Fragile X SyndromePhase 2NCT06261450
Effect of CBD on the Brain
A Phase 2 study testing CBD Oral Solution (eCBD system Target) in people with Fragile X Syndrome, run by Université de Sherbrooke.
Trial summary
This proposal focuses on the therapeutic relevance of the endocannabinoid (eCB) system for the treatment of Fragile-X syndrome (FXS), the primary hereditary cause of autism spectrum disorder (ASD). Read more about this study
- Who is it for?
- Ages 7 to 55 yearsHealthy volunteers can take part
- Conditions the study is for
- Fragile X Syndrome
- What is being tested?
- CBD Oral Solution (eCBD system Target)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 50 participants
- Where
- No sites listed yet
- Study dates
- 1 May 2025to 15 Dec 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 7 to 55 years
- All sexes
- Healthy volunteers accepted
- Child, Adult
Who may be able to join
- Eligibility criteria for FXS participants will include:
- age between 7 and 55 years, molecular diagnosis of FXS,
- intelligence quotient (IQ) <70,
- aberrant behavior questionnaire score (ABC-C) > 20,
- <3 psychoactive drugs, drug stable for > 3 months.
- Eligibility criteria for the control group:
Show all 11Show fewer
- 18 and 55 years old,
- be in good general health, with no history of neurological or psychiatric disorders.
- Eligibility Criteria for all Participants:
- A minimum weight of 60 kg;
- no history of liver problems (A complete blood profile to measure liver enzyme levels (bilirubin, aspartate aminotransferase (AST), argininosuccinate lyase (ASL), alanine transaminase (ALT), alkaline phosphatase (ALP), gamma-glutamyl transferase (GGT)) will be obtained before randomization for all participants).
Who cannot join
- The presence of an absolute contraindication to the use of TMS and MRI / MRS (ie presence of metal in the head).
- Individuals with ALT / ASL levels greater than 3 times the upper normal baseline, or if bilirubin exceeds 2 times the upper baseline,
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Effect of CBD on the GABAergic System in Patients with Fragile X Syndrome.
This proposal focuses on the therapeutic relevance of the endocannabinoid (eCB) system for the treatment of Fragile-X syndrome (FXS), the primary hereditary cause of autism spectrum disorder (ASD). Although most individuals with FXS have moderate to severe intellectual disability (ID), caregivers are mainly concerned about aggressive behavior and anxiety problems, hallmark features of the condition. Concurrent lines of evidence suggest that targeting the endocannabinoid (eCB) system by administration of cannabidiol (CBD) could upregulate GABAergic functions and correct inhibitory deficits presumed responsible for the neuropsychiatric phenotype of FXS. However, the eCB system and its effect on the brain remains unexplored in FXS patients. This clinical trial aims to define the therapeutic relevance of the eCB system for FXS using a multimodal neuroimaging approach to finely characterize the acute effects of oral CBD on the principal inhibitory neurotransmitter system (GABA) in a large cohort of FXS patients.
- Conditions
- Fragile X Syndrome
- Being tested
- CBD Oral Solution (eCBD system Target), Placebo
- Keywords
- MRI, Cannabidiol, GABA, FXS, MRS, TMS
- Medical subject headings
- Fragile X Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CBD Oral Solution (eCBD system Target)Drug
Participants receive orally 6 ml of CBD Oral Solution (100 mg / ml; 60 mg / kg; max 600 mg of CBD) followed by 6 ml of a placebo composed of the inactive ingredients of CBD Oral Solution 3 weeks later.
PlaceboDrug
Participants receive orally 6 ml of a placebo composed of the inactive ingredients of CBD Oral Solution followed by 6 ml of Oral CBD Solution (100 mg / ml; 60 mg / kg; max 600 mg of CBD)
| Study group | Role | What they receive |
|---|---|---|
| CBD first | Experimental | A single dose of CBD dose administered followed by a dose of placebo 3 weeks later. Receives: Drug: CBD Oral Solution (eCBD system Target); Drug: Placebo |
| Placebo first | Experimental | A single dose of placebo dose administered followed by a dose of CBD 3 weeks later. Receives: Drug: CBD Oral Solution (eCBD system Target); Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Short Intracortical Inhibition
Transcranial Magnetic Stimulation (TMS)-derived measure of Intracortical inhibition: The degree of decrease of peak-to-peak motor evoked potential (MEP) amplitude induced by the administration of a conditioning stimulus (set at 70% of resting motor threshold) 2-4 ms before the test stimulus (stimulation intensity required to produce an MEP of 1 millivolt (mV), approximately 120% of resting motor threshold)
Measured: Comparison between pre and 2 hours post administration of Oral CBD solution and placebo
Secondary outcomes (2)
Intracortical Facilitation
TMS-derived measure of Intracortical Facilitation: The degree of increase of peak-to-peak motor evoked potential (MEP) amplitude induced by the administration of a conditioning stimulus (set at 80% of resting motor threshold) 12-24 ms before the test stimulus (stimulation intensity required to produce an MEP of 1 mV, approximately 120% of resting motor threshold).
Measured: Comparison between pre and 2 hours post administration of Oral CBD solution and placebo
Gaba concentration levels
Estimation of GABA concentrations in the brain from magnetic resonance spectroscopy (MRS)
Measured: Comparison between pre and 2 hours post administration of Oral CBD solution and placebo
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Feb 2024
- Study start1 May 2025 (estimated)
- Primary completion15 Dec 2027 (estimated)
- Study completion15 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fragile X SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Basic science
- Allocation
- Randomised
- Intervention model
- Crossover
- Model description
- This study is a double-blind-crossover placebo control comparing the acute effect of oral CBD in patients with FXS to controls.
- Masking
- Triple blind
- Who is masked
- Participant, Investigator, Outcomes assessor
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Université de Sherbrooke
- Sponsor type
- University, hospital or other
- Collaborators
- Canadian Institutes of Health Research (CIHR), Jazz Pharmaceuticals, Centre de recherche du Centre hospitalier universitaire de Sherbrooke
- Responsible party
- Principal investigator, Francois Corbin, Université de Sherbrooke
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06261450
- Sponsor's protocol ID
- 2020-3424
- Other IDs
- 236114, 202010PJT-451514
- Status last verified
- Feb 2025
- First submitted
- 26 Jan 2024
- Last updated
- 17 Feb 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effect of CBD on the GABAergic System in Patients with Fragile X Syndrome.
- ClinicalTrials.gov ID
- NCT06261450
- Acronym / sponsor ID
- 2020-3424
- Conditions
- Fragile X Syndrome
- Keywords
- MRI, Cannabidiol, GABA, FXS, MRS, TMS
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Université de SherbrookeUniversity, hospital or other
- Collaborators
- Canadian Institutes of Health Research (CIHR), Jazz Pharmaceuticals, Centre de recherche du Centre hospitalier universitaire de Sherbrooke
- Study start
- 1 May 2025 (estimated)
- Primary completion
- 15 Dec 2027 (estimated)
- Study completion
- 15 Dec 2027 (estimated)
- First posted
- 15 Feb 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This proposal focuses on the therapeutic relevance of the endocannabinoid (eCB) system for the treatment of Fragile-X syndrome (FXS), the primary hereditary cause of autism spectrum disorder (ASD). Although most individuals with FXS have moderate to severe intellectual disability (ID), caregivers are mainly concerned about aggressive behavior and anxiety problems, hallmark features of the condition. Concurrent lines of evidence suggest that targeting the endocannabinoid (eCB) system by administration of cannabidiol (CBD) could upregulate GABAergic functions and correct inhibitory deficits presumed responsible for the neuropsychiatric phenotype of FXS. However, the eCB system and its effect on the brain remains unexplored in FXS patients. This clinical trial aims to define the therapeutic relevance of the eCB system for FXS using a multimodal neuroimaging approach to finely characterize the acute effects of oral CBD on the principal inhibitory neurotransmitter system (GABA) in a large cohort of FXS patients.
Study design
- Primary purpose
- Basic science
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind (participant, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- This study is a double-blind-crossover placebo control comparing the acute effect of oral CBD in patients with FXS to controls.
| Arm | Type | Receives |
|---|---|---|
| CBD first | Experimental | Drug: CBD Oral Solution (eCBD system Target), Drug: PlaceboA single dose of CBD dose administered followed by a dose of placebo 3 weeks later. |
| Placebo first | Experimental | Drug: CBD Oral Solution (eCBD system Target), Drug: PlaceboA single dose of placebo dose administered followed by a dose of CBD 3 weeks later. |
Who may qualify
- Ages
- 7 to 55 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
Key inclusion criteria (11 listed)
- age between 7 and 55 years, molecular diagnosis of FXS,
- intelligence quotient (IQ) <70,
- aberrant behavior questionnaire score (ABC-C) > 20,
- <3 psychoactive drugs, drug stable for > 3 months.
- 18 and 55 years old,
Key exclusion criteria (2 listed)
- The presence of an absolute contraindication to the use of TMS and MRI / MRS (ie presence of metal in the head).
- Individuals with ALT / ASL levels greater than 3 times the upper normal baseline, or if bilirubin exceeds 2 times the upper baseline,
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CBD Oral Solution (eCBD system Target)Participants receive orally 6 ml of CBD Oral Solution (100 mg / ml; 60 mg / kg; max 600 mg of CBD) followed by 6 ml of a placebo composed of the inactive ingredients of CBD Oral Solution 3 weeks later. | Drug | CBD first, Placebo first |
| PlaceboParticipants receive orally 6 ml of a placebo composed of the inactive ingredients of CBD Oral Solution followed by 6 ml of Oral CBD Solution (100 mg / ml; 60 mg / kg; max 600 mg of CBD) | Drug | CBD first, Placebo first |
Primary outcome
Short Intracortical Inhibition
Time frame: Comparison between pre and 2 hours post administration of Oral CBD solution and placebo
Transcranial Magnetic Stimulation (TMS)-derived measure of Intracortical inhibition: The degree of decrease of peak-to-peak motor evoked potential (MEP) amplitude induced by the administration of a conditioning stimulus (set at 70% of resting motor threshold) 2-4 ms before the test stimulus (stimulation intensity required to produce an MEP of 1 millivolt (mV), approximately 120% of resting motor threshold)
2 secondary outcomes
Intracortical Facilitation
Time frame: Comparison between pre and 2 hours post administration of Oral CBD solution and placebo
Gaba concentration levels
Time frame: Comparison between pre and 2 hours post administration of Oral CBD solution and placebo
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Feb 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing CBD Oral Solution (eCBD system Target) in people with Fragile X Syndrome, run by Université de Sherbrooke.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 17 Feb 2025. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact François Corbin, MD, Ph.D. (819-346-1110 ext. 15801, Francois.Corbin@USherbrooke.ca), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Short Intracortical Inhibition (time frame: Comparison between pre and 2 hours post administration of Oral CBD solution and placebo). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Feb 2025.