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Mesial Temporal Lobe EpilepsyPhase 1/2GenTLENCT06063850

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

A Phase 1/2 study testing AAV9-hSyn1-miGRIK2 in people with Mesial Temporal Lobe Epilepsy, run by UniQure Biopharma B.V. at 18 sites in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial 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). Read more about this study

Who is it for?
Ages 18 to 75 years
Conditions the study is for
Mesial Temporal Lobe Epilepsy
What is being tested?
AAV9-hSyn1-miGRIK2
Could I receive a placebo?
No, everyone receives an active treatment
How many people
12 participants
Where
18 sitesUnited States
Study dates
12 Jun 2024to Dec 2031
Phase
Phase 1/2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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.

Who cannot join

  • 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.

Trial locations and contacts

18 sites in 1 country. Contact the study team or a site near you to ask about taking part.

uniQure contact

1-866-520-1257

medinfo@uniqure.com

18 sites in 1 country

United States18 sites, 16 recruiting

  • Baylor Scott & White Medical Center

    Austin, Texas, 78735

    Victor H Gonzalez Montoya, 512-654-1234, Victor.gonzalezmontoya@bswhealth.org

    Victor H Gonzalez Montoya, MD, FAES

    Recruiting

  • Johns Hopkins School of Medicine

    Baltimore, Maryland, 21287

    Joon Kang, MD, 410-955-2822, Jkang50@jh.edu

    Joon Kang, MD

    Recruiting

  • Midatlantic Epilepsy and Sleep Center

    Bethesda, Maryland, 20817

    Completed

  • University of Alabama at Birmingham

    Birmingham, Alabama, 35294-0021

    Jennifer Pilkington, 205-934-8352, jlpilkington@uabmc.edu

    Jerzy P Szaflarski, MD, PhD

    Recruiting

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114

    Erin Donahue, 617.643.3732, ekdonahue@mgh.harvard.edu

    Mark Richardson, MD, PhD

    Recruiting

  • Cleveland Clinic

    Cleveland, Ohio, 44195

    Xiaoming Zhang, 216-445-7510, zhangx6@ccf.org

    Imad Najm, MD

    Recruiting

  • Ohio State University

    Columbus, Ohio, 43210

    James Elder, MD, 614-685-1965, james.elder@osumc.edu

    James Elder, MD

    Recruiting

  • Corewell Health

    Grand Rapids, Michigan, 49503

    David Burdette, MD, (616) 267-0301, david.burdette@corewellhealth.org

    David Burdette, MD

    Recruiting

  • Northeast Regional Epilepsy Group

    Hackensack, New Jersey, 07601

    Hardik Rana, 551-497-5000, hrana@epilepsygroup.com

    Asfi Rafiuddin

    Recruiting

  • Mayo Clinic Florida

    Jacksonville, Florida, 32224

    Megan J Gauthier, 904-953-2000, Gauthier.Megan2@mayo.edu

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

    Recruiting

  • Kansas University Medical Center

    Kansas City, Kansas, 66160

    Laura Crabtree, 913-574-0412, lcrabtree2@kumc.edu

    P Landazuri, MD

    Recruiting

  • Dartmouth Hitchcock Medical Center

    Lebanon, New Hampshire, 03756

    Anastasia Kanishcheva, MPH, CCRC, 603-650-0260, Anastasia.Kanishcheva@Hitchcock.ORG

    Barbara Jobst, MD

    Recruiting

  • Medical College of Wisconsin

    Milwaukee, Wisconsin, 53226

    Sarah Young, 414-955-0989, scyoung@mcw.edu

    Kunal Gupta, MD, PhD

    Recruiting

  • Robert Wood Johnson Hospital

    New Brunswick, New Jersey, 08901

    Christine Yohn, 908-328-4210, cy253@rwjms.rutgers.edu

    Robert E Gross, MD, PhD

    Recruiting

  • Stanford University

    Palo Alto, California, 94304

    Completed

  • Hospital of the University of Pennsylvania

    Philadelphia, Pennsylvania, 19104

    Melissa Johnston Esparza, MS, 215-614-0520, melissa.johnstonesparza@pennmedicine.upenn.edu

    Michael Gelfand, MD, PhD

    Recruiting

  • Mayo Clinic Arizona

    Phoenix, Arizona, 85054

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

    Jonathon Parker, MD

    Recruiting

  • Allegheny Health Network

    Pittsburgh, Pennsylvania, 15212

    Sarah Kimutis, 412-359-3565, sarah.kimutis@ahn.org

    Alexander Whiting, MD

    Recruiting

Other trials in United States

About this study

Official title: A Multi-center, Phase 1/2a, First-in-human (FIH) Study Investigating the Safety, Tolerability, and Efficacy of AMT-260 in Adults With Unilateral Refractory Mesial Temporal Lobe Epilepsy (MTLE) Administered Via Magnetic Resonance Imaging (MRI)-Guided Convection-enhanced Delivery (CED)

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).

Read the detailed description

In this first clinical study of AMT-260, two dose levels will be studied to find the best dose of AMT-260. All eligible participants will receive AMT-260 at one of the two dose levels (i.e., there is no placebo in this study).

AMT-260 is intended for a one-time administration, without the need to remove or destroy any part of the brain. Participation in this study would not prevent later pursuit of other treatment options.

Participants will be monitored through study site visits, telephone calls, blood tests, and questionnaires about how their seizures affect daily life. Participants will record seizures using an electronic seizure diary.

Being tested
AAV9-hSyn1-miGRIK2
Keywords
Epilepsy, Temporal Lobe, Hippocampal Sclerosis, Epileptic Syndromes
Medical subject headings
Epilepsy, Hippocampal Sclerosis, Epileptic Syndromes

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • AAV9-hSyn1-miGRIK2Genetic

    Also called AMT-260

    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.

Study groups in NCT06063850
Study groupRoleWhat they receive
AMT-260ExperimentalCohort 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).
Receives: Genetic: AAV9-hSyn1-miGRIK2

What is being measured

The primary outcome is the main question the study is designed to answer.

  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.

    Measured: 1 year

Secondary outcomes (2)

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

    Change in seizure frequency, comparing baseline to the 1 year period after AMT-260 administration.

    Measured: 1 year

  2. To evaluate the biodistribution properties of AMT-260.

    Blood, urine, saliva, and CSF samples will be collected and evaluated for vector DNA shedding at each timepoint.

    Measured: 1 year

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted3 Oct 2023
  2. Study start12 Jun 2024
  3. Primary completion30 Nov 2026 (estimated)
  4. Study completionDec 2031 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Sequential
Masking
None (open label)
Enrolment
12 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06063850
Sponsor's protocol ID
CT-AMT-260-01
Acronym
GenTLE
Status last verified
Aug 2026
First submitted
1 Sep 2023
Last updated
24 Aug 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing AAV9-hSyn1-miGRIK2 in people with Mesial Temporal Lobe Epilepsy, run by UniQure Biopharma B.V. at 18 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 24 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 18 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact uniQure (1-866-520-1257, medinfo@uniqure.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

To evaluate the safety and tolerability of AMT-260 in adults with unilateral refractory MTLE. (time frame: 1 year). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06063850.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Aug 2026.

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