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Gene therapy for diabetic retinopathy and diabetic macular edema

A Phase 2, Randomized, Controlled, Dose-escalation Study to Evaluate the Efficacy, Safety, and Tolerability of RGX-314 Gene Therapy Delivered Via a Single Suprachoroidal Space (SCS) Injections in Participants With Diabetic Retinopathy (DR) With and Without Center Involved-Diabetic Macular Edema (CI-DME)(ALTITUDE)

Phase 2 Interventional AbbVie · NCT04567550

This study is testing a new gene therapy for people with diabetic retinopathy and diabetic macular edema to see if it can improve their vision and reduce the need for frequent treatments.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment130 (estimated)
Ages25 Years to 89 Years
SexAll
SponsorAbbVie Industry-sponsored
Locations25 sites (Phoenix, Arizona and 24 other locations)
Trial IDNCT04567550 on ClinicalTrials.gov

What this trial studies

This phase 2 clinical trial evaluates the efficacy, safety, and tolerability of ABBV-RGX-314, a novel gene therapy, administered in the suprachoroidal space for patients with diabetic retinopathy (DR) with and without center-involved diabetic macular edema (CI-DME). The study includes two parts: one for participants with DR without CI-DME, where they will be randomized to receive different doses of ABBV-RGX-314 or be observed, and another for those with CI-DME, comparing ABBV-RGX-314 to a control treatment. The trial aims to reduce the treatment burden associated with current therapies, which often require frequent injections. Participants will be monitored for their visual acuity and overall safety throughout the study.

Who should consider this trial

Good fit: Ideal candidates are adults aged 25-89 with a diagnosis of diabetic retinopathy who can safely defer current treatments for at least six months.

Not a fit: Patients with active center-involved diabetic macular edema or those with neovascularization from causes other than diabetic retinopathy may not benefit from this study.

Why it matters

Potential benefit: If successful, this gene therapy could significantly reduce the need for frequent injections and improve vision outcomes for patients with diabetic retinopathy.

How similar studies have performed: Other studies have shown promise with gene therapy approaches for retinal diseases, but this specific application is novel.

Eligibility criteria

Show full inclusion / exclusion criteria 
Part 1 (DR without CI-DME):

Inclusion Criteria:

* Patients 25-89 years of age with a diabetic retinopathy (DR) diagnosis of nonproliferative diabetic retinopathy (NPDR) and proliferative diabetic retinopathy (PDR) secondary to diabetes mellitus Type 1 or 2 for which PRP or anti-VEGF injections can be safely deferred for at least 6 months
* HbA1c \< 12%.
* Best corrected visual acuity (BCVA) Early Treatment Diabetic Retinopathy Study (ETDRS) letter score in the study eye of ≥69 letters (approximate Snellen equivalent of 20/40 or better).
* Prior history of CI-DME in the study eye is acceptable.
* Must be willing and able to provide written, signed informed consent.

Exclusion Criteria:

* Neovascularization in the study eye from a cause other than DR.
* Presence of any active CI-DME.
* Active or history of retinal detachment in the study eye.
* Any evidence or documented history of PRP or retinal laser in the study eye.
* Patients who had a prior vitrectomy surgery.
* Women of childbearing potential.

Part 2 (DR with CI-DME):

Inclusion Criteria:

* Patients 25-89 years of age with diabetic retinopathy secondary to diabetes mellitus Type 1 or 2.
* HbA1c \< 12%
* Macular thickening secondary to DME involving the center of the fovea, CST on SD-OCT (≥ 325 μm)
* Best corrected visual acuity (BCVA) Early Treatment Diabetic Retinopathy Study (ETDRS) letter score in the study eye of 78-25 letters (approximate Snellen equivalent of 20/32 to 20/320)
* Participants must have demonstrated a meaningful response to anti-VEGF therapy.
* Must be willing and able to provide written, signed informed consent

Exclusion Criteria:

* Neovascularization in the study eye from a cause other than DR.
* Active or history of retinal detachment in the study eye.
* Any evidence or documented history of PRP or retinal laser in the study eye.
* Patients who had a prior vitrectomy surgery.
* Women of childbearing potential.

Note: Other inclusions/exclusions criteria apply.

Where this trial is running

Phoenix, Arizona and 24 other locations

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 → Find more trials like this →
Conditions Diabetic RetinopathyCenter-Involved Diabetic Macular EdemaDRCI-DME
Last reviewed 2026-06-13 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.