Pivotal 2 Study of RGX-314 Gene Therapy in Participants With nAMD
a study on Macular Degeneration WetAMD Copy Number Variation
Summary
- Eligibility
- for people ages 50-89 (full criteria)
- Location
- at UC Davis UCSD
- Dates
- study startedstudy ends around
Description
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.
Official Title
A Randomized, Partially Masked, Controlled, Phase 3 Clinical Study to Evaluate the Efficacy and Safety of RGX-314 Gene Therapy in Participants With nAMD
Details
This randomized, partially masked, controlled, Phase 3 clinical study will evaluate the efficacy and safety of ABBV-RGX-314 gene therapy in participants with nAMD. The study will evaluate 2 dose levels of RGX-314 gene therapy relative to an active comparator. The primary endpoint of this study is mean change in best-corrected visual acuity (BCVA) of ABBV-RGX-314 relative to aflibercept. Approximately 660 participants who meet the inclusion/exclusion criteria, will be enrolled into one of 3 arms.
A bilateral treatment substudy conducted at US sites is an open-label, partially randomized, parallel arm study to evaluate the safety and efficacy of subretinal ABBV-RGX-314 administered bilaterally in participants who have bilateral nAMD. Previously treated crossover participants from the control arm of the main study who crossed over and received ABBV-RGX-314 in the study eye will receive the same ABBV-RGX-314 dose in the contralateral eye (ie, same dose as in the study eye), and newcomers (participants who have not been randomized in an ABBV-RGX-314 study) and untreated crossover participants (ongoing control participants in the main study who have completed Week 54 but have not crossed over to receive ABBV-RGX-314 in the main study) will be randomized in a 2:1 ratio to receive ABBV-RGX-314 Dose 1 or ABBV-RGX-314 Dose 2 in both eyes. Up to 15 participants who qualify for the substudy will be enrolled and followed for a minimum of 50 weeks.
Keywords
AMD, nAMD, Wet Age-related Macular Degeneration, wAMD, WetAMD, CNV, Age-Related Macular Degeneration, Neovascular Age-Related Macular Degeneration, Wet Macular Degeneration, Choroidal Neovascularization, Retinal Degeneration, Retinal Diseases, Eye Diseases, Ranibizumab, Aflibercept, Angiogenesis Inhibitors, Angiogenesis Modulating Agents, Growth Substances, Physiological Effects of Drugs, Growth Inhibitors, Antineoplastic Agents, Gene Therapy, Anti-vascular endothelial grown factory therapy, Anti-VEGF therapy, Macular Degeneration, ABBV-RGX-314 Dose 1, ABBV-RGX-314 Dose 2, Aflibercept (EYLEA®)
Eligibility
You can join if…
Open to people ages 50-89
- Age ≥ 50 years and ≤ 89 years
- An ETDRS BCVA letter score between ≤ 78 and ≥ 40 in the study eye
- Diagnosis of subfoveal choroidal neovascularization (CNV) secondary to AMD in the study eye previously treated with anti-VEGF
- Must be pseudophakic (at least 12 weeks postcataract surgery) in the study eye
- Willing and able to provide written, signed informed consent for this study
- Participants must have demonstrated a meaningful response to anti-VEGF therapy at study entry
Inclusion Criteria (Bilateral Treatment Substudy)*:
- An ETDRS BCVA letter score between ≤ 83 and ≥ 40 in both eyes
- Diagnosis of subfoveal choroidal neovascularization (CNV) secondary to AMD in both eyes
- Must be pseudophakic (at least 12 weeks postcataract surgery) in both eyes
- Willing and able to provide written, signed informed consent for this study
- Newcomers must have active disease in the study eye; crossover participants must have active disease in the eye not treated in the main study
You CAN'T join if...
- CNV or macular edema in the study eye secondary to any causes other than AMD
- Subfoveal fibrosis or atrophy in the study eye
- Any condition in the investigator's opinion that could limit VA improvement in the study eye
- Active or history of retinal detachment, or current retinal tear that cannot be treated, in the study eye
- Advanced glaucoma or history of secondary glaucoma in the study eye
- Myocardial infarction, cerebrovascular accident, or transient ischemic attack within the past 6 months
- History of intraocular surgery in the study eye within 12 weeks prior to randomization
- 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
- Prior treatment with gene therapy
Exclusion Criteria (Bilateral Treatment Substudy)*:
- CNV or macular edema in either eye secondary to any causes other than AMD
- Subfoveal fibrosis or atrophy in either eye
- Any condition in the investigator's opinion that could limit VA improvement in either eye
- Active or history of retinal detachment, or current retinal tear that cannot be treated in either eye
- Advanced glaucoma or history of secondary glaucoma in either eye
- Myocardial infarction, cerebrovascular accident, or transient ischemic attack within the past 6 months
- History of intraocular surgery in either eye within 12 weeks prior to randomization
- 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
- 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
Locations
- Hamilton Glaucoma Center Shiley Eye Center UCSD /ID# 256320
accepting new patients
La Jolla 5363943 California 5332921 92093 United States - University of California Davis Health /ID# 256317
accepting new patients
Sacramento 5389489 California 5332921 95817 United States - Retina Vitreous Assoc Med Grp /ID# 256246
accepting new patients
Beverly Hills 5328041 California 5332921 90211 United States - The Retina Partners - Encino /ID# 259660
accepting new patients
Encino 5346649 California 5332921 91436 United States - Orange County Retina Medical Group /ID# 256286
accepting new patients
Santa Ana 5392900 California 5332921 92705 United States - Retina Consultants of San Diego /ID# 256258
accepting new patients
Poway 5384690 California 5332921 92064-2530 United States - Macula Retina Vitreous Center - Torrance /ID# 270247
accepting new patients
Torrance 5403022 California 5332921 90503 United States - Retina Consultants of Orange County /ID# 256267
accepting new patients
Fullerton 5351247 California 5332921 92835 United States - Retinal Consultants Medical Group, Inc. - Greenback /ID# 256353
accepting new patients
Sacramento 5389489 California 5332921 95841 United States - Bay Area Retina Associates - Walnut Creek - Lennon Lane /ID# 268513
accepting new patients
Walnut Creek 5406990 California 5332921 94598 United States
Details
- Status
- accepting new patients
- Start Date
- Completion Date
- (estimated)
- Sponsor
- AbbVie
- Links
- Related Info
- ID
- NCT05407636
- Phase
- Phase 3 research study
- Study Type
- Interventional
- Participants
- Expecting 660 study participants
- Last Updated