NCT04850118

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

Active, enrolled Phase 2, PHASE3 Last updated 7 April 2026

What this trial tests

Phase 2, PHASE3 trial testing rAAV2tYF-GRK1-hRPGRco in X-Linked Retinitis Pigmentosa in 85 participants. Participants enrolled and being followed up; not accepting new ones.

Timeline

14 March 2024

Primary endpoint
30 June 2026

31 October 2031

Quick facts

Lead sponsor	Beacon Therapeutics
Phase	Phase 2, PHASE3
Status	Active, enrolled
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	quadruple
Primary purpose	treatment
Enrollment	85
Start date	14 March 2024
Primary completion	30 June 2026
Estimated completion	31 October 2031
Sites	25 locations across United Kingdom, Canada, United States, Australia

Drugs / interventions tested

• rAAV2tYF-GRK1-hRPGRco — full drug profile →
• Control

Conditions studied

• X-Linked Retinitis Pigmentosa — all drugs for X-Linked Retinitis Pigmentosa →

Sponsor

Beacon Therapeutics — full company profile →

Who can join

Adults 12 to 50, male only, with X-Linked Retinitis Pigmentosa. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Current clinical applications of AAV-mediated gene therapy.
   Byrne BJ, Flanigan KM, Matesanz SE, Finkel RS, et al · · 2025 · cited 43× · PMID 40329530 · DOI 10.1016/j.ymthe.2025.04.045
2. Gene Therapy in Inherited Retinal Diseases: An Update on Current State of the Art.
   Amato A, Arrigo A, Aragona E, Manitto MP, et al · · 2021 · cited 43× · PMID 34722588 · DOI 10.3389/fmed.2021.750586
3. Update on Viral Gene Therapy Clinical Trials for Retinal Diseases.
   Cheng SY, Punzo C. · · 2022 · cited 42× · PMID 36074935 · DOI 10.1089/hum.2022.159
4. Viral and non-viral vectors in gene therapy: current state and clinical perspectives.
   Geng G, Xu Y, Hu Z, Wang H, et al · · 2025 · cited 38× · PMID 40602323 · DOI 10.1016/j.ebiom.2025.105834
5. Ocular Drug Delivery: Advancements and Innovations.
   Tian B, Bilsbury E, Doherty S, Teebagy S, et al · · 2022 · cited 28× · PMID 36145679 · DOI 10.3390/pharmaceutics14091931
6. Update on Clinical Trial Endpoints in Gene Therapy Trials for Inherited Retinal Diseases.
   Igoe JM, Lam BL, Gregori NZ. · · 2024 · cited 18× · PMID 39336999 · DOI 10.3390/jcm13185512
7. X-Linked Retinitis Pigmentosa Gene Therapy: Preclinical Aspects.
   Mansouri V. · · 2023 · cited 18× · PMID 36346573 · DOI 10.1007/s40123-022-00602-y
8. Gene Therapies in Clinical Development to Treat Retinal Disorders.
   McClements ME, Elsayed MEAA, Major L, de la Camara CM, et al · · 2024 · cited 17× · PMID 38955952 · DOI 10.1007/s40291-024-00722-0