In short

Currently, there are 2 ongoing clinical trials investigating treatments for Fragile X Syndrome. Both studies are testing ZYN002 Transdermal Gel containing cannabidiol (CBD) in children, adolescents, and young adults. The trials are being conducted in Ireland and focus on evaluating the safety, tolerability, and effectiveness of this skin-applied gel in managing behavioral symptoms associated with this genetic condition.

Clinical trial locations

Ireland
- Study on the Effects and Safety of ZYN002 Gel for Children, Adolescents, and Young Adults with Fragile X Syndrome
- Study on Long-Term Safety of ZYN002 Gel for Children, Adolescents, and Young Adults with Fragile X Syndrome

Study on Long-Term Safety of ZYN002 Gel for Children, Adolescents, and Young Adults with Fragile X Syndrome

This clinical trial is investigating the long-term safety of ZYN002 Transdermal Gel, which contains cannabidiol (CBD), in treating Fragile X Syndrome. The medication is applied to the skin, allowing it to be absorbed into the body over an extended period of up to 48 months.

Who can participate:

Children, adolescents, and young adults aged between 3 and less than 23 years old who have completed a previous study with the same treatment
Participants must be in generally good health based on medical history, physical examination, heart tests, and laboratory results
Those taking seizure medications must be on a stable treatment plan with no more than two medications for at least four weeks, or must have been seizure-free for one year without medication
If taking mental health medications for conditions like anxiety, depression, ADHD, or sleep issues, participants should be on a stable plan with no more than three such medications for at least four weeks
Any non-drug treatments or special diets must be stable for three months before joining
Body mass index (BMI) between 12 and 30, or if between 30 and 40, must have normal liver function tests
Females who can have children must have negative pregnancy tests at all required visits

Who cannot participate:

Patients with any serious medical condition that might interfere with the study
Those currently participating in another clinical trial
Patients who have had a significant allergic reaction to any ingredients in the study medication
Those unable to follow study procedures or instructions
Patients with a history of substance abuse
Pregnant or breastfeeding patients
Those with skin conditions that could interfere with gel application
Patients with a history of seizures not meeting the stability criteria

What the study involves:

The trial focuses on assessing the long-term safety and tolerability of ZYN002 Transdermal Gel over an extended period. Participants will regularly apply the gel to their skin and attend follow-up visits to monitor their health. Throughout the study, various safety assessments will be conducted, including physical and neurological examinations, heart tests using 12-lead ECG, and laboratory tests to check blood and urine. The study will also observe any changes in behavior and symptoms to understand the long-term impact of the treatment. Participants and their caregivers will provide feedback on any improvements or side effects they notice. The study is expected to conclude by August 31, 2027.

Investigational drug: ZYN002 is a transdermal gel containing cannabidiol that is applied to the skin. It interacts with the endocannabinoid system, which plays a role in regulating mood and behavior.

Study on the Effects and Safety of ZYN002 Gel for Children, Adolescents, and Young Adults with Fragile X Syndrome

This clinical trial is testing ZYN002 Transdermal Gel containing cannabidiol (CBD) for its effectiveness in managing behavioral symptoms associated with Fragile X Syndrome. The study aims to determine how well the treatment works and how safe it is over a 16-week period.

Who can participate:

Children, adolescents, and young adults aged between 3 and less than 23 years old
Must have a confirmed diagnosis of Fragile X Syndrome through genetic testing
Must live with a caregiver who will provide consistent care during the study
Should be in generally good health as determined by a doctor, based on medical history, physical check-up, heart test, and lab results
If the patient has a history of seizures, they must be on a stable treatment plan with no more than two anti-seizure medications for at least four weeks, or have been seizure-free for one year
Those taking mental health medications should be on a stable plan with no more than three such medications for at least four weeks
Body mass index (BMI) between 12 and 30, or if between 30 and 40, must have normal liver function and no family history of fatty liver disease

Who cannot participate:

Patients without a confirmed diagnosis of Fragile X Syndrome
Those younger than 3 years old or older than 22 years old
Patients unable to use the medication as a transdermal gel applied to the skin
Those with other medical conditions that might interfere with the study results
Patients currently participating in another clinical trial
Those with a history of allergic reactions to similar medications
Patients unable to follow study procedures or attend study visits

What the study involves:

Participants will be randomly assigned to receive either ZYN002 gel or a placebo gel, which looks the same but does not contain the active ingredient. The treatment will be administered daily for 18 weeks by applying the gel to the skin. Throughout this period, participants will be monitored regularly to assess changes in behavior and ensure their safety. Researchers will specifically look at improvements in social interactions and irritability, comparing measurements from the start of the study to the end. The goal is to provide valuable information on whether ZYN002 gel can be a helpful treatment option for managing symptoms of this condition.

Investigational drug: ZYN002 is a transdermal gel containing cannabidiol that is applied to the skin and absorbed into the body. It is believed to work by interacting with the endocannabinoid system, which helps regulate mood and behavior.

Summary

Both ongoing clinical trials for Fragile X Syndrome are being conducted in Ireland and focus on the same investigational treatment: ZYN002 Transdermal Gel containing cannabidiol. One trial evaluates the long-term safety and tolerability over up to 48 months in patients who have previously participated in studies with this treatment. The other trial assesses the effectiveness and safety of the gel over a shorter 16-week period in a broader patient population. Together, these studies aim to provide comprehensive data on both the immediate effects and long-term safety profile of this cannabinoid-based therapy for managing behavioral symptoms associated with Fragile X Syndrome in young patients aged 3 to under 23 years.

Fragile X syndrome Trials in Disease

Clinical trials for Fragile X syndrome