Purpose of this Study
We are doing this study to find out if an experimental drug called Zorevunersen (the study drug) can effectively reduce the frequency of major motor seizures for children who have Dravet syndrome.
Who Can Participate?
Eligibility
Children ages 2-17 who:
- Are diagnosed with Dravet syndrome based on mutations to the SCNA1 gene
- Have experienced at least 6 seizures in the 21 days before joining the study
- Do not have any spinal cord deformities
- Have never received any form of gene therapy
Age Range
2-17
Sex/Genders
Male (cisgender)
Female (cisgender)
Looking for Healthy Participants
No
What is Involved?
Description
If you choose to enroll your child in the study, they will:
Get a random assignment (like a coin flip) to either get the study drug or undergo sham procedures (phony injections that do not deliver any study drug)
Get 4 doses of the study drug or sham procedures over the course of about 40 weeks
Visit our clinic 9 times over the course of the study (approximately 60 weeks)
The study drug is given as an injection (shot) into the fluid-filled area between the tissues that cover and protect the spinal cord. All participants will have fluid (cerebrospinal fluid) samples taken from this area at four different timepoints. Participants who are assigned to get the study drug will also have the drug administered immediately after the sample is drawn. Participants who are assigned to the get the sham procedure will have no drug delivered after the sample is drawn.
Locations
Duke University Hospital
Other
Visit Timing
Weekdays
Compensation
No
Spanish Materials Available
No
Study Details
Full Title
A Multicenter, Randomized, Double-blind, Sham-controlled, Parallel Group, Phase 3 Study Evaluating the Efficacy, Safety, and Tolerability of Zorevunersen (STK-001) in Patients with Dravet Syndrome
Principal Investigator
Muhammad
Zafar
Protocol Number
PRO00117594
NCT ID
NCT06872125
Phase
III
Enrollment Status
Open to Enrollment