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NCT Number: NCT04514653

RGX-314 Gene Therapy Administered in the Suprachoroidal Space for Participants With Neovascular Age-Related Macular Degeneration (nAMD)

This interventional study is being conducted with an investigational gene therapy treatment called ABBV-RGX-314 (also known as RGX-314) and is being developed as a potential one-time gene therapy treatment for neovascular (wet) age-related macular degeneration (wet AMD or nAMD). The typical treatment for nAMD is frequent injections of anti-VEGF therapy. Researchers are testing ABBV-RGX-314 to see if it has similar effects as the current approved standard of care, such as Lucentis® or Eylea® injections.

The duration of this study will be up to 52 weeks or for ranibizumab control participants who cross over to ABBV-RGX-314 after week 52, up to 80 weeks post-randomization.

The primary outcome measure for this investigational study is to evaluate the mean change in best-corrected visual acuity (BCVA) for ABBV-RGX-314 compared with ranibizumab monthly at the Week 40 visit.

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Key information

Conditions

Neovascular Age-related Macular Degeneration (nAMD)

Age range

50 year–89 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Retinal Research Institute /ID# 255925, Phoenix, Arizona, United States

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About this study

This phase 2, randomized, dose-escalation study is designed to evaluate the efficacy, safety and tolerability of ABBV-RGX-314 gene therapy in subjects with nAMD.

ABBV-RGX-314 will be injected into the SCS part of the eye. Approximately 140 participants who meet the inclusion/exclusion criteria will be enrolled into one of 7 cohorts. Participants will be randomized in Cohorts 1 and 2 to receive ABBV-RGX-314 or the intravitreal ranibizumab control. Participants enrolled in Cohorts 3 through 5 will receive ABBV-RGX-314. Participants enrolled in Cohort 6 will receive ABBV-RGX-314 and will be randomized to one of two different post-procedural steroid regimens. Participants randomized in Cohort 7 will receive ABBV-RGX-314 with a protocol mandated steroid regimen or the intravitreal ranibizumab control. Cohort 1 will evaluate ABBV-RGX-314 Dose 1, Cohorts 2 and 3 will evaluate ABBV-RGX-314 Dose 2, Cohorts 4, 5, and 6 will evaluate ABBV-RGX-314 Dose 3, and Cohort 7 will evaluate ABBV-RGX-314 Dose 4.

The duration of the study will be up to 52 weeks or for ranibizumab control participants who cross over to ABBV-RGX-314, up to 80 weeks post-randomization.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Age >/= 50 and </= 89
  • Diagnosis of CNV secondary to age-related macular degeneration in the study eye.
  • Participants must have demonstrated a meaningful response to anti-VEGF therapy.
  • Willing and able to provide written, signed informed consent for this study.

Exclusion criteria

  • CNV or macular edema in the study eye secondary to any causes other than AMD.
  • Subfoveal fibrosis or atrophy in study eye.
  • Participants who have had a prior vitrectomy.
  • Active or history of retinal detachment in the study eye.
  • History of intravitreal therapy in the study eye, such as intravitreal steroid injection or investigational product (IP), other than anti-VEGF therapy, in the 6 months prior to study entry.
  • Received any gene therapy.
  • Any condition preventing visualization of the fundus or VA improvement in the study eye, eg, cataract.
  • History of intraocular surgery in the study eye within 12 weeks of study entry.
  • Receipt of any IP within 30 days of study entry or 5 half-lives of the IP.
  • Myocardial infarction, cerebrovascular accident, or transient ischemic attacks within 6 months of study entry.
  • Cohorts 1 - 5 only: Uncontrolled glaucoma in the study eye.

COHORT 6 AND 7 ONLY:

  • Active or history of glaucoma or ocular hypertension (steroid-induced ocular hypertension (Cohort 7 only)) in the study eye.
  • Certain OCT characteristics including: Large Pigment Epithelial Detachments (PED), clinically significant Epiretinal Membrane (ERM) in the study eye at Visit 1.

Note: Other inclusion/exclusion criteria apply.

Treatment and study plan

ranibizumab

Biological

Ranibizumab (anti-VEGF agent)

ABBV-RGX-314 Dose 1

Genetic

AAV8 vector containing a transgene for anti-VEGF fab (Dose 1)

Other names: Combination Product

ABBV-RGX-314 Dose 2

Genetic

AAV8 vector containing a transgene for anti-VEGF fab (Dose 2)

Other names: Combination Product

ABBV-RGX-314 Dose 3

Genetic

AAV8 vector containing a transgene for anti-VEGF fab (Dose 3)

Other names: Combination Product

Local Steroid

Drug

Local steroid

Topical Steroid

Drug

Topical steroid

ABBV-RGX-314 Dose 4

Genetic

AAV8 vector containing a transgene for anti-VEGF fab (Dose 4)

Other names: Combination Product

Primary outcomes

  1. Mean change from baseline in BCVA to Week 40 based on the Early Treatment Diabetic Retinopathy Study (ETDRS) score

    Time frame: 40 weeks

    The scale used is the early treatment diabetic retinopathy study (ETDRS) letter score from 0-100 and higher score being better vision.

Secondary outcomes

  1. Incidence of overall and ocular adverse events (AEs) and serious adverse events (SAEs)

    Time frame: 52 weeks

    Incidence of overall and ocular adverse events (AEs) and serious adverse events (SAEs)

  2. Vector shedding analysis in serum, urine, and tears

    Time frame: 52 Weeks

    Evaluate the safety and tolerability of ABBV-RGX-314

  3. Proportion of participants who experience ocular inflammation following SCS ABBV-RGX-314 administration

    Time frame: 52 weeks

    To evaluate the incidences of ocular inflammation following SCS ABBV-RGX-314 administration

  4. Mean change from baseline in choroidal neovascularization (CNV) lesion size and leakage area based on fluorescein angiography (FA)

    Time frame: Up to Week 80

    To evaluate the effect of ABBV-RGX-314 on CNV lesion growth and leakage as measured by FA

  5. Mean change from baseline in BCVA based on ETDRS score

    Time frame: Up to Week 80

    Evaluate the effect of ABBV-RGX-314 on BCVA

  6. Proportion of participants (1) gaining ≥ 15, ≥ 10, ≥ 5, or ≥ 0 letters; (2) losing ≥ 15, ≥ 10, ≥ 5, or > 0 letters (3) maintaining vision (not losing ≥ 15 letters) compared with baseline as per BCVA

    Time frame: Up to Week 52

    To evaluate the effect of ABBV-RGX-314 on BCVA

  7. Mean change from baseline in CRT as measured by SD-OCT

    Time frame: Up to Week 80

    To evaluate the effect of ABBV-RGX-314 on CRT, as measured by SD-OCT

  8. Mean supplemental anti-VEGF injection annualized rate in the ABBV-RGX-314 treatment arms

    Time frame: Up to Week 80

    To assess the need for supplemental anti-VEGF therapy in participants who receive ABBV-RGX-314 treatment

  9. Proportion of participants who have a reduction of ≥ 50% and ≥ 75% in supplemental anti-VEGF- injection annualized rate

    Time frame: 52 Weeks

    Proportion of participants who have a reduction of ≥ 50% and ≥ 75% in supplemental anti-VEGF- injection annualized rate through Week 40 and Week 52 compared with the prior 52 weeks preceding the first intravitreal ranibizumab injection received as part of the Screening Period (ABBV-RGX-314 administered participants)

  10. Proportion of participants with 0, ≤ 1, and ≤ 2 supplemental injections

    Time frame: Up to Week 80

    To assess the need for supplemental anti-VEGF therapy in participants who receive ABBV-RGX-314 treatment

  11. Mean percent reduction in supplemental anti-VEGF injection annualized rate

    Time frame: Up to Week 80

    To assess the need for supplemental anti-VEGF therapy in participants who receive ABBV-RGX-314 treatment

  12. Time from ABBV-RGX-314 administration to first supplemental anti-VEGF injection

    Time frame: 80 Weeks

    To assess the need for supplemental anti-VEGF therapy in participants who receive ABBV-RGX-314 treatment

  13. Aqueous humor ABBV-RGX- 314 TP concentration over time

    Time frame: 52 Weeks

    To evaluate the concentration of ABBV-RGX-314 TP in aqueous humor and serum

  14. Mean change from baseline in serum ABBV-RGX-314 TP concentration over time

    Time frame: 80 Weeks

    To evaluate the concentration of ABBV-RGX-314 TP in aqueous humor and serum

Sponsors and collaborators

Lead sponsor

AbbVie

Industry

Collaborators

  • REGENXBIO Inc.

Registry information

Official study title

A Phase 2, Randomized, Dose-escalation, Ranibizumab-controlled Study to Evaluate the Efficacy, Safety, and Tolerability of RGX-314 Gene Therapy Delivered Via One or Two Suprachoroidal Space (SCS) Injections in Participants With Neovascular Age-Related Macular Degeneration (nAMD) (AAVIATE)

Acronym: AAVIATE

Important dates

Study start
2020
Primary completion
2026
Study completion
2027
First posted
Aug 17, 2020
Registry last updated
Feb 27, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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