Rare Daily Staff

Researchers said results from Zynerba Pharmaceuticals open-label trial indicate that the company’s experimental cannabidiol therapy for Fragile X syndrome may be an effective treatment for many behavioral and emotional symptoms associated with FXS.

The study’s findings, published in the August 2 online edition of the Journal of Neurodevelopmental Disorders, are limited by the open label design and small sample size. These data were recently presented at the 2019 Annual Meeting of the American Psychiatric Association.

The trial evaluated ZYN002 cannabidiol (CBD) gel in pediatric and adolescent patients with Fragile X syndrome (FXS).

“Fragile X is a family diagnosis, impacting not only the diagnosed child but also the entire family unit,” said Honey Heussler, associate professor, University of Queensland and medical director of child development, Children’s Health Queensland, and an investigator in the study. “Children with Fragile X syndrome exhibit a number of developmental and behavioral symptoms including anxiety, social avoidance, hyperactivity, and socially unresponsive behaviors that significantly impact the family, and the child’s capacity to interact with them, their peers, and care providers.”

She said if they can successfully treat these symptoms, they can enhance the child’s ability to engage and interact.

The open-label study enrolled 20 male and female pediatric patients with FXS, six through 17 years of age at screening, with a diagnosis of FXS confirmed through molecular documentation of FMR1 full mutation. Eligible patients were provided 12 weeks of ZYN002 as an adjunct to their existing treatments. Safety and tolerability were assessed bi-weekly through various tests and patients were monitored for adverse events.

Results from the efficacy analyses suggest a pattern of clinical improvement in a range of key parent and clinician rated emotional and behavioral symptoms of FXS, which included improvement over baseline scores for the majority of study efficacy endpoints, including anxiety, and social avoidance. Basically, improvements were generally greater than those demonstrated for placebo in prior controlled clinical trials in FXS. ZYN002 was well tolerated in this study and no serious adverse events were reported.

Enrollment is ongoing in CONNECT-FX, a multi-national, randomized, double blind placebo controlled pivotal clinical trial of Zygel (ZYN002) in FXS. Zygel has been designated a Fast Track development program by the U.S. Food and Drug Administration for treatment of behavioral symptoms of FXS.

Photo: Honey Heussler, associate professor, University of Queensland and medical director of child development, Children’s Health Queensland with a child from the study

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