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 17 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.

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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 17 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.

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).

Contact

To find out more, call (833) 680-1155 or visit FRAGILEXHELP.com

Frequently Asked Questions

No. There will not be a therapy intervention used along with the study medication, but if a patient is undergoing physical, speech, or occupational therapy, they will be able to continue while participating in the study. The study is placebo controlled, meaning that enrolled participants in the trial will be randomized (assigned by chance, not choice) and given either the active study drug or placebo.

Your child may be able to be on other medications; the study doctor will evaluate the different medications that each patient is currently taking, and, based on trial Inclusion and Exclusion criteria, and will decide if your child’s current medication(s) will be allowed during the trial.

There will be behavioral assessments performed by the parent/caregiver throughout the study during each study visit. Your doctor will also perform some assessments. To monitor safety, there will be:

  • Physical and neurological exam at the beginning and a limited physical exam at the end of the study.
  • Blood work two or three times (beginning, middle, end) during the 18-week study. If a patient is on medications for seizures or certain other medicine such as risperidone or aripiprazole, blood draws will be required three times.
  • A 12-lead ECG 2 times during the study at the beginning and end of the study.
  • Plus, several other assessments to ensure that the patient is not experiencing adverse events.
  • The study does not include MRIs or EEGs.

All of this work, as part of the study, will be done at no cost to the participant.

There are eight scheduled visits during the study. The first three visits and the last visit will be in person at the study site.  Visits at weeks 4, 6, 10 and 14 will be virtual video visits.  Some participants may require a additional visit(s) if they do not enroll in the follow-on open label study. The length of time for the visits can vary, depending on the number of assessments/procedures that are conducted but in general the visits may be 1 to 2 hours in length.

Participants who participate may receive reimbursement for reasonable study-related costs such as travel and incidental expenses.

Participants in this study 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.

ZYN002 is an experimental treatment for certain symptoms of Fragile X Syndrome. This means that it is currently not approved by government regulatory bodies, including the FDA. In order to make it available to patients, it must be first tested to see if it is an effective and safe treatment. The purpose of RECONNECT is to evaluate the efficacy and safety of ZYN002 in improving certain behavioral symptoms that patients with FXS exhibit. RECONNECT is enrolling eligible patients to help researchers find a potential treatment for these patients and their families.

Healthy volunteers participate in trials to help others and contribute to moving science forward so doctors and researchers can learn more about a disease and improve health care for others in the future.

Participants with an illness, disease, or condition also participate in trials, like RECONNECT, to help others, but also to possibly receive new, experimental drugs or devices—especially if there are no other treatment options or if all of other treatment options have failed.

No. Please do not post about your experiences on Facebook, and other social media outlets. As this study is a double-blind, placebo-controlled study, we would strongly encourage parents/caregivers not to post either positive or negative experiences on social media. This will help us avoid any type of study bias. We want to do everything possible to have a successful study and potentially bring this product to market.

No. Participants enrolled in this study cannot participate in another trial of another investigational drug simultaneously.

Yes, ZYN002 was studied in a double-blind, placebo-controlled study, CONNECT-FX, and many participants who participated in that study are currently receiving ZYN002 in an open-label follow-on study.

The RECONNECT study is being conducted as it is common for regulatory agencies like the US FDA to require more than one study to confirm a medicine works and is safe before the medicine can be marketed and made available for use outside of clinical trials.

No, only individuals who have not previously participated in a study of ZYN002 may participate in the RECONNECT study.

ZYN002 is the only investigational pharmaceutically produced cannabidiol and is not available outside of clinical trials.  ZYN002 also contains no THC.  The amount of cannabidiol in the ZYN002 is significantly more than in most product containing cannabidiol that may be purchased and the ZYN002 gel is made to ensure the cannabidiol can be absorbed through the skin.

No, ZYN002 is an investigational pharmaceutically produced cannabidiol (not plant based) and does not contain THC.

Interested in Participating?

If you would like to learn more about the RECONNECT trial, fill out this form and your information will be sent to the recruitment coordinator, who will reach out to you.

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