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

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
18

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • 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 Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
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

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Sierra Eye Associates

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
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)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
March 18, 2025

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 1, 2027

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 1, 2027

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
December 3, 2024

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 16, 2025

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
April 24, 2025

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
April 24, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 16, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
April 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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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