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New treatment option for Sanfilippo Syndrome
Official title Study of Cannabidiol in Sanfilippo Syndrome
ClinicalTrials.gov ID: NCT06333041
What this study is testing
What is Epidiolex?
Epidiolex is an investigational medicine, given as a pill taken by mouth, being studied as a potential treatment for sanfilippo syndrome.
Also referred to as Cannabidiol; CBD.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 3: a large, late-stage study
- You might receive a placebo (an inactive treatment) instead of the study drug, decided by chance. You may not know which one you got.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 4 and older
You may be able to join if
- 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...
- If taking any of the following, no dose changes for the last 8 weeks:
- Anakinra
You likely can't join if
- Equitable selection will be used when choosing participants. An individual who meets any of the following criteria will be excluded from...
- 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
See the full eligibility 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:
- Anakinra
- Fluoxetine
- Probiotic supplement
- One of the following criteria are met:
- Previous participation in a gene/cell therapy or enzyme restorative clinical trial.
- Previously ineligible to participate in a gene/cell therapy or enzyme restorative clinical trial due to advanced disease state.
- Functional age, as measured by the Vineland, is ≤ 0.5 chronological age
- 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
The study team makes the final eligibility decision.
Where it's taking place
- Torrance, California, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 4 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Torrance, California, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.