Efficacy and Safety Study of Ixoberogene Soroparvovec (Ixo-vec) in Participants With Neovascular Age-related Macular Degeneration (AQUARIUS)
AQUARIUS
A Multi-center, Randomized, Double-masked, Active-comparator-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Ixoberogene Soroparvovec (Ixo-vec) in Participants With Neovascular Age-related Macular Degeneration (AQUARIUS)
1 other identifier
interventional
284
1 country
46
Brief 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_3
Started Mar 2026
Longer than P75 for phase_3
46 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
March 16, 2026
CompletedStudy Start
First participant enrolled
March 16, 2026
CompletedFirst Posted
Study publicly available on registry
March 19, 2026
CompletedPrimary Completion
Last participant's last visit for primary outcome
November 15, 2027
ExpectedStudy Completion
Last participant's last visit for all outcomes
October 20, 2031
May 20, 2026
May 1, 2026
1.7 years
March 16, 2026
May 19, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Mean Change from Baseline in BCVA Based on an Average at Weeks 52 and 56
BCVA will be measured using an Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity chart.
Baseline, Week 52 and Week 56
Secondary Outcomes (26)
Mean Number of Aflibercept IVT Injections Received
Week 4 through Week 56
Percentage of Participants with Worsened BCVA
Through Week 56
Percentage of Participants with Improved BCVA
Through Week 56
Mean Change from Baseline in BCVA Over Time Based on an Average at Weeks 52 and 56
Baseline Through Week 56
Mean Change from Week 1 in BCVA Based on an Average at Weeks 52 and 56
Week 1 through Week 56
- +21 more secondary outcomes
Study Arms (2)
Ixo-vec
EXPERIMENTALParticipants will receive 3 monthly loading doses of aflibercept 2 mg IVT, a single IVT injection of Ixo-vec 6 x 10\^10 vg/eye at Week 1, and sham injections every 8 weeks.
Aflibercept
ACTIVE COMPARATORParticipants will receive 3 monthly loading doses of aflibercept 2 mg IVT, a sham injection at Week 1, and aflibercept 2 mg IVT every 8 weeks.
Interventions
Eligibility Criteria
You may qualify if:
- Able and willing to provide informed consent (or have a legally authorized representative who is able and willing to provide informed consent) prior to any study assessments and procedures and comply with the study requirements and visits.
- Male or female with a diagnosis of CNV secondary to nAMD in the study eye, with nAMD disease activity at Screening Visit 1.
- At least 50 years old at Screening Visit 1.
- An ETDRS BCVA letter score of 35 - 78 (approximate Snellen equivalent of 20/200 to 20/32) in the study eye at Screening Visit 1.
- Demonstrated a meaningful anatomic response to anti-VEGF therapy during screening.
- Able to reliably use eye drops per protocol.
You may not qualify if:
- Received any prior gene therapy.
- Prior treatment with any non-gene therapy investigational medicinal product (IMP) or medical device in the study eye within 3 months of Screening Visit 1 or 5 half-lives of the IMP prior to dosing with Ixo-vec, whichever is longer.
- Female participants who are pregnant or breastfeeding or who intend to become pregnant or breastfeed in the future.
- History or evidence of any of the following cardiovascular diseases:
- Myocardial infarction in the 6-month period prior to Week 1.
- Uncontrolled hypertension defined as systolic blood pressure \> 160 mmHg or diastolic blood pressure \> 100 mmHg during screening.
- Stroke in the 6-month period prior to Week 1.
- History of ongoing bleeding disorders. The use of aspirin or other anticoagulants (e.g., Factor Xa inhibitors) is permitted.
- Use of systemic immunosuppressive drugs within 90 days prior to Screening Visit 1. Short courses of oral corticosteroids are permitted, as well as any inhaled, intra-articular, nasal or dermal steroid use.
- Evidence of poorly controlled diabetes or glycated hemoglobin (HbA1c) ≥ 8.0% during screening.
- Any active ocular or periocular infection in the study eye from Screening Visit 1.
- History or evidence of the following in the study eye:
- Intraocular or refractive surgery within 5 months prior to Week 1.
- Any previous penetrating keratoplasty or vitrectomy.
- Any previous panretinal photocoagulation.
- +11 more criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (46)
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 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 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 202
Torrance, California, 90503, United States
Adverum Clinical Site 189
Walnut Creek, California, 94598, 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 214
Lakeland, Florida, 33805, United States
Adverum Clinical Site 213
Orlando, Florida, 32806, United States
Adverum Clinical Site 183
South Miami, Florida, 33143, United States
Adverum Clinical Site 182
Augusta, Georgia, 30909, United States
Adverum Clinical Site 195
Carmel, Indiana, 46032, United States
Adverum Clinical Site 204
Hagerstown, Maryland, 21740, United States
Adverum Clinical Site 216
Jackson, Mississippi, 39202, United States
Adverum Clinical Site 225
Liverpool, New York, 13088, United States
Adverum Clinical Site 196
Asheville, North Carolina, 28803, 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 241
Bluffton, South Carolina, 29910, United States
Adverum Clinical Site 222
Ladson, South Carolina, 29456, United States
Adverum Clinical Site 127
Austin, Texas, 78705, 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 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
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Adam Turpcu, PhD
Adverum Biotechnologies, Inc.
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- DOUBLE
- Who Masked
- PARTICIPANT, INVESTIGATOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
March 16, 2026
First Posted
March 19, 2026
Study Start
March 16, 2026
Primary Completion (Estimated)
November 15, 2027
Study Completion (Estimated)
October 20, 2031
Last Updated
May 20, 2026
Record last verified: 2026-05
Data Sharing
- IPD Sharing
- Will share
Adverum is committed to transparency. Appropriately de-identified participant-level data and supporting documents may be shared under appropriate conditions (e.g. when contractually permitted and when participants provide appropriate consent). Individual patient-level data would only be shared following completion of this study (and any associated studies), completion of applicable regulatory submissions, and in accordance with applicable regulations and laws, as well as criteria established by Adverum, our collaborators and/or industry best practices. Shared datasets and/or documents may be de-identified and/or redacted to protect participant identity and to protect sensitive and confidential information. For inquiries, please contact us at datasharing@adverum.com.