Consider participating in a research study for patients with Dravet syndrome
Your loved one may be eligible to take part in a new clinical study
A clinical research study for children and adolescents with Dravet syndrome, called the EMPEROR Study, will evaluate a new investigational medicine. The study will enroll participants between 2 and 17 years of age with a confirmed diagnosis of Dravet syndrome. The study will assess whether the investigational medicine may help to reduce or prevent motor seizures when given in addition to standard of care medicines. The study will also assess impacts of the investigational medicine on other symptoms of Dravet syndrome, including behavior and cognition.
People who take part may receive*:
- Study-related care from doctors at no cost
- Study medicine at no cost
For more information on the EMPEROR Study, please contact 
info@emperorstudy.com
About the EMPEROR StudyWhat is the EMPEROR Study?
The EMPEROR Study is a clinical research study (also called a clinical trial). The study will assess the investigational medicine to see:
- whether it can reduce or prevent motor seizures when given in addition to standard anti-seizure medicines
- whether it can improve overall health condition and non-seizure symptoms, including behavior and cognition
- how safe it is and how well it is tolerated
- how it is processed by the body.
About 150 children and adolescents from around the world are expected to take part in the study.
About Dravet syndrome
Dravet syndrome is a rare, severe form of epilepsy that causes seizures and developmental delays. Symptoms often begin during the first year of life and continue to affect children as they age into adolescence, and throughout their life. There is currently no cure for Dravet syndrome. The current treatment options are limited and do not address the underlying cause of the disease. Dravet syndrome is commonly caused by a mutation in a specific gene called SCN1A.
People with Dravet syndrome usually have a spontaneous mutation in 1 of their SCN1A genes. Genes are the codes that the body uses to produce proteins. The SCN1A gene gives instructions to make the Naᵥ1.1 protein. People with Dravet syndrome have only 1 fully functional SCN1A gene, so they do not make enough Naᵥ1.1 protein, which is important for the brain’s nerves to work properly. When there is not enough Naᵥ1.1 in the brain, it can cause motor seizures and other symptoms of Dravet syndrome.
EMPEROR Study quick facts
The study will last for about 14 months.
The participant and their caregiver will need to visit the study center up to 10 times.
The caregiver will need to speak by telephone (or other virtual platform) to the study team about 11 times.
Participants will receive 4 doses of either the investigational medicine or sham procedure. The investigational medicine and sham procedure are both given in the participant’s lower back, through a procedure called a lumbar puncture.
Participants will continue taking their regular anti-seizure medicines during the study, as well as any other seizure treatments they were receiving before the study (for example, a ketogenic diet or vagus nerve stimulation).
Several tests and assessments will be performed to monitor the participant’s health at each visit to the study center.
At the end of the study, participants may have the opportunity to enroll in an open label extension study and receive the investigational medicine.
What to expect in the EMPEROR Study?
The study periods
The EMPEROR Study is split into 2 parts: The baseline period and the study treatment period.
1
Baseline period (about 2 months)
The participant and their caregiver will visit the study center up to 3 times. During these visits, the study doctor will perform assessments and collect health information about the participant. This information will help to decide whether the study is right for them. If the decision is made to join the study, participants will begin the 6-week observation period (part of the baseline period). There will be no change to the participant’s current anti-seizure medicine or treatment during the observation period. During the 6-week observation period, caregivers will collect information about participants’ motor seizures at home. This information will help us check again if the study is right for the participant.
2
Study treatment period (about 1 year)
The participants and their caregivers will need to visit the study center up to 7 times. This will include 4 visits (Visit 1, Visit 3, Visit 4, and Visit 6) where the participant will receive a lumbar puncture. The participant and their caregiver will then visit the study center 3 additional times for follow-up visits to check the participant’s health. There will also be about 11 phone calls or virtual visits with the study team.
The investigational medicine
The investigational medicine is designed to help the body to make more of the Naᵥ1.1 protein. The investigational medicine works differently from anti-seizure medicines because it is designed to target the cause of Dravet syndrome by helping the body produce more protein. By doing this, the goal is to reduce motor seizures and improve other non-seizure symptoms such as cognition and behavior.
This study is randomized, which means that participants will have a 50% chance of being assigned to 1 of 2 groups. One group will receive the investigational medicine and the other will receive a sham procedure. In this study, the investigational medicine and sham procedure are given through a thin needle in the lower back, known as a lumbar puncture. All participants will have the lumbar puncture and all will have cerebrospinal fluid (fluid surrounding the brain and spinal cord) collected and tested. Participants receiving the sham procedure will not receive the investigational medicine.
All participants will receive 4 lumbar punctures throughout the duration of the study. Every effort will be made to reduce pain and discomfort. Participants may be given a sedative (a medicine that makes you feel sleepy) or local anesthetic (a medicine that numbs an area of the body) and pain medicine, as needed. The type of medicines given for the procedure will be decided by the study doctor. Participants may have the opportunity to continue receiving the study medicine in an open-label extension study at the end of the study. There will be no sham procedure in the extension study.
To be eligible to take part in the EMPEROR Study, participants will need to meet certain criteria. They must:
A clinical research study, also called a clinical trial or research study, is a carefully designed scientific evaluation of a potential new (investigational) medicine. Clinical research studies are conducted by doctors and researchers. A clinical research study helps to answer important questions about a potential new medicine, such as:
- How safe is the potential new (investigational) medicine?
- Does it work?
A potential new (investigational) medicine, also sometimes called an investigational drug or a study medicine, has been tested in a laboratory. Based on those results, it has been approved by health regulatory authorities for testing in people. An investigational medicine may also be a medicine that is approved for the treatment of one disease or condition but is still under investigation in other diseases or conditions. An investigational medicine can only be used in clinical research studies
The study doctor will provide more details on what to expect throughout the study if your loved one participates in the study.
If you would like to know whether your loved one might be able to take part in the study, please contact a participating clinical research study center in your area using the “Study Locations” finder below, where you can find active study sites. Please note: during the screening process, the study doctor or study team will check additional criteria before someone can join the study.
The study-required investigational medicine or sham procedure will be provided at no cost. The person taking part in the study will also receive study-related care from a team of experienced doctors and nurses throughout the study at no cost.
All personal information will remain confidential, and data will only be collected and used as necessary to support an individual’s participation in a study. Participants’ names will not be included in any data reported. For more information, including how and why we process personal data, please read our Privacy Policy.
Clinical research studies must undergo rigorous reviews and follow strict rules. These rules help to ensure that the rights, safety, and well-being of participants are at the forefront of any study. Participating in a clinical research study comes with risks as well as benefits. Before joining a study, potential participants must make sure they understand the benefits, such as getting access to study-related medical care and maybe helping others in the future. They must also understand the risks, such as unknown side effects.
Potential participants may take all the time they need to decide whether to take part in this study. They may gather as much information as possible to help them make a decision. Before joining a study, the potential participant will be asked to sign an informed consent form, which will include a full explanation of the study, including its potential risks. If the potential participant is not capable of giving informed consent, a parent or legally authorized representative may provide informed consent on their behalf. In this situation, the potential participant will provide informed assent if they are able to.
It is a personal decision to take part. Participation in the study is voluntary. Please also consider:
- If a decision is made to take part, participants can withdraw at any time during the study
- The study team will explain the possible benefits and risks of the study during the informed consent process.
- A person does not have to join any study if they don’t want to.
- A team of doctors and nurses will carefully monitor the health of people taking part during the study.
- The potential new (investigational) medicine or sham procedure will be provided at no cost.
People taking part in the study will need to follow all the instructions from the study doctor and nurses.
For the latest information on participating study centers and locations, please visit https://clinicaltrials.gov/study/NCT06872125. You can also reach out to info@emperorstudy.com for more information and to receive notifications when new study centers and locations are added.
Want to know more? For more information on the EMPEROR Study, please contact info@emperorstudy.com