Menu
Not yet recruiting NCT06261450

Effect of CBD on the Brain

Phase II Interventional Fragile X Syndrome

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: CBD Oral Solution (eCBD system Target), Placebo.
Who it may be relevant to
Registry conditions: Fragile X Syndrome. Basic parameters: 7 years — 55 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Center list to be confirmed — check the primary protocol.
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

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

Overview

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.

Interventions

  • Drug 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 Placebo
    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 outcome measures

  • Short Intracortical Inhibition [Time frame: Comparison between pre and 2 hours post administration of Oral CBD solution and placebo]
Secondary outcome measures (2)
  • 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]

Eligibility criteria

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,

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: Yes

Study design

Allocation
Randomized
Model
Crossover
Masking
Triple blind
Primary purpose
Basic science

Study locations

Center list to be confirmed — check the primary protocol.

Identifiers

NCT: NCT06261450 · 2020-3424 · 236114 · 202010PJT-451514

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗