Neovascular Age-related Macular Degeneration, Wet Macular Degeneration
Conditions
Keywords
gene therapy, wet AMD, nAMD
Brief summary
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.
Interventions
GENETICRGX-314
AAV8 vector containing a transgene for anti-VEGF Fab
Sponsors
AbbVie
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
1 main Observational study arm, 1 RGX-314 Fellow Eye Treatment substudy
Eligibility
Sex/Gender
ALL
Age
50 Years to No maximum
Healthy volunteers
No
Inclusion criteria
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence of ocular adverse events and any serious adverse events in the study eye | 5 years inclusive of parent study |
| Fellow eye substudy: Incidence of ocular adverse events and any serious adverse events in the fellow eye | 54 weeks |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of supplemental anti-VEGF injections in the study eye | 5 years inclusive of parent study | Mean number of supplemental anti-VEGF injections based on chart review |
| Number of retinal specialist visits for the study eye | 5 years inclusive of parent study | Mean number of retinal specialist visits attended for nAMD based on chart review |
| Fellow eye substudy: Change from baseline in best corrected visual acuity | 54 weeks | Mean change from baseline in BCVA in the fellow eye |
| Change from baseline in best corrected visual acuity (BCVA) in the study eye | 5 years inclusive of parent study | Mean change and proportion of participants losing greater than or equal to 5, 10, and 15 letters |
| Fellow eye substudy: Number of supplemental anti-VEGF injections in the fellow eye | 54 weeks | Mean number of supplemental anti-VEGF injections in the fellow eye |
| Fellow eye substudy: Aqueous and Serum RGX-314 transgene product (TP) concentrations in the fellow eye | 54 weeks | Fellow eye aqueous and serum RGX-314 TP concentrations at assessed time points |
| Fellow eye substudy: Immunogenicity measurements in the fellow eye | 54 weeks | Fellow eye immunogenicity measurements at assessed timepoints |
| Fellow eye substudy: Change from baseline in Central Retinal Thickness in the fellow eye | 54 weeks | Mean change from baseline in CRT in the fellow eye as measured by SD-OCT |
| Change from baseline in Central Retinal Thickness (CRT) in the study eye | 5 years inclusive of parent study | Mean change from baseline in CRT as measured by spectral domain optical coherence tomography (SD-OCT) |
Countries
United States
Outcome results
None listed