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NCT03066258COMPLETEDanonymous

A Phase I/IIa (Phase 1/Phase 2a), Open-label, Multiple-cohort, Dose-escalation Study to Evaluate the Safety and Tolerability of Gene Therapy With RGX-314 in Subjects With Neovascular AMD (nAMD)

Sponsor

Source record

REGENXBIO Inc.

Phase

Source record

PHASE1

Modality

AI-normalized

gene therapy

Target

AI-normalized

RGX-314

Indication / condition

AI-normalized

Neovascular Age-related Macular Degeneration

Intervention

Source record

RGX-314

Source & freshness

Source record

NCT ID

NCT03066258

Original source

ClinicalTrials.gov

Source last updated

May 16, 2023

Ingested at

Jun 11, 2026

Internal sync

Jun 11, 2026

Model version

trialsignal-ai-v1

Normalized confidence

96%

Validation status

validated

Open original registry record
View original source fields

NCT ID

NCT03066258

Title

A Phase I/IIa (Phase 1/Phase 2a), Open-label, Multiple-cohort, Dose-escalation Study to Evaluate the Safety and Tolerability of Gene Therapy With RGX-314 in Subjects With Neovascular AMD (nAMD)

Sponsor

REGENXBIO Inc.

Status

COMPLETED

Phase

PHASE1

Condition raw

Neovascular Age-related Macular Degeneration, Wet Age-related Macular Degeneration

Condition normalized

Neovascular Age-related Macular Degeneration, Wet Age-related Macular Degeneration

Modality raw

gene therapy

Modality normalized

gene therapy

Target raw

RGX-314

Target normalized

RGX-314

Interventions

RGX-314

Public preview

Source record

Excessive vascular endothelial growth factor (VEGF) plays a key part in promoting neovascularization and edema in neovascular (wet) age-related macular degeneration (nAMD). VEGF inhibitors (anti-VEGF), including ranibizumab (LUCENTIS®, Genentech) and aflibercept (EYLEA®, Regeneron), have been shown to be safe and effective for treating nAMD and have demonstrated improvement in vision. However, anti-VEGF therapy is administered frequently via intravitreal injection and can be a significant burden to the patients. RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein. The long-term, stable delivery of this therapeutic protein following a 1 time gene therapy treatment for nAMD could potentially reduce the treatment burden of currently available therapies while maintaining vision with a favorable benefit:risk profile.

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