AMT-260 Gene Therapy Study in Adults With Unilateral Refractory Mesial Temporal Lobe Epilepsy
Launched by UNIQURE BIOPHARMA B.V. · Sep 25, 2023
Trial Information
Current as of July 09, 2025
Recruiting
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.
About Uniqure Biopharma B.V.
uniQure Biopharma B.V. is a leading gene therapy company focused on developing innovative treatments for severe diseases, leveraging its proprietary AAV vector platform. With a commitment to advancing the field of genetic medicine, uniqure aims to provide transformative therapies for patients with unmet medical needs, particularly in the areas of rare genetic disorders and neurological conditions. The company is dedicated to rigorous research and development, fostering collaboration with academic institutions and industry partners to bring its groundbreaking therapies from the laboratory to clinical practice. Through its pioneering efforts, uniqure strives to improve the quality of life for patients worldwide.
Contacts
Jennifer Cobb
Immunology at National Institute of Allergy and Infectious Diseases (NIAID)
Locations
Philadelphia, Pennsylvania, United States
Columbus, Ohio, United States
Boston, Massachusetts, United States
Lebanon, New Hampshire, United States
Milwaukee, Wisconsin, United States
Boston, Massachusetts, United States
Palo Alto, California, United States
Cleveland, Ohio, United States
Baltimore, Maryland, United States
Kansas City, Kansas, United States
New Brunswick, New Jersey, United States
Jacksonville, Florida, United States
Phoenix, Arizona, United States
Hackensack, New Jersey, United States
Pittsburgh, Pennsylvania, United States
Bethesda, Maryland, United States
Grand Rapids, Michigan, United States
Birmingham, Alabama, United States
Austin, Texas, United States
Patients applied
Trial Officials
Clinical Development Lead
Study Director
uniQure France SAS
Timeline
First submit
Trial launched
Trial updated
Estimated completion
Not reported