This study will evaluate the effectiveness and safety of ZYN002, a clear cannabidiol gel applied to the skin (called transdermal application) twice a day for the treatment of behavioral symptoms of Fragile X syndrome (FXS). Eligible participants will participate in up to an 18-week study treatment period, where all participants will receive placebo or active study drug. Patients ages 3 to 22 years, will be eligible to participate if they meet all entry criteria.
ZYN002, is the first and only investigational pharmaceutically-produced cannabidiol, 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, 22Q deletion syndrome, and autism spectrum disorder (ASD). 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 study treatment.
About the Study
Eligible participants will be randomized 1:1 to either active study drug or placebo and will undergo an 18-week treatment period. All participants may receive placebo during the trial.
The assignment to active study drug or placebo will be done by a computer-generated system and neither the study doctor nor the participant or their caregivers will know which treatment is being given to them. The dose of the study treatment will depend on the weight of the participants and the study drug will be applied twice a day (approximately every 12 hours). 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.
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 FDA and other country regulatory agencies, to determine if a drug should or should not be approved for marketing and made available to patients.
Blood samples (at the beginning, middle [only participants on certain medications], and end of the study) 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.
At the end of the 18-week treatment period, 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 a participant does not enroll in the follow-on study AND the patient is on medications for seizures, the study drug may need to be discontinued over a one to three-week period depending upon the dose. Four weeks after the final dose, these patients will complete a follow up visit via the telephone prior to discharge from the study.
Why are we doing this research?
Zynerba Pharmaceuticals is conducting a clinical trial to learn about effectiveness and safety of ZYN002, an investigational pharmaceutically produced cannabidiol transdermal gel. The trial is being conducted to confirm previous results from the CONNECT-FX study and will provide data to be reviewed by the FDA to determine the potential for approval to market the medicine for treatment of behaviors of FXS.
Who can participate?
Males and females, age 3 to 22 years of age with full mutation FXS may be eligible to participate.
What will happen in the study?
If the individual qualifies and decides to be in this research study, they will have study visits in person at a study site and virtually with the site staff over the course of eight visits conducted over approximately 20 weeks. Four of the visits will be in person (Visits 1, 2, 3 and 8) and four of the visits will be virtual (Visits 4, 5, 6 and 7). Individuals will undergo screening procedures to ensure eligibility at Visit 1 and then come back within two to three weeks for Visit 2 at which time they will receive blinded study medication.
Some of the initial assessment to determine eligibility for the trial may now be done remotely after discussion with the clinical site. If eligibility is met on these initial remote assessments, an in-person clinic visit will be scheduled to complete the remaining screening assessments.
The following is a list of some of the study procedures that will happen during the study:
- Blood samples for genetic testing to confirm diagnosis of full mutation FXS and methylation status
- Blood samples and electrocardiogram (ECG) for safety monitoring
- Parent/caregiver questionnaires to assess the severity of symptoms of FXS
What are the good things that can happen from this research?
Caregivers and their child will receive medical care associated with the conduct of the study at no charge.
Their participation will provide important information to help determine whether the study medication is safe and effective in FXS. This information will be reviewed by the FDA. If the study medication is deemed safe and effective, the medication may become available for use by other individuals with FXS.
What are the bad things that can happen from this research?
Overall the active study medication has been well tolerated in previous studies in individuals with FXS. The most common adverse events are application site reactions such as pain or redness. Individuals may also experience discomfort related to the procedure to take blood samples.
There may be other risks that we do not know about yet.
Will I/my child be paid to be in this research study?
Participants will not receive payment for participation in the study. Participants may receive reimbursement for reasonable study-related costs such as travel and incidental expenses (parking, meals).
To find out more, call (833) 680-1155 or visit FRAGILEXHELP.com