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An Observational Study in Subjects to Follow the Progression of Stargardt Disease Type 1 (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ABCA4 Gene

We observe Stargardt Disease Type 1 progression in patients.

Recruiting
12-65 years
All
Phase N/A
  • Simplified description (AI rewritten)
  • Technical description (Original)

Stargardt Disease Type 1 (STGD1) is a rare eye condition caused by changes in the ABCA4 gene. This is an observational study, meaning doctors will watch and record how the disease progresses without giving any treatment. The study aims to learn more about STGD1 to help design future treatments.

About 75 people, aged 12 to 65, with a confirmed diagnosis of STGD1 can join. Participants must have experienced worsening of their condition recently, and they need to have specific eye conditions to be eligible. They cannot have other eye diseases that might interfere with the study, nor can they have had recent eye surgeries.

  • The study is long-term but will not involve any treatment.
  • Participants will need to visit study centers for evaluations.
  • There are no direct health risks from the study itself, as no treatments are given.

Joining this study can help researchers understand STGD1 better, possibly leading to new therapies in the future.

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Study details
    Stargardt
    Stargardt's Disease
    Stargardt Disease
    STGD1

NCT06435000

Splice Bio

3 May 2025

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