Gene therapy for diabetic macular edema in adults

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

PHASE2 · Sierra Eye Associates · NCT06942520

This study is testing a new gene therapy called RGX-314 to see if it can improve vision in adults with diabetic macular edema compared to the standard treatment.

Quick facts

PhasePHASE2
Study typeInterventional
Enrollment18 (estimated)
Ages25 Years to 89 Years
SexAll
SponsorSierra Eye Associates (other)
Locations1 site (Reno, Nevada)
Trial IDNCT06942520 on ClinicalTrials.gov

What this trial studies

This Phase 2 trial evaluates the safety and efficacy of RGX-314, a gene therapy, delivered subretinally in adults with Center Involved Diabetic Macular Edema (CI-DME). The study is open label, randomized, and actively controlled, comparing two doses of RGX-314 against the standard treatment, Aflibercept. Participants will be monitored for clinical responses and visual acuity improvements over the course of the trial.

Who should consider this trial

Good fit: Ideal candidates include adults with Type I or Type II diabetes who have previously been treated for CI-DME and have a specific range of visual acuity.

Not a fit: Patients with concurrent ocular conditions or those who have had certain prior treatments may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a novel therapeutic option for patients suffering from diabetic macular edema, potentially improving their vision and quality of life.

How similar studies have performed: While gene therapy approaches are emerging, this specific application for diabetic macular edema is relatively novel and has not been extensively tested in prior studies.

Eligibility criteria

Show full inclusion / exclusion criteria
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

Where this trial is running

Reno, Nevada

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.

View on ClinicalTrials.gov →

Conditions: Diabetic Macular Edema, diabetic macular edema, gene therapy

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.