Status:

RECRUITING

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

Lead Sponsor:

UniQure Biopharma B.V.

Conditions:

Mesial Temporal Lobe Epilepsy

Eligibility:

All Genders

18-75 years

Phase:

PHASE1

PHASE2

Brief Summary

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).

Detailed Description

In this first clinical study of AMT-260, two dose levels will be studied to find the best dose of AMT-260. All eligible participants will receive AMT-260 at one of the two dose levels (i.e., there is ...

Eligibility Criteria

Inclusion

  • 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 in the 3 months prior to screening.
  • On a stable type and dose regimen of up to a maximum of 4 approved Anti Seizure Drugs for at least 6 months prior to screening.
  • Confirmed unilateral hippocampal pathology and concordant unilateral seizure focus
  • No evidence of focal neurocognitive dysfunction, inconsistent with disease pathology- related MRI and/or (18F)FDG-PET findings.
  • Women of childbearing potential (WOCBP) and fertile male subjects must be willing and able to use highly effective methods of birth control consistently and correctly throughout the study.
  • For WOCBP only: Negative pregnancy test.

Exclusion

  • 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 for generalized anesthesia or surgery.
  • 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.
  • Any seizures with contralateral or extra-temporal icta onset captured on EEG.
  • Dementia or other progressive neurological disorders and progressive brain lesions.
  • Previous major disease-unrelated neurosurgical intervention due to intracranial tumor, trauma, or bleeding and/or history of previous intracranial surgery for treatment of epileptic seizures, not including diagnostic stereo-EEG.
  • Magnetic resonance imaging evidence of epileptogenic, extra-temporal lesions, or dual temporal lobe pathology.
  • Inadequate vaccination status (including flu shots and other relevant immunizations such as COVID-19 per local guidelines and regulations).

Key Trial Info

Start Date :

June 12 2024

Trial Type :

INTERVENTIONAL

Allocation :

ESTIMATED

End Date :

December 1 2031

Estimated Enrollment :

12 Patients enrolled

Trial Details

Trial ID

NCT06063850

Start Date

June 12 2024

End Date

December 1 2031

Last Update

December 10 2025

Active Locations (18)

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Page 1 of 5 (18 locations)

1

University of Alabama at Birmingham

Birmingham, Alabama, United States, 35294-0021

2

Mayo Clinic Arizona

Phoenix, Arizona, United States, 85054

3

Stanford University

Palo Alto, California, United States, 94304

4

Mayo Clinic Florida

Jacksonville, Florida, United States, 32224