This study evaluates the safety, tolerability, and efficacy of HG005 in patients with Stargardt disease (an inherited cause of vision loss). It is open to patients aged 6-17 who carry changes in the ABCA4 gene and have well-defined atrophic lesions in the macula. The study is being conducted in Early Phase 1 and is 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: * Patient ≥ 6 and ≤17 years at the time of signing informed consent, with clinical diagnosis of Stargardt disease; * At least one ABCA4 allele on each chromosome; * Both eyes must have well-defined macular atrophic lesions consistent with the diagnosis of Stargardt macular dystrophy. * Meet visual acuity criteria based on ETDRS letter chart * Subject must agree to contraception during the study. * Acceptable hematology, clinical chemistry, urine laboratory, and protocol required eye examination. Exclusion Criteria: * Presence of active intraocular inflammation or uveitis history in either eye; * Presence of ocular or periocular infection history in either eye within 2 weeks prior to selection; * History or presence of corneal dystrophy in the study eye; * History of HIV or hepatitis A, B, or C infection; * Previous treatment with any gene therapy or cell therapy (e.g., stem cell transplantation); * Additional intraocular surgery in study eye 3 months prior to baseline visit; * Participation in an oral therapeutic STGD clinical trial within 3 months (or within 5 half-lives after last dose) prior to Screening * Any concomitant treatment that, in the opinion of the investigator, might interfere with the surgical procedure or healing process of the eye * Any other conditions that would not allow the potential subject to complete follow-up examinations during the study and would, in the opinion of the investigator, make the potential subject unsuitable for the study.