An Open-label Study to Evaluate the Long-term Safety and Tolerability of LUM001 in Patients With Primary Biliary Cirrhosis (CASCADE)

An Open-label, Multicenter Extension Study to Evaluate the Long-term Safety and Tolerability of LUM001, an Apical Sodium-dependent Bile Acid Transporter Inhibitor (ASBTi), in Patients With Primary Biliary Cirrhosis

Open-label, multicenter study in adults with Primary Biliary Cirrhosis (PBC) designed to evaluate the long-term safety and tolerability of daily dosing with LUM001.

Study Overview

• Status: Withdrawn
• Conditions: Primary Biliary Cirrhosis
• Intervention / Treatment: Drug: LUM001

Detailed Description

This is an open-label study in subjects with PBC who previously completed the LUM001 201 (CLARITY) study. The study is designed to investigate the long-term safety and tolerability of LUM001 treatment, with daily dosing for up to 2 years. Changes over time, compared to baseline, in liver enzymes, other biochemical markers associated with PBC, pruritus, and other quality of life measures will also be assessed.

• Study Type: Interventional
• Phase: Phase 2

Contacts and Locations

Study Locations

• United Kingdom
  • London, United Kingdom, NW3 2QG
    • Royal Free Hospital
  • England
    • Birmingham, England, United Kingdom, B15 2TT
      • University of Birmingham
    • Liverpool, England, United Kingdom, L7 8XP
      • Royal Liverpool & Broadgreen University Hospital
    • Newcastle Upon Tyne, England, United Kingdom, NE1 4LP
      • Newcastle University
    • Oxford, England, United Kingdom, OX3 9DU
      • Oxford University Hospitals (John Radcliffe)
• United States
  • California
    • La Jolla, California, United States, 92037
      • Scripps Clinic
    • Sacramento, California, United States, 95817
      • University of California at Davis
  • Florida
    • Miami, Florida, United States, 33136
      • University of Miami
  • Indiana
    • Indianapolis, Indiana, United States, 46202
      • Indiana University
  • Kentucky
    • Louisville, Kentucky, United States, 40202
      • University of Louisville
  • Michigan
    • Detroit, Michigan, United States, 48202
      • Henry Ford Health System
  • Minnesota
    • Saint Paul, Minnesota, United States, 55114
      • Minnesota Gastroenterology
  • Missouri
    • Saint Louis, Missouri, United States, 63104
      • St. Louis University
  • New York
    • New York, New York, United States, 10021
      • Weill Cornell Medical College
  • Texas
    • Dallas, Texas, United States, 75390
      • University of Texas Southwestern Medical Center
  • Virginia
    • Newport News, Virginia, United States, 23602
      • Liver Institute of Virginia
    • Richmond, Virginia, United States, 23249
      • Hunter Holmes McGuire VA Medical Center
  • Washington
    • Seattle, Washington, United States, 98104
      • University of Washington Harborview Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Completed a core treatment protocol of LUM001 in the treatment of Primary Biliary Cirrhosis.
2. Ability to understand and willingness to sign informed consent/assent prior to initiation of any study procedures.

Exclusion Criteria:

1. Presence of advanced liver disease.
2. History of non-adherence during the subject's participation in the core LUM001 treatment protocol.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: LUM001; LUM001 administered orally once each day.	Drug: LUM001; LUM001 administered orally once each day.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants with Adverse Events as a Measure of Safety	Adverse events, serious adverse events, changes in vital signs, laboratory and physical exam findings, and concommitant medication usage from baseline to Week 104	2 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104	Alkaline phosphatase	2 years
Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104	Liver enzymes (ALT, AST)	2 years
Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104	Fasting serum bile acid	2 years
Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104	C4	2 years
Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104	Quality of life assessments (itch and sleep scores)	2 years

Collaborators and Investigators

• Sponsor: Mirum Pharmaceuticals, Inc.

Study record dates

Study Major Dates

• Study Start: May 1, 2015
• Primary Completion (Anticipated): December 1, 2017
• Study Completion (Anticipated): March 1, 2018

Study Registration Dates

• First Submitted: December 12, 2014
• First Submitted That Met QC Criteria: December 16, 2014
• First Posted (Estimate): December 22, 2014

Study Record Updates

• Last Update Posted (Actual): March 19, 2019
• Last Update Submitted That Met QC Criteria: March 15, 2019
• Last Verified: March 1, 2019

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Liver Diseases; Biliary Tract Diseases; Bile Duct Diseases; Cholestasis, Intrahepatic; Cholestasis; Fibrosis; Liver Cirrhosis; Liver Cirrhosis, Biliary
• Other Study ID Numbers: LUM001-202; 2014-003433-26 (EudraCT Number)