An Observational Study in Children and Adults With Stargardt Disease

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

80 participants

Study Classification

OBSERVATIONAL

Study Start Date

2024-05-01

Study Completion Date

2027-06-30

Brief Summary

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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.

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Detailed Description

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Conditions

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Stargardt Disease

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Eligibility Criteria

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Inclusion Criteria

1. Male and female subjects between 8 and 50 years of age at the time of enrolment.
2. 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).
3. Confirmed mutation in the ABCA4 gene.

Exclusion Criteria

1. History of uveitis.
2. Any ocular disease in either eye that may confound assessment of the retina morphologically and functionally.
3. Any pathology of the posterior segment other than ABCA4 retinopathy.
4. Presence of any other genetic mutation(s) that have been associated with retinal or macular dystrophy.

Minimum Eligible Age

8 Years

Maximum Eligible Age

50 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No