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Neovascular Age-related Macular DegenerationPhase 2NCT04832724

RGX-314 Gene Therapy Pharmacodynamic Study for Neovascular Age-related Macular Degeneration (nAMD)

A Phase 2 study testing RGX-314 in people with Neovascular Age-related Macular Degeneration and Wet Macular Degeneration, run by AbbVie at 13 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

RGX-314 is being developed as a novel one-time gene therapy for the treatment of neovascular (wet) age-related macular degeneration (nAMD) also referred to as Wet AMD. Wet AMD is characterized by loss of vision due to new, leaky blood vessel formation in the retina. Read more about this study

Who is it for?
Ages 50 to 89 years
What is being tested?
RGX-314
Could I receive a placebo?
No, everyone receives an active treatment
How many people
60 participants
Where
13 sitesUnited States
Study dates
22 Feb 2021to 18 Mar 2024
Phase
Phase 2
Are there results?
None posted in the registry

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

  • Males or females, aged ≥ 50 years and ≤ 89 years.
  • An Early Treatment Diabetic Retinopathy Study (ETDRS) BCVA-letter score between ≤ 78 and ≥ 40 in the study eye at Screening.
  • Diagnosis of choroidal neovascularization (CNV) secondary to age-related macular degeneration in the study eye previously treated with anti-VEGF.
  • Participants must have demonstrated a meaningful response to anti-VEGF therapy at study entry.
  • Willing and able to provide written, signed informed consent for this study.
  • Must be pseudophakic (at least 12 weeks postcataract surgery) in the study eye.

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.
  • Any condition in the investigator's opinion that could limit visual acuity improvement in the study eye.
  • Active or history of retinal detachment or retinal tear in the study eye.
  • Advanced glaucoma in the study eye.
  • Prior treatment with gene therapy.
  • Myocardial infarction, cerebrovascular accident, or transient ischemic attack within the past 6 months.

Trial locations and contacts

13 sites in 1 country.

13 sites in 1 country

United States13 sites

  • Eye Associates of New Mexico

    Albuquerque, New Mexico, 87109

  • Wilmer Eye Institute

    Baltimore, Maryland, 21287

  • Ophthalmic Consultants of Boston

    Boston, Massachusetts, 02114

  • Texas Retina Associates

    Dallas, Texas, 75231

  • Charles Retina Institute

    Germantown, Tennessee, 38138

  • Wills Eye Hospital

    Philadelphia, Pennsylvania, 19107

  • Retinal Consultants of Arizona

    Phoenix, Arizona, 85014

  • Sierra Eye Associates

    Reno, Nevada, 89502

  • Retina Consultants of San Diego

    San Diego, California, 92064

  • California Retina Consultants

    Santa Barbara, California, 93103

  • Retina Vitreous Associates of Florida

    St. Petersburg, Florida, 33711

  • Barnet Dulaney Perkins Eye Center

    Sun City, Arizona, 85351

  • Retina Consultants of Texas

    The Woodlands, Texas, 77384

Other trials in United States

About this study

Official title: A Phase 2, Open-label Study to Explore the Pharmacodynamics of Two Doses in Two Formulations of RGX-314 Gene Therapy Administered Via Subretinal Delivery in Participants With Neovascular Age-related Macular Degeneration

RGX-314 is being developed as a novel one-time gene therapy for the treatment of neovascular (wet) age-related macular degeneration (nAMD) also referred to as Wet AMD. Wet AMD is characterized by loss of vision due to new, leaky blood vessel formation in the retina. The purpose of this phase 2, open label study is to evaluate whether different doses of RGX-314 from two different formulations (clinical versus eventual commercial formulation) perform the same in humans when delivered by subretinal administration

Read the detailed description

Wet AMD is a significant cause of vision loss in the United States, Europe and Japan, with up to 2 million people living with wet AMD in these geographies alone. Current anti-VEGF therapies have significantly changed the landscape for treatment of wet AMD, becoming the standard of care due to their ability to prevent progression of vision loss in the majority of patients. These therapies, however, require life-long repeated intraocular injections to maintain efficacy. Due to the burden of treatment, patients often experience a decline in vision with reduced frequency of treatment over time. RGX-314 is being developed as a potential one-time treatment for wet AMD. RGX-314 uses an AAV8 vector that contains a gene that encodes for a monoclonal antibody fragment which binds to and neutralizes VEGF activity. This phase 2, open label study will explore the pharmacodynamics of two doses in two formulations of RGX-314 gene therapy via subretinal delivery in patients with neovascular Age-related Macular Degeneration. Approximately 60 patients (15 per cohort) who meet the inclusion/exclusion criteria will be enrolled in 4 sequential dose cohorts. A dose cohort will be comprised of 1 of 2 doses of RGX-314 in 1 of 2 formulations in order to explore the pharmacodynamics of RGX-314 based on aqueous humor transgene product (TP) concentrations. If the participants meet the study criteria and choose to participate in the study, their participation in the study will last about 12 months.

Being tested
RGX-314
Keywords
AMD, wet AMD, wAMD, neovascular AMD, nAMD, gene therapy
Medical subject headings
Wet Macular Degeneration

What participants receive

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

  • RGX-314Genetic

    RGX-314 is a recombinant adeno-associated virus gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein

Study groups in NCT04832724
Study groupRoleWhat they receive
Commercial Formulation Dose 1ExperimentalDose 1 of RGX-314
Receives: Genetic: RGX-314
Clinical Formulation Dose 1ExperimentalDose 1 of RGX-314
Receives: Genetic: RGX-314
Commercial Formulation Dose 2ExperimentalDose 2 of RGX-314
Receives: Genetic: RGX-314
Clinical Formulation Dose 2ExperimentalDose 2 of RGX-314
Receives: Genetic: RGX-314

What is being measured

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

  1. RGX-314 target protein concentration in aqueous humor

    Measured: At Week 24

Secondary outcomes (4)

  1. Incidence and severity of ocular Adverse Events (AEs) and overall AEs

    Evaluate the safety and tolerability of RGX-314

    Measured: Through Week 48

  2. Changes in Best Corrected Visual Acuity (BCVA)

    BCVA measured by Early Treatment Diabetic Retinopathy Study (ETDRS)

    Measured: At Week 24 and 48

  3. Changes in Central Retinal Thickness (CRT)

    CRT is measured by spectral domain optical coherence tomography (SD-OCT)

    Measured: At Week 24 and 48

  4. Mean Supplemental anti-VEGF injection annualized rate through week 24 and week 48

    To assess the need for supplemental anti-VEGF therapy over 48 weeks

    Measured: Through Week 24 and week 48

Timeline

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

  1. First posted6 Apr 2021
  2. Study start22 Feb 2021
  3. Primary completion2 Oct 2023
  4. Study completion18 Mar 2024

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
Non-randomised
Intervention model
Sequential
Masking
None (open label)
Enrolment
60 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04832724
Sponsor's protocol ID
RGX-314-2103
Status last verified
Sep 2024
First submitted
30 Mar 2021
Last updated
5 Sep 2024
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing RGX-314 in people with Neovascular Age-related Macular Degeneration and Wet Macular Degeneration, run by AbbVie at 13 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 6 inclusion and 7 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: finished, as of the sponsor's last update on 5 Sep 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 13 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?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

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?

RGX-314 target protein concentration in aqueous humor (time frame: At Week 24). 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 NCT04832724.

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 5 Sep 2024.

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