RecruitingPhase 3ACTRN12621000874819

Clinical study of cannabidiol in children, adolescents and young adults with Fragile X syndrome (RECONNECT)

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

Sponsor

Zynerba Pharmaceuticals Pty Ltd

Enrollment

250 participants

Start Date

Dec 16, 2021

Study Type

Interventional

Conditions

Fragile X Syndrome

Summary

This study is evaluating the efficacy and safety of ZYN002, a pharmaceutically manufactured form of Cannabidiol (CBD) that is a clear gel that can be applied to the skin (called transdermal application). It is applied twice a day for treatment of symptoms of Fragile X Syndrome (FXS) Who is it for? Patients who have been diagnosed with Fragile X Syndrome with full mutation of FMR1 gene and are aged between 3 and less than 30 years old. Study details All participants will undergo a screening process. Eligible participants will be randomized 1:1 to drug or placebo and will undergo up to a 18-week treatment period. Participants who are taking anti-epileptic drugs may undergo an additional 1-3 weeks of blinded treatment to taper off study drug treatment. During the treatment period, all participants may be either assigned to ZYN002 or placebo. 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 30kg, they will receive 2 sachets of the gel through the day (1 sachet approximately every 12 hours), if they weigh more than 30kg but less than 50 kg they will receive 4 sachets of gel per day (2 sachets approximately every 12 hours) and if they weigh more than 50kg, they 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 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 and/or with the participant and their parents/caregivers. Participation in this study may help the child’s/adolescent’s FXS symptoms; however, we cannot guarantee that he/she will get any benefits from this study. The results of this study may benefit future patients.

Eligibility

Sex: Both males and femalesMin Age: 3 YearssMax Age: 29 Yearss

Plain Language Summary

Simplified for easier understanding

This study is testing a CBD (cannabidiol) gel applied to the skin twice a day as a treatment for symptoms of Fragile X Syndrome (FXS). FXS is the most common inherited cause of intellectual disability and autism, and it can cause significant behavioural challenges including anxiety, irritability, hyperactivity, and social difficulties. The CBD gel, called ZYN002, is absorbed through the skin and does not contain THC (the component in cannabis that causes a high). The trial is open to patients aged 3 to under 30 years old who have a confirmed genetic diagnosis of FXS. Participants will be randomly assigned to receive either ZYN002 gel or a placebo gel for up to 18 weeks. Neither participants, caregivers, nor study doctors will know which treatment is being given. The dose is adjusted based on the child's weight. Parents and caregivers will apply the gel to the upper arms and shoulders and complete questionnaires about their child's symptoms. To be eligible, the patient must have a confirmed full mutation FMR1 gene, live at home with a consistent caregiver, and be medically stable. Patients currently using cannabis products, certain anti-epileptic drugs, or specific enzyme-inhibiting medications are not eligible. This Phase 3 trial is run by Zynerba Pharmaceuticals.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

Interested in this trial?

Get notified about updates and connect with the research team.

Interventions

This is a randomized, double-blind, placebo-controlled, multiple-center study, to assess the efficacy and safety of Cannabidiol (CBD) administered as ZYN002. ZYN002 is a pharmaceutically manufactured

This is a randomized, double-blind, placebo-controlled, multiple-center study, to assess the efficacy and safety of Cannabidiol (CBD) administered as ZYN002. ZYN002 is a pharmaceutically manufactured CBD that is developed as a clear gel that can be applied to the skin (called transdermal delivery). The gel will be applied to clean, dry, intact skin of the shoulders and/or upper arms. All participants will undergo a screening process. Eligible participants will then participate in a 18 week treatment period, and may receive placebo or active study drug Treatment group A (ZYN002): Parents/caregivers will apply the study gel twice daily for the treatment period. Participants who weigh less than or equal to 30 kg, will receive 1 sachet of ZYN002, applied every 12 hours. Participants who weigh more than 30 kg but less than or equal to 50 kg will receive 2 sachets of ZYN002, applied every 12 hours. Participants who weigh more than 50 kg will receive 3 sachets of ZYN002, applied every 12 hours. Participants who are taking Anti-epileptic drugs may have an additional one, two or three weeks of blinded treatment to taper off study treatment, depending upon their weight. Four weeks after the final dose, patients will be followed up by a telephone call, prior to discharge from the study. Patient compliance with the intervention will be monitored by the study coordinator through drug accountability at each study visit.