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A Phase 2 Open-label Study to Evaluate the Safety of Laruparetigene Zovaparvovec Administered Bilaterally in Male Participants With X-Linked Retinitis Pigmentosa

NCT07174726

The purpose of this Phase 2 Study is to see if the investigational study drug, laruparetigene zovaparvovec, also known as AGTC-501, given in both eyes, is safe and works to preserve and/or improve vision and other symptoms of XLRP.

X-Linked Retinitis Pigmentosa (XLRP)

Beacon Therapeutics10 participantsStarted Sep 2025

Jacksonville, Florida, United States • Miami, Florida, United States • Durham, North Carolina, United States +3 more locations

RECRUITINGPHASE1/PHASE2

Gene Therapy for RPGR Gene Mutation-associated X-linked Retinitis Pigmentosa

NCT06492850

The aim of this study was to evaluate the safety, tolerability, and efficacy of one-time subretinal injection of FT-002 in male subjects (8-45 years of age) with RPGR (Retinitis Pigmentosa GTPase Regulator) gene mutation-associated X-linked retinitis pigmentosa, of XLRP. This study includes Phase I (dose escalation phase) and Phase II (dose expansion phase).

X-Linked Retinitis Pigmentosa (XLRP)

Frontera Therapeutics32 participantsStarted Apr 2024

Beijing, Beijing Municipality, China

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A Phase 2 Open-label Study to Evaluate the Safety of Laruparetigene Zovaparvovec Administered Bilaterally in Male Participants With X-Linked Retinitis Pigmentosa

Gene Therapy for RPGR Gene Mutation-associated X-linked Retinitis Pigmentosa

A Phase 2 Open-label Study to Evaluate the Safety of Laruparetigene Zovaparvovec Administered Bilaterally in Male Participants With X-Linked Retinitis Pigmentosa

Gene Therapy for RPGR Gene Mutation-associated X-linked Retinitis Pigmentosa