NCT04977986

Clinical Study of Cannabidiol in Children, Adolescents, and Young Adults With Fragile X Syndrome

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

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 215 (actual)
• Sponsor: Zynerba Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 3 Years – 29 Years
• Healthy volunteers: Not accepted

Summary

This is a randomized, double-blind, placebo-controlled, multiple-center study, to assess the efficacy and safety of Cannabidiol administered as ZYN002 for the treatment of children, adolescent, and young adult patients with Fragile X Syndrome (FXS). Eligible participants will participate in up to an 18-week treatment period, where all participants will receive placebo or active study drug. Patients ages 3 to \< 30 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 clear gel that can be applied to the skin (called transdermal delivery), in children, adolescents, and young adults with FXS. 250 male and female patients, ages 3 to \< 30 years, will be randomized 1:1 to either trial drug or placebo and will undergo an 18-week treatment period. Randomization will be stratified by gender, methylation status and weight. The study will be comprised 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. The dose of the treatment will depend on the weight of the participants. If the participants weigh less than or equal to 30 kg, they will receive 2 sachets of the gel per day (1 sachet approximately every 12 hours). If the participant weighs more than 30 kg but less than or equal to 50 kg, they will receive 4 sachets of gel per day (2 sachets approximately every 12 hours). Participants who weigh more than 50 kg will receive 6 sachets of gel per day (3 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 and study doctor will be asked to complete some questionnaires for efficacy and safety assessment.

Conditions

• Fragile X Syndrome

Interventions

Type	Name	Description
DRUG	ZYN002 - transdermal gel	Pharmaceutically manufactured cannabidiol formulated as a clear gel that can be applied to the skin (transdermal delivery)
DRUG	Placebo	Placebo formulated as a clear gel that can be applied to the skin (transdermal delivery) Other Names: Placebo Comparator Matching Placebo

Timeline

• Start date: 2021-09-13
• Primary completion: 2025-08-29
• Completion: 2025-08-29
• First posted: 2021-07-27
• Last updated: 2025-12-17

Locations

27 sites across 5 countries: United States, Australia, Ireland, New Zealand, United Kingdom

Regulatory

• FDA-regulated drug study