Endpoints and Design for Clinical Trials in USH2A-Related Retinal Degeneration: Results and Recommendations From the RUSH2A Natural History Study.

ABSTRACT:

Purpose

To evaluate functional and structural assessments as endpoints for clinical trials for USH2A-related retinal degeneration.

Methods

People with biallelic disease-causing variants in USH2A, visual acuity ≥ 20/80, and visual field ≥ 10° diameter were enrolled in a 4-year, natural history study. Participants underwent static perimetry, microperimetry, visual acuity, fullfield stimulus testing (FST), and optical coherence tomography annually. Rates of change estimated from mixed-effects linear models and percentages of eyes with changes exceeding the coefficient of repeatability (CoR) or thresholds conforming with U.S. Food and Drug Administration (FDA) guidelines were evaluated.

Results

Rates of change were generally more sensitive to change than proportions of ey

SUBMITTER: Maguire MG 

PROVIDER: S-EPMC11469320 | biostudies-literature | 2024 Oct

REPOSITORIES: biostudies-literature