PBCPhase 2CLARITYNCT01904058

Phase 2 Study to Evaluate LUM001 in Combination With Ursodeoxycholic Acid in Patients With Primary Biliary Cirrhosis

A Phase 2 study testing LUM001 and Ursodeoxycholic Acid in people with PBC and Primary Biliary Cirrhosis, run by Mirum Pharmaceuticals, Inc. at 24 sites in 3 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial 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. Read more about this study

Who is it for?: Ages 18 to 80 years
Conditions the study is for: PBC, Primary Biliary Cirrhosis
What is being tested?: LUM001 and Ursodeoxycholic Acid
Type of treatment: Pill or oral medicine
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 66 participants
Where: 24 sitesin 3 countries
Study dates: Aug 2013to Apr 2015
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

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 to 80 years
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

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

Who cannot join

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

Trial locations and contacts

24 sites in 3 countries.

Country

City, postcode or hospital

24 sites in 3 countries

United States17 sites

University of Chicago Medical Center
Chicago, Illinois, 60637
University of Texas Southwestern Medical Center
Dallas, Texas, 75390
Henry Ford Health System
Detroit, Michigan, 48202
Advanced Liver Therapies at St. Lukes Episcopal Hospital
Houston, Texas, 77030
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
Mayo Clinic
Rochester, Minnesota, 55905
University of California at Davis
Sacramento, California, 95817
Minnesota Gastroenterology
Saint Paul, Minnesota, 55114
University of Utah Health Science Center
Salt Lake City, Utah, 84132
University of Washington Harborview Medical Center
Seattle, Washington, 98104
St. Louis University
St Louis, Missouri, 63104

Other trials in United States

United Kingdom6 sites

University of Birmingham
Birmingham, England, B15 2TT
Royal Liverpool & Broadgreen University Hospital
Liverpool, England, L7 8XP
Royal Free Hospital
London, NW3 2QG
Imperial College London St Mary's Hospital
London, W2 1NY
Newcastle University
Newcastle upon Tyne, England, NE1 4LP
Oxford University Hospitals (John Radcliffe)
Oxford, England, OX3 9DU

Other trials in United Kingdom

Canada1 site

University Health Network, Toronto Western Hospital
Toronto, Ontario, M5T 2S8

Other trials in Canada

About this study

Official title: A Phase 2, Randomized, Double-blind, Placebo-controlled Study to Evaluate LUM001, an Apical Sodium-dependent Bile Acid Transporter Inhibitor (ASBTi) in Combination With Ursodeoxycholic Acid (UDCA) in Patients With Primary Biliary Cirrhosis

Conditions: PBC, Primary Biliary Cirrhosis
Being tested: LUM001, Placebo, Ursodeoxycholic Acid
Keywords: PBC
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
PlaceboDrug
Ursodeoxycholic AcidDrug
Also called UDCA

Study groups in NCT01904058
Study group	Role	What they receive
LUM001 and Ursodeoxycholic Acid (UDCA)	Experimental	Administered orally once daily Receives: Drug: LUM001; Drug: Ursodeoxycholic Acid
Placebo and Ursodeoxycholic Acid (UDCA)	Placebo comparator	Administered orally once daily Receives: Drug: Placebo; Drug: Ursodeoxycholic Acid

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: 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
ItchRO scores had a range 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.
Measured: 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)
ItchRO scores had a range 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. Adult ItchRO average daily score was the sum of daily scores divided by the number of days adult ItchRO was completed, using the 7 days prior to the reported visit date.
Measured: 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)
Laboratory serum ALP enzyme levels were evaluated using blood samples collected.
Measured: 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)
The 5-D itch (validated instrument to measure pruritus) scale was developed for the multidimensional quantification of pruritus that is sensitive to change over time. The 5-D itch scale included 5 domains (duration, degree, direction, disability, and distribution of pruritus). The total 5-D score was obtained by scoring each of the domains separately and then summing them together. 5-D total scores ranged between 5 (no pruritus) and 25 (most severe pruritus).
Measured: 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)
Laboratory serum bile acid level levels were evaluated using blood samples collected.
Measured: Baseline, Weeks 4, 8, 13 and Last Post-baseline visit (Week 13/ET)
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)
C4 7 alpha-hydroxy-4-cholesten-3-one is an intermediate in the biochemical synthesis of bile acids from cholesterol and its concentrations reflect the activity of the bile acid synthetic pathway. Elevated levels of C4 indicate bile acid malabsorption. Laboratory C4 levels were evaluated using blood samples collected.
Measured: Baseline, Weeks 4, 8, 13 and Last Post-baseline Visit (Week 13/ET)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Aug 2013Apr 2015

First posted22 Jul 2013 (estimated)
Study startAug 2013
Primary completionApr 2015
Study completionApr 2015

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for PBCStudies 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: Randomised
Intervention model: Parallel groups
Masking: Double blind
Who is masked: Participant, Investigator
Enrolment: 66 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01904058
Sponsor's protocol ID: LUM001-201
Other IDs: 2013-000482-36
Acronym: CLARITY
Status last verified: Mar 2019
First submitted: 17 Jul 2013
Last updated: 27 Mar 2019
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

Enrolling by invitationPhase 2/3
A Multicenter, Randomized, Controlled Trial of Prednisone Combined With Ursodeoxycholic Acid in the Treatment of Primary Biliary Cholangitis With Moderate to Severe Interface Hepatitis Characteristics
Primary Biliary Cholangitis (PBC)
RecruitingPhase 3
A Long-Term Study of Elafibranor in Adult Participants With Primary Biliary Cholangitis
Primary Biliary Cholangitis (PBC)
Active, not recruitingPhase 3
Evaluation of Maralixibat in Pruritus Associated With General Cholestatic Liver Disease (EXPAND)
Cholestatic Liver Disease (Except ALGS, PFIC, PBC and PSC)
Active, not recruitingObservational study
Epidemiology of PrImary Biliary Cholangitis in Spain
PBC, Primary Biliary Cholangitis (PBC)
Active, not recruitingPhase 2
A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Biliary Cholangitis
Primary Biliary Cholangitis, PBC

All PBC trials

Study at a glance

From the registry record, last updated by the sponsor on 27 Mar 2019. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 2, Randomized, Double-blind, Placebo-controlled Study to Evaluate LUM001, an Apical Sodium-dependent Bile Acid Transporter Inhibitor (ASBTi) in Combination With Ursodeoxycholic Acid (UDCA) in Patients With Primary Biliary Cirrhosis
ClinicalTrials.gov ID: NCT01904058
Acronym / sponsor ID: CLARITY / LUM001-201
Conditions: PBC, Primary Biliary Cirrhosis
Keywords: PBC
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 66 participants (actual)
Lead sponsor: Mirum Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
Study start: Aug 2013
Primary completion: Apr 2015 (actual)
Study completion: Apr 2015 (actual)
First posted: 22 Jul 2013 (estimated)
Results: Posted 6 Jul 2016

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Double blind (participant, investigator)
Number of arms: 2

Arm	Type	Receives
LUM001 and Ursodeoxycholic Acid (UDCA)	Experimental	Drug: LUM001, Drug: Ursodeoxycholic AcidAdministered orally once daily
Placebo and Ursodeoxycholic Acid (UDCA)	Placebo comparator	Drug: Placebo, Drug: Ursodeoxycholic AcidAdministered orally once daily

Who may qualify

Ages: 18 to 80 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (4 listed)

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

Key exclusion criteria (4 listed)

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

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
LUM001	Drug	LUM001 and Ursodeoxycholic Acid (UDCA)
Placebo	Drug	Placebo and Ursodeoxycholic Acid (UDCA)
Ursodeoxycholic AcidAlso: UDCA	Drug	LUM001 and Ursodeoxycholic Acid (UDCA), Placebo and Ursodeoxycholic Acid (UDCA)

Primary outcome

Change From Baseline in Pruritus Using Adult Itch Reported Outcome (ItchRO) Weekly Sum Score at Week 13/ Early Termination (ET)
Time frame: Baseline and Week 13/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.

6 secondary outcomes

Change From Baseline in Pruritus Using Adult ItchRO Weekly Sum Scores at Weeks 4, 8 and 13
Time frame: 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)
Time frame: 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)
Time frame: 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)
Time frame: 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)
Time frame: Baseline, Weeks 4, 8, 13 and Last Post-baseline visit (Week 13/ET)
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)
Time frame: Baseline, Weeks 4, 8, 13 and Last Post-baseline Visit (Week 13/ET)

Results and safety

Results posted to the registry on 6 Jul 2016, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
LUM001 10 mg + UDCA (Cohort A)	21	18
LUM001 20 mg + UDCA (Cohort B)	21	21
Placebo + UDCA (Cohort A)	11	10
Placebo + UDCA (Cohort B)	13	12

Primary outcome: Change From Baseline in Pruritus Using Adult Itch Reported Outcome (ItchRO) Weekly Sum Score at Week 13/ Early Termination (ET)

Time frame: Baseline and Week 13/ET. Mean (Standard Deviation). Unit: units on a scale

Measure	LUM001 10 mg + UDCA (Cohort A n = 21	LUM001 20 mg + UDCA (Cohort B) n = 21	Placebo + UDCA (Cohort A) n = 11	Placebo + UDCA (Cohort B) n = 13
Baseline	48.11 (13.363)	52.10 (13.780)	54.64 (9.157)	49.46 (14.110)
Change at Week 13/ET	-24.59 (15.240)	-27.67 (19.888)	-26.18 (18.313)	-22.77 (17.123)

LUM001 10 mg + UDCA (Cohort A vs Placebo + UDCA (Cohort A) vs Placebo + UDCA (Cohort B): Least squares mean difference: -2.28 (95% CI -12.59 to 8.03), p = 0.6603, ANCOVA
LUM001 20 mg + UDCA (Cohort B) vs Placebo + UDCA (Cohort A) vs Placebo + UDCA (Cohort B): Least squares mean difference: -3.99 (95% CI -14.20 to 6.23), p = 0.4380, ANCOVA

Population analysed: The mITT population included all participants who were randomized, received at least 1 dose of treatment, and had at least 1 post-baseline ItchRO assessment.

6 secondary outcomes are reported in the full results.

Adverse events

Time frame: From the first dose of study drug until the 13 weeks of treatment period (or ET) + 14 days (approximately 15 weeks). Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
LUM001 5 mg + UDCA	0 of 1 (0%)	1 of 1 (100%)	Not reported
LUM001 10 mg + UDCA	2 of 20 (10%)	18 of 20 (90%)	Not reported
LUM001 20 mg + UDCA	1 of 21 (4.8%)	20 of 21 (95.2%)	Not reported
Placebo + UDCA	0 of 24 (0%)	16 of 24 (66.7%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 27 Mar 2019.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related InfoLink provided by the sponsor.

Related trials for PBC

Enrolling by invitationPhase 2/3
A Multicenter, Randomized, Controlled Trial of Prednisone Combined With Ursodeoxycholic Acid in the Treatment of Primary Biliary Cholangitis With Moderate to Severe Interface Hepatitis Characteristics
Primary Biliary Cholangitis (PBC)
RecruitingPhase 3
A Long-Term Study of Elafibranor in Adult Participants With Primary Biliary Cholangitis
Primary Biliary Cholangitis (PBC)
Active, not recruitingPhase 3
Evaluation of Maralixibat in Pruritus Associated With General Cholestatic Liver Disease (EXPAND)
Cholestatic Liver Disease (Except ALGS, PFIC, PBC and PSC)
Active, not recruitingObservational study
Epidemiology of PrImary Biliary Cholangitis in Spain
PBC, Primary Biliary Cholangitis (PBC)
Active, not recruitingPhase 2
A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Biliary Cholangitis
Primary Biliary Cholangitis, PBC

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

24 sites in 3 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing LUM001 and Ursodeoxycholic Acid in people with PBC and Primary Biliary Cirrhosis, run by Mirum Pharmaceuticals, Inc. at 24 sites in 3 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 4 inclusion and 4 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: finished, as of the sponsor's last update on 27 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 24 sites in United States, United Kingdom, Canada. 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. Some participants in this study may receive a placebo. 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?

Yes. Results are posted in the registry (first posted 6 Jul 2016). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Change From Baseline in Pruritus Using Adult Itch Reported Outcome (ItchRO) Weekly Sum Score at Week 13/ Early Termination (ET) (time frame: Baseline and Week 13/ET). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01904058.

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 27 Mar 2019; AzTrials last refreshed its copy on 30 Oct 2026.