This study will evaluate the efficacy and safety of ZYN002, a clear cannabidiol (CBD) gel that can be applied to the skin (called transdermal application) twice a day for the treatment of behavioral symptoms of Fragile X syndrome (FXS). Eligible participants will then participate in up to a 14-week treatment period, where all participants will receive placebo or active study drug. Patients ages 3 to < 18 years, will be eligible to participate.
ZYN002, is the first and only pharmaceutically-produced CBD, a non-psychoactive cannabinoid, formulated as a patent-protected permeation-enhanced gel for transdermal delivery through the skin and into the circulatory system. ZYN002 is being developed for patients suffering from Fragile X syndrome and certain refractory epilepsies including DEEs. The U.S. Food and Drug Administration has granted Orphan Drug designation to Zynerba for the use of CBD as treatment of patients with FXS.
About the Study
Eligible participants will be randomized 1:1 to either trial drug or placebo and will undergo a 14-week treatment period. Randomization will be stratified by gender, weight category, and geographic region. All participants may receive placebo during the trial.
The assignment will be done by a computer-generated system and neither the study doctor or the participant or their caregivers will know which treatment is being given to them. The dose of the treatment will depend on the weight of the participants. If the participants weigh less than or equal to 35 kg [approximately 77 lbs.], they will receive 2 sachets of the gel twice a day (1 sachet approximately every 12 hours) and if they weigh more than 35 kg, they will receive 4 sachets of gel per day (2 sachets approximately every 12 hours).
These types of placebo-controlled, double blind studies are important to help investigators establish the benefits and risks of treatment and to allow government regulatory bodies, such as the U.S. Food and Drug Administration (FDA) and other country regulatory agencies, to determine if a drug should or should not be approved for marketing and made available to patients.
Parents/caregivers will be instructed on proper application of the gel. The gel will be applied to clean, dry, intact skin of the upper arms/ shoulders.
Blood samples will be collected for safety analysis of ZYN002. An independent analytical laboratory will also perform CGG repeat and methylation status analyses. Additionally, the parents/caregivers will be asked to complete some questionnaires. There will be other questionnaires and scales that will be completed at the site by the study doctor.
After the final dose, patients will be followed weekly for four weeks by telephone, prior to discharge from the study.
All participants in this study, whether on study drug or placebo, may have an opportunity to enroll in a one-year follow-on study, in which everyone will receive active study drug, study-related medical exams and reimbursement of reasonable study-related travel.
If you’d like to learn more or apply for this opportunity, please fill out the form to send your contact information to the study coordinators.
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