{"type":"trials","record":{"id":41816,"source_id":"clinicaltrials-gov","external_id":"NCT03144999","title":"Treatment of Advanced Dry Age Related Macular Degeneration With AAVCAGsCD59","brief_summary":"Age related macular degeneration (AMD) is the leading cause of vision loss in individuals over age 60. AMD is classified as wet and dry. Wet AMD constitutes 10 to 15% of all cases of AMD and occurs when an abnormal blood vessel grows in or under the retina leading to central vision loss. Wet AMD is successfully treated with injections in the eye on a monthly basis that stop the blood vessel from growing and leaking. The most common form of AMD is the dry variant or dry AMD that affects 85 to 90% of all patients with AMD. In dry AMD, there is loss of retinal pigment, formation of deposits called drusen, and loss of the vessels in a layer of the retina called the choriocapillaris. In the most severe forms of dry AMD there is loss of retinal tissue called geographic atrophy. Over time retinal tissue degenerates in the area responsible for central vision leading to vision loss leading to legal blindness. Currently no treatment for dry AMD exists so that there is a significant unmet need in patients with this ocular disease. Recently, evidence has implicated an overactive inflammatory cascade called the complement system as playing a pivotal role in the development of dry AMD. The complement cascade consists of 3 arms that converge to form a pore-like complex on the surface of cells called the membrane attack complex (MAC). Accumulation of MAC on cell surfaces leads to cell damage and death causing the clinical findings seen in AMD. Normal cells within the human body produce a protein on their cell surfaces called CD59 that blocks the MAC from forming. In AMD, the complement cascade is upregulated and leads to more MAC formation than the body can protect itself against leading to cell destruction. AAVCAGsCD59, an ocular gene therapy product that is injected in to the eye in the physician's office, causes normal retinal cells to increase the expression of a soluble form of CD59 (sCD59). This soluble recombinant version of the naturally occurring CD59 is designed and intended to protect retinal cells that are responsible for central vision by inhibiting the formation of the membrane attack complex (MAC), the terminal step of complement-mediated cell lysis. In gene therapy the cells of the retina are potentially permanently altered to make sCD59 for the life of the patient. With gene therapy only one injection is needed for the drug to be effective for the patient's entire life. This study will evaluate the safety after a single injection of AAVCAGsCD59 administered in an office setting for patients whose enrolled eye has advanced dry AMD with geographic atrophy. The initial study is 26 weeks followed by an additional 18-month safety evaluation.","overall_status":"Completed","phases":["PHASE1"],"study_type":"INTERVENTIONAL","sponsor":"Janssen Research & Development, LLC","enrollment":17,"countries":["United States"],"start_date":"2017-03-29","completion_date":"2019-12-09","last_update_date":"2025-03-30","source_url":"https://clinicaltrials.gov/study/NCT03144999","editorial_summary":"A registered PHASE1 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","comparator":null,"organization":"Janssen Research & Development, LLC","interventions":["AAVCAGsCD59"],"registry_source":"ClinicalTrials.gov","outcome_measures":["Number of participants experiencing ocular and systemic adverse events as graded by CTCAE v4.0 — 26 Weeks","Evaluate the change in area of GA in eyes with dry AMD — 26 Weeks","Evaluate the rate of growth of GA in eyes with dry AMD — 26 Weeks","Incidence of conversion of dry AMD to wet AMD — 26 Weeks","Change in drusen volume measured by spectral domain OCT — 26 Weeks","Prevention of loss of 15 or more letters on an ETDRS visual acuity chart — 26 Weeks"],"design_description":"NON RANDOMIZED · SEQUENTIAL · OTHER · NONE","source_has_results":false},"controlled_terms":["Dry Age-related Macular Degeneration","AAVCAGsCD59"],"relevance_confidence":60,"source_quality_score":100,"freshness_score":65,"publication_state":"published","match_explanation":"Source terminology contains: age-related macular degeneration.","quality_checked_at":"2026-09-25T06:20:59.355028+00:00","duplicate_cluster_key":"id:nct03144999","duplicate_of_id":null,"evidence_snapshot":{"status":"structured","version":1,"duration":"2017-03-29 to 2019-12-09","comparator":null,"confidence":"structured-source","population":"ALL · 50 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":"2026-09-25T06:20:47.875Z","intervention":["AAVCAGsCD59"],"participants":17,"study_design":"NON RANDOMIZED · SEQUENTIAL · OTHER · NONE","subject_scope":"Human clinical study registration","evidence_stage":"PHASE1","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":["Number of participants experiencing ocular and systemic adverse events as graded by CTCAE v4.0 — 26 Weeks","Evaluate the change in area of GA in eyes with dry AMD — 26 Weeks","Evaluate the rate of growth of GA in eyes with dry AMD — 26 Weeks","Incidence of conversion of dry AMD to wet AMD — 26 Weeks","Change in drusen volume measured by spectral domain OCT — 26 Weeks","Prevention of loss of 15 or more letters on an ETDRS visual acuity chart — 26 Weeks"],"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":["Source terminology contains: age-related macular degeneration.","Source terminology supplies longevity context: age-related.","Study type is explicitly identified as INTERVENTIONAL."],"matched_fields":["controlled terminology","controlled context","study type"],"relevance_score":60,"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/41816","automation_disclosure":"Generated automatically from cited source metadata. No scientist, clinician, researcher, editor, or human reviewer evaluates this publication before release."}