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Compensation: Varies

Number of Visits: 14

865 Participants Needed

RGX-314 for Age-Related Macular Degeneration
(RGX-314 SRLTFU Trial)

Recruiting at 16 trial locations

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: REGENXBIO, Inc.

Must not be taking: Steroids, Investigational products

Disqualifiers: Glaucoma, Recent heart attack, others

No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

Prior Safety DataThis treatment has passed at least one previous human trial

Trial Summary

What is the purpose of this trial?

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.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. However, if you have had certain eye treatments or surgeries recently, you may not be eligible for the substudy.

How is the drug RGX-314 different from other treatments for age-related macular degeneration?

RGX-314 is unique because it is a gene therapy designed to provide a long-term solution by delivering a gene that produces an anti-VEGF (vascular endothelial growth factor) protein, potentially reducing the need for frequent injections required by other treatments.¹ ² ³ ⁴ ⁵

Eligibility Criteria

This trial is for people up to 93 years old with a condition called age-related macular degeneration (AMD) in both eyes. They must have previously received RGX-314 treatment in one eye and be willing to consent. The study excludes those who don't meet the vision criteria or haven't been part of the main observational study.

Inclusion Criteria

BCVA between ≤ 80 and ≥ 20 letters in the fellow eye

I am 93 years old or younger.

I have been diagnosed with a specific eye condition due to aging.

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Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Enrollment

Participants enroll after completing the parent study's end or early termination visit

Immediate

Fellow Eye Treatment Substudy

Subretinal administration of RGX-314 in the fellow eye with 13 study visits over 54 weeks

54 weeks

13 visits (in-person)

Main Observational Study

Participants are followed for long-term safety and efficacy of RGX-314

5 years

Annual visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

6 months

1 visit (in-person)

Treatment Details

Interventions

RGX-314

Trial OverviewThe long-term effects and safety of RGX-314 are being studied over five years in patients who've had it before. A subgroup will also get RGX-314 in their other eye, which hasn't been treated yet, to see how well it works there.

Participant Groups

2Treatment groups

Experimental Treatment

Active Control

Group I: RGX-314 Fellow Eye Treatment SubstudyExperimental Treatment1 Intervention

RGX-314 Fellow Eye Treatment

Group II: Main Observational StudyActive Control1 Intervention

All subjects that previously received RGX-314 in a subretinal administration parent study are enrolled into this arm.

Find a Clinic Near You

Who Is Running the Clinical Trial?

REGENXBIO, Inc.

Lead Sponsor

Trials

Recruited

2,800+

Regenxbio Inc.

Lead Sponsor

Trials

Recruited

2,800+

AbbVie

Lead Sponsor

Trials

1,079

Recruited

535,000+

Founded

2013

Headquarters

North Chicago, USA

Known For

Immunology treatments

Findings from Research

In the VISION-1 trial involving 1186 patients, pegaptanib, a VEGF inhibitor, significantly helped maintain visual acuity in patients with neovascular age-related macular degeneration, with 70% of those receiving the 0.3 mg dose losing fewer than 15 letters of visual acuity compared to 55% in the sham group.

While pegaptanib showed no serious systemic side effects, it did lead to more ocular adverse effects, such as vitreous floaters and inflammation, indicating a need for ongoing improvements in treatment options for this condition.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pegaptanib: the first antiangiogenic agent approved for neovascular macular degeneration.Doggrell, SA.[2019]