Ignite Creation Date:
2025-12-25 @ 12:19 AM
Ignite Modification Date:
2026-07-13 @ 5:18 PM
Study NCT ID:
NCT03066258
Status:
COMPLETED
Last Update Posted:
2023-05-16
First Post:
2017-02-17
Is Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Safety and Tolerability of RGX-314 (Investigational Product) Gene Therapy for Neovascular AMD Trial
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'submissionTracking': {'firstMcpInfo': {'postDateStruct': {'date': '2023-03-10', 'type': 'ACTUAL'}}}}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'svaneveren@regenxbio.com', 'phone': '240.552.8181', 'title': 'Sherri Van Everen, PharmD/ Vice President of Clinical Development', 'organization': 'Regenxbio Inc.'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801).', 'description': 'Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \\>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population\n\nSAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population', 'eventGroups': [{'id': 'EG000', 'title': 'Cohort 1', 'description': '3E9 GC/eye of RGX-314\n\nRGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein', 'otherNumAtRisk': 6, 'deathsNumAtRisk': 6, 'otherNumAffected': 5, 'seriousNumAtRisk': 6, 'deathsNumAffected': 1, 'seriousNumAffected': 2}, {'id': 'EG001', 'title': 'Cohort 2', 'description': '1E10 GC/eye of RGX-314\n\nRGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein', 'otherNumAtRisk': 6, 'deathsNumAtRisk': 6, 'otherNumAffected': 6, 'seriousNumAtRisk': 6, 'deathsNumAffected': 0, 'seriousNumAffected': 2}, {'id': 'EG002', 'title': 'Cohort 3', 'description': '6E10 GC/eye of RGX-314\n\nRGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein', 'otherNumAtRisk': 6, 'deathsNumAtRisk': 6, 'otherNumAffected': 6, 'seriousNumAtRisk': 6, 'deathsNumAffected': 0, 'seriousNumAffected': 1}, {'id': 'EG003', 'title': 'Cohort 4', 'description': '1.6E11 GC/eye of RGX-314\n\nRGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein', 'otherNumAtRisk': 12, 'deathsNumAtRisk': 12, 'otherNumAffected': 12, 'seriousNumAtRisk': 12, 'deathsNumAffected': 0, 'seriousNumAffected': 3}, {'id': 'EG004', 'title': 'Cohort 5', 'description': '2.5E11 GC/eye of RGX-314\n\nRGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein', 'otherNumAtRisk': 12, 'deathsNumAtRisk': 12, 'otherNumAffected': 12, 'seriousNumAtRisk': 12, 'deathsNumAffected': 1, 'seriousNumAffected': 4}], 'otherEvents': [{'term': 'Conjunctival Haemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 6, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 4, 'numAffected': 4}, {'groupId': 'EG003', 'numAtRisk': 12, 'numEvents': 9, 'numAffected': 9}, {'groupId': 'EG004', 'numAtRisk': 12, 'numEvents': 11, 'numAffected': 11}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Intraocular Pressure Increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG003', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 12, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Visual Acuity Reduced/Impairment', 'notes': 'Visual Acuity Reduced \\& Impairment will be presented as a group term including the AE preferred terms (PTs) of "Visual Acuity Reduced" and "Visual Impairment"', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 6, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG004', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Eye Irritation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 12, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG004', 'numAtRisk': 12, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Eye Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 12, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG004', 'numAtRisk': 12, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Retinal Degeneration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 12, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG004', 'numAtRisk': 12, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Photopsia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG004', 'numAtRisk': 12, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Conjunctival Hyperaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG003', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 12, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Vision Blurred', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 12, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Retinal pigmentary changes (depigmentation, pigment epitheliopathy & pigmentation)', 'notes': "Retinal pigmentary changes is a group term including AE preferred terms of 'Retinal Depigmentation', 'Retinal Pigment Epitheliopathy', and 'Retinal Pigmentation'.", 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 6, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG003', 'numAtRisk': 12, 'numEvents': 12, 'numAffected': 12}, {'groupId': 'EG004', 'numAtRisk': 12, 'numEvents': 11, 'numAffected': 11}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Retinal Haemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 4, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 12, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG004', 'numAtRisk': 12, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Intraocular Inflammation', 'notes': "Intraocular Inflammation is a group term including AE preferred terms of 'Anterior Chamber Cell', 'Anterior Chamber Flare', and 'Anterior Chamber Inflammation'.", 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 4, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 6, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG003', 'numAtRisk': 12, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG004', 'numAtRisk': 12, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Metamorphopsia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 12, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG004', 'numAtRisk': 12, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Retinal Thickening', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 12, 'numEvents': 4, 'numAffected': 4}, {'groupId': 'EG004', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Dry Age-Related Macular Degeneration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 12, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Erythema Of Eyelid', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 12, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG004', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Photophobia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 12, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG004', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Posterior Capsule Opacification', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG004', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Vitreous Floaters', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 12, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG004', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}], 'seriousEvents': [{'term': 'Retinal Detachment', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Endophthalmitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG004', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Visual Impairment', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Atrial Fibrillation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Colitis Ischaemic', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Asthenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Cholelithiasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG004', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Diverticulitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Urinary Tract Infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG004', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Deep Vein Thrombosis Postoperative', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Femoral Neck Fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG004', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Hypervolaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Arthritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Encephalopathy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Delirium', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Respiratory Failure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Hypertension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 6, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Safety (Participants With Ocular and Non-ocular AEs (Adverse Events) and SAEs (Serious Adverse Events))', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}, {'value': '6', 'groupId': 'OG001'}, {'value': '6', 'groupId': 'OG002'}, {'value': '12', 'groupId': 'OG003'}, {'value': '12', 'groupId': 'OG004'}]}], 'groups': [{'id': 'OG000', 'title': 'Cohort 1', 'description': '3E9 GC/eye of RGX-314\n\nRGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein'}, {'id': 'OG001', 'title': 'Cohort 2', 'description': '1E10 GC/eye of RGX-314\n\nRGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein'}, {'id': 'OG002', 'title': 'Cohort 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[{'categories': [{'title': 'Female', 'measurements': [{'value': '4', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}, {'value': '7', 'groupId': 'BG003'}, {'value': '6', 'groupId': 'BG004'}, {'value': '22', 'groupId': 'BG005'}]}, {'title': 'Male', 'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '4', 'groupId': 'BG002'}, {'value': '5', 'groupId': 'BG003'}, {'value': '6', 'groupId': 'BG004'}, {'value': '20', 'groupId': 'BG005'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'title': 'White', 'categories': [{'measurements': [{'value': '6', 'groupId': 'BG000'}, {'value': '6', 'groupId': 'BG001'}, {'value': '6', 'groupId': 'BG002'}, {'value': '12', 'groupId': 'BG003'}, {'value': '11', 'groupId': 'BG004'}, {'value': '41', 'groupId': 'BG005'}]}]}, {'title': 'Asian', 'categories': [{'measurements': [{'value': '0', 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'interventionModelDescription': 'dose escalation'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 42}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2017-03-29', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2023-05', 'completionDateStruct': {'date': '2021-06-17', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2023-05-12', 'studyFirstSubmitDate': '2017-02-17', 'resultsFirstSubmitDate': '2023-02-13', 'studyFirstSubmitQcDate': '2017-02-22', 'lastUpdatePostDateStruct': {'date': '2023-05-16', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2023-05-12', 'studyFirstPostDateStruct': {'date': '2017-02-28', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2023-05-16', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2019-11-24', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Safety (Participants With Ocular and Non-ocular AEs (Adverse Events) and SAEs (Serious Adverse 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A higher score represents better vision.'}, {'measure': 'Change From Baseline in CRT (Central Retinal Thickness)', 'timeFrame': '106 weeks (104 weeks following RGX-314 administration)', 'description': 'Retinal fluid status of the study eye was evaluated using spectral domain OCT (Optical Coherence Tomography). A decrease in value indicates a decrease in fluid'}, {'measure': 'Supplemental Injections (Annualized Rate of Supplemental Injections)', 'timeFrame': '106 weeks (104 weeks following RGX-314 administration)', 'description': 'The number of supplemental anti-VEGF injections given after RGX-314 was administered. Injections per year which were determined by the number of supplemental injections divided total follow-up in study days which is annualized to a per year rate. Injections were given for signs of worsening disease at a study visit, per the discretion of the investigator.'}, {'measure': 'Mean Change From Baseline in Area of CNV (Choroidal Neovascularization)', 'timeFrame': '106 weeks (104 weeks following RGX-314 administration)', 'description': 'Area of Choroidal Neovascularization of the study eye was assessed with color fundus photography. Analysis was performed by the central reading center. An increase in value represents an increase in CNV.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['nAMD', 'wet AMD', 'gene therapy'], 'conditions': ['Neovascular Age-related Macular Degeneration', 'Wet Age-related Macular Degeneration']}, 'referencesModule': {'references': [{'pmid': '38554726', 'type': 'DERIVED', 'citation': 'Campochiaro PA, Avery R, Brown DM, Heier JS, Ho AC, Huddleston SM, Jaffe GJ, Khanani AM, Pakola S, Pieramici DJ, Wykoff CC, Van Everen S. Gene therapy for neovascular age-related macular degeneration by subretinal delivery of RGX-314: a phase 1/2a dose-escalation study. Lancet. 2024 Apr 20;403(10436):1563-1573. doi: 10.1016/S0140-6736(24)00310-6. Epub 2024 Mar 27.'}]}, 'descriptionModule': {'briefSummary': 'Excessive vascular endothelial growth factor (VEGF) plays a key part in promoting neovascularization and edema in neovascular (wet) age-related macular degeneration (nAMD). VEGF inhibitors (anti-VEGF), including ranibizumab (LUCENTIS®, Genentech) and aflibercept (EYLEA®, Regeneron), have been shown to be safe and effective for treating nAMD and have demonstrated improvement in vision. However, anti-VEGF therapy is administered frequently via intravitreal injection and can be a significant burden to the patients. RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein. The long-term, stable delivery of this therapeutic protein following a 1 time gene therapy treatment for nAMD could potentially reduce the treatment burden of currently available therapies while maintaining vision with a favorable benefit:risk profile.', 'detailedDescription': 'This Phase I/IIa, open-label, multiple-cohort, dose-escalation study was designed to evaluate the safety and tolerability of RGX-314 gene therapy in subjects with previously treated nAMD. Five doses were studied in approximately 42 subjects. Subjects who met the inclusion/exclusion criteria and had an anatomic response to an initial anti-VEGF injection received a single dose of RGX-314 administered by subretinal delivery. RGX-314 uses an AAV8 vector that contains a gene that encodes for a monoclonal antibody fragment which binds to and neutralizes VEGF activity. Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '89 Years', 'minimumAge': '50 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Patients ≥ 50 and ≤ 89 years with a diagnosis of subfoveal CNV (Choroidal neovascularization) secondary to AMD in the study eye receiving prior intravitreal anti-VEGF therapy.\n2. BCVA (Best Corrected Visual Acuity) between ≤20/63 and ≥20/400 (≤63 and ≥19 Early Treatment Diabetic Retinopathy Study \\[ETDRS\\] letters) for the first patient in each cohort followed by BCVA between ≤20/40 and ≥20/400 (≤73 and ≥19 ETDRS letters) for the rest of the cohort.\n3. History of need for and response to anti-VEGF therapy.\n4. Response to anti-VEGF at trial entry (assessed by SD-OCT (Spectral Domain Optical Coherence Tomography) at week 1)\n5. Must be pseudophakic (status post cataract surgery) in the study eye.\n6. AST (Aspartate aminotransferase)/ALT (Alanine aminotransferase) \\< 2.5 × ULN (Upper limit of normal); TB (Total bilirubin) \\< 1.5 × ULN; PT (Prothrombin time) \\< 1.5 × ULN; Hb \\> 10 g/dL (males) and \\> 9 g/dL (females); Platelets \\> 100 × 10\\^3/µL; eGFR (Estimated glomerular filtration rate) \\> 30 mL/min/1.73 m\\^2\n7. Must be willing and able to provide written, signed informed consent.\n\nExclusion Criteria:\n\n1. CNV or macular edema in the study eye secondary to any causes other than AMD.\n2. Any condition preventing visual acuity improvement in the study eye, eg, fibrosis, atrophy, or retinal epithelial tear in the center of the fovea.\n3. Active or history of retinal detachment in the study eye.\n4. Advanced glaucoma in the study eye.\n5. History of intravitreal therapy in the study eye, such as intravitreal steroid injection or investigational product, other than anti-VEGF therapy, in the 6 months prior to screening.\n6. Presence of an implant in the study eye at screening (excluding intraocular lens).\n7. Myocardial infarction, cerebrovascular accident, or transient ischemic attacks within the past 6 months.\n8. Uncontrolled hypertension (systolic blood pressure \\[BP\\] \\>180 mmHg, diastolic BP \\>100 mmHg) despite maximal medical treatment.'}, 'identificationModule': {'nctId': 'NCT03066258', 'briefTitle': 'Safety and Tolerability of RGX-314 (Investigational Product) Gene Therapy for Neovascular AMD Trial', 'organization': {'class': 'INDUSTRY', 'fullName': 'REGENXBIO Inc.'}, 'officialTitle': 'A Phase I/IIa (Phase 1/Phase 2a), Open-label, Multiple-cohort, Dose-escalation Study to Evaluate the Safety and Tolerability of Gene Therapy With RGX-314 in Subjects With Neovascular AMD (nAMD)', 'orgStudyIdInfo': {'id': 'RGX-314-001'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Cohort 1', 'description': '3E9 GC (genome copies)/eye of RGX-314 (E means the exponential constant)', 'interventionNames': ['Genetic: RGX-314']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 2', 'description': '1E10 GC/eye of RGX-314', 'interventionNames': ['Genetic: RGX-314']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 3', 'description': '6E10 GC/eye of RGX-314', 'interventionNames': ['Genetic: RGX-314']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 4', 'description': '1.6E11 GC/eye of RGX-314', 'interventionNames': ['Genetic: RGX-314']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 5', 'description': '2.5E11 GC/eye of RGX-314', 'interventionNames': ['Genetic: RGX-314']}], 'interventions': [{'name': 'RGX-314', 'type': 'GENETIC', 'description': 'RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein', 'armGroupLabels': ['Cohort 1', 'Cohort 2', 'Cohort 3', 'Cohort 4', 'Cohort 5']}]}, 'contactsLocationsModule': {'locations': [{'zip': '93103', 'city': 'Santa Barbara', 'state': 'California', 'country': 'United States', 'facility': 'Santa Barbara location', 'geoPoint': {'lat': 34.42083, 'lon': -119.69819}}, {'zip': '21287', 'city': 'Baltimore', 'state': 'Maryland', 'country': 'United States', 'facility': 'Baltimore location', 'geoPoint': {'lat': 39.29038, 'lon': -76.61219}}, {'zip': '02114', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'Boston location', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '89502', 'city': 'Reno', 'state': 'Nevada', 'country': 'United States', 'facility': 'Reno location', 'geoPoint': {'lat': 39.52963, 'lon': -119.8138}}, {'zip': '19104', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'Philadelphia location 1', 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '19107', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'Philadelphia location 2', 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '38138', 'city': 'Germantown', 'state': 'Tennessee', 'country': 'United States', 'facility': 'Memphis location', 'geoPoint': {'lat': 35.08676, 'lon': -89.81009}}, {'zip': '77030', 'city': 'Houston', 'state': 'Texas', 'country': 'United States', 'facility': 'Houston location', 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}], 'overallOfficials': [{'name': 'Jeffrey Heier, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Ophthalmic Consultants of Boston'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'REGENXBIO Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}