A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With X-linked Retinitis Pigmentosa Caused by RPGR Mutations
X-Linked Retinitis Pigmentosa
About this trial
This is an interventional treatment trial for X-Linked Retinitis Pigmentosa focused on measuring XLRP, retinal degeneration, RPGR, adeno-associated virus, AAV, gene therapy
Eligibility Criteria
Inclusion Criteria:
- Males between the ages of 8-50 years old with a clinical diagnosis of XLRP with a confirmed RPGR mutation who also meet the other requirements of the study.
- Have at least one documented pathogenic or likely pathogenic variant in the RPGR gene within exons 1-14 and/or ORF15
- Have a BCVA no better than 75 letters (20/32) and no worse than 35 letters (20/200) in the study eye based on an Early Treatment of Diabetic Retinopathy Study (ETDRS) chart at each screening visit
Exclusion Criteria:
- Have other known retinal disease mutations or previously received an AAV gene therapy product, as well as being unable or unwilling to meet the requirements of the study.
Sites / Locations
Arms of the Study
Arm 1
Arm 2
Arm 3
Active Comparator
Active Comparator
Other
Group 1: Low Dose
Group 2: High Dose
Group 3: Control
Male subjects 8-50 years of age treated by subretinal injection with the low dose of AGTC-501
Male subjects 8-50 years of age treated by subretinal injection with the high dose of AGTC-501
Male subjects 8-50 years of age in the untreated control group. Subjects randomized to the untreated control group will be followed for a minimum of 12 months. After this time, they will be treated by subretinal injection with the high dose of AGTC-501.