An Observational Study in Children and Adults With Stargardt Disease
Recruiting
- Conditions
- Stargardt Disease
- Registration Number
- NCT06591806
- Lead Sponsor
- AAVantgarde Bio Srl
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 80
Inclusion Criteria
- Male and female subjects between 8 and 50 years of age at the time of enrolment.
- Willingness to adhere to the protocol as evidenced by written informed consent if the subject is 18 years or older. If the subject is under 18 years of age, written assent must be obtained from the subject and written informed consent must be obtained from the subject's legally authorized representative (parent or legal guardian).
- Confirmed mutation in the ABCA4 gene.
Exclusion Criteria
- 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) that have been associated with retinal or macular dystrophy.
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method To measure the change from baseline in Fundus Autofluorescence 24 months
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (1)
Retina Foundation of the Southwest
🇺🇸Dallas, Texas, United States