Clinical trial NCT02321306: An Open-label Study to Evaluate the Long-term Safety and Tolerability of LUM001 in Patients With Primary Biliary Cirrhosis
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
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: PHASE2
Conditions: Primary Biliary Cirrhosis
Lead sponsor: Mirum Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2014-12-22
Enrollment: 0
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Number of Participants with Adverse Events as a Measure of Safety
timeFrame: 2 years
Secondary outcomes:
measure: Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104
timeFrame: 2 years
measure: Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104
timeFrame: 2 years
measure: Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104
timeFrame: 2 years
measure: Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104
timeFrame: 2 years
measure: Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104
timeFrame: 2 years
Other outcomes:
Locations:
city: La Jolla
country: United States
facility: Scripps Clinic
state: California
city: Sacramento
country: United States
facility: University of California at Davis
state: California
city: Miami
country: United States
facility: University of Miami
state: Florida
city: Indianapolis
country: United States
facility: Indiana University
state: Indiana
city: Louisville
country: United States
facility: University of Louisville
state: Kentucky
city: Detroit
country: United States
facility: Henry Ford Health System
state: Michigan
city: Saint Paul
country: United States
facility: Minnesota Gastroenterology
state: Minnesota
city: Saint Louis
country: United States
facility: St. Louis University
state: Missouri
city: New York
country: United States
facility: Weill Cornell Medical College
state: New York
city: Dallas
country: United States
facility: University of Texas Southwestern Medical Center
state: Texas
city: Newport News
country: United States
facility: Liver Institute of Virginia
state: Virginia
city: Richmond
country: United States
facility: Hunter Holmes McGuire VA Medical Center
state: Virginia
city: Seattle
country: United States
facility: University of Washington Harborview Medical Center
state: Washington
city: Birmingham
country: United Kingdom
facility: University of Birmingham
state: England
city: Liverpool
country: United Kingdom
facility: Royal Liverpool & Broadgreen University Hospital
state: England
city: Newcastle Upon Tyne
country: United Kingdom
facility: Newcastle University
state: England
city: Oxford
country: United Kingdom
facility: Oxford University Hospitals (John Radcliffe)
state: England
city: London
country: United Kingdom
facility: Royal Free Hospital
Study officials:
affiliation: Mirum
name: Study Director
role: STUDY_DIRECTOR
References: