Contact SCGE




Gene Therapy Trial Report

Summary

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


NCTID NCT06063850 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Mesial Temporal Lobe Epilepsy
Disease Ontology Term DOID:3328
Compound Name AMT-260
Sponsor UniQure Biopharma B.V.
Funder Type Industry
Recruitment Status
Enrollment Count 12 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant MiGRIK2
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action MiRNA knockdown of mutant/aberrant gene
Route of Administration Intraparenchymal
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAV9
Editor Type none
Dose 1 Undisclosed low dose
Dose 2 Undisclosed high dose

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2023-09-01
Completion Date 2031-12
Last Update 2025-12-10

Participation Criteria


Eligible Age 18 Years - 75 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
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 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. 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 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 icta 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. * Inadequate vaccination status (including flu shots and other relevant immunizations such as COVID-19 per local guidelines and regulations).
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 18
Locations United States

Regulatory Information


Has US IND True
FDA Designations
Recent Updates First 3 patients in first cohort enrolled September 2025

Resources/Links