Efficacy and Safety Study of Ixoberogene Soroparvovec (Ixo-vec) in Participants With Neovascular Age-Related Macular Degeneration

NCT06856577 | Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: ADVM-022-12
• Study Type: interventional
• Target: 284
• Locations: 1 country
• Sites: 79

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 an 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 at an average of Weeks 52 and 56. Safety, tolerability, and efficacy will be evaluated throughout the study.

Conditions

Neovascular Age-Related Macular Degeneration (nAMD); Wet AMD

Keywords

Ixoberogene soroparvovec; Ixo-vec; Aflibercept; Neovascular age-related macular degeneration; nAMD; ADVM; ADVM-022; ADVM-022-12; Wet AMD; wAMD; Wet Age-related Macular Degeneration; CNV; Neovascular AMD; AAV; AAV vector; AAV.7m8-aflibercept; AAV.7m8; Gene therapy; Eye disease; Blindness; Adeno-associated viruses

Outcome Measures

Primary Outcomes (1)

• Mean change from Baseline in Best-Corrected Visual Acuity (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 (25)

• 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

  Through Week 56

• Mean change from Week 1 in BCVA based on an average at Weeks 52 and 56

  Week 1 through Week 56

Study Arms (2)

Ixo-vec

EXPERIMENTAL

Participants 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

Genetic: Ixo-vec; Drug: Aflibercept

Aflibercept

ACTIVE COMPARATOR

Participants 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.

Drug: Aflibercept

Interventions

Ixo-vec GENETIC

Ixo-vec will be administered intravitreally.

Ixo-vec

Aflibercept DRUG

Aflibercept will be administered intravitreally.

Aflibercept; Ixo-vec

Eligibility Criteria

• Age: 50 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

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 Early Treatment Diabetic Retinopathy Study (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.

Sponsors & Collaborators

• Adverum Biotechnologies, Inc.: lead

Study Sites (79)

Adverum Clinical Site 223

Gilbert, Arizona, 85297, United States

RECRUITING

Adverum Clinical Site 178

Phoenix, Arizona, 85021, United States

RECRUITING

Adverum Clinical Site 126

Phoenix, Arizona, 85053, United States

RECRUITING

Adverum Clinical Site 229

Scottsdale, Arizona, 85255, United States

RECRUITING

Adverum Clinical Site 159

Tucson, Arizona, 85704, United States

RECRUITING

Adverum Clinical Site 198

Springdale, Arkansas, 72764, United States

RECRUITING

Adverum Clinical Site 109

Bakersfield, California, 93309, United States

RECRUITING

Adverum Clinical Site 100

Beverly Hills, California, 90211, United States

RECRUITING

Adverum Clinical Site 201

Campbell, California, 95008, United States

RECRUITING

Adverum Clinical Site 172

Encino, California, 91436, United States

RECRUITING

Adverum Clinical Site 169

Fullerton, California, 92835, United States

RECRUITING

Adverum Clinical Site 224

Huntington Beach, California, 92647, United States

RECRUITING

Adverum Clinical Site 215

Redlands, California, 92374, United States

RECRUITING

Adverum Clinical Site 164

Riverside, California, 92505, United States

RECRUITING

Adverum Clinical Site 140

Sacramento, California, 95825, United States

RECRUITING

Adverum Clinical Site 212

Sacramento, California, 95841, United States

RECRUITING

Adverum Clinical Site 175

Santa Barbara, California, 93103, United States

RECRUITING

Adverum Clinical Site 202

Torrance, California, 90503, United States

RECRUITING

Adverum Clinical Site 189

Walnut Creek, California, 94598, United States

RECRUITING

Adverum Clinical Site 200

Denver, Colorado, 80222, United States

RECRUITING

Adverum Clinical Site 116

Lakewood, Colorado, 80228, United States

RECRUITING

Adverum Clinical Site 165

Waterford, Connecticut, 06385, United States

RECRUITING

Adverum Clinical Site 184

Deerfield Beach, Florida, 33064, United States

RECRUITING

Adverum Clinical Site 176

Fort Lauderdale, Florida, 33308, United States

RECRUITING

Adverum Clinical Site 221

Fort Myers, Florida, 33912, United States

RECRUITING

Adverum Clinical Site 236

Gainesville, Florida, 32605, United States

RECRUITING

Adverum Clinical Site 168

Jacksonville, Florida, 32216, United States

RECRUITING

Adverum Clinical Site 214

Lakeland, Florida, 33805, United States

RECRUITING

Adverum Clinical Site 213

Orlando, Florida, 32806, United States

RECRUITING

Adverum Clinical Site 230

Pensacola, Florida, 32503, United States

RECRUITING

Adverum Clinical Site 124

Pompano Beach, Florida, 33064, United States

RECRUITING

Adverum Clinical Site 183

South Miami, Florida, 33143, United States

RECRUITING

Adverum Clinical Site 120

St. Petersburg, Florida, 33711, United States

RECRUITING

Adverum Clinical Site 193

Tampa, Florida, 33617, United States

RECRUITING

Adverum Clinical Site 182

Augusta, Georgia, 30909, United States

RECRUITING

Adverum Clinical Site 149

‘Aiea, Hawaii, 96701, United States

RECRUITING

Adverum Clinical Site 179

Oak Forest, Illinois, 60452, United States

RECRUITING

Adverum Clinical Site 207

Oak Park, Illinois, 60304, United States

RECRUITING

Adverum Clinical Site 195

Carmel, Indiana, 46032, United States

RECRUITING

Adverum Clinical Site 205

Carmel, Indiana, 46032, United States

RECRUITING

Adverum Clinical Site 197

Hagerstown, Maryland, 21740, United States

RECRUITING

Adverum Clinical Site 204

Hagerstown, Maryland, 21740, United States

RECRUITING

Adverum Clinical Site 167

Detroit, Michigan, 48201, United States

RECRUITING

Adverum Clinical Site 216

Madison, Mississippi, 39110, United States

RECRUITING

Adverum Clinical Site 163

Southaven, Mississippi, 38671, United States

RECRUITING

Adverum Clinical Site 190

St Louis, Missouri, 63128, United States

RECRUITING

Adverum Clinical Site 171

Teaneck, New Jersey, 07666, United States

RECRUITING

Adverum Clinical Site 225

Liverpool, New York, 13088, United States

RECRUITING

Adverum Clinical Site 196

Asheville, North Carolina, 28803, United States

RECRUITING

Adverum Clinical Site 234

Cary, North Carolina, 27511, United States

RECRUITING

Adverum Clinical Site 186

Durham, North Carolina, 27705, United States

RECRUITING

Adverum Clinical Site 211

Greensboro, North Carolina, 27401, United States

RECRUITING

Adverum Clinical Site 220

Hickory, North Carolina, 28602, United States

RECRUITING

Adverum Clinical Site 209

Wake Forest, North Carolina, 27587, United States

RECRUITING

Adverum Clinical Site 219

Winston-Salem, North Carolina, 27103, United States

RECRUITING

Adverum Clinical Site 181

Erie, Pennsylvania, 16507, United States

RECRUITING

Adverum Clinical Site 110

Philadelphia, Pennsylvania, 19107, United States

RECRUITING

Adverum Clinical Site 208

Charleston, South Carolina, 29414, United States

RECRUITING

Adverum Clinical Site 222

Ladson, South Carolina, 29456, United States

RECRUITING

Adverum Clinical Site 122

West Columbia, South Carolina, 29169, United States

RECRUITING

Adverum Clinical Site 144

Rapid City, South Dakota, 57701, United States

RECRUITING

Adverum Clinical Site 123

Abilene, Texas, 79606, United States

RECRUITING

Adverum Clinical Site 127

Austin, Texas, 78705, United States

RECRUITING

Adverum Clinical Site 108

Bellaire, Texas, 77401, United States

RECRUITING

Adverum Clinical Site 227

Burleson, Texas, 76028, United States

RECRUITING

Adverum Clinical Site 194

Dallas, Texas, 75231, United States

RECRUITING

Adverum Clinical Site 188

Houston, Texas, 77030, United States

RECRUITING

Adverum Clinical Site 218

Katy, Texas, 77494, United States

RECRUITING

Adverum Clinical Site 162

McAllen, Texas, 78503, United States

RECRUITING

Adverum Clinical Site 151

San Antonio, Texas, 78240, United States

RECRUITING

Adverum Clinical Site 185

San Antonio, Texas, 78240, United States

RECRUITING

Adverum Clinical Site 232

San Marcos, Texas, 78666, United States

RECRUITING

Adverum Clinical Site 228

Schertz, Texas, 78154, United States

RECRUITING

Adverum Clinical Site 107

The Woodlands, Texas, 77384, United States

RECRUITING

Adverum Clinical Site 231

The Woodlands, Texas, 77384, United States

RECRUITING

Adverum Clinical Site 199

Lynchburg, Virginia, 24502, United States

RECRUITING

Adverum Clinical Site 131

Spokane, Washington, 99204, United States

RECRUITING

Adverum Clinical Site 152

Morgantown, West Virginia, 26506, United States

RECRUITING

Adverum Clinical Site 187

Wausau, Wisconsin, 54403, United States

RECRUITING

MeSH Terms

Conditions

Eye Diseases; Blindness

Interventions

aflibercept

Condition Hierarchy (Ancestors)

Vision Disorders; Sensation Disorders; Neurologic Manifestations; Nervous System Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Officials

• Adam Turpcu, PhD

  Adverum Biotechnologies, Inc.

  STUDY DIRECTOR

Central Study Contacts

Adverum Study Contact

CONTACT

650-656-9323; ADVM02212ClinOp@adverum.com

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: February 26, 2025
• First Posted: March 4, 2025
• Study Start: February 28, 2025
• Primary Completion (Estimated): December 22, 2026
• Study Completion (Estimated): November 23, 2030
• Last Updated: November 18, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will share

Locations

US (79)