Primary Biliary CirrhosisPhase 2CASCADENCT02321306
An Open-label Study to Evaluate the Long-term Safety and Tolerability of LUM001 in Patients With Primary Biliary Cirrhosis
A Phase 2 study testing LUM001 in people with Primary Biliary Cirrhosis, run by Mirum Pharmaceuticals, Inc. at 18 sites in 2 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Primary Biliary Cirrhosis
- What is being tested?
- LUM001
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 18 sitesin 2 countries
- Study dates
- May 2015to Mar 2018
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- Presence of advanced liver disease.
- History of non-adherence during the subject's participation in the core LUM001 treatment protocol.
Trial locations and contacts
18 sites in 2 countries.
18 sites in 2 countries
United States13 sites
University of Texas Southwestern Medical Center
Dallas, Texas, 75390
Henry Ford Health System
Detroit, Michigan, 48202
Indiana University
Indianapolis, Indiana, 46202
Scripps Clinic
La Jolla, California, 92037
University of Louisville
Louisville, Kentucky, 40202
University of Miami
Miami, Florida, 33136
Weill Cornell Medical College
New York, New York, 10021
Liver Institute of Virginia
Newport News, Virginia, 23602
Hunter Holmes McGuire VA Medical Center
Richmond, Virginia, 23249
University of California at Davis
Sacramento, California, 95817
Minnesota Gastroenterology
Saint Paul, Minnesota, 55114
University of Washington Harborview Medical Center
Seattle, Washington, 98104
St. Louis University
St Louis, Missouri, 63104
United Kingdom5 sites
University of Birmingham
Birmingham, England, B15 2TT
Royal Liverpool & Broadgreen University Hospital
Liverpool, England, L7 8XP
Royal Free Hospital
London, NW3 2QG
Newcastle University
Newcastle upon Tyne, England, NE1 4LP
Oxford University Hospitals (John Radcliffe)
Oxford, England, OX3 9DU
About this study
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
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.
Read the 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.
- Conditions
- Primary Biliary Cirrhosis
- Being tested
- LUM001
- Medical subject headings
- Liver Cirrhosis, Biliary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LUM001Drug
LUM001 administered orally once each day.
| Study group | Role | What they receive |
|---|---|---|
| LUM001 | Experimental | LUM001 administered orally once each day. Receives: Drug: LUM001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 2 years
Secondary outcomes (5)
Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104
Alkaline phosphatase
Measured: 2 years
Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104
Liver enzymes (ALT, AST)
Measured: 2 years
Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104
Fasting serum bile acid
Measured: 2 years
Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104
C4
Measured: 2 years
Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104
Quality of life assessments (itch and sleep scores)
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Dec 2014 (estimated)
- Study startMay 2015
- Primary completionDec 2017 (estimated)
- Study completionMar 2018 (estimated)
Why it stopped: The study stopped early, before enrolling its first participant.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Biliary CirrhosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mirum Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Mirum
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02321306
- Sponsor's protocol ID
- LUM001-202
- Other IDs
- 2014-003433-26, 2014
- Acronym
- CASCADE
- Status last verified
- Mar 2019
- First submitted
- 12 Dec 2014
- Last updated
- 19 Mar 2019
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Mar 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02321306
- Acronym / sponsor ID
- CASCADE / LUM001-202
- Conditions
- Primary Biliary Cirrhosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: The study stopped early, before enrolling its first participant.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Mirum Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- May 2015
- Primary completion
- Dec 2017 (estimated)
- Study completion
- Mar 2018 (estimated)
- First posted
- 22 Dec 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| LUM001 | Experimental | Drug: LUM001LUM001 administered orally once each day. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- 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.
Key exclusion criteria (2 listed)
- Presence of advanced liver disease.
- History of non-adherence during the subject's participation in the core LUM001 treatment protocol.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| LUM001LUM001 administered orally once each day. | Drug | LUM001 |
Primary outcome
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
5 secondary outcomes
Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104
Time frame: 2 years
Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104
Time frame: 2 years
Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104
Time frame: 2 years
Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104
Time frame: 2 years
Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104
Time frame: 2 years
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Mar 2019.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Study Director study director
Mirum
18 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing LUM001 in people with Primary Biliary Cirrhosis, run by Mirum Pharmaceuticals, Inc. at 18 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 19 Mar 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 18 sites in United States, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants with Adverse Events as a Measure of Safety (time frame: 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Mar 2019.