NCT03316560

Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations

A Phase 1/2 Open-Label Dose Escalation Study to Evaluate the Safety and Efficacy of AGTC-501 (rAAV2tYF-GRK1-RPGR) and a Phase 2 Randomized, Controlled, Masked, Multi-center Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Confirmed by a Pathogenic Variant in the RPGR Gene

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 29 (actual)
• Sponsor: Beacon Therapeutics · Industry
• Sex: Male
• Age: 6 Years – 50 Years
• Healthy volunteers: Not accepted

Summary

This study will evaluate the safety and efficacy of a recombinant adeno-associated virus vector (rAAV2tYF-GRK1-RPGR) in patients with X-linked retinitis pigmentosa caused by RPGR mutations.

Detailed description

This protocol includes a non-randomized, open-label, Phase 1/2 study (HORIZON). Approximately 30 participants will be enrolled into the dose escalation study (HORIZON). Each participant will receive the study agent by subretinal injection in one eye on a single occasion. Enrollment will begin with the lowest dose and will proceed to higher doses only after review of safety data by a Data and Safety Monitoring Committee (DSMC). There are a total of 15 visits over approximately 36 months, and long-term follow-up evaluations annually at years 4 and 5.

Conditions

• X-Linked Retinitis Pigmentosa

Interventions

Type	Name	Description
BIOLOGICAL	rAAV2tYF-GRK1-RPGR	Adeno-associated virus vector expressing a human RPGR gene

Timeline

• Start date: 2018-04-16
• Primary completion: 2023-11-09
• Completion: 2025-03-01
• First posted: 2017-10-20
• Last updated: 2024-05-20

Locations

4 sites across 1 country: United States

Regulatory

• FDA-regulated drug study