Document Type
Article
Publication Date
5-2025
Identifier
DOI: 10.1080/23995270.2025.2492473
Abstract
This is a summary describing a phase 3 clinical trial, called ‘ASSERT’, which studied the medicine odevixibat in people with Alagille syndrome.
Alagille syndrome is a rare disease that can affect different parts of the body, but mainly affects how well the liver works. Symptoms usually start at a young age, but the age of first symptoms and seriousness of the disease can vary, and the disease can affect both children and adults. People who have Alagille syndrome may have a build-up of bile acids in the liver and this can lead to organ damage. People with Alagille syndrome may also have a build-up of bile acids in the blood and may suffer from feelings of intense itch, called pruritus. Pruritus contributes to poor day-to-day quality of life in people with Alagille syndrome. In serious cases of the disease, some people might need surgery to alter bile flow or a liver transplantation.
Previous options for treating pruritus in Alagille syndrome have been limited and have not worked very well for some people. Odevixibat is a treatment which improves pruritus in people who have Alagille syndrome and lowers the levels of bile acids in the blood by helping bile acids to be removed through poo.
Journal Title
Future Rare Diseases
Volume
5
Issue
1
Keywords
Alagille syndrome; Odevixibat; pruritus; Rare liver disease; Cholestasis: ASSERT clinical trial; phase 3 clinical trial; plain language summary
Recommended Citation
Ovchinsky, N., Aumar, M., Baker, A., Bufler, P., Cananzi, M., Czubkowski, P., … Sturm, E. (2025). The effects of odevixibat in people with Alagille syndrome: a plain language summary. Future Rare Diseases, 5(1). https://doi.org/10.1080/23995270.2025.2492473
Comments
Link to original article: https://scholarlyexchange.childrensmercy.org/papers/5988/
This is an Open Access article distributed under the terms of the Creative Commons Attribution-NonCommercial License (http://creativecommons.org/licenses/by-nc/4.0/), which permits unrestricted non-commercial use, distribution, and reproduction in any medium, provided the original work is properly cited. The terms on which this article has been published allow the posting of the Accepted Manuscript in a repository by the author(s) or with their consent.
Publisher's Link: https://www.tandfonline.com/doi/abs/10.1080/23995270.2025.2492473