- Patients must be between 2 and 17 years of age
- Patients must have a clinical diagnosis of DS confirmed by the Epilepsy Study Consortium, Inc. (ESCI)
- Patient must experience the required number of major motor seizures during the 6-week Observation Period. Major motor seizure types included are Seizure types included in counts are Hemiclonic, Focal with Motor Signs, Focal to Bilateral Tonic-Clonic, Generalized Tonic-Clonic, Tonic, Tonic/Atonic (Drop Attacks with fall or risk of fall), and Bilateral Clonic.
- Patient must have used at least 2 prior interventions for seizures. These can include anti-seizure medications (ASMs), ketogenic diet and/or vagus nerve stimulation (VNS) with either lack of adequate seizure control or discontinued due to an AE(s). These interventions can be ongoing therapies.
Detailed eligibility will be discussed when reaching out to the study team.
Dravet syndrome is a severe form of epilepsy (a brain disorder that causes seizures). There are several types of therapies available to help reduce the number of seizures. However, even with treatment using current anti-seizure therapies, the number of seizures may not be reduced enough, and seizures may still happen.
This study will look at a possible new investigational therapy for Dravet syndrome, called zorevunersen (STK-001). Investigational means that zorevunersen is not approved by the U.S. Food and Drug Adminstration (FDA).
The main purposes of this study are to:
• evaluate the effect of the study drug on how often children with Dravet syndrome have seizures
• see if/how the study drug effects the damage caused by Dravet syndrome, including behavioral and developmental delays, and difficulties in language and speech
• evaluate how long the study drug may reduce the frequency of seizures in children and adolescents with Dravet syndrome (called “durability”)
• see how safe the study drug is in children and adolescents and how well a child can handle receiving it (called “tolerability”)
* This involves checking for side effects, vital signs, overall health, movement and balance, and heart rhythm among other safety checks
*how the participant’s immune system (body’s defense system) responds to the study drug.
• look at how the study drug is taken up and broken down by the body (called “pharmacokinetics” or PK).
The EMPEROR Study is split into 2 parts: The baseline period and the study treatment period.
In the baseline period, 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.
In the study treatment period, 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.