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An Open-label Study to Evaluate the Long-term Safety and Tolerability of LUM001 in Patients With Primary Biliary Cirrhosis (CASCADE)

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ClinicalTrials.gov Identifier: NCT02321306
Recruitment Status : Withdrawn (The study stopped early, before enrolling its first participant.)
First Posted : December 22, 2014
Last Update Posted : March 19, 2019
Sponsor:
Information provided by (Responsible Party):
Mirum Pharmaceuticals, Inc.

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.

Condition or disease Intervention/treatment Phase
Primary Biliary Cirrhosis Drug: LUM001 Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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 Start Date : May 2015
Estimated Primary Completion Date : December 2017
Estimated Study Completion Date : March 2018


Arm Intervention/treatment
Experimental: LUM001
LUM001 administered orally once each day.
Drug: LUM001
LUM001 administered orally once each day.




Primary Outcome Measures :
  1. Number of Participants with Adverse Events as a Measure of Safety [ Time Frame: 2 years ]
    Adverse events, serious adverse events, changes in vital signs, laboratory and physical exam findings, and concommitant medication usage from baseline to Week 104


Secondary Outcome Measures :
  1. Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104 [ Time Frame: 2 years ]
    Alkaline phosphatase

  2. Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104 [ Time Frame: 2 years ]
    Liver enzymes (ALT, AST)

  3. Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104 [ Time Frame: 2 years ]
    Fasting serum bile acid

  4. Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104 [ Time Frame: 2 years ]
    C4

  5. Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104 [ Time Frame: 2 years ]
    Quality of life assessments (itch and sleep scores)



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02321306


Locations
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United States, California
Scripps Clinic
La Jolla, California, United States, 92037
University of California at Davis
Sacramento, California, United States, 95817
United States, Florida
University of Miami
Miami, Florida, United States, 33136
United States, Indiana
Indiana University
Indianapolis, Indiana, United States, 46202
United States, Kentucky
University of Louisville
Louisville, Kentucky, United States, 40202
United States, Michigan
Henry Ford Health System
Detroit, Michigan, United States, 48202
United States, Minnesota
Minnesota Gastroenterology
Saint Paul, Minnesota, United States, 55114
United States, Missouri
St. Louis University
Saint Louis, Missouri, United States, 63104
United States, New York
Weill Cornell Medical College
New York, New York, United States, 10021
United States, Texas
University of Texas Southwestern Medical Center
Dallas, Texas, United States, 75390
United States, Virginia
Liver Institute of Virginia
Newport News, Virginia, United States, 23602
Hunter Holmes McGuire VA Medical Center
Richmond, Virginia, United States, 23249
United States, Washington
University of Washington Harborview Medical Center
Seattle, Washington, United States, 98104
United Kingdom
University of Birmingham
Birmingham, England, United Kingdom, B15 2TT
Royal Liverpool & Broadgreen University Hospital
Liverpool, England, United Kingdom, L7 8XP
Newcastle University
Newcastle Upon Tyne, England, United Kingdom, NE1 4LP
Oxford University Hospitals (John Radcliffe)
Oxford, England, United Kingdom, OX3 9DU
Royal Free Hospital
London, United Kingdom, NW3 2QG
Sponsors and Collaborators
Mirum Pharmaceuticals, Inc.
Investigators
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Study Director: Study Director Mirum

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Responsible Party: Mirum Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT02321306     History of Changes
Other Study ID Numbers: LUM001-202
2014-003433-26 ( EudraCT Number )
First Posted: December 22, 2014    Key Record Dates
Last Update Posted: March 19, 2019
Last Verified: March 2019
Additional relevant MeSH terms:
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Liver Cirrhosis
Liver Cirrhosis, Biliary
Fibrosis
Pathologic Processes
Liver Diseases
Digestive System Diseases
Cholestasis, Intrahepatic
Cholestasis
Bile Duct Diseases
Biliary Tract Diseases