GREENVILLE, SC – The Greenwood Genetic Center (GGC) is pleased to announce it has been selected as one of approximately 20 sites in the United States,
Australia, and New Zealand now enrolling patients in a clinical trial for Fragile X syndrome (FXS). The study will include more than 200 male and female
individuals ages three through 17.
FXS is a genetic condition that causes intellectual disability, behavioral and learning challenges, and is also the most common known single gene cause
of autism spectrum disorder.
The newly-initiated study, CONNECT-FX (Clinical Study of Cannabidiol (CBD) in Children and Adolescents with Fragile X), is a 14-week randomized, double-blind,
placebo-controlled trial to assess the efficacy and safety of an investigational CBD gel (ZYN002) in children with full mutation FXS. CONNECT-FX is
evaluating delivery of the drug through the skin as a potential treatment for some common behavioral symptoms associated with FXS.
“Patients with Fragile X syndrome deal with a number of challenging behaviors including irritability, anxiety, social withdrawal, and aggression that make
home life and social interactions very difficult,” said Carrie Buchanan, MD
, a developmental-behavioral
pediatrician in GGC’s Greenville office and study investigator. “This drug shows great promise in alleviating many of these debilitating behaviors,
thus improving the quality of life and social functioning of both patients and their families.”
Potential patients will undergo a screening visit and, if eligible, will be randomly assigned to either the ZYN002 treatment group or placebo group for
up to 14 weeks. Study visits will occur every two to four weeks and there will be up to six visits for patients and their parent/caregiver. Participants
in the CONNECT-FX trial may have the opportunity to enroll in an open-label extension study, whereby all participants will receive study drug for up
to 12 months.
The Greenwood Genetic Center in Greenville was the first site to enroll patients into this study in the US this fall.
The CONNECT-FX trial is sponsored by Zynerba Pharmaceuticals. Patients who participate may receive reimbursement for study-related costs such as travel
and incidental expenses.
About Fragile X Syndrome
FXS is a rare genetic disability that is the leading known cause of both inherited intellectual disability and autism spectrum disorder, affecting 1 in 3,600 to 4,000 males and 1 in 4,000 to 6,000 females. It is the most common inherited intellectual disability in males and a significant cause of intellectual disability in females. It is caused by a mutation in the Fragile X syndrome gene (FMR1) located on the X chromosome and leads to dysregulation of the endocannabinoid pathway and a reduction in the endogenous cannabinoid 2-AG. The disorder negatively affects synaptic function, plasticity and neuronal connections, and results in a spectrum of intellectual disabilities and behavioral symptoms. In the US, there are about 71,000 patients suffering with FXS.
Zynerba’s ZYN002 CBD gel is a pharmaceutically-produced CBD formulated as a permeation-enhanced transdermal gel and is being studied in children and adolescents with FXS. ZYN002 is an experimental treatment. This means that it is not approved by government regulatory bodies, including the United States Food and Drug Administration (FDA) and must be tested to see if it is an effective and safe treatment.