What side effects are associated with this type of intervention?

Common treatments for Mesial Temporal Lobe Epilepsy (MTLE) include antiepileptic drugs (AEDs), surgical interventions, and emerging therapies like gene therapy delivered via MRI-guided Convection-enhanced Delivery (CED). Side effects of AEDs can include dizziness, fatigue, weight gain, and cognitive impairment. Surgical treatments may lead to complications such as infection, bleeding, and neurological deficits. For targeted delivery methods like CED, potential side effects could include localized infection, inflammation, and damage to surrounding tissues. The AMT-260 trial specifically aims to assess the safety, tolerability, and efficacy of such targeted gene therapy in adults with MTLE.

What prior FDA approvals exist?

There is limited information on FDA-approved treatments specifically using MRI-guided Convection-enhanced Delivery (CED) for Mesial Temporal Lobe Epilepsy (MTLE). Traditional FDA-approved treatments for MTLE typically include antiseizure medications such as levetiracetam, carbamazepine, and lacosamide. These medications are generally administered orally and do not involve targeted delivery methods like CED. For more advanced or refractory cases, surgical interventions such as resective surgery or laser interstitial thermal therapy (LITT) may be considered. The AMT-260 study represents a novel approach by investigating the safety, tolerability, and efficacy of gene therapy delivered via MRI-guided CED, which is not yet a standard FDA-approved treatment for MTLE.

What other types of research exist?

Promising novel alternatives to AMT-260 for MTLE patients include: 1) Responsive Neurostimulation (RNS), which involves implanting a device that monitors brain activity and delivers electrical stimulation to prevent seizures. 2) Deep Brain Stimulation (DBS), targeting specific brain regions to modulate neural activity and reduce seizure frequency. 3) Transcranial Magnetic Stimulation (TMS), a non-invasive method using magnetic fields to stimulate nerve cells in the brain. These alternatives have shown efficacy in clinical studies and may offer viable options for MTLE treatment in 2024.

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Gene Therapy

Gene Therapy for Epilepsy

Phase 1 & 2

Recruiting

Research Sponsored by uniQure France SAS

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Adult, 18-65 years of age, capable of giving informed consent

Diagnosis of unilateral refractory MTLE for ≥360 days, confirmed by an Epilepsy Monitoring Unit

Must not have

Any psychogenic nonepileptic seizures within the last year

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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 1 year

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new treatment called AMT-260 for adults with a type of epilepsy that doesn't respond to usual treatments. The treatment is delivered directly to the affected part of the brain using MRI guidance. The goal is to see if this method can better control seizures and improve safety and tolerability.

Who is the study for?

Adults aged 18-65 with unilateral refractory mesial temporal lobe epilepsy, having at least 2 seizures per month and on stable anti-seizure drugs. They must have normal cognitive function, no major unrelated neurosurgery history, and agree to use birth control. MRI-compatible devices are okay if implanted over 3 months ago.

What is being tested?

The trial tests AMT-260 gene therapy for safety and effectiveness in reducing seizures in adults with MTLE. It involves injecting the treatment directly into the brain using MRI-guided techniques while monitoring patients' responses.

What are the potential side effects?

Potential side effects aren't specified but may include typical risks associated with gene therapies such as immune reactions, headaches or discomfort from the delivery method (MRI-guided injection), and general anesthesia-related risks.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am between 18 and 65 years old and can make my own medical decisions.

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I have been diagnosed with hard-to-treat epilepsy in one side of my brain for over a year.

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I have had at least 2 seizures a month in the last 3 months.

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My cognitive function score is 26 or higher.

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My brain function is normal and matches my MRI and PET scan results.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have had seizures not caused by epilepsy in the past year.

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I have had brain surgery for reasons not related to my current disease.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 1 year

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~1 year

This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 year for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Adverse Events

Secondary study objectives

AAV9 vector in human biofluid (copies/qPCR reaction).

Patient Health Questionnaire (PHQ9)

Pittsburgh Sleep Quality Index (PSQI)

+4 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Experimental Treatment

Group I: Cohort 2: AMT-260 adapted doseExperimental Treatment1 Intervention

N# of treated - 6 Dose is dependent on the DSMB recommendation.

Group II: Cohort 1: AMT-260 starting doseExperimental Treatment1 Intervention

N# of treated - 6

0 / 7Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Mesial Temporal Lobe Epilepsy (MTLE) often involve targeted delivery methods like MRI-guided Convection-enhanced Delivery (CED), as seen in the AMT-260 study. This approach allows for precise administration of therapeutic agents directly to the affected brain regions, ensuring higher local drug concentrations and minimizing systemic exposure. This is particularly important for MTLE patients as it can effectively reduce seizure frequency and severity by directly modulating the hyperexcitable neuronal circuits in the mesial temporal lobe, while also minimizing side effects and improving overall treatment outcomes.