Ignite Creation Date:
2024-05-06 @ 8:19 PM
Last Modification Date:
2024-10-26 @ 3:25 PM
Study NCT ID:
NCT06333041
Status:
NOT_YET_RECRUITING
Last Update Posted:
2024-04-19
First Post:
2024-03-20
Brief Title:
Study of Cannabidiol in Sanfilippo Syndrome
Sponsor:
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Organization:
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Study Overview
Official Title:
Randomized Double-Blind Placebo-Controlled Cross-Over Study to Evaluate the Effects of Cannabidiol on Neurobehavioral and Function Outcomes in Sanfilippo Syndrome
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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 participants caregiver will be asked to complete surveys related to the participants 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 11 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
Each participants caregiver will be asked to complete surveys related to the participants 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 11 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
Detailed Description:
Sanfilippo syndrome or Mucopolysaccharidosis type III MPS III is a rare genetic lysosomal storage disease characterized by the accumulation of heparan sulfate due to insufficient production of lysosomal enzymes Consequently a buildup of heparan sulfate causes progressive neurodegeneration leading to significant neurobehavioral problems These neurobehavioral symptoms are highly disruptive and distressing to families have a significant impact on the quality of life of the patients and their families and likely interfere with adjunctive therapeutic attempts at supporting the child To date there are no approved therapies for the treatment of the neurobehavioral symptoms of Sanfilippo syndrome Cannabidiol CBD a compound derived from the cannabis plant but without the psychoactive effects has been shown to be safe and effective in the treatment of children with severe epilepsy disorders Studies have shown that CBD improves behavior in children with autism This study will use Epidiolex a pharmaceutical-grade purified oral solution of cannabidiol that was approved by the FDA for the treatment of severe pediatric epilepsy disorders Study subjects will include approximately 35 participants with Sanfilippo syndrome Participants will undergo baseline clinical evaluations and neurobehavioral and functional outcomes will be collected from parent-reported questionnaires Participants will then receive either Epidiolex or placebo for 16 weeks Following this period and an 8-week washout participants will cross over and receive the opposite treatment for 16 weeks Safety labs and questionnaires will be collected throughout these periods to assess safety and efficacy Participants and study personnel will be blinded to the treatment status of each participant When this blinded portion of the trial is completed all participants will receive Epidiolex open-label for 52 weeks to measure long-term safety The type and severity of adverse events will be collected to measure safety and different behavioral and functional outcomes will be collected to measure efficacy
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None