STK-001-DS-301 (Dravet Syndrome) - Clinical Trial

¿Cuál es el Propósito de este estudio?

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.

¿Cuál es la Condición que se está estudiando?

Dravet Syndrome

¿Quién puede participar en el Estudio?

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

For more information, contact the study team at gloria.pinero@duke.edu.

Grupo etario

Niños

¿Qué Implica?

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.

Detalles del Estudio

Título Completo

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

Investigador Principal