Ixoberogene Soroparvovec (Ixo-vec) Contralateral Dosing Study in Participants With Neovascular Age-related Macular Degeneration

NCT07630649 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: ADVM-022-14
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 4

Brief Summary

The purpose of this study is to evaluate safety, effectiveness and durability of a gene therapy called Ixo-vec (Ixoberogene soroparvovec) when administered to the contralateral (second) eye of adult participants (≥ 50 years of age) who have been diagnosed with bilateral neovascular (wet) age related macular degeneration (nAMD). The study will enroll adults with nAMD in both eyes, including participants who previously received Ixo-vec treatment in one (initial) eye and/or participants who will receive Ixo-vec treatment for the first time. This study focuses on how the treatment works when both eyes are treated at different times and how effective and long-lasting Ixo-vec treatment is in the second (contralateral) eye. Participants will receive a single administration of Ixo-vec in the contralateral eye and will be followed for approximately 5 years to evaluate safety, efficacy and durability of contralateral treatment. Secondary objectives include assessments that will evaluate clinical activity, including visual and anatomic outcomes, as well as the need for supplemental anti-VEGF therapy. The study is intended to provide additional information on the safety, tolerability, and use of Ixo-vec in bilateral treatment.

Conditions

Neovascular Age-Related Macular Degeneration (nAMD) Wet AMD

Keywords

macular degeneration; AMD; neovascular macular degeneration; gene therapy; contralateral; Ixoberogene soroparvovec; Ixo-vec; Aflibercept; Neovascular age-related macular degeneration; nAMD; wAMD; ADVM; ADVM-022; wet AMD; Wet Age-related Macular Degeneration; CNV; Neovascular AMD; AAV; AAV vector; AAV.7m8-aflibercept; AAV.7m8; Eye disease; Blindness; Adeno-associated viruses; ADVM-022-14

Outcome Measures

Primary Outcomes (3)

• Incidence of ocular adverse events (AEs)

  The number of participants who experience an ocular adverse event will be summarized.

  Baseline through Week 28

• Incidence of ocular serious adverse events (SAEs).

  The number of participants who experience ocular serious adverse event will be summarized.

  Baseline through Week 28

• Incidence of non-ocular serious adverse events (SAEs).

  The number of participants who experience non-ocular serious adverse event will be summarized.

  Baseline through Week 28

Secondary Outcomes (4)

• Mean change from baseline in best corrected visual acuity (BCVA) over time through Week 28.

  Baseline through Week 28

• Mean change from baseline in central subfield thickness (CST) over time through Week 28.

  Baseline through Week 28

• Mean number of supplemental aflibercept IVT injections received from Week 4 through Week 28

  Week 4 through Week 28

• Percentage of participants who are supplemental aflibercept injection-free through Week 28.

  Baseline through Week 28

Study Arms (1)

Ixo-vec dosing in Contralateral Eye

EXPERIMENTAL

Population of Ixo-vec-Experienced or Ixo-vec Naïve Participants.

Genetic: Ixo-vec

Interventions

Ixo-vec GENETIC

Ixo-vec (6 × 10\^10 vg/eye) will be administered intravitreally

Ixo-vec dosing in Contralateral Eye

Eligibility Criteria

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

You may qualify if:

• Male or female, aged ≥ 50 years at Screening Visit 1.
• Must agree to use an acceptable form of contraception.
• Have diagnosis of bilateral CNV secondary to nAMD.
• Meet ETDRS BCVA letter score criteria:
• For Ixo-vec-experienced participants: Contralateral eye has an ETDRS BCVA letter score within a protocol-defined range appropriate for nAMD clinical trials.
• For Ixo-vec-naïve participants: Both the initial eye and contralateral eye have ETDRS BCVA letter score within a protocol-defined range appropriate for nAMD clinical trials
• Have prior treatment history consistent with protocol-defined requirements, including prior anti-VEGF therapy and, where applicable, prior Ixo-vec exposure.
• Demonstrate a meaningful anatomic response to anti-VEGF in the contralateral eye (all participants) and in the initial eye (Ixo-vec-naïve participants only). Meaningful anatomic response to prior anti-VEGF therapy as defined in the protocol.
• Able to comply with study procedures according to the Investigator's judgment.

You may not qualify if:

• Received prior gene therapy (other than Ixo-vec, where applicable)
• Currently receiving steroids at Screening Visit 1 in the previously treated eye (Ixo-vec-experienced participants) or in either eye (Ixo-vec-naïve participants).
• Received any non-gene IMP or medical device in the contralateral eye (and/or initial eye for Ixo-vec-naïve participants) within 3 months of Screening Visit 1
• Have history of allergy, hypersensitivity, or contraindications to the use of any product or its excipients administered as part of this study.
• Have evidence of poorly controlled diabetes or glycated hemoglobin (HbA1c) ≥ 8.0%
• Have history or evidence of cardiovascular diseases and ongoing bleeding disorders as defined by the protocol.
• Use of systemic immunosuppressive therapy within a protocol-defined period.
• Received systemic anti-VEGF therapy within a protocol-defined period.
• Have history of malignancy within the 5 years prior to Screening Visit 1, except for adequately treated malignancies as defined by the protocol.
• Have any active ocular or periocular infection in the contralateral eye in the contralateral eye (all participants) or in the initial eye (Ixo-vec-naïve participants only).
• Have any history or evidence of a concurrent intraocular condition in the contralateral eye (all participants) or in the initial eye (Ixo-vec-naïve participants only), including retinal diseases other than nAMD, that, in the judgment of the Investigator, could reduce the potential for visual improvement, confound assessment of the macula or require medical or surgical intervention during the study.
• Have prior ocular surgery or treatment that may confound assessment or increase risk.
• Have any history or evidence of retinal detachment (with or without repair) or retinal pigment epithelium rip/tear in the contralateral eye (all participants) or in the initial eye (Ixo-vec-naïve participants only), as determined by the Investigator.
• Have uncontrolled ocular hypertension or glaucoma as defined by the protocol.
• Have any history of inflammatory anterior chamber cell or vitreous cell ≥ 2+ in the initial eye, that, in the opinion of the Investigator and Sponsor, predisposes the contralateral eye to a heightened risk of intraocular inflammation. Enrollment of a participant with prior anterior chamber cell or vitreous cell ≥2+ in the initial eye requires discussion with the medical monitor.

Sponsors & Collaborators

• Adverum Biotechnologies, Inc.: lead

Study Sites (4)

Adverum site 124

Deerfield Beach, Florida, 33064, United States

NOT YET RECRUITING

Adverum site 176

Fort Lauderdale, Florida, 33308, United States

RECRUITING

Adverum site 168

Jacksonville, Florida, 32216, United States

NOT YET RECRUITING

Adverum site 122

West Columbia, South Carolina, 29169, United States

RECRUITING

MeSH Terms

Conditions

Macular Degeneration; Eye Diseases; Blindness

Condition Hierarchy (Ancestors)

Retinal Degeneration; Retinal Diseases; 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; ADVM02214ClinOp@adverum.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Participants previously treated in one eye (initial eye) and/or treatment-naive participants will receive sequential bilateral administration, with dosing in the contralateral eye.

Study Record Dates

• First Submitted: June 2, 2026
• First Posted: June 5, 2026
• Study Start: June 2, 2026
• Primary Completion (Estimated): June 1, 2031
• Study Completion (Estimated): June 1, 2031
• Last Updated: June 5, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will share

Locations

US (4)