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This study investigates the effectiveness and safety of a treatment called LX101 in inherited retinal dystrophy (an inherited vision-loss condition) caused by biallelic mutations in the RPE65 gene. It can include people aged 6 and over who have a biallelic RPE65 mutation and reduced visual acuity or visual field below a certain level. The study is in Phase 3 and is active but not currently recruiting participants.
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: * Subject and/or their guardian signing a written informed consent. Diagnosed with biallelic RPE65 mutation-associated inherited retinal dystrophy. Subjects are 6 years of age or older. Visual acuity of ≤ 20/63 or visual field less than 20 degrees in the eye to be injected. Exclusion Criteria: * Prior gene therapy for IRD and other hereditary eye diseases. Pre-existing eye conditions that would interfere with interpretation of study endpoints. Active intraocular or periocular infections in the study eye. Lacking of sufficient surviving retinal cells. Prior ocular surgery within six months. Complicating systemic diseases or clinically significant abnormal baseline laboratory values. Pre-existing systemic diseases that should not discontinue the use of any retinal toxic compounds. Complicating systemic diseases or clinically significant abnormal baseline laboratory values.