Clinical Trial: Safety and Efficacy Study of LUM001 in the Treatment of Cholestatic Liver Disease in Patients With Alagille Syndrome

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A Randomized, Double-blind, Placebo-controlled Study to Evaluate the Safety and Efficacy of LUM001, an Apical Sodium-dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic

Brief Summary: The study is a randomized, double-blind, placebo-controlled study to evaluate the safety and tolerability of LUM001. Efficacy will be assessed by evaluating the effect of LUM001 versus placebo on the biochemical markers and pruritus associated with Alagille Syndrome.

Detailed Summary:
Sponsor: Shire

Current Primary Outcome: Change From Baseline to Week 13 (End of Treatment) in Fasting Serum Bile Acid Level [ Time Frame: Baseline to 13 weeks or end of treatment ]

Participants were required to fast for at least 4 hours; only water was permitted prior to collection. A negative change from baseline indicates that the level of bile acid decreased.


Original Primary Outcome: Efficacy [ Time Frame: 13 weeks ]

Change from baseline in fasting serum bile acids compared to placebo


Current Secondary Outcome:

  • Change From Baseline to Week 13 (End of Treatment) in Liver Enzymes [ Time Frame: Baseline to 13 weeks or end of treatment ]
    Analysis of liver enzymes included alanine aminotransferase (ALT), aspartate aminotransferase (AST), and alkaline phosphatase (ALP). A negative change from baseline indicates that the level of that enzyme decreased.
  • Change From Baseline to Week 13 (End of Treatment) in Pruritus as Measured by The Patient And Observer Itch Reported Outcome (ItchRO) Average Daily Scores [ Time Frame: Baseline to 13 weeks or end of treatment ]
    The ItchRO was administered as a twice daily electronic diary (eDiary). Children ≥9 years of age completed the patient ItchRO; those between the ages of 5 and 8 completed the patient ItchRO with the assistance of their caregiver. There was no patient report for subjects under the age of 5. ItchRO scores range from 0 to 4, with the higher score indicating increasing itch severity. ItchRO average daily scores were calculated as the sum of daily scores (ie, the maximum of morning and evening scores) divided by the number of days. The average daily score was calculated by using the 7 days pre-treatment for baseline, and the last 7 days of treatment for Week 13. A negative change from Baseline indicates that itch severity decreased.


Original Secondary Outcome: Efficacy [ Time Frame: 13 weeks ]

Change from baseline in liver enzymes and pruritus compared to placebo


Information By: Shire

Dates:
Date Received: July 16, 2013
Date Started: August 2013
Date Completion:
Last Updated: November 2, 2015
Last Verified: June 2015