Clinical trial NCT01904058: Phase 2 Study to Evaluate LUM001 in Combination With Ursodeoxycholic Acid in Patients With Primary Biliary Cirrhosis
Official title: A Phase 2, Randomized, Double-blind, Placebo-controlled Study to Evaluate LUM001, an Apical Sodium-dependent Bile Acid Transporter Inhibitor (ASBTi) in Combination With Ursodeoxycholic Acid (UDCA) in Patients With Primary Biliary Cirrhosis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: PBC
Primary Biliary Cirrhosis
Keywords: PBC
Lead sponsor: Mirum Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2013-07-22
Enrollment: 66
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The study is a randomized, double-blind, placebo-controlled, multicenter study. It is a 13-week Phase 2 study in adults with primary biliary cirrhosis designed to compare the effect of daily dosing with UDCA in combination with LUM001 or placebo.


Eligibility criteria: Inclusion Criteria:

1. Diagnosis of Primary Biliary Cirrhosis
2. Moderate to severe pruritus
3. Taking ursodeoxycholic acid (UDCA) for at least 6 months, or unable to tolerate UDCA
4. Ability to understand and willingness to sign informed consent prior to initiation of any study procedures

Exclusion Criteria:

1. History or presence of other concomitant significant liver disease
2. Liver transplant
3. Known HIV infection
4. Women who are pregnant or lactating

Primary outcomes:
measure: Change From Baseline in Pruritus Using Adult Itch Reported Outcome (ItchRO) Weekly Sum Score at Week 13/ Early Termination (ET)
timeFrame: Baseline and Week 13/ET
Secondary outcomes:
measure: Change From Baseline in Pruritus Using Adult ItchRO Weekly Sum Scores at Weeks 4, 8 and 13
timeFrame: Baseline, Weeks 4, 8 and 13
measure: Change From Baseline in Pruritus Using Adult ItchRO Average Daily Scores at Weeks 4, 8, 13, and Last Post-baseline Visit (Week 13/ET)
timeFrame: Baseline, Weeks 4, 8, 13 and Last Post-baseline visit (Week 13/ET)
measure: Change From Baseline in Alkaline Phosphatase (ALP) at Weeks 4, 8, 13, and Last Post-baseline Visit (Week 13/ET)
timeFrame: Baseline, Weeks 4, 8, 13 and Last Post-baseline (Week 13/ET)
measure: Change From Baseline in 5-D Itch Score at Weeks 4, 8, 13, and Last Post -Baseline Visit (Week 13/ET)
timeFrame: Baseline, Weeks 4, 8, 13 and Last Post-baseline visit (Week 13/ET)
measure: Change From Baseline in Fasting Serum Bile Acid Level at Weeks 4, 8, 13, and Last Post -Baseline Visit (Week 13/ET)
timeFrame: Baseline, Weeks 4, 8, 13 and Last Post-baseline visit (Week 13/ET)
measure: Change From Baseline in Bile Acid Synthesis as Measured by Serum 7 Alpha-Hydroxy-4-Cholesten-3-One C4 Level [7 Alpha C4]) at Weeks 4, 8, 13, and Last Post -Baseline Visit (Week 13/ET)
timeFrame: Baseline, Weeks 4, 8, 13 and Last Post-baseline Visit (Week 13/ET)
Other outcomes:
measure: Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)
timeFrame: From the first dose of study drug until the 13 weeks of treatment period (or ET) + 14 days (approximately 15 weeks)
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: Chicago
country: United States
facility: University of Chicago Medical Center
state: Illinois
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: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
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: Houston
country: United States
facility: Advanced Liver Therapies at St. Lukes Episcopal Hospital
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah Health Science Center
state: Utah
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: Toronto
country: Canada
facility: University Health Network, Toronto Western Hospital
state: Ontario
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
city: London
country: United Kingdom
facility: Imperial College London St Mary's Hospital
Study officials:
affiliation: Mirum
name: Study Director
role: STUDY_DIRECTOR
References: