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Active, not recruiting Phase 1Phase 2 Interventional

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

ClinicalTrials.gov ID: NCT03316560

Public ClinicalTrials.gov record NCT03316560. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

ClinicalTrials.gov public records Last synced Sep 27, 2026, 10:10 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

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

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

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.

Study identification

NCT ID
NCT03316560
Recruitment status
Active, not recruiting
Study type
Interventional
Phase
Phase 1, Phase 2
Lead sponsor
Beacon Therapeutics
Industry
Enrollment
29 participants

Conditions and interventions

Interventions

Biological

Eligibility (public fields only)

Age range
6 Years to 50 Years
Sex
Male
Healthy volunteers
Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date
Apr 15, 2018
Primary completion
Nov 8, 2023
Completion
Feb 28, 2025
Last update posted
May 19, 2024

2018 – 2025

United States locations

U.S. sites
4
U.S. states
4
U.S. cities
4
Facility City State ZIP Site status
Duke University Durham North Carolina 27701 —
Cincinnati Eye Institute Cincinnati Ohio 45242 —
Casey Eye Institute Portland Oregon 97239 —
Retina Foundation of the Southwest Dallas Texas 75231 —

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

About this trial record page

What this page shows
Public field values for ClinicalTrials.gov record NCT03316560, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do
No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from
Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh
Last update posted May 19, 2024 · Synced Sep 27, 2026

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Open the official record

The complete protocol, eligibility criteria, and contact information for NCT03316560 live on ClinicalTrials.gov.

View official ClinicalTrials.gov record →