Clinical Trial: A Long-Term, Open-Label Study of LUM001 With a Double-Blind, Placebo Controlled, Randomized Drug Withdrawal Period to Evaluate Safety and Efficacy in Children With Alagille Syndrome

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

Official Title: Long-Term, Open-Label Study With a Double-Blind, Placebo-Controlled, Randomized Drug Withdrawal Period of LUM001, an Apical Sodium-Dependent Bile Acid Transporter Inhibitor (ASBTi), in Patients With <

Brief Summary: This is a long-term, open-label study with a double-blind, placebo-controlled, randomized drug withdrawal period in children with ALGS designed to evaluate the safety and efficacy of LUM001. The study is divided into 5 parts: a 6-week open-label, dose escalation period, a 12-week stable dosing period, a 4-week randomized, double-blind, placebo-controlled drug withdrawal period, a 26-week long-term stable dosing period, and an optional 52 week follow-up treatment period for eligible subjects who choose to stay on treatment with LUM001. Subjects' participation in the optional follow-up treatment period will continue until the first of the following occur: (i) completion of 52 weeks of additional treatment (Week 100), or (ii) in the event that a new study of LUM001 opens to enrollment.

Detailed Summary:
Sponsor: Shire

Current Primary Outcome:

  • Evaluate effect of LUM001 on serum bile acid levels in children with ALGS using an analysis of covariance (ANCOVA) model treatment [ Time Frame: 48 weeks ]
  • Evaluate effect of LUM001 on biochemical markers of cholestasis in children with ALGS [ Time Frame: 48 weeks ]
    Change from Baseline in Liver enzymes (ALT, ALP) and bilirubin (total and direct) and other biochemical markers of cholestasis [total cholesterol, low-density lipoprotein cholesterol (LDL-C)].
  • Evaluate effect of LUM001 on pruritus in children with ALGS as measured by the average daily Itch Reported Outcome (ItchRO) [ Time Frame: 48 weeks ]


Original Primary Outcome: Efficacy [ Time Frame: 4 weeks ]

Mean change in serum bile acid levels from week 18 to week 22


Current Secondary Outcome:

Original Secondary Outcome: Efficacy [ Time Frame: 4 weeks ]

Change in liver enzymes and pruritus from week 18 to week 22


Information By: Shire

Dates:
Date Received: June 9, 2014
Date Started: September 2014
Date Completion: September 2017
Last Updated: January 19, 2017
Last Verified: January 2017