ENDEAVOR Part 2
Research type
Research Study
Full title
ENDEAVOR: A Clinical Study To Evaluate The Safety And Efficacy Of ETX101, An AAV9-Delivered Gene Therapy In Infants And Children With SCN1a-Positive Dravet Syndrome
IRAS ID
1010854
Contact name
John Spoden
Contact email
Sponsor organisation
Encoded Therapeutics, Inc.
Clinicaltrials.gov Identifier
Research summary
This study will test the safety of a new gene therapy drug EXT101 and see if ETX101 shows signs of working for the treatment of Dravet Syndrome. The first part of the study was done in the United States (US) only and the second part of this study will be performed in the US, Australia and the UK. People with Dravet Syndrome do not make enough sodium channels with the cells of their brain. Sodium channels allow brain cells to communicate correctly and so people with Dravet Syndrome have brain cells that do not communicate with each other as they should, which causes the symptoms of the illness. This gene therapy will introduce genetic material, chemical structures carrying genetic information, so that the body may make more sodium channels within the brain. It is possible that the brain cells of participants with Dravet Syndrome who receive EXT101 then start to communicate with each other as they should and this will reduce/stop some of the symptoms of the illness. The study will be performed in children who are between the age of 6 and 48 months old at the time of their first study visit. It is anticipated that 18 children across the 3 countries will be enrolled in part 2 of the study. For the UK, 3 hospital sites are involved in recruiting and performing the study with patients. The study is designed so that children that take part may receive the study drug or they may receive a sham surgical treatment. For children who do receive the sham surgical treatment, they will be able to receive the study drug 1 year later. It is planned for the study to be a minimum of 5 years long and most of the visits occurring in the first year. The study involves tests to be completed first to make sure children meet the criteria to take part in the study. If able to take part, participants will receive the study drug via an injection directly into the brain. After getting ETX101 or sham, participants will have tests performed to measure their health and Dravet symptoms.
REC name
London - West London & GTAC Research Ethics Committee
REC reference
24/LO/0766
Date of REC Opinion
6 Dec 2024
REC opinion
Further Information Favourable Opinion