Active not recruitingPHASE2, PHASE3INTERVENTIONAL

Open Label Extension to Assess the Long-Term Safety and Tolerability of ZYN002 in Children and Adolescents With FXS

This study is an ongoing look at how safe and well-tolerated a special gel called ZYN002 is for children and teenagers with Fragile X Syndrome over a longer period. ZYN002 contains cannabidiol and is designed to be absorbed through the skin. If your child previously took part in the ZYN2-CL-016 or ZYN2-CL-033 studies and meets certain health criteria, they might be able to join this next phase. In this study, parents or carers will apply the gel to their child's skin twice a day for at least 52 weeks (about a year). The main goal is to understand how safe this treatment is when used over a long time, and how participants' bodies handle it.

At a glance

Status

Active not recruiting

Phase

PHASE2, PHASE3

Sponsor

Zynerba Pharmaceuticals, Inc.

Enrolment target

476

Start

09 Nov 2018

Estimated completion

30 Jun 2026

Fragile X Syndrome

What is this study about?

This research is specifically looking at children and teenagers who have Fragile X Syndrome, a genetic condition that can affect a person's learning, behaviour, and development. The study builds on previous research and aims to understand the long-term effects of a gel called ZYN002.

ZYN002 is a medicine that contains cannabidiol, a substance found in the cannabis plant. However, it's manufactured carefully to be a specific amount, and it's designed to be absorbed through the skin rather than taken by mouth. By applying it as a gel to the shoulders or upper arms, researchers hope it can help manage some of the challenges linked with Fragile X Syndrome. This study is important because it will give healthcare professionals and families a better understanding of how safe and effective ZYN002 is when used over a longer period, potentially for several years.

It's important to remember that this is a research study, and while ZYN002 contains cannabidiol, it's a carefully controlled and manufactured medicine. The goal is to see if it can be a safe and helpful long-term option for people with Fragile X Syndrome. The information gathered will contribute to medical knowledge and could help shape future treatment options.

Key takeaways

This study is for children and teenagers with Fragile X Syndrome who have already been in a ZYN002 study.
The treatment is a gel (ZYN002) applied to the skin twice a day.
It aims to check the long-term safety and how well the body tolerates the gel.
The study lasts for at least 52 weeks, with potential to continue for several years.
Regular clinic visits, blood tests, and questionnaires are part of taking part.
Your child's dose may be adjusted based on their weight.

Who may be eligible?

To be able to join this study, your child must have already taken part in one of the earlier ZYN002 studies (either ZYN2-CL-016 or ZYN2-CL-033). They also need to be between 3 and 29 years old. Both boys and girls are welcome to participate. It's really important that you, as their parent or carer, can commit to all the study rules and procedures.

Your child won't be able to join if they are currently taking part in another medical study using a different experimental drug or device. Also, if your child had a serious side effect in the previous ZYN002 studies that the study doctor thinks could be a problem, they won't be eligible. If your child has certain abnormal levels in their liver blood tests, they would also not be able to join.

For girls or women who might get pregnant, they will need to have a negative pregnancy test at each visit. If they are pregnant, breastfeeding, or planning to get pregnant, they cannot take part. Similarly, if you or your child (if they have a partner) are of childbearing age and not able or willing to use effective contraception for the duration of the study and for three months afterwards, they won't be able to join.

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.

Has your child been in a previous ZYN002 study (ZYN2-CL-016 or ZYN2-CL-033)?
Is your child between 3 and 29 years old?
Are you, as a parent/carer, able to commit to applying the gel and attending all visits?
Is your child able to use effective contraception if they could get pregnant?
Does your child have certain normal liver blood test results?

Answer every question to see your result.

What does participation involve?

If your child joins this study, you, as their parent or carer, will be responsible for applying the ZYN002 gel to their shoulders or upper arms twice a day, every day. This will be for at least 52 weeks (around one year). Some participants might continue treatment for up to 72 months (6 years) if they meet certain criteria after the first year.

The amount of gel applied will depend on your child's weight and which previous study they were in. The study doctor might adjust the dose during the study, for example, if your child's weight changes. For children taking epilepsy medication, there might be an extra week or two of treatment at the end to slowly reduce the study gel.

Throughout the study, blood samples will be taken to check on your child's safety. You and your child will also be asked to complete various questionnaires at home and during visits to the study clinic. These questionnaires help the research team understand how your child is doing and how they are responding to the treatment. The research team will regularly check in with you and your child, and your child will have annual check-ups after the initial 12 months if they continue in the study.

Potential risks and benefits

Like all medical studies, there are potential benefits and risks. A potential benefit of taking part is that your child might receive a potential treatment for Fragile X Syndrome that could help manage their symptoms. You'll also have regular medical checks throughout the study. However, there's no guarantee the gel will work or provide any direct benefit to your child. Potential risks include side effects from the gel, which will be carefully monitored. The study involves regular clinic visits, blood tests, and completing questionnaires, which might be time-consuming or uncomfortable for your child. You are free to withdraw your child from the study at any time, for any reason, without it affecting their usual medical care.

Locations (26)

Southwest Autism Research and Resource Center
Verified postcode
Phoenix, United States
Science 37
Verified postcode
Culver City, United States
Amnova Clinical Research, LLC
Verified postcode
Irvine, United States
UC Davis Health System, MIND Institute
Verified postcode
Sacramento, United States
Children's Hospital of Colorado
Verified postcode
Denver, United States
Children's Research Institute
Verified postcode
Washington D.C., United States
University of Miami
Verified postcode
Miami, United States
Han Phan
Verified postcode
Atlanta, United States
Rush University Medical Center
Verified postcode
Chicago, United States
Kennedy Krieger Institute
Verified postcode
Baltimore, United States
Boston Children's Hospital
Verified postcode
Boston, United States
Masonic Institute of Developing Brain
Verified postcode
Minneapolis, United States

Common questions

What is Fragile X Syndrome?

Fragile X Syndrome is a genetic condition that can impact a person's learning ability, behaviour, and development. It's the most common inherited cause of learning difficulties.

What is ZYN002?

ZYN002 is a specially made gel that contains cannabidiol. It's designed to be absorbed through the skin when applied, rather than taken by mouth.

How long will my child be on the gel?

Parents or carers will apply the gel for at least 52 weeks (about a year). Some children might continue for up to 6 years if they continue to qualify.

Will my child have to visit the clinic often?

Yes, there will be regular visits for check-ups, blood tests, and to fill in questionnaires. You'll also be asked to complete questionnaires at home.

Can I take my child out of the study if we change our mind?

Yes, you can withdraw your child from the study at any time, for any reason, and it won't affect their regular medical care.