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

Launched by UNIQURE BIOPHARMA B.V. · Sep 25, 2023

Keywords

ClinConnect Summary

The AMT-260 Gene Therapy Study is a clinical trial that aims to test a new treatment called AMT-260 for adults suffering from a specific type of epilepsy known as unilateral refractory mesial temporal lobe epilepsy (MTLE). This condition means that patients have seizures that do not respond well to standard medications. The study will evaluate how safe and effective this new gene therapy is when delivered using a special imaging technique called MRI-guided convection-enhanced delivery.

To participate in this study, individuals must be between 18 and 65 years old and have been diagnosed with MTLE for at least one year. They should have a history of experiencing at least two seizures each month and have been on a stable medication regimen for the past three months. Participants will undergo procedures and evaluations to ensure their safety throughout the trial. It’s important for potential participants to know that they will need to use birth control during the study and for some time after, to prevent pregnancy. Overall, this trial offers hope for those who have not found relief from their epilepsy through other treatments.

Gender

ALL

Eligibility criteria

• 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 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 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 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 ictal 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.