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ACTIVE NOT RECRUITING
NCT06333249
PHASE2

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

Sponsor: Beacon Therapeutics

View on ClinicalTrials.gov

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.

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

Key Details

Gender

MALE

Age Range

8 Years - 50 Years

Study Type

INTERVENTIONAL

Enrollment

14

Start Date

2021-04-13

Completion Date

2027-02

Last Updated

2024-08-12

Healthy Volunteers

No

Interventions

BIOLOGICAL

rAAV2tYF-GRK1-RPGR

Adeno-associated virus vector expressing a human RPGR gene

Locations (6)

University of Florida

Jacksonville, Florida, United States

Boston Children's Hosptial

Boston, Massachusetts, United States

Cincinnati Eye Institute

Cincinnati, Ohio, United States

Cleveland Clinic

Cleveland, Ohio, United States

Casey Eye Institute

Portland, Oregon, United States

Retina Foundation of the Southwest

Dallas, Texas, United States