AMT-260 Gene Therapy Study in Adults With Unilateral Refractory Mesial Temporal Lobe Epilepsy

Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18-75
SponsorUniQure Biopharma B.V.

About this trial

The main goals of this clinical study are to learn if AMT-260 is safe and tolerable and works to reduce the frequency of seizures in adults with unilateral mesial temporal lobe epilepsy (MTLE).

Eligibility criteria

Qualifiers

Diagnosis of unilateral refractory MTLE

History of seizures with an average of ≥ 2 documented focal impaired awareness seizures or focal to bilateral tonic-clonic seizures per 30-day period over the 3 month Retrospective Period.

On a stable type and dose regimen of up to a maximum of 4 approved Anti Seizure Drugs for at least 1 month prior to the Retrospective Period.

Confirmed unilateral hippocampal pathology and concordant unilateral seizure focus

Disqualifiers

Implanted devices that would contraindicate MRI; MRI-compatible devices must be implanted ≥3 months prior to Screening (vagus nerve stimulation devices will be up to discretion of the Investigator).

Any other contraindications to general anesthesia, surgery, or intra-operative magnetic resonance imaging (MRI).

Medications that could confound clinical (e.g., antipsychotic medication and anti-viral therapy) and laboratory evaluations or could affect a participant's safety or their ability to undergo the neurosurgical procedure or comply with the procedures and study visit schedule.

Bilateral or multifocal epilepsy; or inability to confidently lateralize seizure onset.

Trial design

Treatments tested in this trial

  • AAV9-hSyn1-miGRIK2

Treatment groups

12 Participants
are divided into 1 treatment group

Sponsors and collaborators