RGX-314 for Age-Related Macular Degeneration

(RGX-314 SRLTFU Trial)

Enrolling by invitation 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 Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 14

What You Need to Know Before You Apply

What is the purpose of this trial?

The trial aims to assess the safety and effectiveness of RGX-314, a gene therapy for age-related macular degeneration, a condition that causes vision loss. It targets individuals who previously received RGX-314 in one eye during an earlier study. Participants will be monitored for up to five years to observe long-term effects. Additionally, the study includes a segment for those with the condition in both eyes, administering RGX-314 to the other eye. Individuals who have experienced vision problems due to age-related macular degeneration and participated in the prior RGX-314 study may be suitable candidates. As a Phase 2 trial, this research focuses on evaluating the treatment's efficacy in an initial, smaller group.

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.

Is there any evidence suggesting that RGX-314 is likely to be safe for humans?

Research has shown that RGX-314, a treatment for age-related macular degeneration, is generally safe. Most patients tolerate the treatment well. In one study, 20 serious side effects occurred among 13 participants, but only one was possibly related to RGX-314, involving changes in the eye's pigment.

Other studies have found that RGX-314 reduced the need for other treatments by 97% after nine months, with consistent safety results, indicating that side effects are uncommon.

These findings can reassure those considering participation in clinical trials for RGX-314. However, staying informed and discussing any concerns with a doctor is important. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatment?

Researchers are excited about RGX-314 for age-related macular degeneration (AMD) because it offers a novel approach compared to current treatments like anti-VEGF injections. Unlike those standard treatments, which require frequent injections into the eye, RGX-314 uses gene therapy to deliver a one-time dose designed to produce a therapeutic protein directly within the eye, potentially reducing or eliminating the need for repeated treatments. This innovative delivery method aims to maintain vision improvements over a longer period with fewer interventions, making it a promising option for those affected by AMD.

What evidence suggests that RGX-314 might be an effective treatment for age-related macular degeneration?

Research has shown that RGX-314 could be a promising treatment for age-related macular degeneration (AMD). In earlier studies, patients experienced a 97% reduction in the need for other treatments nine months after receiving RGX-314. This gene therapy reduces the activity of VEGF-A, a protein that can cause harmful blood vessel growth in the eye. By controlling this protein, RGX-314 helps maintain vision and prevent fluid leaks in the eye. These findings suggest that RGX-314 might be an effective one-time treatment for people with this condition. Participants in this trial may join the RGX-314 Fellow Eye Treatment Substudy or the Main Observational Study, which involves those who previously received RGX-314 in a subretinal administration parent study. ^{1 2 3 4 6}

Are You a Good Fit for This Trial?

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.

Timeline for a Trial Participant

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)

What Are the Treatments Tested in This Trial?

Interventions

• RGX-314

Trial Overview The 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.

How Is the Trial Designed?

Group I: RGX-314 Fellow Eye Treatment Substudy Experimental Treatment 1 Intervention

RGX-314 Fellow Eye Treatment

Group II: Main Observational Study Active Control 1 Intervention

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

Who Is Running the Clinical Trial?

REGENXBIO, Inc.

Lead Sponsor

Trials

20

Recruited

2,800+

Regenxbio Inc.

Lead Sponsor

Trials

20

Recruited

2,800+

AbbVie

Lead Sponsor

Trials

1,079

Recruited

535,000+

Founded

2013

Headquarters

North Chicago, USA

Known For

Immunology treatments

Top Products

Humira (adalimumab), Skyrizi (risankizumab), Rinvoq (upadacitinib)

Dr. Roopal Thakkar

AbbVie

Chief Medical Officer since 2023

MD from Wayne State University School of Medicine

Robert A. Michael

AbbVie

Chief Executive Officer

Bachelor's degree in Finance from the University of Illinois

REGENXBIO Inc.

Lead Sponsor

Trials

25

Recruited

3,100+

Published Research Related to This Trial

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]

Citations

1. ir.regenxbio.com ir.regenxbio.com/news-releases/news-release-details/regenxbio-presents-positive-data-phase-ii-study-subretinal-abbv

Release Details 97% reduction in treatment burden at nine months after treatment with ABBV-RGX-314; Data consistent with that from multiple previous studies ...

2. thelancet.com thelancet.com/journals/lancet/article/PIIS0140-6736(24)00310-6/abstract

Gene therapy for neovascular age-related macular ... We report results on the safety and efficacy of subretinal injection of RGX-314 in patients with nAMD.

3. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/38554726/

Gene therapy for neovascular age-related macular ... RGX-314 gene therapy provides a novel approach for sustained VEGF-A suppression in patients with nAMD that has potential to control exudation, maintain vision, ...

4. news.abbvie.com news.abbvie.com/2025-01-13-AbbVie-and-REGENXBIO-Announce-Updates-on-the-ABBV-RGX-314-Clinical-Program

AbbVie and REGENXBIO Announce Updates on the ABBV ... ABBV-RGX-314 is being investigated as a potential one-time treatment for wet AMD, diabetic retinopathy and potentially other chronic retinal conditions.

5. clinicaltrials.gov clinicaltrials.gov/study/NCT05407636

NCT05407636 | Pivotal 2 Study of RGX-314 Gene Therapy ... This randomized, partially masked, controlled, Phase 3 clinical study will evaluate the efficacy and safety of ABBV-RGX-314 gene therapy in participants with ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT03066258

NCT03066258 | Safety and Tolerability of RGX-314 ... This Phase I/IIa, open-label, multiple-cohort, dose-escalation study was designed to evaluate the safety and tolerability of RGX-314 gene therapy in subjects ...