. The trial was first listed on 5/1/2024 and last updated on 3/20/2024. While recruitment is paused for this particular trial, there are a total of 1523 other trials actively seeking volunteers."
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