RecruitingPHASE3INTERVENTIONAL

A Double-blind Study Evaluating the Efficacy, Safety, and Tolerability of Zorevunersen in Patients With Dravet Syndrome

This research study is looking at a new medicine called zorevunersen for children and teenagers aged 2 to 17 who have Dravet syndrome. Dravet syndrome is a type of epilepsy that starts in infancy. The medicine aims to help the body make more of a protein called Nav1.1, which is often too low in people with Dravet syndrome, and this might help control seizures. This isn't a gene therapy, but a way to help the body use its own genes better. We'll be comparing zorevunersen to a dummy treatment (placebo) to see if it's effective, safe, and well-tolerated. The study will look at how often seizures happen, as well as changes in behaviour, thinking skills, and quality of life. Participants may have the chance to continue on the medicine after the main study ends.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

Stoke Therapeutics, Inc

Enrolment target

170

Start

04 Jun 2025

Estimated completion

01 Oct 2028

Dravet Syndrome

What is this study about?

This study is about a new medicine called zorevunersen for children and teenagers with Dravet syndrome. Dravet syndrome is a serious form of epilepsy that often begins in babies. The medicine is designed to help the body work better at a very basic level. People with Dravet syndrome often have lower levels of a specific protein called Nav1.1, which is important for how brain cells communicate. Zorevunersen aims to help the body make more of this useful protein. It’s important to know this isn't gene therapy, which changes your genes, but rather an approach that helps your body use its existing genetic information more effectively to produce the needed protein.

The main goal of this study is to find out if zorevunersen can reduce the number of major seizures in people with Dravet syndrome. We also want to see if it can make a difference in other important areas, like how a child behaves, their learning and thinking abilities, their overall health, and their quality of life. The study will compare zorevunersen to a ‘sham comparator’ (a dummy treatment that looks like the real medicine but has no active ingredients). This is a standard way to make sure any improvements we see are truly due to the medicine and not other factors.

This study is global, involving many different centres, and will take place in two main parts. After the first part, we will look at how well the medicine is working and how safe it is. These checks will be done again after the second part. The hope is that zorevunersen could not just manage symptoms but potentially change how the disease progresses, which would be a significant step forward for people living with Dravet syndrome.

Key takeaways

This study is testing a new medicine, zorevunersen, for children and teenagers with Dravet syndrome.
The medicine aims to increase a protein (Nav1.1) that is usually low in people with Dravet syndrome.
It's a 'blinded' study, meaning participants will receive either the new medicine or a dummy treatment.
Researchers will check for changes in seizure frequency, behaviour, thinking skills, and quality of life.
Participants must be aged 2 to 17 with a confirmed Dravet syndrome diagnosis and previously tried other treatments.

Who may be eligible?

This study is looking for children and teenagers with Dravet syndrome who are between 2 and 17 years old. To be included, your child must have been formally diagnosed with Dravet syndrome by experts, and this diagnosis needs to be linked to a specific change (mutation) in a gene called SCN1A. They also need to have experienced a certain number of major seizures during a 6-week observation period before the study starts.

Your child should have tried at least two different treatments for their seizures in the past, such as other seizure medicines, a special diet (ketogenic diet), or a device called a vagus nerve stimulator (VNS). These previous treatments might not have worked well enough or might have caused side effects. They must also be taking at least one seizure medicine regularly. Any current medicines or treatments your child is on, including cannabis-based products, must have been stable for a while before the study begins.

However, your child will not be able to join if their SCN1A gene change is known to cause a different type of problem (called a 'gain-of-function' variant). They also can't be taking certain seizure medicines that primarily block sodium channels, like phenytoin. The study doctors will carefully check all these requirements to ensure the study is safe and appropriate for your child.

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.

Is your child between 2 and 17 years old?
Does your child have a confirmed diagnosis of Dravet Syndrome with a specific gene change (SCN1A)?
Has your child had a certain number of major seizures in the last 6 weeks?
Has your child tried at least two other seizure treatments, like medicines or special diets?
Is your child currently taking at least one seizure medicine regularly?
Is your child NOT taking certain sodium channel blocking seizure medicines?

Answer every question to see your result.

What does participation involve?

If your child takes part in this study, they will be given either the new medicine, zorevunersen, or a dummy treatment (placebo). Neither you nor the study team will know which one they are receiving until after the main parts of the study are finished. Your child will have regular visits to the clinic where they will have various assessments, including checks on their seizure frequency, behaviour, thinking skills, and overall health. They will also have physical exams and blood tests. The study is divided into two treatment periods. After the study, if your child meets certain conditions, they may have the opportunity to continue receiving zorevunersen in a follow-up study where everyone gets the active medicine. The total length of your child's participation in the main study will be discussed by the study team.

Potential risks and benefits

Participating in a clinical trial may offer potential benefits, such as access to a new investigational medicine that might improve your child's condition, as well as close medical monitoring. However, there are also potential risks, including side effects from the investigational medicine, the inconvenience of study visits, and the possibility that the medicine may not work or could even worsen the condition. You have the right to withdraw your child from the study at any time, for any reason, without it affecting their future medical care.

Locations (61)

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

Phoenix Children's Hospital
Verified postcode
Phoenix, United States· Recruiting
Arkansas Children's Hospital
Verified postcode
Little Rock, United States· Recruiting
Cedars Sinai Medical Center
Verified postcode
Los Angeles, United States· Recruiting
Children's Hospital of Orange County
Verified postcode
Orange, United States· Recruiting
USCF Medical Center
Verified postcode
San Francisco, United States· Recruiting
Children's Hospital Colorado
Verified postcode
Aurora, United States· Recruiting
Children's National Medical Center
Verified postcode
Washington D.C., United States· Recruiting
Nemours Children's Health
Verified postcode
Jacksonville, United States· Recruiting
Nicklaus Children's Hospital
Verified postcode
Miami, United States· Recruiting
Advent Health Neuroscience Research Institute
Verified postcode
Orlando, United States· Recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago
Verified postcode
Chicago, United States· Recruiting
University of Iowa Hospital and Clinics
Verified postcode
Iowa City, United States· Not yet recruiting

Common questions

What is Dravet syndrome?

Dravet syndrome is a rare and severe form of epilepsy that usually starts in babies. It causes frequent and long-lasting seizures, and can affect a child's development.

What is zorevunersen and how does it work?

Zorevunersen is an investigational medicine that aims to help the body make more of a protein called Nav1.1, which is often too low in people with Dravet syndrome. By increasing this protein, it might help control seizures.

Will my child get the actual medicine or a dummy treatment?

In this study, your child will either receive zorevunersen or a dummy treatment (sham comparator). This is a 'blinded' study, meaning neither you nor the study team will know which one your child is getting until later.

How long will the study last?

The study has two main treatment periods. The total duration of your child's participation will be explained by the study team. There might also be an option to continue receiving the medicine after the main study ends.

What if the medicine doesn't work for my child?

Participation in a study does not guarantee a positive outcome. The study is designed to learn if the medicine is effective. Your child will continue to receive medical care throughout the study, and your doctor will discuss all treatment options with you.

How to find out more

Emperor Information Center

info@emperorstudy.com 1-781-430-8200 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.