Clinical trials for Wolfram syndrome neurodegeneration: Novel design, endpoints, and analysis models.

ABSTRACT:

Objective

Wolfram syndrome, an ultra-rare condition, currently lacks effective treatment options. The rarity of this disease presents significant challenges in conducting clinical trials, particularly in achieving sufficient statistical power (e.g., 80%). The objective of this study is to propose a novel clinical trial design based on real-world data to reduce the sample size required for conducting clinical trials for Wolfram syndrome.

Methods

We propose a novel clinical trial design with three key features aimed at reducing sample size and improve efficiency: (i) Pooling historical/external controls from a longitudinal observational study conducted by the Washington University Wolfram Research Clinic. (ii) Utilizing run-in data to estimate model parameters. (iii) Simultane

SUBMITTER: Wang G 

PROVIDER: S-EPMC12064034 | biostudies-literature | 2025

REPOSITORIES: biostudies-literature