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Effectiveness of CBD on behavioural problems in patients with TSC, FXS and Sanfilippo
EU CTIS ID: 2025-520846-31-00
What this study is testing
To investigate the effectiveness of CBD on irritability and other behavioural problems in children and adults with TSC, Sanfilippo and FXS
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Subjects must be at least 6 years old and have a clinically confirmed diagnosis of FXS, TSC or Sanfilippo syndrome. Participants must also exhibit severe behavioral manifestations, defined as a minimum score of 4 on the Clinical Global Impressions (CGI) scale. All medications or interventions for epilepsy and behavioral symptoms must have been stable for at least one month prior to the screening, and participants must be willing to maintain this regimen throughout the duration of the study. Finally, a consistently available patient caregiver is required to provide proxy reports.
- Minimum age of 6 years old
- Clinically definite diagnosis of TSC, Sanfilippo syndrome or FXS (modified Gomez criteria or positive genetic test (such as FISH test, microarray, WES-analysis))
- Suffering from severe behavioural manifestation with a minimum score of 4 on the CGI scale
- All medications or interventions for epilepsy and behavioural manifestations must have been stable dosed for one month prior to screening and the participant is willing to maintain the current regimen throughout the trial
- Presence of a consistently available patient caregiver for proxy-reports
You likely can't join if
- Use of valproate should be stable three months prior to enrolment
- Subjects who have had changes in non-exclusionary psychotropic medications within 4 weeks of initiation of trial
- Any planned major surgery within the duration of the trial
- Expected inability to take blood samples due to anxiety or resistance
- Unable to swallow the study drug (or placebo)
- The patient is unwilling or, in the investigator’s opinion, unable to adhere to the requirements of the study
See the full eligibility criteria
Who can join
- Subjects must be at least 6 years old and have a clinically confirmed diagnosis of FXS, TSC or Sanfilippo syndrome. Participants must also exhibit severe behavioral manifestations, defined as a minimum score of 4 on the Clinical Global Impressions (CGI) scale. All medications or interventions for epilepsy and behavioral symptoms must have been stable for at least one month prior to the screening, and participants must be willing to maintain this regimen throughout the duration of the study. Finally, a consistently available patient caregiver is required to provide proxy reports.
- Minimum age of 6 years old
- Clinically definite diagnosis of TSC, Sanfilippo syndrome or FXS (modified Gomez criteria or positive genetic test (such as FISH test, microarray, WES-analysis))
- Suffering from severe behavioural manifestation with a minimum score of 4 on the CGI scale
- All medications or interventions for epilepsy and behavioural manifestations must have been stable dosed for one month prior to screening and the participant is willing to maintain the current regimen throughout the trial
- Presence of a consistently available patient caregiver for proxy-reports
What rules you out
- Use of valproate should be stable three months prior to enrolment
- Subjects who have had changes in non-exclusionary psychotropic medications within 4 weeks of initiation of trial
- Any planned major surgery within the duration of the trial
- Expected inability to take blood samples due to anxiety or resistance
- Unable to swallow the study drug (or placebo)
- The patient is unwilling or, in the investigator’s opinion, unable to adhere to the requirements of the study
- Any condition or abnormality which may, in opinion of the investigator, compromise the safety of patients
- Use of any interfering medication within 30 days prior to enrolment or planning to take in-terfering medication during the trial
- Any known or suspected hypersensitivity to cannabinoids or any of the excipients of the Investigational Medicinal Product (IMP), such as sesame oil
- Participant is currently using or has in the past used recreational or medicinal cannabis, or cannabinoid-based medications, within three months prior to screening and is unwilling to abstain for the duration of the study
- Treatment with CBD or other cannabinoid within the previous two months
- History or current evidence of significantly impaired liver function, defined as 1) Alanine aminotransferase (ALT) and / or aspartate aminotransferase (AST) > 5 × upper limit of nor-mal (ULN); 2) ALT or AST > 3 × ULN with concomitant total bilirubin > 2.0 × ULN; or 3) ALT or AST ≥ 3 × ULN with the appearance of fatigue, nausea, vomiting, right upper quadrant pain or tenderness, fever, rash, and/or eosinophilia
- Pregnancy or breastfeeding
- Glaucoma
- Participant has undergone general anaesthesia in the four weeks prior to randomization
- Planned changes in use of anti-epileptics
The study team makes the final eligibility decision.
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, 0-17 years, 18-64 years. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.