Efficacy and Safety Study of Ixoberogene Soroparvovec (Ixo-vec) in Participants With Neovascular Age-related Macular Degeneration (AQUARIUS)
Start Date
3/16/2026
Completion Date
10/20/2031
Summary
This is a multi-center, randomized, double-masked, active-comparator-controlled, Phase 3 study in a broad participant population (treatment-naïve and treatment-experienced) with neovascular (wet) age-related macular degeneration (nAMD). The study will evaluate a single intravitreal (IVT) injection of Ixo-vec compared to intravitreal aflibercept (active comparator). The primary endpoint of this study is the mean change in best corrected visual acuity (BCVA) of Ixo-vec compared to an active comparator measured as an average at Weeks 52 and 56. Safety, tolerability, and efficacy will be evaluated throughout the study.
Detailed Description
The primary objective of this study is to evaluate the non-inferiority in efficacy of a single IVT injection of Ixo-vec 6 x 10\^10 vector genome (vg)/eye compared to an active comparator. Non-inferiority will be evaluated using a pre-specified margin defined in the protocol. Neovascular AMD is a degenerative ocular disease associated with the infiltration of abnormal blood vessels in the retina from the underlying choroid layer and is a leading cause of blindness in patients over 65 years of age. The abnormal angiogenic process in nAMD is stimulated and modulated by vascular endothelial growth factor (VEGF). Treatment of nAMD requires frequent IVT injections of VEGF inhibitors (anti-VEGF) administered every 4-16 weeks. Ixo-vec (also known as ADVM-022 or AAV.7m8-aflibercept) is an adeno-associated virus (AAV)-based gene therapy product being developed for the treatment of nAMD. Ixo-vec is designed to reduce the current treatment burden which often results in undertreatment and vision loss in patients with nAMD receiving anti-VEGF therapy in clinical practice. Safety, tolerability, and efficacy will be evaluated throughout this study. The primary endpoint of this study is the mean change in BCVA of Ixo-vec compared to an active comparator measured as an average at Weeks 52 and 56 post-treatment. Due to the long duration of the Screening period, this study will be considered fully enrolled when randomization has been completed.
Eligibility Criteria
Age Range: 50 years to No maximum
Interventions
Ixo-vec
Aflibercept
Conditions
Locations
Adverum Clinical Site 245
Mesa, Arizona 85206
United States
Adverum Clinical Site 126
Phoenix, Arizona 85014
United States
Adverum Clinical Site 178
Phoenix, Arizona 85020
United States
Adverum Clinical Site 223
Phoenix, Arizona 85020
United States
Adverum Clinical Site 229
Scottsdale, Arizona 85255
United States
Adverum Clinical Site 242
Sun City, Arizona 85351
United States
Adverum Clinical Site 198
Springdale, Arkansas 72764
United States
Adverum Clinical Site 109
Bakersfield, California 93309
United States
Adverum Clinical Site 100
Beverly Hills, California 90211
United States
Adverum Clinical Site 201
Campbell, California 95008
United States
Adverum Clinical Site 172
Encino, California 91436
United States
Adverum Clinical Site 169
Fullerton, California 92835
United States
Adverum Clinical Site 224
Huntington Beach, California 92647
United States
Adverum Clinical Site 130
Poway, California 92064
United States
Adverum Clinical Site 215
Redlands, California 92374
United States
Adverum Clinical Site 140
Sacramento, California 95825
United States
Adverum Clinical Site 212
Sacramento, California 95841
United States
Adverum Clinical Site 175
Santa Barbara, California 93103
United States
Adverum Clinical Site 202
Torrance, California 90503
United States
Adverum Clinical Site 189
Walnut Creek, California 94598
United States
Adverum Clinical Site 200
Denver, Colorado 800222
United States
Adverum Clinical Site 116
Denver, Colorado 80207
United States
Adverum Clinical Site 165
Waterford, Connecticut 06385
United States
Adverum Clinical Site 124
Deerfield Beach, Florida 33064
United States
Adverum Clinical Site 184
Deerfield Beach, Florida 33064
United States
Adverum Clinical Site 176
Fort Lauderdale, Florida 33308
United States
Adverum Clinical Site 221
Fort Myers, Florida 33912
United States
Adverum Clinical Site 236
Gainesville, Florida 32607
United States
Adverum Clinical Site 214
Lakeland, Florida 33805
United States
Adverum Clinical Site 168
Miami Springs, Florida 33166
United States
Adverum Clinical Site 213
Orlando, Florida 32806
United States
Adverum Clinical Site 206
Plantation, Florida 33324
United States
Adverum Clinical Site 183
South Miami, Florida 33143
United States
Adverum Clinical Site 120
St. Petersburg, Florida 33711
United States
Adverum Clinical Site 148
Tampa, Florida 33609
United States
Adverum Clinical Site 238
Winter Haven, Florida 33880
United States
Adverum Clinical Site 182
Augusta, Georgia 30909
United States
Adverum Clinical Site 246
Chicago, Illinois 60616
United States
Adverum Clinical Site 179
Oak Forest, Illinois 60452
United States
Adverum Clinical Site 207
Oak Park, Illinois 60304
United States
Adverum Clinical Site 195
Carmel, Indiana 46032
United States
Adverum Clinical Site 205
Carmel, Indiana 46032
United States
Adverum Clinical Site 253
Carmel, Indiana 46032
United States
Adverum Clinical Site 197
Hagerstown, Maryland 21740
United States
Adverum Clinical Site 204
Hagerstown, Maryland 21740
United States
Adverum Clinical Site 216
Jackson, Mississippi 39202
United States
Adverum Clinical Site 240
Bloomfield, New Jersey 07003
United States
Adverum Clinical Site 171
Teaneck, New Jersey 07666
United States
Adverum Clinical Site 225
Liverpool, New York 13088
United States
Adverum Clinical Site 244
Rochester, New York 14620
United States
Adverum Clinical Site 196
Asheville, North Carolina 28803
United States
Adverum Clinical Site 234
Cary, North Carolina 27511
United States
Adverum Clinical Site 211
Greensboro, North Carolina 27401
United States
Adverum Clinical Site 219
Greensboro, North Carolina 27401
United States
Adverum Clinical Site 220
Hickory, North Carolina 28602
United States
Adverum Clinical Site 209
Wake Forest, North Carolina 27587
United States
Adverum Clinical Site 248
Beachwood, Ohio 44122-5846
United States
Adverum Clinical Site 247
Cleveland, Ohio 44130
United States
Adverum Clinical Site 254
Edmond, Oklahoma 73013
United States
Adverum Clinical Site 252
Portland, Oregon 97225
United States
Adverum Clinical Site 181
Erie, Pennsylvania 16505
United States
Adverum Clinical Site 239
Philadelphia, Pennsylvania 19102
United States
Adverum Clinical Site 110
Philadelphia, Pennsylvania 19107
United States
Adverum Clinical Site 241
Bluffton, South Carolina 29910
United States
Adverum Clinical Site 222
Ladson, South Carolina 29456
United States
Adverum Clinical Site 101
Nashville, Tennessee 37203
United States
Adverum Clinical Site 145
Arlington, Texas 76012
United States
Adverum Clinical Site 127
Austin, Texas 78705
United States
Adverum Clinical Site 243
Austin, Texas 78750
United States
Adverum Clinical Site 107
Conroe, Texas 77384
United States
Adverum Clinical Site 194
Dallas, Texas 75231
United States
Adverum Clinical Site 162
McAllen, Texas 78503
United States
Adverum Clinical Site 185
San Antonio, Texas 78240
United States
Adverum Clinical Site 232
San Marcos, Texas 78666
United States
Adverum Clinical Site 228
Schertz, Texas 78154
United States
Adverum Clinical Site 231
The Woodlands, Texas 77384
United States
Adverum Clinical Site 237
Tyler, Texas 75703
United States
Adverum Clinical Site 199
Lynchburg, Virginia 24502
United States
Adverum Clinical Site 249
Silverdale, Washington 98383
United States