Phase 2 Trial of RGX-314 in Adults With Center Involved - Diabetic Macular Edema (CI - DME) (ELAAVATE)

April 16, 2025 updated by: Sierra Eye Associates

A Randomized, Open Label, Controlled, Phase 2 Clinical Study to Evaluate the Efficacy and Safety of RGX-314 AAV Gene Therapy Administered Via Subretinal Delivery in Participants With Center Involved Diabetic Macular Edema

Phase 2 open label, randomized, active controlled, dose-ranging trial in adults with Center Involved - Diabetic Macular Edema (CI - DME)

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

This Phase 2 trial is an open label, randomized, active controlled, dose-ranging trial to allow for preliminary safety and efficacy data on subretinal (SR) delivery of RGX-314 in participants with CI - DME

Study Type

Interventional

Enrollment (Estimated)

18

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Type I or Type II diabetes mellitus with center involving diabetic macular edema with previous treatment
  • HbA1c ≤12%
  • BCVA in study eye 78 to 25 ETDRS letters (~20/32 to 20/320) at screening
  • Decreased visual acuity attributable primarily due to CI - DME
  • Demonstrate clinical response to aflibercept injection in the study eye
  • Provide written informed consent

Exclusion Criteria:

  • Women of childbearing potential
  • Neovascularization in the study eye from a cause other than DR
  • Evidence in the study eye of optic nerve pallor on clinical examination
  • History of pan retinal photocoagulation in the study eye
  • Any concurrent ocular condition in the study eye other than CI-DME that could require surgical intervention within 6 months or any condition in the study eye that may increase the risk to the participant, require either medical or surgical intervention during the study to prevent or treat vision loss, or interfere with the study procedures or assessments
  • Presence of an implant in the study eye at screening (excluding intraocular lens)
  • Any condition in the investigator's opinion that could limit VA improvement in the study eye
  • Active or history of glaucoma, steroid response, or ocular hypertension
  • Any prior intravitreal steroid injection in the study eye within 6 months prior to screening, administration in the study eye of Ozudrex® within 12 months prior to screening, or administration in the study eye if Iluven® within 36 months prior to screening
  • Diabetic macular edema diagnosis ≥ 7 years
  • History of chronic renal failure requiring dialysis or kidney transplant
  • Participation in any other gene therapy study or receipt of any investigational product within 30 days prior to enrollment or 5 half-lives of the investigational product, whichever is longer, or any plans to use an investigational product within 6 months following enrollment

Note: Other inclusion/exclusion criteria apply

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: RGX-314 Treatment Arm (Dose 1)

Genetic: RGX-314 Dose 1

- AAV8 vector containing a transgene for anti-VEGF fab (Dose 1)
Genetic: RGX-314 Dose 1
AAV8 vector containing a transgene for anti-VEGF fab (Dose 1)
Experimental: Treatment Arm (Dose 2)

Genetic: RGX-314 Dose 2

-AAV8 vector containing a transgene for anti-VEGF fab (Dose 2)
Genetic: RGX-314 Dose 2
AAV8 vector containing a transgene for anti-VEGF fab (Dose 2)
Active Comparator: Aflibercept Treatment Arm

Biological: Aflibercept IVT

  • Commercially available Active Comparator
  • Other Names: Eylea
Biological: Aflibercept (2.0 mg)
Commercially available Active Comparator

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Mean change from baseline in BCVA in the study eye at Week 52
Time Frame: 52 Weeks
To evaluate the effect of RGX-314 on BCVA at Week 52
52 Weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Mean change from baseline in BCVA in the study eye over time
Time Frame: 52 Weeks
To evaluate the effect of RGX-314 on BCVA over time
52 Weeks
Proportion of participants with BCVA of 20/40 or better (ETDRS ≥ 69 letters) in the study eye over time
Time Frame: 52 Weeks
To evaluate the effect of RGX-314 on BCVA over time
52 Weeks
Mean change from baseline in central subfield thickness (CST) in the study at week 52
Time Frame: 52 Weeks
To evaluate the effect of RGX-314 on anatomic outcomes and CST (as determined by SD OCT measurement) at Week 52
52 Weeks
Mean change from baseline in CST in the study eye on SD-OCT at Week 26 and Week 52
Time Frame: 52 Weeks
To evaluate the effect of RGX-314 on anatomic outcomes and CST (as determined by SD OCT measurement) at Week 52
52 Weeks
Proportion of participants with an absence of CI-DME in the study eye at Week 26 and Week 52
Time Frame: 52 Weeks
To evaluate the effect of RGX-314 on anatomic outcomes and CST (as determined by SD OCT measurement) at Week 52
52 Weeks
Proportion of participants achieving a 2-step or greater improvement or any improvement (a 1-step or greater improvement) in DR in the study eye by ETDRS DRSS on 4 widefield digital stereoscopic fundus photography at Week 52
Time Frame: 52 Weeks
To evaluate the effect of RGX-314 on DR by the ETDRS DRSS at Week 52
52 Weeks
Incidences of overall and ocular adverse events (AEs)
Time Frame: 52 Weeks
To assess the safety and tolerability of RGX-314
52 Weeks
Proportion of participants requiring any additional intervention for ocular diabetic complications to Week 52
Time Frame: 52 Weeks
To evaluate the need for additional standard of care intervention due to ocular diabetic complications
52 Weeks
Proportion of participants with any sight-threatening ocular diabetic complications to Week 52
Time Frame: 52 Weeks
To evaluate the need for additional standard of care intervention due to ocular diabetic complications
52 Weeks
Proportion of participants developing ocular diabetic complications requiring treatment per standard of care (anti-VEGF treatment, steroid treatment, pan retinal photocoagulation, laser, or surgery) through Week 52
Time Frame: 52 Weeks
To evaluate the need for additional standard of care intervention due to ocular diabetic complications
52 Weeks
Proportion of participants developing ocular diabetic complications (e.g., neovascularization due to DR) requiring treatment per standard of care (pan retinal photocoagulation or anti-VEGF treatment) through Week 52
Time Frame: 52 Weeks
To evaluate the need for additional standard of care intervention due to ocular diabetic complications
52 Weeks
Proportion of participants developing ocular diabetic complications (e.g., retinal detachments) requiring surgical intervention per standard of care through Week 52
Time Frame: 52 Weeks
To evaluate the need for additional standard of care intervention due to ocular diabetic complications
52 Weeks
Aqueous RGX-314 transgene (TP) concentration at assessed time points
Time Frame: 52 Weeks
To measure aqueous RGX-314 TP concentrations
52 Weeks
Proportion of participants with visual stability in the study eye from baseline to Week 52
Time Frame: 52 Weeks
To evaluate the effect of RGX-314 on vision outcomes
52 Weeks
Proportion of participants with vision gain or vision loss ≥ to 5, 10, or 15 ETDRS letters in the study eye from baseline to Week 52
Time Frame: 52 Weeks
To evaluate the effect of RGX-314 on vision outcomes
52 Weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sierra Eye Associates

Collaborators

REGENXBIO Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

March 18, 2025

Primary Completion (Estimated)

December 1, 2027

Study Completion (Estimated)

December 1, 2027

Study Registration Dates

First Submitted

December 3, 2024

First Submitted That Met QC Criteria

April 16, 2025

First Posted (Actual)

April 24, 2025

Study Record Updates

Last Update Posted (Actual)

April 24, 2025

Last Update Submitted That Met QC Criteria

April 16, 2025

Last Verified

April 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SEA1979

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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