AzTrials
AzTrials

Neovascular Age-Related Macular Degeneration (nAMD)Phase 2AAVIATENCT04514653

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

A Phase 2 study testing Ranibizumab, ABBV-RGX-314 Dose 1 and ABBV-RGX-314 Dose 2 in people with Neovascular Age-Related Macular Degeneration (nAMD), run by AbbVie at 18 sites in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
Search all trials

Trial summary

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). Read more about this study

Who is it for?
Ages 50 to 89 years
What is being tested?
Ranibizumab, ABBV-RGX-314 Dose 1 and ABBV-RGX-314 Dose 2
Could I receive a placebo?
No, everyone receives an active treatment
How many people
146 participants
Where
18 sitesUnited States
Study dates
25 Aug 2020to Feb 2027
Phase
Phase 2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 50 to 89 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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.

Who cannot join

  • 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.
Show all 15Show fewer
  • 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.

Trial locations and contacts

18 sites in 1 country.

18 sites in 1 country

United States18 sites

  • Eye Associates of New Mexico /ID# 255915

    Albuquerque, New Mexico, 87109

  • Southeast Retina Center /ID# 255912

    Augusta, Georgia, 30909

  • California Retina Consultants - Bakersfield /ID# 255910

    Bakersfield, California, 93309-1677

  • Johns Hopkins Hospital /ID# 255919

    Baltimore, Maryland, 21287

  • Retina Vitreous Assoc Med Grp /ID# 255921

    Beverly Hills, California, 90211

  • Ophthalmic Consultants of Boston /ID# 255917

    Boston, Massachusetts, 02114

  • Retinal Diagnostic Center /ID# 272275

    Campbell, California, 95008

  • Duke University Medical Center /ID# 267646

    Durham, North Carolina, 27710

  • Charles Retina Institute /ID# 255922

    Germantown, Tennessee, 38138

  • Northern California Retina Vitreous Associates Medical Group, Inc /ID# 255920

    Mountain View, California, 94040-4101

  • Tennessee Retina - Nashville /ID# 255918

    Nashville, Tennessee, 37203-1513

  • Mid Atlantic Retina /ID# 255906

    Philadelphia, Pennsylvania, 19107

  • Retinal Research Institute /ID# 255925

    Phoenix, Arizona, 85053

  • Retina Consultants of San Diego /ID# 255911

    Poway, California, 92064-2530

  • Sierra Eye Associates /ID# 255908

    Reno, Nevada, 89502

  • California Retina Consultants - Santa Barbara /ID# 255923

    Santa Barbara, California, 93103

  • Springfield Clinic - First /ID# 272274

    Springfield, Illinois, 62702

  • Retina Consultants - The Woodlands /ID# 255924

    The Woodlands, Texas, 77384

Other trials in United States

About this study

Official 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)

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.

Read the detailed description

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.

Being tested
Ranibizumab, ABBV-RGX-314 Dose 1, ABBV-RGX-314 Dose 2, ABBV-RGX-314 Dose 3, Local Steroid
Keywords
AMD, wet AMD, wAMD, nAMD

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • RanibizumabBiological

    Ranibizumab (anti-VEGF agent)

  • ABBV-RGX-314 Dose 1Genetic

    Also called Combination Product

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

  • ABBV-RGX-314 Dose 2Genetic

    Also called Combination Product

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

  • ABBV-RGX-314 Dose 3Genetic

    Also called Combination Product

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

  • Local SteroidDrug

    Local steroid

  • Topical SteroidDrug

    Topical steroid

  • ABBV-RGX-314 Dose 4Genetic

    Also called Combination Product

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

Study groups in NCT04514653
Study groupRoleWhat they receive
Ranibizumab controlActive comparatorControl treatment arm
Receives: Biological: Ranibizumab
ABBV-RGX-314 Treatment Arm (Dose 1)ExperimentalABBV-RGX-314 Dose 1
Receives: Genetic: ABBV-RGX-314 Dose 1
ABBV-RGX-314 Treatment Arm (Dose 2)ExperimentalABBV-RGX-314 Dose 2
Receives: Genetic: ABBV-RGX-314 Dose 2
ABBV-RGX-314 Treatment Arm (Dose 3)ExperimentalABBV-RGX-314 Dose 3
Receives: Genetic: ABBV-RGX-314 Dose 3
ABBV-RGX-314 Treatment Arm (Dose 3) and Local SteroidExperimentalABBV-RGX-314 Dose 3 and Local Steroid
Receives: Genetic: ABBV-RGX-314 Dose 3; Drug: Local Steroid
ABBV-RGX-314 Treatment Arm (Dose 3) and Topical SteroidExperimentalABBV-RGX-314 Dose 3 and Topical Steroid
Receives: Genetic: ABBV-RGX-314 Dose 3; Drug: Topical Steroid
ABBV-RGX-314 Treatment Arm (Dose 4) and Topical SteroidExperimentalABBV-RGX-314 Dose 4 and Topical Steroid
Receives: Drug: Topical Steroid; Genetic: ABBV-RGX-314 Dose 4

What is being measured

The primary outcome is the main question the study is designed to answer.

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

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

    Measured: 40 weeks

Secondary outcomes (14)

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

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

    Measured: 52 weeks

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

    Evaluate the safety and tolerability of ABBV-RGX-314

    Measured: 52 Weeks

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

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

    Measured: 52 weeks

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

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

    Measured: Up to Week 80

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

    Evaluate the effect of ABBV-RGX-314 on BCVA

    Measured: Up to Week 80

  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

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

    Measured: Up to Week 52

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

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

    Measured: Up to Week 80

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

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

    Measured: Up to Week 80

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

    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)

    Measured: 52 Weeks

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

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

    Measured: Up to Week 80

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

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

    Measured: Up to Week 80

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

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

    Measured: 80 Weeks

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

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

    Measured: 52 Weeks

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

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

    Measured: 80 Weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted17 Aug 2020
  2. Study start25 Aug 2020
  3. Primary completionAug 2026 (estimated)
  4. Study completionFeb 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Sequential
Masking
Single blind
Who is masked
Outcomes assessor
Enrolment
146 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04514653
Sponsor's protocol ID
RGX-314-2102 (M23-411)
Other IDs
M23-411
Acronym
AAVIATE
Status last verified
Feb 2026
First submitted
4 Aug 2020
Last updated
27 Feb 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
Yes
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Ranibizumab, ABBV-RGX-314 Dose 1 and ABBV-RGX-314 Dose 2 in people with Neovascular Age-Related Macular Degeneration (nAMD), run by AbbVie at 18 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 27 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 18 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Mean change from baseline in BCVA to Week 40 based on the Early Treatment Diabetic Retinopathy Study (ETDRS) score (time frame: 40 weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04514653.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Feb 2026.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in