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An Observational Study in Children and Adults With Stargardt Disease

Recruiting
Conditions
Stargardt Disease
Registration Number
NCT06591806
Lead Sponsor
AAVantgarde Bio Srl
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
Recruiting
Sex
All
Target Recruitment
80
Inclusion Criteria

Inclusion Criteria:<br><br> 1. Male and female subjects between 8 and 50 years of age at the time of enrolment.<br><br> 2. Willingness to adhere to the protocol as evidenced by written informed consent if<br> the subject is 18 years or older. If the subject is under 18 years of age, written<br> assent must be obtained from the subject and written informed consent must be<br> obtained from the subject's legally authorized representative (parent or legal<br> guardian).<br><br> 3. Confirmed mutation in the ABCA4 gene.<br><br>Exclusion Criteria:<br><br> 1. History of uveitis.<br><br> 2. Any ocular disease in either eye that may confound assessment of the retina<br> morphologically and functionally.<br><br> 3. Any pathology of the posterior segment other than ABCA4 retinopathy.<br><br> 4. Presence of any other genetic mutation(s) that have been associated with retinal or<br> macular dystrophy.

Exclusion Criteria

Not provided

Study & Design

Study Type
Observational
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
To measure the change from baseline in Fundus Autofluorescence
Secondary Outcome Measures
NameTimeMethod
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