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.

NCT Number	NCT06261450
Lead Sponsor	Université de Sherbrooke Collaborators: Canadian Institutes of Health Research (CIHR), Jazz Pharmaceuticals, Centre de recherche du Centre hospitalier universitaire de Sherbrooke
Conditions	Fragile X Syndrome
Enrollment	50 participants
Start Date	2025-05-01
Primary Completion	2027-12-15 (estimated)
Study Completion	2027-12-15 (estimated)
Updated on ClinicalTrials.gov	2025-02-17