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

NCT06942520 | Recruiting Phase 2 FDA Drug

• 1 other identifier: SEA1979
• Study Type: interventional
• Target: 18
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Diabetic Macular Edema

Keywords

diabetic macular edema; gene therapy

Outcome Measures

Primary Outcomes (1)

• Mean change from baseline in BCVA in the study eye at Week 52

  To evaluate the effect of RGX-314 on BCVA at Week 52

  52 Weeks

Secondary Outcomes (15)

• Mean change from baseline in BCVA in the study eye over time

  52 Weeks

• Proportion of participants with BCVA of 20/40 or better (ETDRS ≥ 69 letters) in the study eye over time

  52 Weeks

• Mean change from baseline in central subfield thickness (CST) in the study at week 52

  52 Weeks

• Mean change from baseline in CST in the study eye on SD-OCT at Week 26 and Week 52

  52 Weeks

• Proportion of participants with an absence of CI-DME in the study eye at Week 26 and Week 52

  52 Weeks

Study Arms (3)

RGX-314 Treatment Arm (Dose 1)

EXPERIMENTAL

Genetic: RGX-314 Dose 1 \- AAV8 vector containing a transgene for anti-VEGF fab (Dose 1)

Genetic: RGX-314 Dose 1

Treatment Arm (Dose 2)

EXPERIMENTAL

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

Genetic: RGX-314 Dose 2

Aflibercept Treatment Arm

ACTIVE COMPARATOR

Biological: Aflibercept IVT * Commercially available Active Comparator * Other Names: Eylea

Biological: Aflibercept (2.0 mg)

Interventions

RGX-314 Dose 1 GENETIC

AAV8 vector containing a transgene for anti-VEGF fab (Dose 1)

RGX-314 Treatment Arm (Dose 1)

RGX-314 Dose 2 GENETIC

AAV8 vector containing a transgene for anti-VEGF fab (Dose 2)

Treatment Arm (Dose 2)

Aflibercept (2.0 mg) BIOLOGICAL

Commercially available Active Comparator

Aflibercept Treatment Arm

Eligibility Criteria

• Age: 25 Years - 89 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Sierra Eye Associates: lead
• REGENXBIO Inc.: collaborator

Study Sites (1)

Sierra Eye Associates

Reno, Nevada, 89502, United States

RECRUITING

MeSH Terms

Interventions

aflibercept

Central Study Contacts

Clinical Research Study Coordinator

CONTACT

775-329-0286; jvannavong@sierraeyeassociates.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 3, 2024
• First Posted: April 24, 2025
• Study Start: March 18, 2025
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): December 1, 2027
• Last Updated: April 24, 2025

Record last verified: 2025-04

Locations

US (1)