Pivotal 2 Study of RGX-314 Gene Therapy in Participants With nAMD

ID#: NCT05407636

Age: 50 - 89 years

Gender: All

Healthy Subjects: No

Study Phase: Phase 3

Recruitment Status: Recruiting

Start Date: December 28, 2021

End Date: December 01, 2025

Contact Information:
Patient Advocacy
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Summary: ABBV-RGX-314 (also known as RGX-314) is being developed as a novel one-time gene therapy for the treatment of neovascular (wet) age-related macular degeneration (wet AMD). Wet AMD is characterized by loss of vision due to new, leaky blood vessel formation in the retina. Wet AMD is a significant cause of vision loss in the United States, Europe and Japan, with up to 2 million people living with wet AMD in these geographies alone. Current anti-vascular endothelial growth factor (VEGF) therapies have significantly changed the landscape for treatment of wet AMD, becoming the standard of care due to their ability to prevent progression of vision loss in the majority of patients. These therapies, however, require life-long intraocular injections, typically repeated every four to 12 weeks in frequency, to maintain efficacy. Due to the burden of treatment, patients often experience a decline in vision with reduced frequency of treatment over time. ABBV-RGX-314 is being developed as a potential one-time treatment for wet AMD.
Eligibility:

Inclusion Criteria:

1. Age ≥ 50 years and ≤ 89 years

2. An ETDRS BCVA letter score between ≤ 78 and ≥ 40 in the study eye

3. Diagnosis of subfoveal choroidal neovascularization (CNV) secondary to AMD in the study eye previously treated with anti-VEGF

4. Must be pseudophakic (at least 12 weeks postcataract surgery) in the study eye

5. Willing and able to provide written, signed informed consent for this study

6. Participants must have demonstrated a meaningful response to anti-VEGF therapy at study entry Inclusion Criteria (Bilateral Treatment Substudy)*:

1. An ETDRS BCVA letter score between ≤ 83 and ≥ 40 in both eyes

2. Diagnosis of subfoveal choroidal neovascularization (CNV) secondary to AMD in both eyes

3. Must be pseudophakic (at least 12 weeks postcataract surgery) in both eyes

4. Willing and able to provide written, signed informed consent for this study

5. Newcomers must have active disease in the study eye; crossover participants must have active disease in the eye not treated in the main study

Exclusion Criteria:

1. CNV or macular edema in the study eye secondary to any causes other than AMD

2. Subfoveal fibrosis or atrophy in the study eye

3. Any condition in the investigator's opinion that could limit VA improvement in the study eye

4. Active or history of retinal detachment, or current retinal tear that cannot be treated, in the study eye

5. Advanced glaucoma or history of secondary glaucoma in the study eye

6. Myocardial infarction, cerebrovascular accident, or transient ischemic attack within the past 6 months

7. History of intraocular surgery in the study eye within 12 weeks prior to randomization

8. History of intravitreal therapy in the study eye, such as intravitreal steroid injection or investigational product, other than anti-VEGF therapy, in the 6 months prior to Screening Visit 1

9. Prior treatment with gene therapy Exclusion Criteria (Bilateral Treatment Substudy)*:

1. CNV or macular edema in either eye secondary to any causes other than AMD

2. Subfoveal fibrosis or atrophy in either eye

3. Any condition in the investigator's opinion that could limit VA improvement in either eye

4. Active or history of retinal detachment, or current retinal tear that cannot be treated in either eye

5. Advanced glaucoma or history of secondary glaucoma in either eye

6. Myocardial infarction, cerebrovascular accident, or transient ischemic attack within the past 6 months

7. History of intraocular surgery in either eye within 12 weeks prior to randomization

8. History of intravitreal therapy in either eye, such as intravitreal steroid injection or investigational product, other than anti-VEGF therapy, in the 6 months prior to screening

9. Prior treatment with gene therapy (*) For previously treated crossover participants, criteria apply to the eye not treated in the main study only. Note: Other inclusion/exclusion criteria apply
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