Clinical Trial: Open Label Study to Evaluate Efficacy and Long Term Safety of LUM001 in the Treatment of Cholestatic Liver Disease in Patients With Progressive Familial Intrahepatic Cholestasis

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

Official Title: Open Label Study of the Efficacy and Long Term Safety of LUM001, an Apical Sodium-Dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Pediatric Patients

Brief Summary: This is an open label study in children with Progressive Familial Intrahepatic Cholestasis (PFIC) designed to evaluate the safety and efficacy of LUM001. Efficacy will be assessed by evaluating the effect of LUM001 on the biochemical markers and pruritus associated with PFIC.

Detailed Summary:
Sponsor: Shire

Current Primary Outcome:

  • Evaluate the effect of LUM001 by the mean change from baseline to Week 13 in fasting serum bile acid level [ Time Frame: Baseline to 13 weeks ]
    Exposure to study drug will be measured approximately 2-4 hours post dose and data will be summarized and listed across the treatment period by treatment group.
  • Evaluate the effect of LUM001 by the mean change from baseline to Week 13 on biochemical markers of cholestasis and liver disease [ Time Frame: Baseline to 13 weeks ]
    Alanine aminotransferase (ALT) and bilirubin (total and direct).
  • Evaluate the effect of LUM001 by the mean change from baseline to Week 13 on pruritus in pediatric subjects with PFIC as measured by Itch Reported Outcome (ItchRO). [ Time Frame: Baseline to 13 weeks ]
    Pruritus as measured by ItchRO (Observer ItchRO/patient ItchRO) change.


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

Change in fasting serum bile acids from baseline to Week 13


Current Secondary Outcome:

  • Evaluate Long-Term Safety and Tolerability of LUM001 through 72 weeks and qualifed subjects who continue beyond 72 weeks. [ Time Frame: Approximately 124 weeks ]
    Safety and clinical laboratory evaluations and a physical exam (including collection of vital signs, height and weight measurements) will be completed at each clinic visit.
  • Assess the level of alpha-fetoprotein (AFP), a marker of hepatocellular carcinoma, by means of blood collection [ Time Frame: Approximately 124 weeks ]
    Blood samples to be collected and assessed every 24 weeks


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

Change in liver enzymes and pruritus from baseline to Week 13


Information By: Shire

Dates:
Date Received: February 5, 2014
Date Started: March 2014
Date Completion: August 2018
Last Updated: February 24, 2017
Last Verified: February 2017