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Gene Therapy
Gene Therapy for Epilepsy (GenTLE Trial)
Phase 1 & 2
Recruiting
Research Sponsored by UniQure Biopharma B.V.
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 belowSelect...
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.
Select...
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:Select...
I have had seizures not caused by epilepsy in the past year.
Select...
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
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
To evaluate the safety and tolerability of AMT-260 in adults with unilateral refractory MTLE.
Secondary study objectives
To evaluate first signs of efficacy of AMT-260.
To evaluate the biodistribution properties of AMT-260.
Awards & Highlights
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Trial Design
1Treatment groups
Experimental Treatment
Group I: AMT-260Experimental Treatment1 Intervention
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).
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.
Find a Location
Who is running the clinical trial?
UniQure Biopharma B.V.Lead Sponsor
11 Previous Clinical Trials
252 Total Patients Enrolled
uniQure France SASLead Sponsor
Andreas BortaStudy DirectoruniQure France SAS