stargardt's disease
Conditions
Interventions
Intervention group :The patient was placed in the supine position. After routine sterile preparation and draping, retrobulbar anesthesia was administered, and the eyelids were retracted using a lid sp
Sponsors
Eye and ENT Hospital of Fudan University
Eligibility
Sex/Gender
All
Age
18 Years to 65 Years
Inclusion criteria
Inclusion criteria: 1. Clinically and diagnostically confirmed Stargardt disease; 2. Aged between 18 and 65 years, regardless of gender; 3. The study eye is the eye with poorer visual acuity, with best-corrected visual acuity (BCVA) < 0.1; 4. The subject agrees to and is able to complete all scheduled visits, treatments, and laboratory examinations as required by the study protocol, and has signed the informed consent form.
Exclusion criteria
Exclusion criteria: 1.Presence of active or recent (within 1 month prior to administration) intraocular, extraocular, or periocular infection in either eye, including but not limited to conjunctivitis, keratitis, scleritis, or endophthalmitis, or a history of uveitis. 2.Presence of amblyopia in either eye. 3.Other ocular diseases that may interfere with study outcomes, such as glaucoma, corneal opacity, or severe lens opacity. 4.Any history of vitreous hemorrhage within 12 weeks prior to administration. 5.Other retinal degenerative diseases, including retinal vascular occlusion; diabetic retinopathy with or without macular edema; or any other retinopathy secondary to diseases such as, but not limited to, hypertension, trauma, or acquired inflammatory diseases (e.g., uveitic serologic changes). 6.Any systemic or ocular disease that may cause or potentially lead to vision loss. 7.Contraindications to pharmacologic mydriasis (e.g., history of angle-closure glaucoma). 8.Presence of only one functional eye, even if that eye otherwise meets the enrollment criteria. 9.Relevant history of prior/concomitant therapies or allergies. 10.Any ocular surgery in the study eye within 6 months prior to enrollment (excluding cataract surgery performed more than 3 months earlier, blepharoplasty, or pterygium excision), or any history of laser treatment for retinal or macular lesions. 11.Presence of unhealed wounds (wound severity greater than Stage III), moderate to severe ulcers, or fractures. 12.Contraindications to transient systemic corticosteroid-induced immunosuppression, including uncontrolled hypertension, diabetes mellitus, tuberculosis, renal impairment, osteoporosis, gastric ulcer, severe affective disorders, or immunodeficiency. 13.Use of subconjunctival or intravitreal corticosteroid therapy in the study eye within 3 months prior to administration (including long-acting subconjunctival or intravitreal corticosteroid implants), or use of ocular corticosteroids or nonsteroidal anti-inflammatory drugs (NSAIDs) within 1 month prior to administration. 14.Previous treatment with any gene therapy product in one or both eyes prior to administration. 15.Participation in any investigational drug trial (except vitamins and minerals) or medical device clinical trial within 3 months prior to administration, with receipt of investigational treatment or device intervention. For investigational products with a long half-life, exclusion applies within 5 half-lives if this exceeds 3 months. 16.Current use of prednisone >=15 mg/day (or equivalent corticosteroid) within 7 days prior to screening. Subjects with recent or current use of inhaled corticosteroids will not be excluded. 17.Known hypersensitivity to any component of the investigational treatment, or allergy to anesthetics, antimicrobial agents, corticosteroids, or NSAIDs used during the study. 18.Refusal to sign the informed consent form (ICF). 19.In the investigator’s judgment, evidence of any disease or condition that contraindicates use of the investigational product, may interfere with interpretation of study results, or may increase subject risk, including but not limited to cerebral infarction or myocardial infarction within 6 months prior to administration, or hypertension uncontrolled despite antihypertensive therapy (systolic blood pressure =160 mmHg or diastolic blood pressure >=100 mmHg). 20.Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) levels >2 × the upper limit of normal (ULN), serum creat
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Best-Corrected Visual Acuity; | — |
Secondary
| Measure | Time frame |
|---|---|
| Full-Field Light Sensitivity Threshold Test(FST);Fluorescein Fundus Angiography;OCT scan; | — |
Countries
China
Contacts
Public ContactWu Jihong
Eye and ENT Hospital of Fudan University
Outcome results
None listed