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This study examined a gene therapy called AAV5-hRKp.RPGR in patients with X-linked retinitis pigmentosa associated with disease-causing variants in the RPGR gene. People aged 3 or older with a diagnosis confirmed by a retinal specialist and an RPGR variant confirmed by an accredited laboratory could take part. The study was conducted in Phase 3 and is completed.
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: * Male or female * 3 years of age or older * Has XLRP confirmed by a retinal specialist and has a predicted disease-causing sequence variant in RPGR confirmed by an accredited laboratory Exclusion Criteria: * Has had ocular surgery within 3 months prior to screening or is anticipated to require ocular surgery within 6 months after the study intervention administration * Any investigational ocular treatment or any other ocular treatment that could confound the interpretation of the efficacy results or affect participant compliance with the visit schedule * Has undergone prior retinal surgery involving the macula, macular laser photocoagulation, external-beam radiation therapy, transpupillary thermotherapy, glaucoma filtration surgery or corneal surgery