Zynerba Pharmaceuticals, Inc. has announced their plans to begin a clinical trial of Cannabidiol in children and teenagers who have Fragile X syndrome. The study, called CONNECT-FX, is expected to produce top-line results in the second half of 2019. The source article can be found here, at Globe Newswire.
About Fragile X Syndrome
Fragile X is a common cause of inherited intellectual disability and autism. The condition is thought to occur in approximately one in 4,000 males, and one in 4,000 to 8,000 females, although there are varying estimates.
According to the NIH, males are more often severely affected than females. The majority of males and one-third of females develop mild to moderate intellectual disability. Children with fragile X may also have difficulty focusing, features of autism, and seizures.
About the CBD Gel (ZYN002)
The CBD Gel by Zynerba is an experimental treatment for fragile X that is a clear gel that can be absorbed through the skin into the bloodstream (transdermal delivery).
The use of CBD for the treatment of fragile X has been supported in clinical and pre-clinical studies. CBD (cannabidiol) is a type of cannabinoid that occurs in Cannabis. Cannabinoids are a type of compound that affects signalling systems and receptors within the central nervous system. They occur in Cannabis plants, as well as in the human body. CBD is being studied for its potential as a treatment for conditions including epilepsy and fragile X.
The CONNECT-FX Study
The CONNECT-FX trial is a planned study that is designed to enroll around 200 people with fragile X, and occur over 14 weeks. It is a randomised, double-blind study that will take place in several different countries. In it, the safety and efficacy of the CBD gel will be compared to that of a placebo. The main change the study will look for is a difference in the ABC-CFXS Social Avoidance subscale, although other measures will also be tracked.
Top-line results from the planned study are expected to become available in the second half of 2019.