Zynerba Pharmaceuticals has recently announced that they have finished the patient screening for their clinical trial of Zygel, a treatment for Fragile X syndrome. Zygel is a CBD gel that will be studied in the CONNECT-FX trial, and it is expected to enroll at least 204 patients. Results should be released in the second quarter of 2020.
About Fragile X Syndrome (FXS)
Fragile X syndrome (FXS) is a developmental disability that is characterized by learning disabilities and cognitive impairment. It typically effects males more severely than females, as it is an X-linked disorder. It is caused by a mutation on the FMR1 gene on the X chromosome, which is responsible for the fragile x mental retardation 1 protein. A lack of this protein results in the symptoms of FXS.
Symptoms are noticed when infants begin to miss developmental milestones. Severity varies across affected individuals, and milder cases may not notice symptoms until later in life. Symptoms include developmental delays, stuttering, learning or intellectual disabilities, social and emotional issues, and problems with language and speech. Distinct facial features are another symptom of this disorder, such as a large head, a protruding chin, forehead, and ears, flat feet, and an elongated face.
A genetic blood test diagnoses this condition, called the FMR1 DNA test. After a diagnosis is obtained, treatment is symptomatic. Specialists can help children learn language and social skills. Medications may be prescribed, such as Ritalin, Catapres, and selective serotonin reuptake inhibitors.
Zygel is a pharmaceutically-manufactured CBD clear gel, and it is the only one of its kind. It delivers the drug to the bloodstream through the skin. This treatment comes from research that shows CBD can modulate the EC system and treat social and behavioral symptoms, such as social avoidance and anxiety.
CONNECT-FX is a 14 week, multi-national randomized, placebo-controlled, double-blind trial. It is meant to evaluate safety and efficacy for the treatment of children and adolescents. At least 204 participants will be enrolled in the trial, and they will come from across the United States, Australia, and New Zealand. Participants will take either a placebo or Zygel in a 1:1 ratio.
Results of the study will be released in the second quarter of 2020. Depending on the results of this phase, Zynerba Pharmaceuticals will send in a New Drug Application. Medical professionals hope that Zygel will be able to better the lives of those with FXS.
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