Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

An Open-Label Extension Study to Assess the Long-Term Safety and Tolerability of ZYN002 Administered as a Transdermal Gel to Children, Adolescents and Young Adults with Fragile X Syndrome

This research is an ongoing study checking the long-term safety of a special gel called ZYN002, which contains cannabidiol (CBD). It's being tested on children, teenagers, and young adults who have Fragile X Syndrome. The main goal is to see if the gel is safe and doesn't cause serious side effects over many months and years. Researchers will also be looking closely at how the gel affects common Fragile X symptoms, such as irritability, avoiding social situations, and hyperactivity. This information will help doctors understand more about using this treatment in the future.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Harmony Biosciences Management Inc.

Enrolment target

Start

03 May 2024

Fragile X Syndrome (FXS)

What is this study about?

This study is a continuation of earlier research into a gel called ZYN002, which contains an ingredient called cannabidiol (CBD). It's designed for children, teenagers, and young adults who have Fragile X Syndrome. Fragile X Syndrome is a genetic condition that can cause a range of learning difficulties, anxiety, and challenges with social interaction and behaviour. This gel is applied to the skin, and the aim of this particular study is to see how safe it is to use over a long period, rather than just a few weeks or months.

The main focus of this study is checking for any side effects or problems that might come up with long-term use of the gel. Doctors and nurses will be carefully monitoring participants' health through various tests and checks. This is really important because before any new medicine can be widely used, we need to be sure it's safe for people to take for a long time if they need to.

Beyond just safety, the researchers are also interested in whether the gel helps with some of the common difficulties faced by people with Fragile X Syndrome. They will track changes in things like how often someone is irritable, if they avoid social situations, or if they are very active. This information will help us understand if the gel not only is safe but also makes a positive difference in their daily lives.

Key takeaways

This study is for people with Fragile X Syndrome.
It's testing a CBD gel called ZYN002.
The main focus is long-term safety.
It also checks if the gel helps with symptoms like irritability and social issues.
Participants must have been in a previous ZYN002 study.
It involves regular health checks over several years.

Who may be eligible?

To be considered for this study, individuals must have Fragile X Syndrome. This study is specifically for those who were already taking part in a previous study of ZYN002.

Participants can be children, teenagers, or young adults. There is no upper age limit for adults, as long as they meet the other study requirements. Both males and females can take part in this research.

Because this is an extension of a previous study, new participants who have not used ZYN002 before cannot join this particular trial. It's designed to follow people who have already started the treatment.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Do I/does my child have Fragile X Syndrome?
Was I/was my child already taking part in a study for ZYN002 gel?
Am I/is my child a child, teenager, or young adult (no upper age limit for adults)?
Am I/is my child able to attend regular study visits for several years?
Is my/my child's doctor aware of and supportive of participating?

Answer every question to see your result.

What does participation involve?

If you or your child take part in this study, you'll be using the ZYN002 gel as directed. You'll need to attend regular visits for health checks and assessments. These checks will include physical examinations, maybe some blood tests, and heart monitoring (like an ECG) to make sure everything is okay. The team will also look for any skin irritation from the gel.

During these visits, you'll be asked questions about how you or your child are feeling and any changes noticed, especially concerning mood, social interactions, and behaviour. These visits will happen over a long period, potentially for several years, to track long-term safety and any ongoing effects of the gel. The overall duration means regular follow-ups for potentially up to four years or more.

Potential risks and benefits

Potential benefits of participating include continued access to the ZYN002 gel and close medical monitoring, which could help manage symptoms of Fragile X Syndrome. There is a chance the gel may improve symptoms like irritability or social difficulties. However, like all medicines, there could be risks, such as side effects from the gel or from the tests involved in the study. While safety is a top priority, not everyone experiences benefits, and some might have unwanted reactions. You have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (1)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Ireland

Common questions

What is Fragile X Syndrome?

Fragile X Syndrome is a genetic condition that causes intellectual disability, learning challenges, and can affect behaviour and social skills.

What is ZYN002 gel?

ZYN002 is a gel that contains cannabidiol (CBD). It's being studied as a possible treatment for some symptoms of Fragile X Syndrome.

What does 'transdermal' mean?

'Transdermal' means that the gel is applied to the skin, and the medication then soaks through the skin into the body.

Is this a new medicine for Fragile X Syndrome?

This study is looking at an experimental gel. If it's found to be safe and helpful, it could become a new treatment option in the future.

What is the main goal of this study?

The main goal is to understand how safe the ZYN002 gel is to use over a long period for people with Fragile X Syndrome, and to see if it helps with their symptoms.