Ignite Creation Date:
2025-12-24 @ 5:18 PM
Ignite Modification Date:
2025-12-28 @ 1:53 PM
Study NCT ID:
NCT06063850
Status:
RECRUITING
Last Update Posted:
2025-12-10
First Post:
2023-09-01
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
AMT-260 Gene Therapy Study in Adults With Unilateral Refractory Mesial Temporal Lobe Epilepsy
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D004827', 'term': 'Epilepsy'}, {'id': 'D000092223', 'term': 'Hippocampal Sclerosis'}, {'id': 'D000073376', 'term': 'Epileptic Syndromes'}], 'ancestors': [{'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D065703', 'term': 'Malformations of Cortical Development, Group I'}, {'id': 'D054220', 'term': 'Malformations of Cortical Development'}, {'id': 'D009421', 'term': 'Nervous System Malformations'}, {'id': 'D000013', 'term': 'Congenital Abnormalities'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SEQUENTIAL'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 12}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2024-06-12', 'type': 'ACTUAL'}, 'statusVerifiedDate': '2025-12', 'completionDateStruct': {'date': '2031-12', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-12-03', 'studyFirstSubmitDate': '2023-09-01', 'studyFirstSubmitQcDate': '2023-09-25', 'lastUpdatePostDateStruct': {'date': '2025-12-10', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2023-10-03', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2026-11-30', 'type': 'ESTIMATED'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'To evaluate the long-term safety and efficacy of AMT-260 in adults with unilateral refractory MTLE.', 'timeFrame': 'up to 5 years', 'description': 'Occurrence of Adverse Events during the period of 1 to 5 years after AMT-260 administration, including seriousness.'}], 'primaryOutcomes': [{'measure': 'To evaluate the safety and tolerability of AMT-260 in adults with unilateral refractory MTLE.', 'timeFrame': '1 year', 'description': 'Occurrence of Adverse Events during the period of 1 year after AMT-260 administration, including seriousness, severity, and causal relationship to AMT-260.'}], 'secondaryOutcomes': [{'measure': 'To evaluate first signs of efficacy of AMT-260.', 'timeFrame': '1 year', 'description': 'Change in seizure frequency, comparing baseline to the 1 year period after AMT-260 administration.'}, {'measure': 'To evaluate the biodistribution properties of AMT-260.', 'timeFrame': '1 year', 'description': 'Blood, urine, saliva, and CSF samples will be collected and evaluated for vector DNA shedding at each timepoint.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Epilepsy', 'Temporal Lobe', 'Hippocampal Sclerosis', 'Epileptic Syndromes'], 'conditions': ['Mesial Temporal Lobe Epilepsy']}, 'referencesModule': {'seeAlsoLinks': [{'url': 'https://www.uniqure.com/programs-pipeline/phase-i-iia-clinical-trial-of-amt-260', 'label': ': Sponsor web page about this Phase 1/2a clinical study of AMT-260'}]}, 'descriptionModule': {'briefSummary': '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).', 'detailedDescription': '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).\n\nAMT-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.\n\nParticipants 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.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '75 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Diagnosis of unilateral refractory MTLE\n* 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.\n* 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.\n* Confirmed unilateral hippocampal pathology and concordant unilateral seizure focus\n* No evidence of focal neurocognitive dysfunction, inconsistent with disease pathology- related MRI and/or (18F)FDG-PET findings.\n* 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.\n* For WOCBP only: Negative pregnancy test.\n\nExclusion Criteria:\n\n* 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).\n* Any other contraindications for generalized anesthesia or surgery.\n* 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.\n* Any seizures with contralateral or extra-temporal icta onset captured on EEG.\n* Dementia or other progressive neurological disorders and progressive brain lesions.\n* 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.\n* Magnetic resonance imaging evidence of epileptogenic, extra-temporal lesions, or dual temporal lobe pathology.\n* Inadequate vaccination status (including flu shots and other relevant immunizations such as COVID-19 per local guidelines and regulations)."}, 'identificationModule': {'nctId': 'NCT06063850', 'acronym': 'GenTLE', 'briefTitle': 'AMT-260 Gene Therapy Study in Adults With Unilateral Refractory Mesial Temporal Lobe Epilepsy', 'organization': {'class': 'INDUSTRY', 'fullName': 'UniQure Biopharma B.V.'}, 'officialTitle': '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)', 'orgStudyIdInfo': {'id': 'CT-AMT-260-01'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'AMT-260', 'description': '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).', 'interventionNames': ['Genetic: AAV9-hSyn1-miGRIK2']}], 'interventions': [{'name': 'AAV9-hSyn1-miGRIK2', 'type': 'GENETIC', 'otherNames': ['AMT-260'], 'description': '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.', 'armGroupLabels': ['AMT-260']}]}, 'contactsLocationsModule': {'locations': [{'zip': '35294-0021', 'city': 'Birmingham', 'state': 'Alabama', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Jennifer Pilkington', 'role': 'CONTACT', 'email': 'jlpilkington@uabmc.edu', 'phone': '205-934-8352'}, {'name': 'Jerzy P Szaflarski, MD, PhD', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'University of Alabama at Birmingham', 'geoPoint': {'lat': 33.52066, 'lon': -86.80249}}, {'zip': '85054', 'city': 'Phoenix', 'state': 'Arizona', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Jonathon Parker, MD', 'role': 'CONTACT', 'email': 'Parker.Jonathon@mayo.edu', 'phone': '480-342-2906'}, {'name': 'Jonathon Parker, MD', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Mayo Clinic Arizona', 'geoPoint': {'lat': 33.44838, 'lon': -112.07404}}, {'zip': '94304', 'city': 'Palo Alto', 'state': 'California', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Jordan Seliger', 'role': 'CONTACT', 'email': 'jseliger@stanford.edu', 'phone': '650-460-9260'}, {'name': 'Yi Li, MD, PhD', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Stanford University', 'geoPoint': {'lat': 37.44188, 'lon': -122.14302}}, {'zip': '32224', 'city': 'Jacksonville', 'state': 'Florida', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Megan J Gauthier', 'role': 'CONTACT', 'email': 'Gauthier.Megan2@mayo.edu', 'phone': '904-953-5544'}, {'name': 'Alicia Kissinger-Knox, PhD', 'role': 'CONTACT', 'email': 'Kissinger-Knox.Alicia@mayo.edu', 'phone': '904-953-5544'}, {'name': 'Sanjeet Grewal, MD', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Mayo Clinic Florida', 'geoPoint': {'lat': 30.33218, 'lon': -81.65565}}, {'zip': '66160', 'city': 'Kansas City', 'state': 'Kansas', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Laura Crabtree', 'role': 'CONTACT', 'email': 'lcrabtree2@kumc.edu', 'phone': '913-574-0412'}, {'name': 'P Landazuri, MD', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Kansas University Medical Center', 'geoPoint': {'lat': 39.11417, 'lon': -94.62746}}, {'zip': '21287', 'city': 'Baltimore', 'state': 'Maryland', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Joon Kang, MD', 'role': 'CONTACT', 'email': 'Jkang50@jh.edu', 'phone': '410-955-2822'}, {'name': 'Joon Kang, MD', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Johns Hopkins School of Medicine', 'geoPoint': {'lat': 39.29038, 'lon': -76.61219}}, {'zip': '20817', 'city': 'Bethesda', 'state': 'Maryland', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Pavel Klein, M.D.', 'role': 'CONTACT', 'email': 'kleinp@epilepsydc.com', 'phone': '301-530-9744'}, {'name': 'Pavel Klein, M.D.', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Midatlantic Epilepsy and Sleep Center', 'geoPoint': {'lat': 38.98067, 'lon': -77.10026}}, {'zip': '02114', 'city': 'Boston', 'state': 'Massachusetts', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Erin Donahue', 'role': 'CONTACT', 'email': 'ekdonahue@mgh.harvard.edu', 'phone': '617.643.3732'}, {'name': 'Mark Richardson, MD, PhD', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Massachusetts General Hospital', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '49503', 'city': 'Grand Rapids', 'state': 'Michigan', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'David Burdette, MD', 'role': 'CONTACT', 'email': 'david.burdette@corewellhealth.org', 'phone': '(616) 267-0301'}, {'name': 'David Burdette, MD', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Corewell Health', 'geoPoint': {'lat': 42.96336, 'lon': -85.66809}}, {'zip': '03756', 'city': 'Lebanon', 'state': 'New Hampshire', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Anastasia Kanishcheva, MPH, CCRC', 'role': 'CONTACT', 'email': 'Anastasia.Kanishcheva@Hitchcock.ORG', 'phone': '603-650-0260'}, {'name': 'Barbara Jobst, MD', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Dartmouth Hitchcock Medical Center', 'geoPoint': {'lat': 43.64229, 'lon': -72.25176}}, {'zip': '07601', 'city': 'Hackensack', 'state': 'New Jersey', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Hardik Rana', 'role': 'CONTACT', 'email': 'hrana@epilepsygroup.com', 'phone': '551-497-5000'}, {'name': 'Asfi Rafiuddin', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Northeast Regional Epilepsy Group', 'geoPoint': {'lat': 40.88593, 'lon': -74.04347}}, {'zip': '08901', 'city': 'New Brunswick', 'state': 'New Jersey', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Christine Yohn', 'role': 'CONTACT', 'email': 'cy253@rwjms.rutgers.edu', 'phone': '908-328-4210'}, {'name': 'Robert E Gross, MD, PhD', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Robert Wood Johnson Hospital', 'geoPoint': {'lat': 40.48622, 'lon': -74.45182}}, {'zip': '44195', 'city': 'Cleveland', 'state': 'Ohio', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Xiaoming Zhang', 'role': 'CONTACT', 'email': 'zhangx6@ccf.org', 'phone': '216-445-7510'}, {'name': 'Imad Najm, MD', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Cleveland Clinic', 'geoPoint': {'lat': 41.4995, 'lon': -81.69541}}, {'zip': '43210', 'city': 'Columbus', 'state': 'Ohio', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'James Elder, MD', 'role': 'CONTACT', 'email': 'james.elder@osumc.edu', 'phone': '614-685-1965'}, {'name': 'James Elder, MD', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Ohio State University', 'geoPoint': {'lat': 39.96118, 'lon': -82.99879}}, {'zip': '19104', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Melissa Johnston Esparza, MS', 'role': 'CONTACT', 'email': 'melissa.johnstonesparza@pennmedicine.upenn.edu', 'phone': '215-614-0520'}, {'name': 'Michael Gelfand, MD, PhD', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Hospital of the University of Pennsylvania', 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '15212', 'city': 'Pittsburgh', 'state': 'Pennsylvania', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Sarah Kimutis', 'role': 'CONTACT', 'email': 'sarah.kimutis@ahn.org', 'phone': '412-359-3565'}, {'name': 'Alexander Whiting, MD', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Allegheny Health Network', 'geoPoint': {'lat': 40.44062, 'lon': -79.99589}}, {'zip': '78735', 'city': 'Austin', 'state': 'Texas', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Victor H Gonzalez Montoya', 'role': 'CONTACT', 'email': 'Victor.gonzalezmontoya@bswhealth.org', 'phone': '512-654-1234'}, {'name': 'Victor H Gonzalez Montoya, MD, FAES', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Baylor Scott & White Medical Center', 'geoPoint': {'lat': 30.26715, 'lon': -97.74306}}, {'zip': '53226', 'city': 'Milwaukee', 'state': 'Wisconsin', 'status': 'NOT_YET_RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Sarah Young', 'role': 'CONTACT', 'email': 'scyoung@mcw.edu', 'phone': '414-955-0989'}, {'name': 'Kunal Gupta, MD, PhD', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Medical College of Wisconsin', 'geoPoint': {'lat': 43.0389, 'lon': -87.90647}}], 'centralContacts': [{'name': 'uniQure', 'role': 'CONTACT', 'email': 'medinfo@uniqure.com', 'phone': '1-866-520-1257'}], 'overallOfficials': [{'name': 'Clinical Development Lead', 'role': 'STUDY_DIRECTOR', 'affiliation': 'uniQure France SAS'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'UniQure Biopharma B.V.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}