Oklahoma clinic tests experimental CBD gel
TULSA — An Oklahoma clinic is one of many sites testing an experimental cannabidiol (CBD) gel for treatment of common behaviors in patients with Fragile X Syndrome.
FXS is a genetic condition that causes intellectual disability, behavioral and learning challenges, and is the most common genetic cause of autism. Like many other rare diseases, there is no FDA-approved drug treatment option.
"This study interested us especially with Oklahoma's (medical marijuana) law and people's interest in CBD products," psychiatrist Sarah Land said. "We have a lot of hope for this drug."
Land, medical director of Central States Research in Tulsa, is participating in the clinical trial CONNECT-FX. The study is evaluating a lab-manufactured CBD gel that is applied to the skin and absorbed into the bloodstream to treat some of the common behaviors in patients with FXS.
That is a good way to administer a drug for the test population, which has problems swallowing pills, Land said. She stressed the trial drug does not contain THC, the psychoactive compound in marijuana that gives the high sensation.
The CONNECT-FX trial will include 204 FXS patients ages 3 to 17 years at about 20 clinical sites in the United States, Australia and New Zealand.
One male patient, age 10, currently is enrolled at the Tulsa clinic and four others are scheduled for screenings, Land said.
She won't know which patients are treated with the experimental drug and which will receive a placebo, but after the 12-week trial all those receiving the placebo will be offered a full year of the drug at no cost.
"The hope is that problematic behaviors and symptoms can be alleviated and improved for generally happier children. A big issue is social avoidance, and this could possibly help with that," Land said.
"It is awesome to be on the forefront of this."
Parents of children ages 3 to 17 who have been diagnosed with FXS can learn more at www.centralstatesresearch.com or by calling 918-645-5400.
Fragile X Syndrome
Fragile X syndrome is an inherited condition caused by a change in the genetic or inherited material found in every cell of the body. People with FXS may experience a range of behavioral and health challenges. These can include anxiety, depression, autism, and difficulty with sensory issues, such as receiving and responding to what a person sees, hears, smells, tastes, and touches. Ear infections, difficulty sleeping, seizures, and stomach or intestinal problems also may occur.
FXS is the most common known inherited cause of intellectual disability. Males with FXS usually have some level of intellectual disability, ranging from mild to severe. Females can have a range of symptoms from normal intellectual ability to moderate intellectual disability.
Source: Centers for Disease Control and Prevention