An Observational Study in Subjects to Follow the Progression of Stargardt Disease Type 1 (STGD1) … (NCT06435000) | Clinical Trial Compass
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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
United States75 participantsStarted 2024-03-29
Plain-language 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
Who can participate
Age range12 Years – 65 Years
SexALL
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Inclusion criteria
✓. Provide written consent
✓. Are male or female aged 12-65 years old
✓. 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
✓. Have a history of STGD1 progression within the last 2 years, in the opinion of the investigator.
✓. Eligible eye(s) must have:
✓. BCVA of between 24-88 ETDRS letters, inclusive (20/20 - 20/320 Snellen equivalent, 0.0-1.2 logMAR) at the Screening Visit.
✓. Clinical evidence of a macular lesion phenotypically consistent with Stargardt Disease.
✓. Fundus autofluorescence (FAF) measurement of definitely decreased autofluorescence (DDAF) as measured by the Central Reading Center (CRC).
Exclusion criteria
✕. Are an immediate family member (e.g., child, sibling) of the Sponsor or study site personnel.
✕. Have any concurrent ocular disease that would affect study procedures or outcomes (e.g., cataracts; subjects can be enrolled 90 days after successful cataract surgery) in eligible eyes.
✕. Have two likely pathogenic or pathogenic variants (not STGD1) in autosomal recessive inherited retinal dystrophy (IRD) genes or a single likely pathogenic or pathogenic variant in autosomal dominant or X-linked IRD genes.
What they're measuring
1
Document disease progression based on change from baseline in lesion size as measured by DDAF on FAF imaging
✕. Have had any intraocular surgery or thermal laser within 90 days of study entry or any prior thermal laser in the macular region within the eligible eye(s).
✕. Have any major surgical procedure within 30 days of the Screening Visit or planned or anticipated major surgery during the study period.
✕. Are unwilling to stop taking the following products at Screening and throughout the study:
✕. Supplements containing vitamin A or beta-carotene, liver-based products.