Lentiviral Gene Therapy for Epilepsy

Drug Resistant Epilepsy Clinical Trial

Official title: Phase I/IIa, First-in-human, Open-label, Single-site Trial of In-vivo Lentiviral Engineered Potassium (K+) Channel (EKC) Gene Therapy for Refractory Epilepsy

Status Not yet recruiting

Clinical Trial Summary

This is a phase I/IIa clinical trial investigating the safety of a lentiviral epilepsy gene therapy using an engineered potassium channel in patients with refractory epilepsy.

Clinical Trial Description

Epilepsy affects about 1% of the population. One third of affected individuals continue to have seizures despite optimal medication. The only realistic prospect of seizure freedom, feasible in very few cases, is surgery to remove the brain area where seizures arise.

Patients with refractory neocortical epilepsy who are being evaluated for surgical resection of the seizure focus will be invited to join the trial. The non-integrating lentiviral vector, which has been engineered to deliver an engineered potassium channel, will be administered via intracerebral infusion to the area scheduled for resection.

The primary objective in this study is to test the safety of the lentiviral gene therapy treatment, including the surgical procedures required for vector administration. Secondary objectives will look at delayed onset adverse events and indicators of efficacy. ;

Related Conditions & MeSH terms

• Drug Resistant Epilepsy
• Epilepsy

• NCT number: NCT04601974
• Study type: Interventional
• Source: University College, London
• Contact: Senior Clinical Project Manager
• Phone: +44(0)2031088954
• Email: ekctrial@ucl.ac.uk
• Status: Not yet recruiting
• Phase: Phase 1/Phase 2
• Start date: September 2024
• Completion date: September 2032