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Gene Therapy

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

Phase 2
Recruiting
Research Sponsored by REGENXBIO, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 52 weeks
Awards & highlights

AAVIATE Trial Summary

This trial is evaluating a potential new treatment for a disease that causes vision loss. The current standard of care for this disease requires life-long, repeated injections to maintain efficacy, but this new treatment would only require a single injection.

Who is the study for?
This trial is for people aged 50 to 89 with neovascular age-related macular degeneration (wet AMD) in one eye. They must have had a positive response to previous anti-VEGF therapy and be willing to consent to the study.Check my eligibility
What is being tested?
The trial tests RGX-314, a potential one-time gene therapy for wet AMD. It's compared against standard treatments like Ranibizumab. Participants will receive different doses of RGX-314 or local/topical steroids.See study design
What are the potential side effects?
Possible side effects include inflammation of the eye (choroidal effusion), discomfort at the injection site, and typical risks associated with steroid use such as increased pressure inside the eye.

AAVIATE Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~52 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and 52 weeks for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Ranibizumab
Secondary outcome measures
Assess the need for supplemental anti-vascular endothelial growth factor (VEGF) therapy in participants who receive RGX-314 treatment
Evaluate the concentration of RGX-314 transgene product (TP) in aqueous humor and serum
Evaluate the effect of RGX-314 on BCVA
+4 more

AAVIATE Trial Design

6Treatment groups
Experimental Treatment
Active Control
Group I: RGX-314 Treatment Arm (Dose 3) and Topical SteroidExperimental Treatment2 Interventions
RGX-314 Dose 3 and Topical Steroid
Group II: RGX-314 Treatment Arm (Dose 3) and Local SteroidExperimental Treatment2 Interventions
RGX-314 Dose 3 and Local Steroid
Group III: RGX-314 Treatment Arm (Dose 3)Experimental Treatment1 Intervention
RGX-314 Dose 3
Group IV: RGX-314 Treatment Arm (Dose 2)Experimental Treatment1 Intervention
RGX-314 Dose 2
Group V: RGX-314 Treatment Arm (Dose 1)Experimental Treatment1 Intervention
RGX-314 Dose 1
Group VI: Ranibizumab controlActive Control1 Intervention
Control treatment arm
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Topical steroid
2020
Completed Phase 1
~140

Find a Location

Who is running the clinical trial?

REGENXBIO, Inc.Lead Sponsor
19 Previous Clinical Trials
2,178 Total Patients Enrolled
Regenxbio Inc.Lead Sponsor
19 Previous Clinical Trials
2,178 Total Patients Enrolled
REGENXBIO Inc.Lead Sponsor
23 Previous Clinical Trials
2,420 Total Patients Enrolled

Media Library

RGX-314 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT04514653 — Phase 2
Age-Related Macular Degeneration Research Study Groups: Ranibizumab control, RGX-314 Treatment Arm (Dose 1), RGX-314 Treatment Arm (Dose 2), RGX-314 Treatment Arm (Dose 3), RGX-314 Treatment Arm (Dose 3) and Local Steroid, RGX-314 Treatment Arm (Dose 3) and Topical Steroid
Age-Related Macular Degeneration Clinical Trial 2023: RGX-314 Highlights & Side Effects. Trial Name: NCT04514653 — Phase 2
RGX-314 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04514653 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is this research endeavor enrolling participants that are octogenarian or older?

"This clinical trial is seeking participants aged 50 to 89 as it meets the inclusion criteria. For those under 18 or over 65, there are 11 and 186 trials respectively available for them."

Answered by AI

Are there currently opportunities to participate in this experiment?

"Correct. Information accessible on clinicaltrials.gov suggests that this medical trial, which began recruitment efforts in late August of 2020, is still actively seeking participant enrolment. To date, 115 volunteers are needed to participate at 10 distinct locations."

Answered by AI

How many participants are currently partaking in this research endeavor?

"This study necessitates the recruitment of 115 suitable participants, with multiple sites available such as Philadelphia Location in Pennsylvania and Reno Location in Nevada."

Answered by AI

Has the FDA sanctioned RGX-314 Dose 2 for therapeutic use?

"With preliminary safety data available, our team at Power assigned RGX-314 Dose 2 a score of 2 on their scale from 1 to 3. This rating implies that there is evidence for its security but further studies need to be conducted in order to assess efficacy."

Answered by AI

To what health issue is RGX-314 Dose 2 typically utilized as a treatment?

"RGX-314 Dose 2 has been approved to treat branch vein occlusion, and can also provide relief for wAMD, macular edema, and myopic choroidal neovascularization."

Answered by AI

What sort of participants are desired for this medical experiment?

"This trial is recruiting 115 volunteers with choroidal effusions ranging from 50 to 89 years old. To qualify, the applicants must be within this age range and have a documented diagnosis of CNV resulting from AMD in their study eye. Additionally, they need to have responded positively to anti-VEGF treatment as well as given written consent for the experiment's inclusion."

Answered by AI

Is there widespread implementation of this trial in Canadian healthcare facilities?

"At the present time, this clinical trial is enrolling out of 10 sites. These include Philadelphia, Reno and Augusta amongst other places. To reduce travelling needs should you choose to participate, it's beneficial to select the closest site."

Answered by AI

Are there any other reports regarding the efficacy of RGX-314 Dose 2?

"Since its initial study at Ludwig Boltzmann Institute for Retinology and Biomicroscopic Lasersurgery in 2008, RGX-314 Dose 2 has been the subject of 314 separate clinical trials. At present there are 31 studies actively recruiting patients; many of these initiatives taking place in Philadelphia, Pennsylvania."

Answered by AI
~25 spots leftby Mar 2025