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

Phase 2

Withdrawn

• Conditions: Primary Biliary Cirrhosis
• Interventions: Drug: LUM001

• Registration Number: NCT02321306
• Lead Sponsor: Mirum Pharmaceuticals, Inc.

Brief 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.

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 Timeline

• Study First Submitted: 2014-12-12
• Study First Submitted QC: 2014-12-16
• Study First Posted: 2014-12-22
• Study Start: 2015-05
• Primary Completion: 2017-12
• Study Completion: 2018-03
• Status Verified: 2019-03
• Last Update Submitted: 2019-03-15
• Last Update Posted: 2019-03-19

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
LUM001	LUM001	LUM001 administered orally once each day.

Primary Outcome Measures

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

Secondary Outcome Measures

Name	Time	Method
Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104	2 years	Quality of life assessments (itch and sleep scores)

Trial Locations

Locations (18)

Minnesota Gastroenterology; 🇺🇸 Saint Paul, Minnesota, United States

University of California at Davis; 🇺🇸 Sacramento, California, United States

Henry Ford Health System; 🇺🇸 Detroit, Michigan, United States

Scripps Clinic; 🇺🇸 La Jolla, California, United States

Weill Cornell Medical College; 🇺🇸 New York, New York, United States

Hunter Holmes McGuire VA Medical Center; 🇺🇸 Richmond, Virginia, United States

Royal Free Hospital; 🇬🇧 London, United Kingdom

University of Texas Southwestern Medical Center; 🇺🇸 Dallas, Texas, United States

University of Washington Harborview Medical Center; 🇺🇸 Seattle, Washington, United States

Indiana University; 🇺🇸 Indianapolis, Indiana, United States

St. Louis University; 🇺🇸 Saint Louis, Missouri, United States

Liver Institute of Virginia; 🇺🇸 Newport News, Virginia, United States

University of Louisville; 🇺🇸 Louisville, Kentucky, United States

University of Miami; 🇺🇸 Miami, Florida, United States

Royal Liverpool & Broadgreen University Hospital; 🇬🇧 Liverpool, England, United Kingdom

University of Birmingham; 🇬🇧 Birmingham, England, United Kingdom

Newcastle University; 🇬🇧 Newcastle Upon Tyne, England, United Kingdom

Oxford University Hospitals (John Radcliffe); 🇬🇧 Oxford, England, United Kingdom