An Observational Study in Subjects to Follow the Progression of Stargardt Disease Type 1 (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ABCA4 Gene
Trial Parameters
Brief Summary
This is an Observational Study to Follow the Progression of Stargardt Disease Type 1 (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ABCA4 Gene This is a multicenter study which will enroll approximately 75 subjects
Eligibility Criteria
Inclusion Criteria: 1. Provide written consent 2. Are male or female aged 12-65 years old 3. Have a diagnosis of STGD1 caused by bi-allelic likely pathogenic or pathogenic variants in the ABCA4 gene confirmed genotypically by an accredited genotyping laboratory 4. Have a history of STGD1 progression within the last 2 years, in the opinion of the investigator. 5. Eligible eye(s) must have: 1. BCVA of between 24-88 ETDRS letters, inclusive (20/20 - 20/320 Snellen equivalent, 0.0-1.2 logMAR) at the Screening Visit. 2. Clinical evidence of a macular lesion phenotypically consistent with Stargardt Disease. 3. Fundus autofluorescence (FAF) measurement of definitely decreased autofluorescence (DDAF) as measured by the Central Reading Center (CRC). 4. Total lesion must be imaged in its entirety. 5. All total lesion borders must be ≥300 microns from all image edges. 6. Eligible eye(s) must have clear ocular media and adequate pupillary dilation, including no allergy to dilating eyedrops, to per