A phase 2 trial of short-term intravenous N-acetylcysteine in biliary atresia after Kasai portoenterostomy
This study demonstrates how the two-stage "minimax" trial design can be used to efficiently evaluate potential therapies for BA. Although the primary endpoint was not met, NAC therapy was generally well-tolerated. NAC therapy may prove efficacious in future trials with (i) a less stringent primary endpoint and/or (ii) a longer course of treatment (NCT03499249).
Authors
- Laurel Cavallo (ORCID: https://orcid.org/0000-0002-8531-840X)
- Michael E. Scheurer (ORCID: https://orcid.org/0000-0002-8379-6088)
- Sridevi Devaraj (ORCID: https://orcid.org/0000-0001-9189-7914)
- Benjamin L. Shneider (ORCID: https://orcid.org/0000-0002-7208-0949)
- Mary Elizabeth M. Tessier (ORCID: https://orcid.org/0000-0002-0341-5442)
- Kalyani R. Patel (ORCID: https://orcid.org/0000-0003-2654-5386)
- Dana Cerminara
- Franca Erhiawarie
- Sanjiv Harpavat (ORCID: https://orcid.org/0000-0002-9964-2294)
- Sanjeev A. Vasudevan (ORCID: https://orcid.org/0000-0003-3578-4258)
- Kristin A. Borovsky
- Adam M. Vogel (ORCID: https://orcid.org/0000-0001-9660-6872)
Institutions
- Baylor College of Medicine (US)
- Texas Children's Hospital (US)
Publication Details
- Journal
- Hepatology Communications
- Published
- 2025-06-09
- DOI
- https://doi.org/10.1097/hc9.0000000000000729
- Primary Topic
- Pediatric Hepatobiliary Diseases and Treatments
- Type
- article
- Field-Weighted Citation Impact
- 0.00