Zynerba Pharmaceuticals has commenced the CONNECT-FX trial, a pivotal clinical trial designed to examine the efficacy and safety of ZYN002 to treat patients with Fragile X syndrome (FXS).

The multi-national, randomised, double-blind placebo-controlled clinical study of Cannabidiol (CBD) in Children and Adolescents with Fragile X (CONNECT-FX) aims to enrol around 200 FXS patients aged between three to 17 years.

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The trial will enrol the FXS patients, who have the full mutation of the FMR1 gene, at nearly 20 clinical sites in the US, Australia, and New Zealand.

During the trial, the patients will be randomised in 1:1 ratio to receive either ZYN002 or placebo.

The primary endpoint of the CONNECT-FX trial is the change from baseline to the end of the treatment period in the Aberrant Behaviour Checklist-Community FXS Specific (ABC-CFXS) Social Avoidance subscale.

“We look forward to demonstrating the clinical effects of ZYN002 in treating some of the most common behavioural symptoms of Fragile X syndrome.”

The trial’s key secondary endpoints include the change from baseline to the end of the treatment period in the ABC-CFXS Irritability subscale score, the ABC-CFXS Socially Unresponsive/Lethargic subscale score, and improvement in Clinical Global Impression – Improvement (CGI-I) following the completion of the treatment period.

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Top line results from the trial are expected in the second half of next year.

Zynerba Pharmaceuticals chairman and CEO Armando Anido said: “We look forward to demonstrating the clinical effects of ZYN002 in treating some of the most common behavioural symptoms of Fragile X syndrome.

“If successful, ZYN002 has the potential to become the first product indicated for the treatment of behavioural symptoms of Fragile X syndrome and help address the ongoing needs of the children and families impacted by this syndrome.”

XS is a rare genetic condition that results in intellectual disability, behavioural and learning challenges.

It is also the most common known single gene cause of autism spectrum disorder.