Ignite Creation Date:
2025-12-24 @ 11:04 PM
Ignite Modification Date:
2026-02-24 @ 1:33 AM
Study NCT ID:
NCT02064569
Status:
COMPLETED
Last Update Posted:
2020-07-01
First Post:
2014-02-13
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Safety Evaluation of Gene Therapy in Leber Hereditary Optic Neuropathy (LHON) Patients
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D029242', 'term': 'Optic Atrophy, Hereditary, Leber'}], 'ancestors': [{'id': 'D015418', 'term': 'Optic Atrophies, Hereditary'}, {'id': 'D009896', 'term': 'Optic Atrophy'}, {'id': 'D009901', 'term': 'Optic Nerve Diseases'}, {'id': 'D003389', 'term': 'Cranial Nerve Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D015785', 'term': 'Eye Diseases, Hereditary'}, {'id': 'D005128', 'term': 'Eye Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D028361', 'term': 'Mitochondrial Diseases'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 19}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2014-02-13', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2020-06', 'completionDateStruct': {'date': '2020-06-25', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2020-06-30', 'studyFirstSubmitDate': '2014-02-13', 'studyFirstSubmitQcDate': '2014-02-14', 'lastUpdatePostDateStruct': {'date': '2020-07-01', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2014-02-17', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2020-06-25', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Incidence of local and general adverse events and Serious Adverse Events', 'timeFrame': 'Up to 48 weeks'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'conditions': ['Leber Hereditary Optic Neuropathy']}, 'referencesModule': {'seeAlsoLinks': [{'url': 'http://www.gensight-biologics.com/', 'label': 'Sponsor web site'}]}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to evaluate the safety and tolerability profile of ascending doses of GS010 in Leber Hereditary Optic Neuropathy (LHON) patients.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\nDocumented diagnosis of LHON based on a genetic test confirming the presence of the G11778A mutation in the mitochondrial ND4\n\nAge 18 years old or older at the time of study entry (informed consent signature)\n\nVisual acuity ≤ 1/10 of the less functional eye\n\nExclusion Criteria:\n\nAny known allergy or hypersensibility to one of the product used during the trial\n\nContraindication to IVT surgery (anaemia Hb \\<8g/dl, severe cardiovascular disease, severe coagulopathy…)\n\nDisorder of the ocular humors and of the internal retina involving visual disability\n\nGlaucoma\n\nPresence of other pathology whose symptoms or associated treatments might affect the retina or the optic nerve Vascular retinal occlusion\n\nNarrow angle contra-indicating pupillary dilation\n\nOther cause of optic neuropathy (inflammatory conditions or exposure to toxins...)\n\nPatients presenting known mutation of other genes implicated in pathological retinal conditions'}, 'identificationModule': {'nctId': 'NCT02064569', 'briefTitle': 'Safety Evaluation of Gene Therapy in Leber Hereditary Optic Neuropathy (LHON) Patients', 'organization': {'class': 'INDUSTRY', 'fullName': 'GenSight Biologics'}, 'officialTitle': 'An Open Label Dose Escalation Clinical Trial to Evaluate the Safety and the Tolerability of GS010 (rAAV2/2-ND4) in Patients With Leber Hereditary Optic Neuropathy Due to Mutations in the Mitochondrial NADH Dehydrogenase 4 Gene', 'orgStudyIdInfo': {'id': 'GS-LHON/CLIN/01'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'GS010', 'interventionNames': ['Genetic: GS010']}], 'interventions': [{'name': 'GS010', 'type': 'GENETIC', 'armGroupLabels': ['GS010']}]}, 'contactsLocationsModule': {'locations': [{'zip': '75012', 'city': 'Paris', 'country': 'France', 'facility': 'CIC du CHNO DES QUINZE-VINGTS', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}], 'overallOfficials': [{'name': 'CATHERINE VIGNAL, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'CIC CHNO DES QUINZE VINGTS'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'UNDECIDED'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'GenSight Biologics', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}