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ENROLLING BY INVITATION
NCT06275620
PHASE2

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

Sponsor: Beacon Therapeutics

View on ClinicalTrials.gov

Summary

This Phase 2 study is a non-randomized, open-label, study of the safety of AGTC-501 in participants with XLRP who have previously been treated with a full-length AAV vector-based gene therapy targeting RPGR protein.

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 Participants With X-linked Retinitis Pigmentosa

Key Details

Gender

MALE

Age Range

12 Years - Any

Study Type

INTERVENTIONAL

Enrollment

24

Start Date

2023-11-14

Completion Date

2029-12

Last Updated

2024-10-30

Healthy Volunteers

No

Interventions

BIOLOGICAL

AGTC-501 (high dose and standard corticosteroid regimen)

Adeno-associated virus vector expressing a human RPGR gene

BIOLOGICAL

AGTC-501 (low dose and standard corticosteroid regimen)

Adeno-associated virus vector expressing a human RPGR gene

BIOLOGICAL

AGTC-501 (high dose and modified corticosteroid regimen)

Adeno-associated virus vector expressing a human RPGR gene

Locations (7)

University of Florida

Jacksonville, Florida, United States

Bascom Palmer Eye Institute

Miami, Florida, United States

Boston Children's Hospital

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