Recruiting
Phase 1
Phase 2

AMT-260

Sponsor:

UniQure Biopharma B.V.

Code:

NCT06063850

Conditions

Mesial Temporal Lobe Epilepsy

Eligibility Criteria

Sex: All

Age: 18 - 70+

Healthy Volunteers: Not accepted

Interventions

AAV9-hSyn1-miGRIK2

Study Details

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).

Conditions

Mesial Temporal Lobe Epilepsy

Study ID

NCT06063850

Start date

Jun 12, 2024

Status verified date

Aug, 2026

Completion date

Dec, 2031

Anticipated

Primary completion date

Nov 30, 2026

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18 - 70+

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

12 participants

Anticipated

Intervention Model

Sequential

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: AMT-260

Cohort 1: AMT-260 starting dose (1.0x 10E12 gc/mL). Cohort 2: AMT-260 adapted dose (6.0x 10E11 gc/mL or 3.0x 10E12 gc/mL).

Interventions

AAV9-hSyn1-miGRIK2

AMT-260 is an AAV9 gene therapy product that locally delivers miRNA silencing technology to target the GRIK2 gene and suppress aberrantly expressed GluK2 containing kainate receptors. Intervention will be a one-time intracerebral administration of AMT-260.

Primary outcome measure

  • To evaluate the safety and tolerability of AMT-260 in adults with unilateral refractory MTLE. [ Time Frame: 1 year ]

Central Contacts and Locations

Central contacts

uniQure

1-866-520-1257medinfo@uniqure.com

Locations

University of Alabama at Birmingham

Recruiting

Birmingham, Alabama, United States, 35294-0021

Contacts

Jennifer Pilkington

205-934-8352jlpilkington@uabmc.edu

Principal Investigator:

Jerzy P Szaflarski, MD, PhD

Mayo Clinic Arizona

Recruiting

Phoenix, Arizona, United States, 85054

Contacts

Jonathon Parker, MD

480-342-2906Parker.Jonathon@mayo.edu

Principal Investigator:

Jonathon Parker, MD

Mayo Clinic Florida

Recruiting

Jacksonville, Florida, United States, 32224

Contacts

Megan J Gauthier

904-953-2000Gauthier.Megan2@mayo.edu

Alicia Kissinger-Knox, PhD

904-953-2000Kissinger-Knox.Alicia@mayo.edu

Principal Investigator:

Brin Freund, MD

Kansas University Medical Center

Recruiting

Kansas City, Kansas, United States, 66160

Contacts

Laura Crabtree

913-574-0412lcrabtree2@kumc.edu

Principal Investigator:

P Landazuri, MD

Johns Hopkins School of Medicine

Recruiting

Baltimore, Maryland, United States, 21287

Contacts

Joon Kang, MD

410-955-2822Jkang50@jh.edu

Principal Investigator:

Joon Kang, MD

Massachusetts General Hospital

Recruiting

Boston, Massachusetts, United States, 02114

Contacts

Erin Donahue

617.643.3732ekdonahue@mgh.harvard.edu

Principal Investigator:

Mark Richardson, MD, PhD

Corewell Health

Recruiting

Grand Rapids, Michigan, United States, 49503

Contacts

David Burdette, MD

(616) 267-0301david.burdette@corewellhealth.org

Principal Investigator:

David Burdette, MD

Dartmouth Hitchcock Medical Center

Recruiting

Lebanon, New Hampshire, United States, 03756

Contacts

Anastasia Kanishcheva, MPH, CCRC

603-650-0260Anastasia.Kanishcheva@Hitchcock.ORG

Principal Investigator:

Barbara Jobst, MD

Northeast Regional Epilepsy Group

Recruiting

Hackensack, New Jersey, United States, 07601

Contacts

Hardik Rana

551-497-5000hrana@epilepsygroup.com

Principal Investigator:

Asfi Rafiuddin

Robert Wood Johnson Hospital

Recruiting

New Brunswick, New Jersey, United States, 08901

Contacts

Christine Yohn

908-328-4210cy253@rwjms.rutgers.edu

Principal Investigator:

Robert E Gross, MD, PhD

Cleveland Clinic

Recruiting

Cleveland, Ohio, United States, 44195

Contacts

Xiaoming Zhang

216-445-7510zhangx6@ccf.org

Principal Investigator:

Imad Najm, MD

Ohio State University

Recruiting

Columbus, Ohio, United States, 43210

Contacts

James Elder, MD

614-685-1965james.elder@osumc.edu

Principal Investigator:

James Elder, MD

Hospital of the University of Pennsylvania

Recruiting

Philadelphia, Pennsylvania, United States, 19104

Contacts

Melissa Johnston Esparza, MS

215-614-0520melissa.johnstonesparza@pennmedicine.upenn.edu

Principal Investigator:

Michael Gelfand, MD, PhD

Allegheny Health Network

Recruiting

Pittsburgh, Pennsylvania, United States, 15212

Contacts

Sarah Kimutis

412-359-3565sarah.kimutis@ahn.org

Principal Investigator:

Alexander Whiting, MD

Baylor Scott & White Medical Center

Recruiting

Austin, Texas, United States, 78735

Contacts

Victor H Gonzalez Montoya

512-654-1234Victor.gonzalezmontoya@bswhealth.org

Principal Investigator:

Victor H Gonzalez Montoya, MD, FAES

Medical College of Wisconsin

Recruiting

Milwaukee, Wisconsin, United States, 53226

Contacts

Sarah Young

414-955-0989scyoung@mcw.edu

Principal Investigator:

Kunal Gupta, MD, PhD

More Information

Sponsor

UniQure Biopharma B.V.

Last update posted

Aug 24, 2026

Last verified

Aug, 2026

Keywords

  • Epilepsy
  • Temporal Lobe
  • Hippocampal Sclerosis
  • Epileptic Syndromes

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-23. This information was provided to ClinicalTrials.gov by UniQure Biopharma B.V. on 2026-08-24.