Effect of CANnabidiol on Anxiety and GABAergic Function in Individuals with Fragile-X Syndrome

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

NOT_YET_RECRUITING

Clinical Phase

PHASE2

Total Enrollment

40 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-05-01

Study Completion Date

2027-12-01

Brief Summary

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This study 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). Most individuals with FXS have moderate to severe intellectual disability (ID), and caregivers are mainly concerned about aggressive behavior and anxiety problems. Since FXS individuals have a normal lifespan, the overall lifetime cost for the Canadian society of a single case is estimated at $1.2 to $4.7 millions reaching $18 billions for all FXS cases. There is no cure for FXS, as all clinical trials so far have been unsuccessful.FXS is caused by transcriptional silencing of the Fragile X mental retardation protein (FMR1) gene, making FXS a simple model to study ASD and ID pathophysiological mechanisms. Of those, neuronal hyperexcitability is largely recognized as a core deficit in FXS, and a critical therapeutic target for the disorder. Using transcranial magnetic stimulation (TMS) in FXS patients, our team provided the first direct evidence of Gamma-aminobutyric acid (GABA) receptor a (GABAa) dysfunctions in humans with this disorder and showed that this inhibitory deficit is linked with cortical hyperexcitability (PMID: 31748507). Concurrent lines of evidence suggest that stimulation of the endocannabinoid (eCB) system with the administration of Cannabidiol (CBD) could upregulate GABAergic function and correct inhibitory deficits presumed responsible for the neuropsychiatric phenotype of FXS. CBD has been shown to increase GABA concentration levels in the brains of healthy individuals, an effect that could help correct the hyperexcitability typically found in FXS. Thus, this trial aims to define the therapeutic potential of the eCB system for FXS, by measuring the impacts of oral CBD administration on the principal inhibitory neurotransmitter system of FXS patients, and the severity of the clinical phenotype.

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Detailed Description

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Conditions

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Fragile X Syndrome

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

Randomized, Double-blind, placebo-controlled, single center, cross-over trial

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

The randomization and participant code allocation process will be carried out by the Centre de recherche du Centre hospitalier universitaire de Sherbrooke (CRCHUS) pharmacy. The pharmacy will dispense the medication or placebo based on the randomization. The principal investigators, Drs. Lepage and Corbin and the research, as well as the participants and their caregivers, will never be aware of the randomization. To avoid any suspicions on the part of the investigators, Dr. Artuela Çaku, who is not part of the research team, will have access to the laboratory results. In the event of an adverse drug effect, Dr. Çaku may break the code to make a more informed decision for the patient's well-being, such as discontinuing participation or reducing the dose, etc.

Study Groups

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CBD First

Participants will start with CBD to stimulate the eCB for 12 weeks, undergo an 8-week washout period, and then receive a 12-week placebo.

Group Type EXPERIMENTAL

CBD Oral Solution

Intervention Type DRUG

Participants will start with oral CBD dose of 5 mg/kg/day for two weeks and then increase to 10 mg/kg/day.

Placebo

Intervention Type DRUG

Participants will receive a dose of a placebo composed of the inactive ingredients of CBD of the same volume as the CBD Oral Solution.

Placebo First

Participants will start with a placebo for 12 weeks, undergo an 8-week washout period, and then receive a 12-week CBD to stimulate the eCB system.

Group Type EXPERIMENTAL

CBD Oral Solution

Intervention Type DRUG

Participants will start with oral CBD dose of 5 mg/kg/day for two weeks and then increase to 10 mg/kg/day.

Placebo

Intervention Type DRUG

Participants will receive a dose of a placebo composed of the inactive ingredients of CBD of the same volume as the CBD Oral Solution.

Interventions

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CBD Oral Solution

Participants will start with oral CBD dose of 5 mg/kg/day for two weeks and then increase to 10 mg/kg/day.

Intervention Type DRUG

Placebo

Participants will receive a dose of a placebo composed of the inactive ingredients of CBD of the same volume as the CBD Oral Solution.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Molecular diagnosis of FXS
* Age 7 to 40 inclusively
* Overall ABC-C score \> 20
* Taking up to 3 psychoactive drugs
* No therapeutic change for the last 3 months

Exclusion Criteria

* Taking valproic acid
* Taking clobazam
* History of liver problems
* aspartate aminotransferase (AST) or alanine transaminase (ALT), \> 3 times the reference values
* Bilirubin \> 2 times the reference values
* Absolute contraindication to the use of TMS and MRI (e.g. presence of metal in the body), will also be considered as an exclusion criterion.

Minimum Eligible Age

7 Years

Maximum Eligible Age

40 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No