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
Recruiting
Observational Study
Stargardt
Stargardt's Disease
Stargardt Disease
STGD1
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Age
- 12 – 65
- Sex
- Any
- Study type
- Observational
- Participants needed
- 75 (estimated)
- Sponsor
- Splice Bio · Industry
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About This Trial
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
Trial Locations
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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 progressio…
Contacts