A Randomized, Partially Masked, Controlled, Phase 3 Clinical Study to Evaluate the Efficacy and Safety of RGX-314 Gene Therapy in Participants With nAMD
A Randomized, Partially Masked, Controlled, Phase 3 Clinical Study to Evaluate the Efficacy and Safety of RGX-314 Gene Therapy in Participants With nAMD
ABBV-RGX-314 (also known as RGX-314 and surabgene lomparvovec (sura-vec)) 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.
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 714 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), while 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.
Inclusion Criteria:
Inclusion Criteria (Bilateral Treatment Substudy)*:
Exclusion Criteria:
Exclusion Criteria (Bilateral Treatment Substudy)*:
Note: Other inclusion/exclusion criteria apply
Bakersfield, California 93309-1677, United States
Oxnard, California 93036, United States
Sacramento, California 95841, United States
Walnut Creek, California 94598, United States
St. Petersburg, Florida 33711-1141, United States
Chicago, Illinois 60611-2987, United States
Hagerstown, Maryland 21740-5965, United States
Edina, Minnesota 55435, United States
St Louis, Missouri 63108, United States
New York, New York 10032, United States
Beaufort, South Carolina 29902-6216, United States
Johnson City, Tennessee 37604, United States
La Crosse, Wisconsin 54601, United States
Vancouver, British Columbia V5Z 3N9, Canada
Nice, Alpes-Maritimes 06001, France
Lyon, Auvergne-Rhône-Alpes 69004, France
Nantes, Pays de la Loire Region 44000, France
Cona, Ferrara 44124, Italy
Rome, Roma 00168, Italy
Rome, Roma 00184, Italy
Nishinomiya-shi, Hyōgo 663-8501, Japan
Bunkyo-ku, Tokyo 113-8431, Japan
Santiago de Compostela, A Coruna 15706, Spain
Las Palmas de Gran Canaria, Las Palmas 35016, Spain
Barcelona, 08028, Spain
Southampton, Hampshire SO16 6YD, United Kingdom
Oxford, Oxfordshire OX3 9DU, United Kingdom