RECRUITING

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

Conditions

Mesial Temporal Lobe Epilepsy Epilepsy Temporal Lobe Hippocampal Sclerosis Epileptic Syndromes

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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).

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)

Quick Facts

Study Start:2024-06-12

Study Completion:2031-12

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06063850

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years to 65 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion 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 in the 3 months prior to screening. * On a stable type and dose regimen of up to a maximum of 4 approved Anti Seizure Drugs for at least 6 months prior to screening. * 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.	* 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 for generalized anesthesia or surgery. * 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. * Any seizures with contralateral or extra-temporal ictal onset captured on EEG. * 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. * Magnetic resonance imaging evidence of epileptogenic, extra-temporal lesions, or dual temporal lobe pathology.

Inclusion Criteria

Exclusion 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 in the 3 months prior to screening.
* On a stable type and dose regimen of up to a maximum of 4 approved Anti Seizure Drugs for at least 6 months prior to screening.
* 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.

* 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 for generalized anesthesia or surgery.
* 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.
* Any seizures with contralateral or extra-temporal ictal onset captured on EEG.
* 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.
* Magnetic resonance imaging evidence of epileptogenic, extra-temporal lesions, or dual temporal lobe pathology.

Contacts and Locations

Study Contact

uniQure

CONTACT

1-866-520-1257

medinfo@uniqure.com

Principal Investigator

Clinical Development Lead

STUDY_DIRECTOR

uniQure France SAS

Study Locations (Sites)

University of Alabama at Birmingham

Birmingham, Alabama, 35294-0021

United States

Mayo Clinic Arizona

Phoenix, Arizona, 85054

United States

Stanford University

Palo Alto, California, 94304

United States

Mayo Clinic Florida

Jacksonville, Florida, 32224

United States

Kansas University Medical Center

Kansas City, Kansas, 66160

United States

Johns Hopkins School of Medicine

Baltimore, Maryland, 21287

United States

Midatlantic Epilepsy and Sleep Center

Bethesda, Maryland, 20817

United States

Massachusetts General Hospital

Boston, Massachusetts, 02114

United States

Brigham and Women's Hospital

Boston, Massachusetts, 02115

United States

Corewell Health

Grand Rapids, Michigan, 49503

United States

Dartmouth Hitchcock Medical Center

Lebanon, New Hampshire, 03756

United States

Northeast Regional Epilepsy Group

Hackensack, New Jersey, 07601

United States

Robert Wood Johnson Hospital

New Brunswick, New Jersey, 08901

United States

Cleveland Clinic

Cleveland, Ohio, 44195

United States

Ohio State University

Columbus, Ohio, 43210

United States

Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, 19104

United States

Allegheny Health Network

Pittsburgh, Pennsylvania, 15212

United States

Baylor Scott & White Medical Center

Austin, Texas, 78735

United States

Medical College of Wisconsin

Milwaukee, Wisconsin, 53226

United States

Collaborators and Investigators

Sponsor: UniQure Biopharma B.V.

Clinical Development Lead, STUDY_DIRECTOR, uniQure France SAS

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-06-12

Study Completion Date2031-12

Study Record Updates

Study Start Date2024-06-12

Study Completion Date2031-12

Terms related to this study

Keywords Provided by Researchers

Epilepsy
Temporal Lobe
Hippocampal Sclerosis
Epileptic Syndromes

Additional Relevant MeSH Terms

Mesial Temporal Lobe Epilepsy