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 Pediatric Subjects With Alagille Syndrome

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

Official Title: A Multicenter 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: This is a multicentre, extension study of LUM001 in children diagnosed with Alagille Syndrome who have completed participation in a core LUM001 treatment protocol. The primary objective is to evaluate long-term safety and tolerability of LUM001. Efficacy will be assessed by evaluating the effect of LUM001 on the biochemical markers and pruritus associated with Alagille Syndrome.

Detailed Summary:
Sponsor: Shire

Current Primary Outcome: Safety and tolerability [ Time Frame: 96 weeks ]

Evaluate teh long-term safety and tolerability of LUM001 in pediatric subjects with ALGS. This will include Adverse events, changes in vital signs, laboratory and other safety parameters from baseline to week 96


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 1 [ Time Frame: 96 weeks ]
    Evaluate the long-term effect of LUM001 on serum bile acid levels associated with ALGS from baseline to week 96
  • Efficacy 2 [ Time Frame: 96 weeks ]
    Evaluate the long-term effect of LUM001 on pruritus associated with ALGS from baseline to Week 96
  • Efficacy 3 [ Time Frame: 96 weeks ]
    Explore the long-term effect of LUM001 on other biochemical markers of cholestasis and liver disease
  • Efficacy 4 [ Time Frame: 96 weeks ]
    Evaluate the long-term effect of LUM001 on xanthomas associated with ALGS
  • Dosing [ Time Frame: 96 weeks ]
    Explore expanded dosing range to identify doses necessary to achieve the optimal benefit-to-risk ration for this patient population


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: April 16, 2014
Date Started: March 16, 2015
Date Completion: October 19, 2018
Last Updated: April 19, 2017
Last Verified: April 2017