Trial Summary
Zynerba Pharmaceuticals is recruiting males and females ages 3-22 with Fragile X syndrome for a large-scale Phase 3 clinical trial (RECONNECT) of Zygel.
Twenty-eight sites across the United States, Australia, and the United Kingdom are recruiting 200 participants for this randomized, double-blind, placebo-controlled study. Eligible participants living anywhere in the US, excluding Alaska, Hawaii, and Washington DC may participate from their home.
Zygel is a pharmaceutically manufactured CBD, formulated as a clear gel that can be applied to the skin (transdermal delivery).
New AT-HOME Option for Participation
Zynerba is pleased to announce a new option for families to participate in the RECONNECT trial completely from their home through a combination of at-home and virtual visits.
The at-home option is being introduced to help lessen potential barriers for families who live far from a Fragile X clinical study site or find it too challenging to go to a site for study visits.
Whether you choose for your child to participate through this new at-home option or at a Fragile X clinical study site, your participation is critical to help complete the RECONNECT trial. RECONNECT is evaluating an investigational medicine in the treatment of behavioral symptoms of Fragile X syndrome. If the data is positive, it may lead to the approval of the first medicine specifically for the treatment of behavioral symptoms in Fragile X syndrome.
Is this option right for my family?
If you have a desire for your child (age 3 through 22 years old) to participate in a clinical trial, but going to a study site may be too much of a barrier, this option may be for you.
Eligible participants living anywhere in the US other than Alaska, Hawaii, and Washington DC may participate from their home.
How do I find out if my child is eligible to participate?
Please go to fragilexhelp.com to fill out a form to be contacted or call (833) 680-1155
Qualified participates must be:
- Between the ages of 3 to 22.
- Have a diagnosis of Fragile X syndrome through genetic testing.
- Be experiencing behavioral symptoms of Fragile X syndrome such as social anxiety/avoidance, irritability, and social unresponsiveness.
- Otherwise, be in generally good health.
Will this impact the care of my child by their current doctor?
No. Your child will continue to receive care from their doctor as they currently do. The study visits are focused on conducting assessments for the study. The study doctor may share relevant information with your child’s current doctor.
What is the difference between “at-home” and “virtual” visits?
During an “at-home” visit, a study nurse will visit your home to conduct study assessments along with having some study staff who will join virtually on a call/video communication. During ‘virtual’ visits, all assessments will be done virtually through a live video communication from your home.
Can my child still enroll if I want to participate through a Fragile X clinical study site?
Yes, participation at clinical study site is still an option. Please go to fragilexhelp.com to see the location of clinical study sites and fill out the contact form or call (833) 680-1155 to ask about participation at a specific site.
Is the at-home option available if my child is already participating in RECONNECT?
No, your child will continue to participate at their current study site. As a reminder, current participants and any newly enrolled child who participates at a clinic study site have a combination of 4 in-clinic (office) visits and 4 virtual visits.
Who is Science 37?
Science 37 is the name of the organization providing this virtual trial option. Dr. David Kudrow is the lead investigator for Science 37 for RECONNECT and he is joined by other doctors, nurses and staff to conduct the at-home and virtual visits for the study. Science 37 has conducted many at-home studies, including studies in children, adolescents, and young adults.
About RECONNECT Phase 3 Fragile X Clinical Trial
The study consists of a screening visit and a combination of seven visits both onsite (face-to-face) and virtual. The assignment of study drug or placebo will be done by a computer generated system and neither the study doctor, participant or their caregivers will know which treatment is being given to them. Parents/caregivers will be instructed on proper application of the gel tol be applied to clean, dry, intact skin of the upper arms/ shoulders.
Blood samples will be collected for safety analysis of the gel. Additionally, the parents/caregivers and study doctor will be asked to complete some questionnaires for efficacy and safety assessment.
The primary endpoint for the trial is the change from baseline to the end of the treatment period in the Aberrant Behavior Checklist-Community FXS Specific (ABC-CFXS) Social Avoidance subscale in patients who have complete methylation of their FMR1 gene. The ABC-CFXS Social Avoidance subscale is the same primary endpoint used in the CONNECT-FX trial. While the primary endpoint is in patients with complete methylation, patients with partial methylation of their FMR1 gene are also being enrolled.
A presentation providing a full overview of the trial is available on FRAXA’s YouTube channel.
Hope for the Future
Armando Anido, Chairman and Chief Executive Officer of Zynerba stated “If successful, we believe the study could serve as the basis for Zygel approval in the US for patients with FXS.” in a press release on September 13, 2021.
FRAXA Research Foundation funded teams in France, Spain, and the US, and is funding three new teams this year, to understand the endocannabinoid system in Fragile X syndrome. These studies have paved the way to this Fragile X clinical trial.
Topline results for the RECONNECT trial are expected in the second half of 2023.
Trial Locations
Enrollment Information
fragilexhelp.com
(833) 682-1155
Nancy Tich, PhD
(973) 727-4117
tichn@zynerba.com
Stephen O’Quinn, PharmD
(919) 271-1339
oquinns@zynerba.com
