NCT01904058

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.

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
66

participants targeted

Target at P50-P75 for phase_2

Timeline
Completed

Started Aug 2013

Geographic Reach
3 countries

24 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

July 17, 2013

Completed
5 days until next milestone

First Posted

Study publicly available on registry

July 22, 2013

Completed
10 days until next milestone

Study Start

First participant enrolled

August 1, 2013

Completed
1.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 1, 2015

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

April 1, 2015

Completed
1.3 years until next milestone

Results Posted

Study results publicly available

July 6, 2016

Completed
Last Updated

March 27, 2019

Status Verified

March 1, 2019

Enrollment Period

1.7 years

First QC Date

July 17, 2013

Results QC Date

April 1, 2016

Last Update Submit

March 15, 2019

Conditions

Keywords

PBC

Outcome Measures

Primary Outcomes (1)

  • Change From Baseline in Pruritus Using Adult Itch Reported Outcome (ItchRO) Weekly Sum Score at Week 13/ Early Termination (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.

    Baseline and Week 13/ET

Secondary Outcomes (6)

  • Change From Baseline in Pruritus Using Adult ItchRO Weekly Sum Scores at Weeks 4, 8 and 13

    Baseline, Weeks 4, 8 and 13

  • 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)

  • 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)

  • 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)

  • 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)

  • +1 more secondary outcomes

Other Outcomes (1)

  • Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)

    From the first dose of study drug until the 13 weeks of treatment period (or ET) + 14 days (approximately 15 weeks)

Study Arms (2)

LUM001 and Ursodeoxycholic Acid (UDCA)

EXPERIMENTAL

Administered orally once daily

Drug: LUM001Drug: Ursodeoxycholic Acid

Placebo and Ursodeoxycholic Acid (UDCA)

PLACEBO COMPARATOR

Administered orally once daily

Drug: PlaceboDrug: Ursodeoxycholic Acid

Interventions

LUM001DRUG
LUM001 and Ursodeoxycholic Acid (UDCA)
Placebo and Ursodeoxycholic Acid (UDCA)
Also known as: UDCA
LUM001 and Ursodeoxycholic Acid (UDCA)Placebo and Ursodeoxycholic Acid (UDCA)

Eligibility Criteria

Age18 Years - 80 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • History or presence of other concomitant significant liver disease
  • Liver transplant
  • Known HIV infection
  • Women who are pregnant or lactating

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (24)

Scripps Clinic

La Jolla, California, 92037, United States

Location

University of California at Davis

Sacramento, California, 95817, United States

Location

University of Miami

Miami, Florida, 33136, United States

Location

University of Chicago Medical Center

Chicago, Illinois, 60637, United States

Location

Indiana University

Indianapolis, Indiana, 46202, United States

Location

University of Louisville

Louisville, Kentucky, 40202, United States

Location

Henry Ford Health System

Detroit, Michigan, 48202, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

Minnesota Gastroenterology

Saint Paul, Minnesota, 55114, United States

Location

St. Louis University

St Louis, Missouri, 63104, United States

Location

Weill Cornell Medical College

New York, New York, 10021, United States

Location

University of Texas Southwestern Medical Center

Dallas, Texas, 75390, United States

Location

Advanced Liver Therapies at St. Lukes Episcopal Hospital

Houston, Texas, 77030, United States

Location

University of Utah Health Science Center

Salt Lake City, Utah, 84132, United States

Location

Liver Institute of Virginia

Newport News, Virginia, 23602, United States

Location

Hunter Holmes McGuire VA Medical Center

Richmond, Virginia, 23249, United States

Location

University of Washington Harborview Medical Center

Seattle, Washington, 98104, United States

Location

University Health Network, Toronto Western Hospital

Toronto, Ontario, M5T 2S8, Canada

Location

University of Birmingham

Birmingham, England, B15 2TT, United Kingdom

Location

Royal Liverpool & Broadgreen University Hospital

Liverpool, England, L7 8XP, United Kingdom

Location

Newcastle University

Newcastle upon Tyne, England, NE1 4LP, United Kingdom

Location

Oxford University Hospitals (John Radcliffe)

Oxford, England, OX3 9DU, United Kingdom

Location

Royal Free Hospital

London, NW3 2QG, United Kingdom

Location

Imperial College London St Mary's Hospital

London, W2 1NY, United Kingdom

Location

Related Links

MeSH Terms

Conditions

Liver Cirrhosis, Biliary

Interventions

Ursodeoxycholic Acid

Condition Hierarchy (Ancestors)

Cholestasis, IntrahepaticCholestasisBile Duct DiseasesBiliary Tract DiseasesDigestive System DiseasesLiver DiseasesLiver CirrhosisFibrosisPathologic ProcessesPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Deoxycholic AcidCholic AcidsBile Acids and SaltsSteroidsFused-Ring CompoundsPolycyclic CompoundsCholanes

Results Point of Contact

Title
Study Physician
Organization
Mirum

Study Officials

  • Study Director

    Mirum

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
DOUBLE
Who Masked
PARTICIPANT, INVESTIGATOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 17, 2013

First Posted

July 22, 2013

Study Start

August 1, 2013

Primary Completion

April 1, 2015

Study Completion

April 1, 2015

Last Updated

March 27, 2019

Results First Posted

July 6, 2016

Record last verified: 2019-03

Locations