This study examines BIIB112, a retinal gene therapy, for X-linked retinitis pigmentosa, an inherited eye disease caused by changes in the RPGR gene. It was open to patients with a genetically confirmed RPGR mutation and active disease in the eye. It was conducted in Phase 1/Phase 2 and has been 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.
Key Inclusion Criteria: Part 1: * Participants with genetically confirmed diagnosis of XLRP (with RPGR mutation). * Participant with active disease clinically visible within the macular region in both eyes. Part 2: \- Participant with mean total retinal sensitivity in the study eye as assessed by microperimetry ≥ 0.1 dB and ≤8 dB. Key exclusion Criteria: Parts 1 and 2: * Participant with history of amblyopia in either eye. * Participated in a gene therapy trial previously or a clinical trial with an investigational drug in the past 12 weeks or received a gene/cell-based therapy at any time previously. NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.