Actively Recruiting
An Observational Study in Children and Adults With Stargardt Disease
Led by AAVantgarde Bio Srl · Updated on 2025-08-28
80
Participants Needed
2
Research Sites
165 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This multicenter, prospective, longitudinal, observational study in approximately 80 subjects with Stargardt disease secondary to biallelic mutations in the ABCA4 gene (STGD1) aims to evaluate prognostic factors of disease progression, and to further characterize the patient population for future clinical studies.
CONDITIONS
Official Title
An Observational Study in Children and Adults With Stargardt Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male and female subjects between 8 and 50 years of age at the time of enrolment.
- Willingness to follow the study protocol with written informed consent for those 18 or older.
- For subjects under 18, written assent plus consent from a parent or legal guardian is required.
- Confirmed mutation in the ABCA4 gene.
You will not qualify if you...
- History of uveitis.
- Any eye disease in either eye that may interfere with retina assessment.
- Any posterior segment pathology other than ABCA4 retinopathy.
- Presence of other genetic mutations associated with retinal or macular dystrophy.
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 2 locations
1
Retina Foundation of the Southwest
Dallas, Texas, United States, 75231
Actively Recruiting
2
Oslo University hospital Ullevål
Oslo, Norway, 0450
Actively Recruiting
Research Team
C
Clinical Operations Manager
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
0
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