Clinical Research Details

A Phase 2/3, Randomized, Controlled, Masked, Multi-center Study to Evaluate the Efficacy, Safety and Tolerability of Two Doses of AGTC-501, a Recombinant Adeno-associated Virus Vector Expressing RPGR (rAAV2tYF-GRK1-RPGR), Compared to an Untreated Control Group in Male Subjects with X-linked Retinitis Pigmentosa Confirmed by a Pathogenic Variant in the RPGR Gene (20204485)

Study Description

A Phase 2/3, Randomized, Controlled, Masked, Multi-center Study to Evaluate the Efficacy, Safety and Tolerability of Two Doses of AGTC-501, a Recombinant Adeno-associated Virus Vector Expressing RPGR (rAAV2tYF-GRK1-RPGR), Compared to an Untreated Control Group in Male Subjects with X-linked Retinitis Pigmentosa Confirmed by a Pathogenic Variant in the RPGR Gene 

Inclusion/Exclusion Criteria

Inclusion Criteria:

Be male (XY chromosome) and have at least one documented pathogenic or likely

pathogenic variant in the RPGR gene within exons 1-14 and/or ORF15 from a Clinical

Laboratory Improvement Amendments of 1988 (CLIA)-certified laboratory.

Open Enrollment

Contact Name: Ghulam Hamdani
Contact Phone: (904) 244-9347
Contact Email: ghulam.hamdani@jax.ufl.edu

Investigators


Sandeep Grover, M.D.
Ophthalmology