A Comparison of Randomizing Either One Eye or Both Eyes in Clinical Trials for Stargardt Disease Type 1.

ABSTRACT:

Objective

Designing a clinical trial for rare diseases such as Stargardt disease type 1 is challenging due to the limited patient population. In traditional clinical trial designs for inherited retinal diseases, often only 1 eye of each patient is used as the treated eye or the sham, disregarding half of the available eyes.This study explores a trial design in which both eyes are included, with the fellow eye serving as the control, maximizing the use of available data and enhancing statistical power.

Design

Retrospective analysis of natural history data to conduct sample size calculations.

Participants

Patients with genetically solved Stargardt disease type 1 who had at least 2 fundus autofluorescence measurements obtained within 5 years of each other. Retrospective

SUBMITTER: Pas JAAH 

PROVIDER: S-EPMC12818204 | biostudies-literature | 2026 Feb

REPOSITORIES: biostudies-literature