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