Gene therapy for adults with refractory mesial temporal lobe epilepsy

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This clinical study aims to evaluate the safety, tolerability, and efficacy of AMT-260, a gene therapy, in reducing seizure frequency in adults diagnosed with unilateral mesial temporal lobe epilepsy (MTLE). Participants will receive one of two dose levels of AMT-260, with no placebo involved, and will be closely monitored through site visits, phone calls, blood tests, and questionnaires. The study will also involve participants recording their seizures using an electronic diary to assess the impact on their daily lives.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* 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.

Exclusion Criteria:

* 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's running

• University of Alabama at Birmingham
  Birmingham, Alabama, United States Recruiting
• Mayo Clinic Arizona
  Phoenix, Arizona, United States Recruiting
• Mayo Clinic Florida
  Jacksonville, Florida, United States Recruiting
• Kansas University Medical Center
  Kansas City, Kansas, United States Recruiting
• Johns Hopkins School of Medicine
  Baltimore, Maryland, United States Recruiting
• Massachusetts General Hospital
  Boston, Massachusetts, United States Recruiting

Study contacts

• Study coordinator: uniQure
• Email: medinfo@uniqure.com
• Phone: 1-866-520-1257

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Using Deep Brain Stimulation to Preserve Memory in Epilepsy

  This study is testing whether a new brain stimulation technique can help people with Mesial Temporal Lobe Epilepsy have fewer seizures and better memory.

  Recruiting at 2 sites The University of Texas Health Science Center, Houston · Rochester, Minnesota and 1 other locations · NCT05608408

• Neural cell therapy for drug-resistant epilepsy

  This study is testing a new treatment that involves injecting special nerve cells into the brains of people with hard-to-treat epilepsy to see if it can help reduce their seizures.

  Recruiting at 36 sites Phase 3 Neurona Therapeutics · Birmingham, Alabama and 35 other locations · NCT05135091

• Gene therapy for classic Fabry disease

  This study is testing a new gene therapy called AMT-191 to see if it can help people with classic Fabry disease by improving their enzyme levels and overall health.

  Recruiting at 10 sites Phase 1/2 UniQure Biopharma B.V. · Birmingham, Alabama and 9 other locations · NCT06270316

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.