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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)

Primary Purpose

Primary Biliary Cirrhosis

Status
Withdrawn
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
LUM001
Sponsored by
Mirum Pharmaceuticals, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Primary Biliary Cirrhosis

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Scripps Clinic
  • University of California at Davis
  • University of Miami
  • Indiana University
  • University of Louisville
  • Henry Ford Health System
  • Minnesota Gastroenterology
  • St. Louis University
  • Weill Cornell Medical College
  • University of Texas Southwestern Medical Center
  • Liver Institute of Virginia
  • Hunter Holmes McGuire VA Medical Center
  • University of Washington Harborview Medical Center
  • University of Birmingham
  • Royal Liverpool & Broadgreen University Hospital
  • Newcastle University
  • Oxford University Hospitals (John Radcliffe)
  • Royal Free Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

LUM001

Arm Description

LUM001 administered orally once each day.

Outcomes

Primary Outcome Measures

Number of Participants with Adverse Events as a Measure of Safety
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

Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104
Alkaline phosphatase
Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104
Liver enzymes (ALT, AST)
Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104
Fasting serum bile acid
Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104
C4
Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104
Quality of life assessments (itch and sleep scores)

Full Information

First Posted
December 12, 2014
Last Updated
March 15, 2019
Sponsor
Mirum Pharmaceuticals, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT02321306
Brief Title
An Open-label Study to Evaluate the Long-term Safety and Tolerability of LUM001 in Patients With Primary Biliary Cirrhosis
Acronym
CASCADE
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 Type
Interventional

2. Study Status

Record Verification Date
March 2019
Overall Recruitment Status
Withdrawn
Why Stopped
The study stopped early, before enrolling its first participant.
Study Start Date
May 2015 (undefined)
Primary Completion Date
December 2017 (Anticipated)
Study Completion Date
March 2018 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Mirum Pharmaceuticals, Inc.

4. Oversight

Data Monitoring Committee
No

5. Study Description

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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Primary Biliary Cirrhosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
0 (Actual)

8. Arms, Groups, and Interventions

Arm Title
LUM001
Arm Type
Experimental
Arm Description
LUM001 administered orally once each day.
Intervention Type
Drug
Intervention Name(s)
LUM001
Intervention Description
LUM001 administered orally once each day.
Primary Outcome Measure Information:
Title
Number of Participants with Adverse Events as a Measure of Safety
Description
Adverse events, serious adverse events, changes in vital signs, laboratory and physical exam findings, and concommitant medication usage from baseline to Week 104
Time Frame
2 years
Secondary Outcome Measure Information:
Title
Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104
Description
Alkaline phosphatase
Time Frame
2 years
Title
Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104
Description
Liver enzymes (ALT, AST)
Time Frame
2 years
Title
Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104
Description
Fasting serum bile acid
Time Frame
2 years
Title
Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104
Description
C4
Time Frame
2 years
Title
Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104
Description
Quality of life assessments (itch and sleep scores)
Time Frame
2 years

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Completed a core treatment protocol of LUM001 in the treatment of Primary Biliary Cirrhosis. Ability to understand and willingness to sign informed consent/assent prior to initiation of any study procedures. Exclusion Criteria: Presence of advanced liver disease. History of non-adherence during the subject's participation in the core LUM001 treatment protocol.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Study Director
Organizational Affiliation
Mirum
Official's Role
Study Director
Facility Information:
Facility Name
Scripps Clinic
City
La Jolla
State/Province
California
ZIP/Postal Code
92037
Country
United States
Facility Name
University of California at Davis
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
University of Miami
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
Indiana University
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Facility Name
University of Louisville
City
Louisville
State/Province
Kentucky
ZIP/Postal Code
40202
Country
United States
Facility Name
Henry Ford Health System
City
Detroit
State/Province
Michigan
ZIP/Postal Code
48202
Country
United States
Facility Name
Minnesota Gastroenterology
City
Saint Paul
State/Province
Minnesota
ZIP/Postal Code
55114
Country
United States
Facility Name
St. Louis University
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63104
Country
United States
Facility Name
Weill Cornell Medical College
City
New York
State/Province
New York
ZIP/Postal Code
10021
Country
United States
Facility Name
University of Texas Southwestern Medical Center
City
Dallas
State/Province
Texas
ZIP/Postal Code
75390
Country
United States
Facility Name
Liver Institute of Virginia
City
Newport News
State/Province
Virginia
ZIP/Postal Code
23602
Country
United States
Facility Name
Hunter Holmes McGuire VA Medical Center
City
Richmond
State/Province
Virginia
ZIP/Postal Code
23249
Country
United States
Facility Name
University of Washington Harborview Medical Center
City
Seattle
State/Province
Washington
ZIP/Postal Code
98104
Country
United States
Facility Name
University of Birmingham
City
Birmingham
State/Province
England
ZIP/Postal Code
B15 2TT
Country
United Kingdom
Facility Name
Royal Liverpool & Broadgreen University Hospital
City
Liverpool
State/Province
England
ZIP/Postal Code
L7 8XP
Country
United Kingdom
Facility Name
Newcastle University
City
Newcastle Upon Tyne
State/Province
England
ZIP/Postal Code
NE1 4LP
Country
United Kingdom
Facility Name
Oxford University Hospitals (John Radcliffe)
City
Oxford
State/Province
England
ZIP/Postal Code
OX3 9DU
Country
United Kingdom
Facility Name
Royal Free Hospital
City
London
ZIP/Postal Code
NW3 2QG
Country
United Kingdom

12. IPD Sharing Statement

Learn more about this trial

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

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