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Gene Therapy

RGX-314 Gene Therapy for Age-Related Macular Degeneration (ASCENT Trial)

Phase 3
Recruiting
Research Sponsored by AbbVie
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Age ≥ 50 years and ≤ 89 years
Participants must have demonstrated a meaningful response to anti-VEGF therapy at study entry
Must not have
Prior treatment with gene therapy
Advanced glaucoma or history of secondary glaucoma in the study eye
Timeline
Screening 3 weeks
Treatment Varies
Follow Up week 54, week 74, week 90, and week 108
Awards & highlights
No Placebo-Only Group
Pivotal Trial

Summary

This trial is testing a new one-time gene therapy called RGX-314 for patients with wet AMD. The goal is to reduce the need for regular treatments by helping the eye produce its own protective proteins. This could make treatment easier and more effective for patients.

Who is the study for?
This trial is for people aged 50-89 with wet age-related macular degeneration (AMD) who've had cataract surgery at least 12 weeks ago and responded to previous anti-VEGF treatments. They should have a certain level of vision as measured by the ETDRS score. Those with recent serious heart events, retinal detachment, other eye conditions or surgeries, or prior gene therapy can't join.
What is being tested?
The study tests RGX-314, a one-time gene therapy for wet AMD that could replace frequent injections currently needed for treatment. Two different doses of RGX-314 are being compared against Aflibercept (EYLEA®), an existing standard care medication.
What are the potential side effects?
While specific side effects of RGX-314 aren't listed here, gene therapies can sometimes cause immune reactions, changes in vision, discomfort at the injection site, and potential long-term risks which are still unknown.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am between 50 and 89 years old.
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I responded well to previous anti-VEGF treatment.
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My eye condition was treated with injections due to AMD.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have previously undergone gene therapy.
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I have advanced glaucoma or a history of it in one eye.
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My vision loss is not due to AMD but another cause.
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I have scarring or tissue loss in the center of my vision in one eye.
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I have not had a heart attack, stroke, or mini-stroke in the last 6 months.
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I have or had a detached retina or currently have a tear in my study eye.
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I haven't had any eye injections except for anti-VEGF in the last 6 months.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~week 54, week 74, week 90, and week 108
This trial's timeline: 3 weeks for screening, Varies for treatment, and week 54, week 74, week 90, and week 108 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Mean change from baseline in Best Corrected Visual Acuity (BCVA)
Secondary study objectives
Aqueous ABBV-RGX-314 TP concentration (control arm participants who cross over to ABBV-RGX-314)
Aqueous ABBV-RGX-314 transgene product (TP) concentration (ABBV-RGX-314 randomized participants)
Immunogenicity measurements (ABBV-RGX-314 randomized participants)
+24 more

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

3Treatment groups
Experimental Treatment
Active Control
Group I: ABBV-RGX-314 Dose 2Experimental Treatment1 Intervention
ABBV-RGX-314 Dose 2 administered via subretinal delivery one time.
Group II: ABBV-RGX-314 Dose 1Experimental Treatment1 Intervention
ABBV-RGX-314 Dose 1 administered via subretinal delivery one time.
Group III: Control ArmActive Control1 Intervention
Aflibercept administered via intravitreal injection approximately every 8 weeks

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
The most common treatments for Age-Related Macular Degeneration (AMD) involve anti-VEGF (vascular endothelial growth factor) therapies. These treatments inhibit VEGF, a protein that stimulates the growth of abnormal blood vessels in the retina, which can leak and lead to vision loss in wet AMD. RGX-314, a gene therapy under investigation, aims to provide a sustained anti-VEGF effect through a one-time treatment, potentially reducing the need for frequent intraocular injections. This is crucial for AMD patients as it could enhance treatment adherence and maintain vision more effectively over the long term.

Find a Location

Who is running the clinical trial?

AbbVieLead Sponsor
1,033 Previous Clinical Trials
521,618 Total Patients Enrolled
REGENXBIO, Inc.Lead Sponsor
19 Previous Clinical Trials
1,828 Total Patients Enrolled
Regenxbio Inc.Lead Sponsor
19 Previous Clinical Trials
1,828 Total Patients Enrolled

Media Library

RGX-314 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT05407636 — Phase 3
Age-Related Macular Degeneration Research Study Groups: Control Arm, ABBV-RGX-314 Dose 1, ABBV-RGX-314 Dose 2
Age-Related Macular Degeneration Clinical Trial 2023: RGX-314 Highlights & Side Effects. Trial Name: NCT05407636 — Phase 3
RGX-314 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05407636 — Phase 3
Age-Related Macular Degeneration Patient Testimony for trial: Trial Name: NCT05407636 — Phase 3
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