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Gene therapy breakthrough targets severe epilepsy in first human trial

NCT ID NCT06063850

Disease control Sponsor: UniQure Biopharma B.V. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Aug 25, 2026 · Updated 4 times

Summary

This early-stage study tests a new gene therapy called AMT-260 in 12 adults with a severe form of epilepsy that doesn't respond to standard medications. The treatment is delivered directly into the brain using MRI guidance. The main goals are to check safety and see if it can reduce seizure frequency over one year.

Why investors are watching

uniQure is testing AMT-260, a gene therapy for adults with a severe form of epilepsy that does not respond to standard drugs. The trial is early stage, with only 12 participants, and it is the first time this treatment is given to humans. For a small company like uniQure, this readout matters because it is one of their key pipeline programs, and the result will show whether the therapy is safe enough and has any effect on seizure frequency.

If it works: If the trial shows AMT-260 is safe and reduces seizures, uniQure could advance the drug to later-stage testing. That would validate their approach and strengthen their pipeline with a potential new treatment for a condition with few options.

If it fails: Early-stage trials often fail on safety or efficacy, and this one has a small number of participants, so results may be unclear. A failure or delay could set back uniQure's development plans for AMT-260 and hurt investor confidence in the company's pipeline.

AI-written from the trial record. Speculative, and not investment advice.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 12 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jun 2024

Expected to finish

Dec 2031

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 75 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

epilepsy Epileptic Syndromes Hippocampal Sclerosis mesial temporal lobe epilepsy with hippocampal sclerosis temporal lobe epilepsy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Mesial temporal lobe epilepsy

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    16 sites. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Allegheny Health Network

    RECRUITING

    Pittsburgh, Pennsylvania, 15212, United States

  • Baylor Scott & White Medical Center

    RECRUITING

    Austin, Texas, 78735, United States

  • Cleveland Clinic

    RECRUITING

    Cleveland, Ohio, 44195, United States

  • Corewell Health

    RECRUITING

    Grand Rapids, Michigan, 49503, United States

  • Dartmouth Hitchcock Medical Center

    RECRUITING

    Lebanon, New Hampshire, 03756, United States

  • Hospital of the University of Pennsylvania

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • Johns Hopkins School of Medicine

    RECRUITING

    Baltimore, Maryland, 21287, United States

  • Kansas University Medical Center

    RECRUITING

    Kansas City, Kansas, 66160, United States

  • Massachusetts General Hospital

    RECRUITING

    Boston, Massachusetts, 02114, United States

  • Mayo Clinic Arizona

    RECRUITING

    Phoenix, Arizona, 85054, United States

  • Mayo Clinic Florida

    RECRUITING

    Jacksonville, Florida, 32224, United States

  • Medical College of Wisconsin

    RECRUITING

    Milwaukee, Wisconsin, 53226, United States

  • Midatlantic Epilepsy and Sleep Center

    COMPLETED

    Bethesda, Maryland, 20817, United States

  • Northeast Regional Epilepsy Group

    RECRUITING

    Hackensack, New Jersey, 07601, United States

  • Ohio State University

    RECRUITING

    Columbus, Ohio, 43210, United States

  • Robert Wood Johnson Hospital

    RECRUITING

    New Brunswick, New Jersey, 08901, United States

  • Stanford University

    COMPLETED

    Palo Alto, California, 94304, United States

  • University of Alabama at Birmingham

    RECRUITING

    Birmingham, Alabama, 35294-0021, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.