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RecruitingNCT06063850GenTLEUpdated Aug 24, 2026

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

A Phase 1/2 interventional study of AAV9-hSyn1-miGRIK2 in Mesial Temporal Lobe Epilepsy, sponsored by UniQure Biopharma B.V.. Recruiting at 18 sites in United States. Open to participants aged 18 Years to 75 Years. Per ClinicalTrials.gov, last updated 2026-08-24.

Sponsored by UniQure Biopharma B.V. · Phase 1/2, Interventional, and Treatment

From the registry’s dates

  • Started Jun 2024; still recruiting 2 years 5 months later.
Phase
Phase 1/2
Study type
Interventional
Enrollment
12
Allocation
Not applicable
Ages
18 Years to 75 Years
Sex
All
01

Study 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 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.

02

Conditions studied

  • Mesial Temporal Lobe Epilepsy

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Keywords

  • Epilepsy
  • Temporal Lobe
  • Hippocampal Sclerosis
  • Epileptic Syndromes
03

In context

Epilepsy

1,848 studies on the registry are indexed under Epilepsy; 422 are open to participants now.

This study's planned enrollment of 12 is below the median of 50 across 1,239 interventional studies indexed under Epilepsy.

Browse Epilepsy studies →

Lead sponsor

UniQure Biopharma B.V. is the lead sponsor of 8 studies on the registry; 2 are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years to 75 Years
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  • Diagnosis of unilateral 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 (WOCBP) and fertile male subjects must be willing and able to use highly effective methods of consistently and correctly throughout the study.
  • For WOCBP only: Negative pregnancy test.

Exclusion criteria

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 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.
Terms in these criteria, in plain language(4)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Relapsed and refractory

Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.

Childbearing potential

Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).

Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.

Contraception

Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.

Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.

Contraindication

A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.

05

Study design

Phase
Phase 1 / Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Sequential assignment
Masking
None (open label)
Enrollment
12 participants (estimated)

Study 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).

    Genetic: AAV9-hSyn1-miGRIK2

Interventions

  • GeneticAAV9-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.

    Also known as: AMT-260

06

What researchers measure

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.

    Time frame: 1 year

Secondary outcomes

  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.

    Time frame: 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.

    Time frame: 1 year

Other outcomes

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

    Occurrence of Adverse Events during the period of 1 to 5 years after AMT-260 administration, including seriousness.

    Time frame: up to 5 years

07

Study locations

16 of 18 sites recruiting
08

References and documents

09

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reached

Started Jun 2024 (actual). Primary completion is estimated for Nov 2026, 29 months after the start.

How these figures are worked out, and which studies are left out of them.

Registered
Sep 1, 2023
First posted
Oct 3, 2023
StartActual
Jun 12, 2024
Primary completionEst.
Nov 30, 2026
CompletionEst.
Dec 2031
Last update
Aug 24, 2026
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Aug 24, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT06063850
Lead sponsor
UniQure Biopharma B.V.
Responsible party
Sponsor
First posted
Oct 3, 2023
Start date
Jun 12, 2024
Primary completion
Nov 30, 2026 (estimated)
Completion
Dec 2031 (estimated)
Last update
Aug 24, 2026

Study contacts

uniQure
Contact
medinfo@uniqure.com
1-866-520-1257
Clinical Development Lead
study director · uniQure France SAS

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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