Phase 2 Study to Evaluate LUM001 in Combination With Ursodeoxycholic Acid in Patients With Primary Biliary Cirrhosis
- Conditions
- Primary Biliary CirrhosisPBC
- Interventions
- Registration Number
- NCT01904058
- Lead Sponsor
- Mirum Pharmaceuticals, Inc.
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 66
- Diagnosis of Primary Biliary Cirrhosis
- Moderate to severe pruritus
- Taking ursodeoxycholic acid (UDCA) for at least 6 months, or unable to tolerate UDCA
- Ability to understand and willingness to sign informed consent prior to initiation of any study procedures
- History or presence of other concomitant significant liver disease
- Liver transplant
- Known HIV infection
- Women who are pregnant or lactating
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Placebo and Ursodeoxycholic Acid (UDCA) Placebo Administered orally once daily LUM001 and Ursodeoxycholic Acid (UDCA) LUM001 Administered orally once daily LUM001 and Ursodeoxycholic Acid (UDCA) Ursodeoxycholic Acid Administered orally once daily Placebo and Ursodeoxycholic Acid (UDCA) Ursodeoxycholic Acid Administered orally once daily
- Primary Outcome Measures
Name Time Method Change From Baseline in Pruritus Using Adult Itch Reported Outcome (ItchRO) Weekly Sum Score at Week 13/ Early Termination (ET) Baseline and Week 13/ET Pruritus was assessed using ItchRO measure, administered as an electronic diary (eDiary) which was completed by the participants twice daily (morning and evening). (ItchRO) scores ranged from 0 to 10, with 0 representing no itch and 10 representing very severe itching. The highest score between the morning and evening ItchRO reports represented the daily score: a measure of the worst itching over the previous 24-hour period. The weekly sum score was calculated as the sum of the daily scores for the 7 days prior to the time point being reported: 7 days prior to randomization or 7 days prior to Week 13/ET visit.
- Secondary Outcome Measures
Name Time Method Change From Baseline in Fasting Serum Bile Acid Level at Weeks 4, 8, 13, and Last Post -Baseline Visit (Week 13/ET) Baseline, Weeks 4, 8, 13 and Last Post-baseline visit (Week 13/ET) Laboratory serum bile acid level levels were evaluated using blood samples collected.
Change From Baseline in Pruritus Using Adult ItchRO Weekly Sum Scores at Weeks 4, 8 and 13 Baseline, Weeks 4, 8 and 13 ItchRO scores had a range from 0 to 10, with 0 representing no itch and 10 representing very severe itching. The highest score between the morning and evening ItchRO reports represented the daily score: a measure of the worst itching over the previous 24-hour period. The weekly sum score was calculated as the sum of the daily scores for the 7 days prior to the time point being reported: 7 days prior to randomization or 7 days prior to Week 13/ET visit.
Change From Baseline in Pruritus Using Adult ItchRO Average Daily Scores at Weeks 4, 8, 13, and Last Post-baseline Visit (Week 13/ET) Baseline, Weeks 4, 8, 13 and Last Post-baseline visit (Week 13/ET) ItchRO scores had a range from 0 to 10, with 0 representing no itch and 10 representing very severe itching. The highest score between the morning and evening ItchRO reports represented the daily score: a measure of the worst itching over the previous 24-hour period. Adult ItchRO average daily score was the sum of daily scores divided by the number of days adult ItchRO was completed, using the 7 days prior to the reported visit date.
Change From Baseline in Alkaline Phosphatase (ALP) at Weeks 4, 8, 13, and Last Post-baseline Visit (Week 13/ET) Baseline, Weeks 4, 8, 13 and Last Post-baseline (Week 13/ET) Laboratory serum ALP enzyme levels were evaluated using blood samples collected.
Change From Baseline in 5-D Itch Score at Weeks 4, 8, 13, and Last Post -Baseline Visit (Week 13/ET) Baseline, Weeks 4, 8, 13 and Last Post-baseline visit (Week 13/ET) The 5-D itch (validated instrument to measure pruritus) scale was developed for the multidimensional quantification of pruritus that is sensitive to change over time. The 5-D itch scale included 5 domains (duration, degree, direction, disability, and distribution of pruritus). The total 5-D score was obtained by scoring each of the domains separately and then summing them together. 5-D total scores ranged between 5 (no pruritus) and 25 (most severe pruritus).
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) Baseline, Weeks 4, 8, 13 and Last Post-baseline Visit (Week 13/ET) C4 7 alpha-hydroxy-4-cholesten-3-one is an intermediate in the biochemical synthesis of bile acids from cholesterol and its concentrations reflect the activity of the bile acid synthetic pathway. Elevated levels of C4 indicate bile acid malabsorption. Laboratory C4 levels were evaluated using blood samples collected.
Trial Locations
- Locations (24)
Minnesota Gastroenterology
🇺🇸Saint Paul, Minnesota, United States
Henry Ford Health System
🇺🇸Detroit, Michigan, United States
Scripps Clinic
🇺🇸La Jolla, California, United States
University of California at Davis
🇺🇸Sacramento, California, United States
University of Miami
🇺🇸Miami, Florida, United States
Indiana University
🇺🇸Indianapolis, Indiana, United States
Mayo Clinic
🇺🇸Rochester, Minnesota, United States
University of Chicago Medical Center
🇺🇸Chicago, Illinois, United States
University of Louisville
🇺🇸Louisville, Kentucky, United States
Royal Liverpool & Broadgreen University Hospital
🇬🇧Liverpool, England, United Kingdom
Liver Institute of Virginia
🇺🇸Newport News, Virginia, United States
University of Birmingham
🇬🇧Birmingham, England, United Kingdom
St. Louis University
🇺🇸Saint Louis, Missouri, United States
Weill Cornell Medical College
🇺🇸New York, New York, United States
Advanced Liver Therapies at St. Lukes Episcopal Hospital
🇺🇸Houston, Texas, United States
University of Texas Southwestern Medical Center
🇺🇸Dallas, Texas, United States
University of Utah Health Science Center
🇺🇸Salt Lake City, Utah, United States
Hunter Holmes McGuire VA Medical Center
🇺🇸Richmond, Virginia, United States
Newcastle University
🇬🇧Newcastle Upon Tyne, England, United Kingdom
University Health Network, Toronto Western Hospital
🇨🇦Toronto, Ontario, Canada
Oxford University Hospitals (John Radcliffe)
🇬🇧Oxford, England, United Kingdom
Royal Free Hospital
🇬🇧London, United Kingdom
Imperial College London St Mary's Hospital
🇬🇧London, United Kingdom
University of Washington Harborview Medical Center
🇺🇸Seattle, Washington, United States