Clinical Trial: An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Subjects With Alagille Syndrome (ALGS)

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: A Multicentre Extension Study to Evaluate the Long-Term Safety and Durability of the Therapeutic Effect of LUM001, an Apical Sodium-Dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment

Brief Summary: The purpose of this extension study is to determine the long-term safety and tolerability of an investigational treatment (LUM001) in children with ALGS who have completed participation in the LUM001-302 study.

Detailed Summary:
Sponsor: Shire

Current Primary Outcome: Safety and tolerability [ Time Frame: 72 weeks ]

Adverse events, changes in vital signs, laboratory and other safety parameters from baseline to week 72


Original Primary Outcome: Safety and tolerability [ Time Frame: 48 weeks ]

Adverse events, changes in vital signs, laboratory and other safety parameters from baseline to week 48


Current Secondary Outcome: Efficacy [ Time Frame: 72 weeks ]

Changes in serum bile acids, pruritus, and other biochemical markers of cholestasis and liver disease from baseline to week 72


Original Secondary Outcome: Efficacy [ Time Frame: 48 weeks ]

Changes in serum bile acids, pruritus, and other biochemical markers of cholestasis and liver disease from baseline to week 48


Information By: Shire

Dates:
Date Received: January 23, 2014
Date Started: December 2013
Date Completion: August 2017
Last Updated: January 19, 2017
Last Verified: January 2017