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This study compares two different doses of a gene therapy called AGTC-501 in male participants with X-linked retinitis pigmentosa caused by RPGR gene mutations. It enrolls male participants aged 12 and older who have already had one eye treated with a gene therapy designed to provide functioning RPGR protein. The study is being conducted in Phase 2 and is enrolling by invitation.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: * Be ≥12 years of age * Have one eye previously treated with an AAV vector-based gene therapy designed to provide full-length functioning RPGR protein. * Have a BCVA no better than 78 letters and no worse than 34 letters * Be able to perform all tests of visual and retinal function and structure in both eyes based on the participant's reliability and fixation, per the Investigator's discretion. * Have detectable baseline mean macular sensitivity measured by MAIA microperimetry, as determined by the Investigator and confirmed by the Central Reading Center (CRC). * Have detectable EZ line in the study eye as assessed by SD-OCT and confirmed by the CRC. Exclusion Criteria: * Have other known disease-causing mutations documented in the participant's medical history or identified through a retinal dystrophy gene panel that, in the opinion of the Investigator, would interfere with the potential therapeutic effect of the study agent or the quality of the assessments. * Have pre-existing eye conditions that would preclude the planned surgery, interfere with the interpretation of study endpoints, or increase the risk of surgical complications * Had intraocular surgery within 90 days of study treatment administration. * Have any active ocular/intraocular infection or inflammation * Have a history of steroid-induced raised IOP of \>25 mmHg following corticosteroid exposure, despite topical IOP-lowering pharmacologic therapy.