X-Linked Retinitis Pigmentosa Clinical Trial
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
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.
This is a Phase 2, open-label, multicenter study to evaluate the safety of 2 doses of AGTC-501 administered as a single subretinal injection in participants with XLRP who have previously been treated with a full-length AAV vector-based gene therapy targeting RPGR protein. The trial includes a screening period of up to 60 days and a 5 year study period. Each participant will receive a single subretinal injection in their previously untreated eye. There will be 2 groups of 6 participants, each receiving one of two doses of the study treatment along with the standard corticosteroid regimen, followed by a third group of ~3-6 participants receiving the high dose but with a modified corticosteroid regimen. The first 6 participants will be enrolled in Group 1, followed by Group 2. Group 3 will open for enrollment after data has been reviewed by the DSMC. DSMC review will occur after all 6 Group 1 (high dose) study participants reach post-operative Month 1 and periodically throughout the study. ;
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