EXPEDITION

• Research type

  Research Study

• Full title

  EXPEDITION: A CLINICAL STUDY TO EVALUATE THE SAFETY AND EFFICACY OF ETX101, AN AAV9-DELIVERED GENE THERAPY IN CHILDREN WITH SCN1A-POSITIVE DRAVET SYNDROME

• IRAS ID

  1008195

• Contact name

  John Spoden

• Contact email

  jspoden@encoded.com

• Sponsor organisation

  Encoded Therapeutics, Inc

• Clinicaltrials.gov Identifier

  NCT06283212

• Research summary

  This study, called the EXPEDITION study, is to test the safety of a new gene therapy drug, ETX101 for the treatment of Dravet Syndrome. The study will also look to see if the drug has signs that it works in treating Dravet Syndrome. People with Dravet Syndrome do not make enough sodium channels within the cells of their brain. Sodium channels allow brain cells to communicate correctly and so people with Dravet Syndrome brain cells don’t communicate with each other as they should causing the symptoms of the disease.
  This gene therapy will introduce genetic material, chemical structures carrying genetic information, so that the body makes more sodium channels within the brain. It is possible that these brain cells will then communicate with each other as they should and reduce/eliminate some of the symptoms of Dravet syndrome.
  The study will be performed in children aged 6 months to 4 years old and it is planned that 4 children will be enrolled in the study. EXPEDITION will be performed in the UK, with 3 hospital sites involved in recruiting patients.
  The plan for the study is that it will be 5 years long with most of the visits occurring in the first year. Over this study there is approximately 5 in hospital stays ranging from 1-4 days each.
  Potential participants would complete a number of screening assessments to ensure they are eligible to take part in the study. For participants who are eligible, they will receive the study drug on day 1 via an injection of the drug directly into the brain, by what is called an ICV injection. Once on the study, a number of assessments will be performed with the participants including taking/collecting blood, urine, stool, saliva samples, as well as fluid from the spinal cord to check participants health and see how/if the drug is working. Assessments to measure blood pressure and looking at the performance of the heart as well as neurological assessments also to check participants health and the effect of the drug.

• REC name

  London - West London & GTAC Research Ethics Committee

• REC reference

  23/LO/0706

• Date of REC Opinion

  11 Jan 2024

• REC opinion

  Further Information Favourable Opinion