NCT03614663

Clinical Study Of caNNabidiol in childrEn and adolesCenTs With Fragile X (CONNECT-FX)

A Randomized, Double-Blind, Placebo-Controlled Multiple-Center, Efficacy and Safety Study of ZYN002 Administered as a Transdermal Gel to Children and Adolescents With Fragile X Syndrome

• Status: Completed
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 212 (actual)
• Sponsor: Zynerba Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 3 Years – 17 Years
• Healthy volunteers: Not accepted

Summary

This trial will evaluate the efficacy and safety of ZYN002, a clear cannabidiol 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.

Detailed description

This is a randomized, double-blind, placebo-controlled, multiple-center study, to assess the efficacy and safety of ZYN002, a pharmaceutically manufactured cannabidiol, formulated as a transdermal gel, for the treatment of children and adolescent with FXS. Approximately 204 male and female patients, ages 3 to \< 18 years, will undergo a screening process. 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. Participants who are taking anti-seizure drugs may undergo an additional 1-2 weeks of blinded treatment to taper off study drug treatment. The assignment will be done by a computer generated system and neither the trial 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, 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). 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 trial doctor. After the final dose, patients will be followed weekly for 4 weeks by telephone, prior to discharge from the study.

Conditions

• Fragile X Syndrome

Interventions

Type	Name	Description
DRUG	ZYN002 - Cannabidiol Transdermal Gel	Pharmaceutically manufactured. Cannabidiol formulated as a clear gel (transdermal delivery)
OTHER	Placebo Transdermal Gel	Placebo formulated as a clear gel (transdermal delivery)

Timeline

• Start date: 2018-06-12
• Primary completion: 2020-06-14
• Completion: 2020-06-14
• First posted: 2018-08-03
• Last updated: 2022-07-06
• Results posted: 2022-06-16

Locations

21 sites across 3 countries: United States, Australia, New Zealand

Regulatory

• FDA-regulated drug study