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A Multicenter, Prospective, Longitudinal, Observational Study in Children and Adults With Stargardt Disease Related Atrophy Secondary to Biallelic Mutations in the ABCA4 Gene
Led by AAVantgarde Bio Srl · Updated on 2025-08-28
80
Participants Needed
2
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are conducting a multicenter, prospective, longitudinal, observational study involving about 80 participants with Stargardt disease caused by biallelic mutations in the ABCA4 gene (STGD1). The study aims to identify factors that predict disease progression and to better understand the patient population to aid future clinical research. Participants include children and adults aged 8 to 50 years with confirmed ABCA4 gene mutations. This observational study does not involve treatment interventions but focuses on monitoring the natural progression of the disease over time. Throughout the study, participants will undergo evaluations including Fundus Autofluorescence imaging to measure changes from baseline over a 24-month period. The study team will collect data on disease progression and patient characteristics to support future research efforts. Participation includes adherence to scheduled visits and assessments, with the overall goal of improving understanding of Stargardt disease.
CONDITIONS
Brief 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 adhere to the protocol as shown by written informed consent or assent as appropriate
- Confirmed mutation in the ABCA4 gene
You will not qualify if you...
- History of uveitis
- Any ocular disease in either eye that may confound assessment of the retina morphologically and functionally
- Any pathology of the posterior segment other than ABCA4 retinopathy
- Presence of any other genetic mutation(s) associated with retinal or macular dystrophy
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - Up to 24 months
Participants are observed over time to monitor changes related to Stargardt Disease without receiving any investigational treatment.
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
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Research Team
C
Clinical Operations Manager
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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