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The RESTORE study evaluated the safety and efficacy of a single intravitreal injection of the gene therapy product MCO-010 in adults with retinitis pigmentosa (RP). MCO-010 is an optogenetic therapy based on virally-carried Multi-Characteristic Opsin; some participants received a sham injection for comparison. The Phase 2 study enrolled people aged 18 and over able to give informed consent, with advanced RP confirmed by clinical and dilated fundus examination plus genetic testing, and best-corrected visual acuity worse than 1.9 LogMAR.
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: 1. Age ≥ 18 years 2. Able to comprehend and give informed consent. 3. Confirmed diagnosis of Advanced Retinitis Pigmentosa (RP) based on clinical examination, dilated fundus examination, and genetic testing. 4. Best-Corrected (Freiburg) Visual Acuity worse than 1.9 LogMAR (Snellen equivalent 20/1600) in the study eye and no better than 1.6 LogMAR (Snellen equivalent 20/800) in the fellow eye during screening. Exclusion Criteria: Subjects are excluded from the study if any of the following criteria apply: 1. Prior participation in gene therapy program 2. Pre-existing conditions in the study eye such as glaucoma, diseases affecting the optic nerve causing significant visual field loss, active uveitis, corneal or lenticular opacities). 3. Presence of any complicating systemic diseases such as malignancies whose treatment could affect central nervous system function. 4. Active ocular inflammation or recurrent history of idiopathic or autoimmune associated uveitis. 5. Having received retinal prothesis (such as ARGUS-II) or any gene or stem cell therapy (ocular or non-ocular).