Zynerba has announced it is now recruiting subjects for a large-scale Phase 3 clinical trial (RECONNECT) of Zygel.
Twenty-five sites across the United States, Australia, the UK and Ireland are recruiting children and adolescents with Fragile X syndrome for this randomized, double-blind, placebo-controlled, multinational study, details below.
Two hundred subjects with Fragile X syndrome aged 3 to 17 will enter an 18-week trial to assess the efficacy and safety of Zygel. Zygel is a pharmaceutically manufactured CBD, formulated as a clear gel that can be applied to the skin (called transdermal delivery).
About RECONNECT Phase 3 Fragile X Clinical Trial
The study will consist of a screening visit and a combination of seven visits both onsite (face-to-face) and virtual. The assignment of study drug or placebo will be done by a computer generated system and neither the study doctor, participant or their caregivers will know which treatment is being given to them. Parents/caregivers will be instructed on proper application of the gel tol be applied to clean, dry, intact skin of the upper arms/ shoulders.
Blood samples will be collected for safety analysis of the gel. Additionally, the parents/caregivers and study doctor will be asked to complete some questionnaires for efficacy and safety assessment.
The primary endpoint for the trial will be the change from baseline to the end of the treatment period in the Aberrant Behavior Checklist-Community FXS Specific (ABC-CFXS) Social Avoidance subscale in patients who have complete methylation of their FMR1 gene. The ABC-CFXS Social Avoidance subscale is the same primary endpoint used in the CONNECT-FX trial.
Hope for the Future
Armando Anido, Chairman and Chief Executive Officer of Zynerba stated “If successful, we believe the study could serve as the basis for Zygel approval in the US for patients with FXS.” in a press release on September 13, 2021.
FRAXA Research Foundation funded teams in France, Spain, and the US, and is funding three new teams this year, to understand the endocannabinoid system in Fragile X syndrome. These studies have paved the way to this Fragile X clinical trial.
Topline results for the RECONNECT trial are expected in the second half of 2023.