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RGX-314 Dose 1

Phase 2

Diabetic Macular Edema | Gene therapy | Ophthalmology |REGENXBIO Inc.|Last Updated: Apr 24, 2025

Success Probability

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Market & Valuation

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Trial Design

RandomizedACTIVE_CONTROLLED
Total Trials1
Total Enrollment18

FDA Designations

RMATORPHAN_DRUGRARE_PEDIATRIC_DISEASEFAST_TRACKACCELERATED_APPROVALPRIORITY_REVIEW

Clinical trial landscape

RGX-314 Dose 1 · 1 trial · 1 indication

Phase 2 1
NCT06942520Phase 2 Trial of RGX-314 in Adults With Center Involved - Diabetic Macular Edema (CI - DME)Diabetic Macular Edema
RECRUITING18 Analytics
PHASE2RECRUITING
Phase 2 Trial of RGX-314 in Adults With Center Involved - Diabetic Macular Edema (CI - DME)
Diabetic Macular EdemaUnlock trial analytics

Study Endpoints

Primary Endpoints

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

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

Secondary Endpoints

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
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Study Design & Arms

AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
RGX-314 Treatment Arm (Dose 1)EXPERIMENTALGenetic: RGX-314 Dose 1 \- AAV8 vector containing a transgene for anti-VEGF fab (Dose 1)
Treatment Arm (Dose 2)EXPERIMENTALGenetic: RGX-314 Dose 2 -AAV8 vector containing a transgene for anti-VEGF fab (Dose 2)
Aflibercept Treatment ArmACTIVE_COMPARATORBiological: Aflibercept IVT * Commercially available Active Comparator * Other Names: Eylea

Interventions

NameTypeDescription
RGX-314 Dose 1GENETICAAV8 vector containing a transgene for anti-VEGF fab (Dose 1)
RGX-314 Dose 2GENETICAAV8 vector containing a transgene for anti-VEGF fab (Dose 2)
Aflibercept (2.0 mg)BIOLOGICALCommercially available Active Comparator
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Eligibility Criteria

Age Range25 Years to 89 Years
SexALL
Healthy VolunteersNo
Study Sites1

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 clin...

Countries:United States
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Frequently asked questions about RGX-314 Dose 1

What is RGX-314 Dose 1 used for?

RGX-314 Dose 1 is an investigational gene therapy being studied for the treatment of diabetic macular edema (DME). It is currently in Phase 2 clinical development for this condition, which affects the center of the retina and can cause vision loss in adults.

Who is developing RGX-314 Dose 1?

RGX-314 Dose 1 is being developed by REGENXBIO Inc., a biopharmaceutical company traded on the Nasdaq under the ticker symbol RGNX. The company is conducting clinical trials to evaluate the safety and efficacy of this gene therapy for diabetic macular edema.

What phase is RGX-314 Dose 1 in?

RGX-314 Dose 1 is in Phase 2 clinical development. It is an investigational therapy, meaning it has not been approved by regulatory authorities and is still being studied in clinical trials for diabetic macular edema.

What clinical trials is RGX-314 Dose 1 in?

RGX-314 Dose 1 is being evaluated in a Phase 2 trial with the identifier NCT06942520. This randomized, active-controlled study is recruiting adults aged 25 years and older with center-involved diabetic macular edema in the United States, with a planned enrollment of 18 participants.

What FDA designations has RGX-314 Dose 1 received?

RGX-314 Dose 1 has received several FDA designations, including Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug, Rare Pediatric Disease, Fast Track, Accelerated Approval, and Priority Review. These designations are intended to expedite the development and review of therapies for serious conditions.