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

Recruiting now · Phase 1/Phase 2

Conditions studied: Mesial Temporal Lobe Epilepsy

In brief

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).

Key facts

Study ID

NCT06063850

Run by

UniQure Biopharma B.V.

People needed

12

Starts

2024-06-12

Expected to finish

2031-12-01

Last updated by the study team

2026-07-29

Who can join

Age: 18 and older, up to 75. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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
• 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.

You may not qualify if…

• 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.
• 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.
• MRI evidence of epileptogenic, extra-temporal lesions, or dual temporal lobe pathology.
• Status epilepticus within the year prior to Screening.

Where it is running

• Medical College of Wisconsin — Milwaukee, Wisconsin, United States (enrolling)
• Mayo Clinic Arizona — Phoenix, Arizona, United States (enrolling)
• Stanford University — Palo Alto, California, United States (enrolling)
• Mayo Clinic Florida — Jacksonville, Florida, United States (enrolling)
• Kansas University Medical Center — Kansas City, Kansas, United States (enrolling)
• Johns Hopkins School of Medicine — Baltimore, Maryland, United States (enrolling)
• University of Alabama at Birmingham — Birmingham, Alabama, United States (enrolling)
• Massachusetts General Hospital — Boston, Massachusetts, United States (enrolling)
• Corewell Health — Grand Rapids, Michigan, United States (enrolling)
• Dartmouth Hitchcock Medical Center — Lebanon, New Hampshire, United States (enrolling)
• Northeast Regional Epilepsy Group — Hackensack, New Jersey, United States (enrolling)
• Robert Wood Johnson Hospital — New Brunswick, New Jersey, United States (enrolling)
• Cleveland Clinic — Cleveland, Ohio, United States (enrolling)
• Ohio State University — Columbus, Ohio, United States (enrolling)
• Hospital of the University of Pennsylvania — Philadelphia, Pennsylvania, United States (enrolling)
• Allegheny Health Network — Pittsburgh, Pennsylvania, United States (enrolling)
• Baylor Scott & White Medical Center — Austin, Texas, United States (enrolling)
• Midatlantic Epilepsy and Sleep Center — Bethesda, Maryland, United States

Full record on ClinicalTrials.gov

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