Gene therapy for wet age-related macular degeneration
A Phase 2, Randomized, Dose-escalation, Ranibizumab-controlled Study to Evaluate the Efficacy, Safety, and Tolerability of RGX-314 Gene Therapy Delivered Via One or Two Suprachoroidal Space (SCS) Injections in Participants With Neovascular Age-Related Macular Degeneration (nAMD) (AAVIATE)
This study is testing a one-time gene therapy called RGX-314 to see if it can help people with wet age-related macular degeneration need fewer injections compared to the standard treatment.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 115 (estimated) |
| Ages | 50 Years to 89 Years |
| Sex | All |
| Sponsor | AbbVie Industry-sponsored |
| Drugs / interventions | ranibizumab |
| Locations | 15 sites (Phoenix, Arizona and 14 other locations) |
| Trial ID | NCT04514653 on ClinicalTrials.gov |
What this trial studies
This phase 2 clinical trial evaluates the efficacy, safety, and tolerability of RGX-314, a one-time gene therapy, for treating neovascular age-related macular degeneration (nAMD). The study involves approximately 115 participants who will be randomized into different cohorts to receive RGX-314 or the standard treatment, ranibizumab. Participants will be monitored for their response to the treatment, with a focus on reducing the need for frequent injections that are currently required for managing nAMD. The trial aims to provide a more sustainable treatment option for patients suffering from this condition.
Who should consider this trial
Good fit: Ideal candidates are individuals aged 50 to 89 with a diagnosis of nAMD who have previously responded well to anti-VEGF therapy.
Not a fit: Patients with other causes of choroidal neovascularization or those who have undergone certain prior ocular surgeries may not benefit from this study.
Why it matters
Potential benefit: If successful, this gene therapy could significantly reduce the treatment burden for patients with wet AMD by potentially eliminating the need for frequent injections.
How similar studies have performed: Other studies have shown promise with gene therapy approaches for retinal diseases, indicating potential for success in this novel treatment.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Age \>/= 50 and \</= 89 * Diagnosis of CNV secondary to age-related macular degeneration in the study eye. * Participants must have demonstrated a meaningful response to anti-VEGF therapy. * Willing and able to provide written, signed informed consent for this study. Exclusion Criteria: * CNV or macular edema in the study eye secondary to any causes other than AMD. * Subfoveal fibrosis or atrophy in study eye. * Participants who have had a prior vitrectomy. * Active or history of retinal detachment in the study eye. * History of intravitreal therapy in the study eye, such as intravitreal steroid injection or investigational product (IP), other than anti-VEGF therapy, in the 6 months prior to study entry. * Received any gene therapy. * Any condition preventing visualization of the fundus or VA improvement in the study eye, eg, cataract. * History of intraocular surgery in the study eye within 12 weeks of study entry. * Receipt of any IP within 30 days of study entry or 5 half-lives of the IP. * Myocardial infarction, cerebrovascular accident, or transient ischemic attacks within 6 months of study entry. * Cohorts 1 - 5 only: Uncontrolled glaucoma in the study eye. COHORT 6 ONLY: * Active or history of glaucoma or ocular hypertension in the study eye. * Certain OCT characteristics including: Large Pigment Epithelial Detachments (PED), clinically significant Epiretinal Membrane (ERM) in the study eye at Visit 1. Note: Other inclusion/exclusion criteria apply.
Where this trial is running
Phoenix, Arizona and 14 other locations
- Phoenix Location — Phoenix, Arizona, United States (Recruiting)
- Bakersfield Location — Bakersfield, California, United States (Active_not_recruiting)
- Beverly Hills Location — Beverly Hills, California, United States (Recruiting)
- Mountain View Location — Mountain View, California, United States (Recruiting)
- Poway Location — Poway, California, United States (Recruiting)
- Santa Barbara Location — Santa Barbara, California, United States (Active_not_recruiting)
- Augusta Location — Augusta, Georgia, United States (Recruiting)
- Baltimore Location — Baltimore, Maryland, United States (Recruiting)
- Boston Location — Boston, Massachusetts, United States (Recruiting)
- Reno Location — Reno, Nevada, United States (Recruiting)
- Albuquerque Location — Albuquerque, New Mexico, United States (Recruiting)
- Philadelphia Location — Philadelphia, Pennsylvania, United States (Recruiting)
- Germantown Location — Germantown, Tennessee, United States (Active_not_recruiting)
- Nashville Location — Nashville, Tennessee, United States (Active_not_recruiting)
- Woodlands Location — The Woodlands, Texas, United States (Active_not_recruiting)
Study contacts
- Study coordinator: Patient Advocacy
- Email: patientadvocacy@regenxbio.com
- Phone: 1-833-711-0349
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.