NCT04850118

A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XLRP

A Randomized, Controlled, Masked, Multi-center Study Evaluating the Efficacy, Safety, and Tolerability of Two Doses of AGTC-501 Compared to an Untreated Control Group in Male Participants With X-linked Retinitis Pigmentosa

• Status: Active Not Recruiting
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 85 (actual)
• Sponsor: Beacon Therapeutics · Industry
• Sex: Male
• Age: 12 Years – 50 Years
• Healthy volunteers: Not accepted

Summary

This study will evaluate and compare the safety, efficacy, and tolerability of 2 doses of a recombinant adeno-associated virus vector (AGTC-501/laruparetigene zovaparvovec )) to an untreated control group in male participants with X-linked retinitis pigmentosa caused by RPGR mutations.

Detailed description

This study is a randomized, controlled, masked, multi-center study evaluating and comparing 2 doses of AGTC-501 to an untreated control group. A single subretinal injection of AGTC-501 Dose 1 or Dose 2 will be administered in participants in 2 treatment groups while participants in the untreated control group will be followed and evaluated, after which they will be evaluated to determine eligibility to receive treatment with AGTC-501 Dose 2. Approximately 75 eligible male participants between 12 and 50 years of age (inclusive) will be randomized in a 1:1:1 ratio to 1 of 3 groups.

Conditions

• X-Linked Retinitis Pigmentosa

Interventions

Type	Name	Description
BIOLOGICAL	rAAV2tYF-GRK1-hRPGRco	Adeno-associated virus vector expressing a human RPGR gene
DRUG	Control	Untreated Control Group 3

Timeline

• Start date: 2024-03-14
• Primary completion: 2026-06-30
• Completion: 2031-10-31
• First posted: 2021-04-20
• Last updated: 2026-04-07

Locations

25 sites across 4 countries: United States, Australia, Canada, United Kingdom

Regulatory

• FDA-regulated drug study