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A Study to Evaluate the Safety and Tolerability of Maralixibat in Infant Participants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille Syndrome (ALGS). — RISE  

Alagille Syndrome Clinical Trial

Official title:
Open-Label, Phase 2 Study to Evaluate the Safety and Tolerability of Maralixibat in the Treatment of Infants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis and Alagille Syndrome

Clinical Trial Summary

This study is designed to assess whether the investigational drug maralixibat, is safe and well tolerated in children <12 months of age with Alagille Syndrome [ALGS] or Progressive Familial Intrahepatic Cholestasis [PFIC].

Clinical Trial Description

This is an open label study where all participants will receive maralixibat treatment. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT04729751
Study type Interventional
Source Mirum Pharmaceuticals, Inc.
Contact
Status Active, not recruiting
Phase Phase 2
Start date September 9, 2021
Completion date December 2024
View Details
See also
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