NCT06333249

A Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (SKYLINE)

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 2
• Study type: Interventional
• Enrollment: 14 (actual)
• Sponsor: Beacon Therapeutics · Industry
• Sex: Male
• Age: 8 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

Approximately 12 participants, who were not part of the Phase 1/2 (HORIZON) study, will be enrolled into the dose expansion portion of the study. These participants will be randomized in a 1:1 ratio to 1 of 2 treatment groups (i.e., Group 1 \[low dose\] and Group 2 \[high dose\]). Each participant will receive the assigned dose of AGTC-501 in one eye on a single occasion.

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: 2021-04-13
• Primary completion: 2023-04-11
• Completion: 2027-02-01
• First posted: 2024-03-27
• Last updated: 2024-08-12

Locations

6 sites across 1 country: United States

Regulatory

• FDA-regulated drug study