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

NCT06063850 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: CT-AMT-260-01
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 18

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

Keywords

Epilepsy; Temporal Lobe; Hippocampal Sclerosis; Epileptic Syndromes

Outcome Measures

Primary Outcomes (1)

• To evaluate the safety and tolerability of AMT-260 in adults with unilateral refractory MTLE.

  Occurrence of Adverse Events during the period of 1 year after AMT-260 administration, including seriousness, severity, and causal relationship to AMT-260.

  1 year

Secondary Outcomes (2)

• To evaluate first signs of efficacy of AMT-260.

  1 year

• To evaluate the biodistribution properties of AMT-260.

  1 year

Other Outcomes (1)

• To evaluate the long-term safety and efficacy of AMT-260 in adults with unilateral refractory MTLE.

  up to 5 years

Study Arms (1)

AMT-260

EXPERIMENTAL

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).

Genetic: AAV9-hSyn1-miGRIK2

Interventions

AAV9-hSyn1-miGRIK2 GENETIC

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.

Also known as: AMT-260

AMT-260

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

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 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.

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 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 icta 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.
• Inadequate vaccination status (including flu shots and other relevant immunizations such as COVID-19 per local guidelines and regulations).

Sponsors & Collaborators

• UniQure Biopharma B.V.: lead

Study Sites (18)

University of Alabama at Birmingham

Birmingham, Alabama, 35294-0021, United States

RECRUITING

Mayo Clinic Arizona

Phoenix, Arizona, 85054, United States

RECRUITING

Stanford University

Palo Alto, California, 94304, United States

RECRUITING

Mayo Clinic Florida

Jacksonville, Florida, 32224, United States

RECRUITING

Kansas University Medical Center

Kansas City, Kansas, 66160, United States

RECRUITING

Johns Hopkins School of Medicine

Baltimore, Maryland, 21287, United States

RECRUITING

Midatlantic Epilepsy and Sleep Center

Bethesda, Maryland, 20817, United States

RECRUITING

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

RECRUITING

Corewell Health

Grand Rapids, Michigan, 49503, United States

RECRUITING

Dartmouth Hitchcock Medical Center

Lebanon, New Hampshire, 03756, United States

RECRUITING

Northeast Regional Epilepsy Group

Hackensack, New Jersey, 07601, United States

RECRUITING

Robert Wood Johnson Hospital

New Brunswick, New Jersey, 08901, United States

RECRUITING

Cleveland Clinic

Cleveland, Ohio, 44195, United States

RECRUITING

Ohio State University

Columbus, Ohio, 43210, United States

RECRUITING

Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

Allegheny Health Network

Pittsburgh, Pennsylvania, 15212, United States

RECRUITING

Baylor Scott & White Medical Center

Austin, Texas, 78735, United States

RECRUITING

Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States

NOT YET RECRUITING

Related Links

• : Sponsor web page about this Phase 1/2a clinical study of AMT-260

MeSH Terms

Conditions

Epilepsy; Hippocampal Sclerosis; Epileptic Syndromes

Condition Hierarchy (Ancestors)

Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Malformations of Cortical Development, Group I; Malformations of Cortical Development; Nervous System Malformations; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Clinical Development Lead

  uniQure France SAS

  STUDY DIRECTOR

Central Study Contacts

uniQure

CONTACT

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

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 1, 2023
• First Posted: October 3, 2023
• Study Start: June 12, 2024
• Primary Completion (Estimated): November 30, 2026
• Study Completion (Estimated): December 1, 2031
• Last Updated: December 10, 2025

Record last verified: 2025-12

Locations

US (18)