Study of Cannabidiol in Sanfilippo Syndrome
Trial statusRecruiting
Trial phasePhase 2, Phase 3
Trial typeInterventional
Biological sexAll
Age4+
SponsorLundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
About this trial
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
This trial does not accept healthy volunteersQualifiers
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.
Anakinra
Disqualifiers
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
Rifampin
Trial design
Design model
Crossover
Treatments tested in this trial
Epidiolex
DrugEpidiolex (cannabidiol) oral solution is a clear, colorless to yellow liquid containing cannabidiol at a concentration of 100 mg/mL. Inactive ingredients include dehydrated alcohol (7.9% w/v), sesame seed oil, strawberry flavor, and sucralose.
Placebo
DrugPlacebo oral solution is a yellow oily solution containing the excipients sesame oil and anhydrous ethanol with added sweetener (sucralose) and strawberry flavoring
Treatment groups
35 Participants
are divided into 2 treatment groupsGroup A: Epidiolex (cannabidiol)Experimental treatment 1 intervention
Group B: Placebo (PBO)Placebo comparator 1 intervention
Trial outcomes
Primary outcomes
1
Sanfilippo Behavior Rating Scale (SBRS)- mood/anger/aggression score
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 0-6 and a total mean score is calculated. The mean mood/anger/aggression score is standardized using the mean and standard deviation from a cohort of Sanfilippo patients, categorized as "slow progressors," ages 81-220 months. This reference cohort was chosen to best match the age distribution of our participants.
Time frame
Changes over 16 weeks of treatment versus 16 weeks of placeboSponsors and contacts
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Source ClinicalTrials.gov