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NCT Number: NCT06261450

Effect of CBD on the Brain

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.

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Key information

Conditions

Fragile X Syndrome Chromosome Disorders Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Heredodegenerative Disorders, Nervous System Intellectual Disability Nervous System Diseases Neurobehavioral Manifestations Neurologic Manifestations Sex Chromosome Disorders X-Linked Intellectual Disability

Age range

7 year–55 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

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:

  • 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).

Exclusion criteria

  • 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,

Treatment and study plan

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.

Placebo

Drug

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)

Primary outcomes

  1. 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)

Secondary outcomes

  1. Intracortical Facilitation

    Time frame: Comparison between pre and 2 hours post administration of Oral CBD solution and placebo

    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).

  2. Gaba concentration levels

    Time frame: Comparison between pre and 2 hours post administration of Oral CBD solution and placebo

    Estimation of GABA concentrations in the brain from magnetic resonance spectroscopy (MRS)

Study contacts

Contact information is provided by the study sponsor or research team.

François Corbin, MD, Ph.D.

CONTACT

[email protected]

819-346-1110 ext. 15801

Samantha Cote

CONTACT

[email protected]

819-346-1110 ext. 70184

Sponsors and collaborators

Lead sponsor

Université de Sherbrooke

Other

Collaborators

  • Canadian Institutes of Health Research (CIHR)
  • Centre de recherche du Centre hospitalier universitaire de Sherbrooke
  • Jazz Pharmaceuticals

Registry information

Official study title

Effect of CBD on the GABAergic System in Patients with Fragile X Syndrome.

Important dates

Study start
2025
Primary completion
2027
Study completion
2027
First posted
Feb 15, 2024
Registry last updated
Feb 17, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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