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

Primary Biliary Cirrhosis Clinical Trial

— CASCADE  

Official title:

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

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT02321306
• Other study ID #: LUM001-202
• Secondary ID: 2014-003433-26
• Status: Withdrawn
• Phase: Phase 2
• Start date: May 2015
• Est. completion date: March 2018

Study information

• Verified date: March 2019
• Source: Mirum Pharmaceuticals, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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.

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.

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. completion date: March 2018
• Est. primary completion date: December 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Fibrosis
• Liver Cirrhosis
• Liver Cirrhosis, Biliary
• Primary Biliary Cirrhosis

Intervention

Drug:
• LUM001
LUM001 administered orally once each day.

Locations

Country	Name	City	State
United Kingdom	University of Birmingham	Birmingham	England
United Kingdom	Royal Liverpool & Broadgreen University Hospital	Liverpool	England
United Kingdom	Royal Free Hospital	London
United Kingdom	Newcastle University	Newcastle Upon Tyne	England
United Kingdom	Oxford University Hospitals (John Radcliffe)	Oxford	England
United States	University of Texas Southwestern Medical Center	Dallas	Texas
United States	Henry Ford Health System	Detroit	Michigan
United States	Indiana University	Indianapolis	Indiana
United States	Scripps Clinic	La Jolla	California
United States	University of Louisville	Louisville	Kentucky
United States	University of Miami	Miami	Florida
United States	Weill Cornell Medical College	New York	New York
United States	Liver Institute of Virginia	Newport News	Virginia
United States	Hunter Holmes McGuire VA Medical Center	Richmond	Virginia
United States	University of California at Davis	Sacramento	California
United States	St. Louis University	Saint Louis	Missouri
United States	Minnesota Gastroenterology	Saint Paul	Minnesota
United States	University of Washington Harborview Medical Center	Seattle	Washington

Sponsors (1)

Lead Sponsor	Collaborator
Mirum Pharmaceuticals, Inc.

Countries where clinical trial is conducted

United States,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104	Alkaline phosphatase	2 years
Secondary	Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104	Liver enzymes (ALT, AST)	2 years
Secondary	Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104	Fasting serum bile acid	2 years
Secondary	Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104	C4	2 years
Secondary	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