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.

MicroscopeZYN002, 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.

Frequently Asked Questions

No. There will not be a therapy intervention used along with the 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 patients 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 scientific evidence and trial Inclusion and Exclusion criteria, make a determination 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 exams.
  • Blood work three times during the 12-week study.
  • A 12-lead ECG 3-4 times during 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 patient.

There are six scheduled visits during the study. Visits are somewhat flexible; each visit has a target date, with a window of three days before and after the target date itself. Some patients may require an additional visit. The length of time for the visits can vary, depending on the number of assessments/procedures that are conducted.

Patients 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 CONNECT-FX is to evaluate the efficacy and safety of ZYN002 in improving certain behavioral symptoms that patients with FXS exhibit. CONNECT-FX is enrolling eligible patients to help researchers find a potential treatment for these patients and their families.

Healthy volunteers participate 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 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. Patients enrolled in this study cannot participate in another trial of another investigational drug simultaneously.

Details

Study Coordinators:
Nancy R Tich, PhD
973-727-4117

Donna Gutterman, PharmD
919-522-8828

Disorder: Full mutation fragile x syndrome
Gender: Male and Female
Ages: 3 to 17 (< 18)
Sponsored by: Zynerba Pharmaceuticals
Clinical Trials Identifiers: 03614663 & ACTRN12618001063202
Cost to Patient: No cost*

Start Date: June 12, 2018
Est Primary Completion: July 23, 2019
Est Study Completion: October 29, 2019

Locations20 sites in the United States, Australia, and New Zealand

Additional Details:

Clinical Study CONNECT-X
Australia/New Zealand Clinical Trials Registry
Transdermal Cannabidiol (CBD) Gel for the Treatment of Fragile X Syndrome (FXS)

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

This clinical trial reached it patient screening target and is no longer accepting participants. Thank you for your interesting and willingness.

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