NCT07174726

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

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 10 (estimated)
• Sponsor: Beacon Therapeutics · Industry
• Sex: Male
• Age: 12 Years – 50 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

XLRP is a genetic (inherited) eye disease that affects cells in the retina (the lining of the back of the eye that detects light). It causes night blindness and gradual worsening of your vision. 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.

Conditions

• X-Linked Retinitis Pigmentosa (XLRP)

Interventions

Type	Name	Description
BIOLOGICAL	Adeno-associated virus vector expressing a human RPGR gene	Male participants 12-50 years of age treated by subretinal injection with the dose of AGTC-501

Timeline

• Start date: 2025-09-10
• Primary completion: 2027-06-30
• Completion: 2030-12-15
• First posted: 2025-09-16
• Last updated: 2026-02-19

Locations

6 sites across 1 country: United States

Regulatory

• FDA-regulated drug study