Phase 2
N=66
Phase 2 Study to Evaluate LUM001 in Combination With Ursodeoxycholic Acid in Patients With Primary Biliary Cirrhosis
PBC · Primary Biliary Cirrhosis
Bottom Line
View on ClinicalTrials.gov: NCT01904058 ↗Enrolled (actual)
66
Serious AEs
4.6%
Results posted
Jul 2016
Primary outcome: Primary: Change From Baseline in Pruritus Using Adult Itch Reported Outcome (ItchRO) Weekly Sum Score at Week 13/ Early Termination (ET) — 48.11; 52.10; 54.64; 49.46 units on a scale — p=0.6603
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- LUM001 (Drug); Placebo (Drug); Ursodeoxycholic Acid (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Mirum Pharmaceuticals, Inc.
- Primary completion
- Apr 2015
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline in Pruritus Using Adult Itch Reported Outcome (ItchRO) Weekly Sum Score at Week 13/ Early Termination (ET) |
48.11; 52.10; 54.64; 49.46; -24.59; -27.67 | 0.6603 |
| SECONDARY Change From Baseline in Pruritus Using Adult ItchRO Weekly Sum Scores at Weeks 4, 8 and 13 |
48.11; 52.10; 54.64; 49.46; -15.62; -22.19 | — |
| SECONDARY Change From Baseline in Pruritus Using Adult ItchRO Average Daily Scores at Weeks 4, 8, 13, and Last Post-baseline Visit (Week 13/ET) |
6.873; 7.442; 7.805; 7.066; -2.231; -3.170 | — |
| SECONDARY Change From Baseline in Alkaline Phosphatase (ALP) at Weeks 4, 8, 13, and Last Post-baseline Visit (Week 13/ET) |
288.2; 257.6; 253.9; 274.2; -22.1; 13.2 | — |
| SECONDARY Change From Baseline in 5-D Itch Score at Weeks 4, 8, 13, and Last Post -Baseline Visit (Week 13/ET) |
18.7; 19.4; 19.6; 19.2; -4.8; -6.3 | — |
| SECONDARY Change From Baseline in Fasting Serum Bile Acid Level at Weeks 4, 8, 13, and Last Post -Baseline Visit (Week 13/ET) |
33.110; 52.460; 52.615; 58.434; -11.204; -14.465 | — |
| SECONDARY 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) |
18.74; 13.17; 22.31; 16.98; 8.66; 17.03 | — |
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
- 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
Exclusion Criteria
- History or presence of other concomitant significant liver disease
- Liver transplant
- Known HIV infection
- Women who are pregnant or lactating
Data sourced from ClinicalTrials.gov (NCT01904058). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.