A First-in-human Study of a New Gene Therapy (AMT-260) in Adults with Unilateral Refractory Mesial Temporal Lobe Epilepsy (MTLE)
Purpose
You are being invited to participate in a study about mesial temporal lobe epilepsy (MTLE) on only 1 side (unilateral) of the brain that has not responded to medication (refractory).
The study will test AMT-260, which is a new gene therapy under investigation for use in treating people like you with MTLE. It is not yet approved as a drug for treating MTLE. It can only be used in a study like this one. This is the first time this gene therapy will be used in humans. It has previously been studied in animals.
AMT-260 is a gene therapy designed to reduce the electrical signals between nerve cells (neurons) in the parts of your brain where nerve cells are abnormally connected. This reduces the extra electrical signals that happen at the same time and is intended to reduce your seizure activity.
The overall goals of this study are:
- To observe whether participants have any side effects from AMT-260 or from the procedure to deliver AMT-260 into the brain and how well participants tolerate any side effects that may occur.
- To measure if participants have changes in:
- frequency of their seizures
- how their seizures affect their life
- how the human body takes up and removes AMT-260
- To find the best dose of AMT-260 for future participants to ensure clinical benefit with minimal or no side effects.
Could this study be right for you?
Adult, 18-65 years of age, inclusive, capable of giving informed consent.
Written informed consent is given freely after the nature of the study has been explained to the participant and the completion of data disclosure.
Willingness to undergo necessary surgical procedures.
Diagnosis of unilateral refractory MTLE for ≥360 days, confirmed by an Epilepsy Monitoring Unit.
Additional Eligibility Criteria to be assessed by neurosurgery team, and as described in the study protocol.