Designing Clinical Trials in Wilson's Disease.

ABSTRACT:

Background and aims

Wilson's disease (WD) is an autosomal-recessive disorder caused by ATP7B gene mutations leading to pathological accumulation of copper in the liver and brain. Adoption of initial treatments for WD was based on empirical observations. These therapies are effective, but there are still unmet needs for which treatment modalities are being developed. An increase of therapeutical trials is anticipated.

Approach and results

The first Wilson Disease Aarhus Symposium (May 2019) included a workshop on randomized clinical trial design. The authors of the article were organizers or presented during this workshop, and this article presents their consensus on the design of clinical trials for WD, addressing trial population, treatment comparators, inclusion and exclus

SUBMITTER: Ott P 

PROVIDER: S-EPMC9291486 | biostudies-literature | 2021 Dec

REPOSITORIES: biostudies-literature