Clinical Trials Logo

Clinical Trial 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.


Clinical Trial Description

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. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT06275620
Study type Interventional
Source Beacon Therapeutics
Contact
Status Enrolling by invitation
Phase Phase 2
Start date November 14, 2023
Completion date August 2029

See also
  Status Clinical Trial Phase
Completed NCT04926129 - Natural History of the Progression of X-Linked Retinitis Pigmentosa
Recruiting NCT05926583 - A Study of AAV5-hRKp.RPGR for the Treatment of Japanese Participants With X-linked Retinitis Pigmentosa Phase 3
Completed NCT03116113 - A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using BIIB112 Phase 1/Phase 2
Recruiting NCT04850118 - A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XLRP Phase 2/Phase 3
Active, not recruiting NCT04517149 - 4D-125 in Patients With X-Linked Retinitis Pigmentosa (XLRP) Phase 1/Phase 2
Enrolling by invitation NCT03584165 - Long-term Safety and Efficacy Follow-up of BIIB111 for the Treatment of Choroideremia and BIIB112 for the Treatment of X-Linked Retinitis Pigmentosa Phase 3
Active, not recruiting NCT04312672 - Long-term Follow-up Gene Therapy Study for RPGR- XLRP
Completed NCT04868916 - An Observational Study of Japanese Participants With X-linked Retinitis Pigmentosa
Completed NCT03252847 - Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) - Retinitis Pigmentosa GTPase Regulator (RPGR) Phase 1/Phase 2
Recruiting NCT05874310 - Gene Therapy for Subjects With RPGR Mutation-associated X-linked Retinitis Pigmentosa Early Phase 1
Active, not recruiting NCT04671433 - Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene Phase 3
Active, not recruiting NCT04794101 - Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene Phase 3
Completed NCT03314207 - Clinical Evaluation of Patients With X-linked Retinitis Pigmentosa (XLRP)
Active, not recruiting NCT03316560 - Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations Phase 1/Phase 2
Active, not recruiting NCT06333249 - A Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (SKYLINE) Phase 2