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Neovascular Age-related Macular DegenerationPhase 2RGX-314 SRLTFUNCT03999801

Long-term Follow-Up Study of RGX-314 and Fellow Eye Substudy

A Phase 2 study testing RGX-314 in people with Neovascular Age-related Macular Degeneration and Wet Macular Degeneration, run by AbbVie at 9 sites in United States.

Enrolling by invitation

Enrolling by invitation. The study team selects and invites participants, usually from an existing group of patients.

Sites and contacts
Official record
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Trial summary

This is a prospective, observational study designed to evaluate the long-term safety and efficacy of RGX-314. Eligible participants are those who were previously enrolled in a clinical study in which they received a single subretinal administration of RGX-314 in their study eye. Read more about this study

Who is it for?
Ages 50 years and older
What is being tested?
RGX-314
Could I receive a placebo?
No, everyone receives an active treatment
How many people
865 participants
Where
9 sitesUnited States
Study dates
31 May 2019to Dec 2028
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 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Able and willing to provide written consent
  • Previously enrolled in a clinical study of RGX-314 and received a single subretinal administration of RGX-314
  • Exclusions Criteria:
  • 1. None
  • Fellow Eye Substudy:
  • Age ≤ 93 years
Show all 10Show fewer
  • Currently or previously enrolled in the main observational study
  • Diagnosis of bilateral choroidal neovascularization secondary to age-related macular degeneration
  • Active nAMD in the fellow eye
  • BCVA between ≤ 80 and ≥ 20 letters in the fellow eye

Who cannot join

  • CNV or macular edema in the fellow eye secondary to causes other than nAMD
  • Subfoveal fibrosis or atrophy in the fellow eye
  • Advanced glaucoma or history of secondary glaucoma in the fellow eye or any invasive procedure to treat glaucoma
  • Myocardial infarction, cerebrovascular accident, or transient ischemic attack within the past 6 months
  • History of intraocular surgery in the fellow eye within 12 weeks of screening
  • History of intravitreal therapy, such as intravitreal steroid injection or investigational product, other than an intravitreal therapy for nAMD, in the fellow eye within 6 months of screening

Trial locations and contacts

9 sites in 1 country. Contact the study team or a site near you to ask about taking part.

9 sites in 1 country

United States9 sites

  • Johns Hopkins University

    Baltimore, Maryland, 21287

  • Ophthalmic Consultants of Boston

    Boston, Massachusetts, 02114

  • Charles Retina Institute

    Germantown, Tennessee, 38138

  • University of Pennsylvania Department of Ophthalmology

    Philadelphia, Pennsylvania, 19104

  • MidAtlantic Retina

    Philadelphia, Pennsylvania, 19107

  • Retinal Research Institute, LCC

    Phoenix, Arizona, 85053

  • Sierra Eye Associates

    Reno, Nevada, 89502

  • California Retina Consultants CRC

    Santa Barbara, California, 93103

  • Retina Consultants of Texas Research Center

    The Woodlands, Texas, 77384

Other trials in United States

About this study

Official title: A Long-term Follow-Up Study to Evaluate the Safety and Efficacy of RGX-314 Following Subretinal Administration in Participants With Neovascular Age-related Macular Degeneration and Fellow Eye Treatment Substudy

This is a prospective, observational study designed to evaluate the long-term safety and efficacy of RGX-314. Eligible participants are those who were previously enrolled in a clinical study in which they received a single subretinal administration of RGX-314 in their study eye. Enrollment of each participant in the current study should occur after the participant has completed either the end of study or early termination visit in the previous (parent) clinical study. Participants will be followed for up to 5 years post-RGX-314 administration (inclusive of the parent study). After enrollment and a 6-month follow-up visit, participants will attend at least annual study visits through the end of the 5-year post-RGX-314 administration follow-up period.

Additionally, an interventional fellow eye treatment substudy will evaluate the safety, efficacy, and immunogenicity of subretinal RGX-314 administration in the fellow eye of participants having bilateral disease who previously received a subretinal injection of RGX-314 in their study eye. Participants who qualify for the substudy will receive subretinal administration of RGX-314 in their fellow eye and complete 13 study visits in a 54-week period. Following completion of the substudy participants will continue in the observational portion of the study for up to 5 years post RGX-314 administration in their fellow eye.

Being tested
RGX-314
Keywords
gene therapy, wet AMD, nAMD
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

    AAV8 vector containing a transgene for anti-VEGF Fab

Study groups in NCT03999801
Study groupRoleWhat they receive
Main Observational StudyNo interventionAll subjects that previously received RGX-314 in a subretinal administration parent study are enrolled into this arm.
RGX-314 Fellow Eye Treatment SubstudyExperimentalRGX-314 Fellow Eye Treatment
Receives: Genetic: RGX-314

What is being measured

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

  1. Incidence of ocular adverse events and any serious adverse events in the study eye

    Measured: 5 years inclusive of parent study

  2. Fellow eye substudy: Incidence of ocular adverse events and any serious adverse events in the fellow eye

    Measured: 54 weeks

Secondary outcomes (9)

  1. Change from baseline in best corrected visual acuity (BCVA) in the study eye

    Mean change and proportion of participants losing greater than or equal to 5, 10, and 15 letters

    Measured: 5 years inclusive of parent study

  2. Change from baseline in Central Retinal Thickness (CRT) in the study eye

    Mean change from baseline in CRT as measured by spectral domain optical coherence tomography (SD-OCT)

    Measured: 5 years inclusive of parent study

  3. Number of supplemental anti-VEGF injections in the study eye

    Mean number of supplemental anti-VEGF injections based on chart review

    Measured: 5 years inclusive of parent study

  4. Number of retinal specialist visits for the study eye

    Mean number of retinal specialist visits attended for nAMD based on chart review

    Measured: 5 years inclusive of parent study

  5. Fellow eye substudy: Change from baseline in best corrected visual acuity

    Mean change from baseline in BCVA in the fellow eye

    Measured: 54 weeks

  6. Fellow eye substudy: Change from baseline in Central Retinal Thickness in the fellow eye

    Mean change from baseline in CRT in the fellow eye as measured by SD-OCT

    Measured: 54 weeks

  7. Fellow eye substudy: Number of supplemental anti-VEGF injections in the fellow eye

    Mean number of supplemental anti-VEGF injections in the fellow eye

    Measured: 54 weeks

  8. Fellow eye substudy: Aqueous and Serum RGX-314 transgene product (TP) concentrations in the fellow eye

    Fellow eye aqueous and serum RGX-314 TP concentrations at assessed time points

    Measured: 54 weeks

  9. Fellow eye substudy: Immunogenicity measurements in the fellow eye

    Fellow eye immunogenicity measurements at assessed timepoints

    Measured: 54 weeks

Timeline

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

  1. First posted27 Jun 2019
  2. Study start31 May 2019
  3. Primary completionDec 2028 (estimated)
  4. Study completionDec 2028 (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
Non-randomised
Intervention model
Single group
Model description
1 main Observational study arm, 1 RGX-314 Fellow Eye Treatment substudy
Masking
None (open label)
Enrolment
865 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03999801
Sponsor's protocol ID
RGX-314-5101
Acronym
RGX-314 SRLTFU
Status last verified
Jun 2024
First submitted
18 Jun 2019
Last updated
17 Jun 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 9 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 10 inclusion and 6 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: enrolling by invitation, as of the sponsor's last update on 17 Jun 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 9 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?

Incidence of ocular adverse events and any serious adverse events in the study eye (time frame: 5 years inclusive of parent study). The study lists 2 primary outcomes in all. 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 NCT03999801.

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 17 Jun 2024.

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