A Long-term Follow-Up Study to Evaluate the Safety and Efficacy of RGX-314 Following Subretinal Administration in Participants With Neovascular Age-related Macular Degeneration and Fellow Eye Treatment Substudy

RGX-314-5101

This is a prospective, observational study designed to evaluate the long-term safety and efficacy of RGX-314. Eligible participants are those who were previously enrolled in a clinical study in which they received a single subretinal administration of RGX-314 in their study eye. Enrollment of each participant in the current study should occur after the participant has completed either the end of study or early termination visit in the previous (parent) clinical study. Participants will be followed for up to 5 years post-RGX-314 administration (inclusive of the parent study). After enrollment and a 6-month follow-up visit, participants will attend at least annual study visits through the end of the 5-year post-RGX-314 administration follow-up period. Additionally, an interventional fellow eye treatment substudy will evaluate the safety, efficacy, and immunogenicity of subretinal RGX-314 administration in the fellow eye of participants having bilateral disease who previously received a subretinal injection of RGX-314 in their study eye. Participants who qualify for the substudy will receive subretinal administration of RGX-314 in their fellow eye and complete 13 study visits in a 54-week period. Following completion of the substudy participants will continue in the observational portion of the study for up to 5 years post RGX-314 administration in their fellow eye.

2019-05-31

2028-12-01

2028-12-01

Early phase I

865

Main Observational Study: Inclusion Criteria: 1. Able and willing to provide written consent 2. Previously enrolled in a clinical study of RGX-314 and received a single subretinal administration of RGX-314 Exclusions Criteria: 1. None Fellow Eye Substudy: Inclusion Criteria 1. Age ≤ 93 years 2. Currently or previously enrolled in the main observational study 3. Diagnosis of bilateral choroidal neovascularization secondary to age-related macular degeneration 4. Active nAMD in the fellow eye 5. BCVA between ≤ 80 and ≥ 20 letters in the fellow eye Exclusion Criteria: 1. CNV or macular edema in the fellow eye secondary to causes other than nAMD 2. Subfoveal fibrosis or atrophy in the fellow eye 3. Advanced glaucoma or history of secondary glaucoma in the fellow eye or any invasive procedure to treat glaucoma 4. Myocardial infarction, cerebrovascular accident, or transient ischemic attack within the past 6 months 5. History of intraocular surgery in the fellow eye within 12 weeks of screening 6. History of intravitreal therapy, such as intravitreal steroid injection or investigational product, other than an intravitreal therapy for nAMD, in the fellow eye within 6 months of screening

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'NON_RANDOMIZED', 'interventionModel': 'SINGLE_GROUP', 'interventionModelDescription': '1 main Observational study arm, 1 RGX-314 Fellow Eye Treatment substudy', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 865, 'type': 'ESTIMATED'}}

2023-05-22