{"type":"trials","record":{"id":42135,"source_id":"clinicaltrials-gov","external_id":"NCT03066258","title":"Safety and Tolerability of RGX-314 (Investigational Product) Gene Therapy for Neovascular AMD Trial","brief_summary":"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.","overall_status":"Completed","phases":["PHASE1","PHASE2"],"study_type":"INTERVENTIONAL","sponsor":"REGENXBIO Inc.","enrollment":42,"countries":["United States"],"start_date":"2017-03-29","completion_date":"2021-06-17","last_update_date":"2023-05-16","source_url":"https://clinicaltrials.gov/study/NCT03066258","editorial_summary":"A registered PHASE1, PHASE2 study indexed because it matched monitored longevity research terms. Registry status: Completed. Registration does not establish safety or effectiveness.","first_seen_at":"2026-09-25T01:25:30.378222+00:00","last_seen_at":"2026-09-25T06:20:47.789+00:00","metadata":{"sex":"ALL","acronym":null,"age_range":"50 Years to 89 Years","comparator":null,"organization":"REGENXBIO Inc.","interventions":["RGX-314"],"registry_source":"ClinicalTrials.gov","outcome_measures":["Safety (Participants With Ocular and Non-ocular AEs (Adverse Events) and SAEs (Serious Adverse Events)) — 26 weeks (24…","Safety (Participants With Ocular and Non-ocular AEs and SAEs) — 106 weeks (104 weeks following RGX-314 administration)","Change From Baseline in BCVA (Best Corrected Visual Acuity) — 106 weeks (104 weeks following RGX-314 administration)","Change From Baseline in CRT (Central Retinal Thickness) — 106 weeks (104 weeks following RGX-314 administration)","Supplemental Injections (Annualized Rate of Supplemental Injections) — 106 weeks (104 weeks following RGX-314 administr…","Mean Change From Baseline in Area of CNV (Choroidal Neovascularization) — 106 weeks (104 weeks following RGX-314 admini…"],"design_description":"NON RANDOMIZED · SEQUENTIAL · TREATMENT · NONE","source_has_results":true},"controlled_terms":["Neovascular Age-related Macular Degeneration","Wet Age-related Macular Degeneration","RGX-314"],"relevance_confidence":100,"source_quality_score":100,"freshness_score":50,"publication_state":"published","match_explanation":"Abstract contains controlled term: age-related macular degeneration.","quality_checked_at":"2026-09-25T06:20:59.355028+00:00","duplicate_cluster_key":"id:nct03066258","duplicate_of_id":null,"evidence_snapshot":{"status":"structured","version":1,"duration":"2017-03-29 to 2021-06-17","comparator":null,"confidence":"structured-source","population":"ALL · 50 Years to 89 Years","provenance":{"duration":"start_date and completion_date","comparator":"registry arm fields","population":"registry eligibility fields","intervention":"registry intervention fields","participants":"enrollment","study_design":"study_type and registry design fields","evidence_stage":"phases","reported_outcome":"not available","outcomes_measured":"registry outcome-measure fields"},"generated_at":"2026-09-25T06:20:47.925Z","intervention":["RGX-314"],"participants":42,"study_design":"NON RANDOMIZED · SEQUENTIAL · TREATMENT · NONE","subject_scope":"Human clinical study registration","evidence_stage":"PHASE1, PHASE2","safety_context":"Eligibility, adverse-event details, and clinical decisions must be checked in the official registry and with qualified clinicians.","source_support":"Structured registry protocol metadata; no finding-level conclusion is generated.","main_limitation":"This is a study registration. No reusable structured result is available here, so it cannot show whether the intervention worked or was safe.","reported_outcome":null,"outcomes_measured":["Safety (Participants With Ocular and Non-ocular AEs (Adverse Events) and SAEs (Serious Adverse Events)) — 26 weeks (24…","Safety (Participants With Ocular and Non-ocular AEs and SAEs) — 106 weeks (104 weeks following RGX-314 administration)","Change From Baseline in BCVA (Best Corrected Visual Acuity) — 106 weeks (104 weeks following RGX-314 administration)","Change From Baseline in CRT (Central Retinal Thickness) — 106 weeks (104 weeks following RGX-314 administration)","Supplemental Injections (Annualized Rate of Supplemental Injections) — 106 weeks (104 weeks following RGX-314 administr…","Mean Change From Baseline in Area of CNV (Choroidal Neovascularization) — 106 weeks (104 weeks following RGX-314 admini…"],"regulatory_context":"Trial registration is not regulatory approval and does not establish that an intervention is available."},"clinical_trial_topics":[{"topic_slug":"vision-aging","is_published":true,"match_reasons":["Abstract contains controlled term: age-related macular degeneration.","Source terminology contains: age-related macular degeneration.","Abstract supplies longevity context: age-related.","Study type is explicitly identified as INTERVENTIONAL."],"matched_fields":["abstract","controlled terminology","abstract context","study type"],"relevance_score":100,"intelligence_topics":{"name":"Vision ageing","slug":"vision-aging"}}],"content_sources":{"name":"ClinicalTrials.gov","homepage_url":"https://clinicaltrials.gov/"}},"canonical_url":"https://www.immortal.life/trials/42135","automation_disclosure":"Generated automatically from cited source metadata. No scientist, clinician, researcher, editor, or human reviewer evaluates this publication before release."}