Primary Sclerosing Cholangitis (PSC)Phase 2CAMEONCT02061540
Open Label Study to Evaluate Safety and Efficacy of LUM001 in Patients With Primary Sclerosing Cholangitis
A Phase 2 study testing LUM001 in people with Primary Sclerosing Cholangitis (PSC), run by Mirum Pharmaceuticals, Inc. at 9 sites in 3 countries.
Trial summary
The study is an open-label study in adults with primary sclerosing cholangitis to evaluate the safety, tolerability, and effect of 14-weeks of daily dosing of LUM001. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Primary Sclerosing Cholangitis (PSC)
- 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
- 27 participants
- Where
- 9 sitesin 3 countries
- Study dates
- Mar 2014to Feb 2016
- 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
- Male or female subjects between the ages of 18-80 years, inclusive.
- Diagnosis of PSC
- If inflammatory bowel disease (IBD) is present, disease activity ≤ 2 (normal to moderate), using the physician assessment on the Mayo ulcerative colitis (UC) disease activity score.
- Patients receiving azathioprine for intestinal bowel disease are eligible to participate in the study provided that they have had no IBD exacerbations for at least 6 months.
- Females of childbearing potential must have a negative serum pregnancy test [β human chorionic gonadotropin (β-hCG)] during screening and negative urine pregnancy test at the baseline/Day 0 visit.
- Sexually active females must be postmenopausal, surgically sterile, or if premenopausal, be prepared to use an effective method (≤ 1% failure rate) of contraception during the trial.
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- Ability to read and understand English in order to use the study-related questionnaires and the text on the eDiary screen.
- Must be willing and able to use an eDiary daily for a minimum of 20 weeks.
- Must digitally accept the licensing agreement in the eDiary software at the outset of the study.
- Must complete at least 10 eDiary Adult ItchRO reports (AM or PM) during each of two consecutive weeks of the screening period prior to allocation to treatment (maximum possible reports = 14 per week).
- Access to phone for scheduled calls from study site.
- Must agree to comply with the study protocol procedures and provide written informed consent.
Who cannot join
- Small duct PSC (clinical biochemical and histological features compatible with PSC, but having a normal cholangiogram).
- Presence of a dominant stricture unless brushings and/or biopsies of the stricture are negative for dysplasia or malignancy within 6 months of screening.
- Surgical or endoscopic biliary tree interventions for treatment of clinically significant strictures within 6 months of screening.
- IBD flare (Mayo UC disease activity score > 5 including endoscopic evaluation) within 3 months prior to screening.
- Secondary cause of sclerosing cholangitis (e.g., choledocholithiasis, post-surgical biliary stricture, intra-arterial chemotherapy, recurrent pancreatitis, IgG4 associated cholangiopathy, AIDS cholangiopathy).
- AST or ALT ≥ 5 x ULN at screening.
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- History or presence of any other concomitant significant liver disease as assessed by the Investigator.
- Medical conditions that may cause nonhepatic increases in ALP (e.g., Paget's disease).
- Known history of human immunodeficiency virus (HIV) infection.
- The anticipated need for a surgical procedure within 20 weeks from randomization.
- Any female who is pregnant or lactating or who is planning to become pregnant within 20 weeks of randomization.
- History of cancer, except for basal or squamous cell carcinoma of the skin, or with any laboratory or physical exam or diagnostic procedure finding suggestive of current malignancy.
- Family history of any documented hereditary cancer syndrome.
- History of alcohol or other substance abuse within 1 year prior to screening.
- Receipt of an investigational drug, biologic, or medical device within 30 days prior to Screening, or 5 half-lives of the study agent, whichever is longer.
- History of noncompliance with medical regimens, unreliability, mental instability or incompetence that could compromise the validity of informed consent or lead to noncompliance with the study protocol.
- Any other conditions or abnormalities which, in the opinion of the Investigator or Medical Monitor, may compromise the safety of the subject, or interfere with the subject participating in or completing the study.
Trial locations and contacts
9 sites in 3 countries.
9 sites in 3 countries
United States5 sites
University of Colorado
Aurora, Colorado, 80045
Duke University
Durham, North Carolina, 27710
Scripps Clinic
La Jolla, California, 92037
University of Miami
Miami, Florida, 33136
University of California at Davis
Sacramento, California, 95817
Canada2 sites
University of Calgary Liver Unit
Calgary, Alberta, T2N 4Z6
University Health Network, Toronto Western Hospital
Toronto, Ontario, M5T 2S8
United Kingdom2 sites
University of Birmingham
Birmingham, England, B15 2TT
Royal Free Hospital
London, NW3 2QG
About this study
Official title: A Pilot, Open-Label Study to Evaluate the Safety, Tolerability and Efficacy of LUM001, an Apical Sodium-dependent Bile Acid Transporter Inhibitor (ASBTi), in Patients With Primary Sclerosing Cholangitis
The study is an open-label study in adults with primary sclerosing cholangitis to evaluate the safety, tolerability, and effect of 14-weeks of daily dosing of LUM001.
- Conditions
- Primary Sclerosing Cholangitis (PSC)
- Being tested
- LUM001
- Medical subject headings
- Cholangitis, Sclerosing
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LUM001Drug
LUM001 oral dose
| 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 Treatment-Emergent Adverse Events (TEAEs)
An Adverse Event (AE) was defined as any unfavorable and unintended sign (including a clinically significant abnormal laboratory finding, for example), symptom, or disease temporally associated with the study or use of investigational drug product, whether or not the AE was considered to be related to the investigational drug product. TEAEs were AEs with a start date on or after the first dose of investigational product and started prior to the last dose of investigational product plus 14 days.
Measured: From start of study drug administration until Week 18
Change From Baseline in Fasting Serum Bile Acid Level at Week 14
Serum bile acid levels were evaluated using blood samples collected.
Measured: Baseline, Week 14
Secondary outcomes (3)
Change From Baseline in Liver Enzyme Levels in Serum
Levels of liver enzymes such as Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST), Alkaline Phosphatase (ALP) in serum were evaluated.
Measured: Baseline, Week 14
Change From Baseline in Bilirubin Levels at Week 14
Total Bilirubin and Direct (Conjugated) Bilirubin levels were evaluated.
Measured: Baseline, Week 14
Change From Baseline in Pruritus as Measured by Adult Itch Reported Outcome (ItchRO) Weekly Sum Score
The Adult ItchRO instrument was completed twice daily using an electronic diary (eDiary). Each morning and evening score had a range from 0-10, with the higher score indicating increasing itch severity. The following was used for assessing the Adult ItchRO daily score: The score which represented the most severe itching for the day (morning or evening) was taken for each day as the daily score (maximum daily score of 10); If only 1 of the 2 scores was available for the day, the score that was available was used as the daily score; If both the morning and the evening scores were missing, the score was considered missing for the day.
Measured: Baseline, Week 14
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Feb 2014 (estimated)
- Study startMar 2014
- Primary completionFeb 2016
- Study completionFeb 2016
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.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Primary Sclerosing Cholangitis (PSC)Studies of the same condition, open and under way.
- More trials run by Mirum Pharmaceuticals, Inc.The sponsor's other registered studies.
- 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
- 27 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
- NCT02061540
- Sponsor's protocol ID
- LUM001-401
- Other IDs
- 2014-005558-21, 2014
- Acronym
- CAMEO
- Status last verified
- Mar 2019
- First submitted
- 11 Feb 2014
- Last updated
- 29 Mar 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Bowlus CL, Eksteen B, Cheung AC, Thorburn D, Moylan CA, Pockros PJ, Forman LM, Dorenbaum A, Hirschfield GM, Kennedy C, Jaecklin T, McKibben A, Chien E, Baek M, Vig P, Levy C. Safety, tolerability, and efficacy of maralixibat in adults with primary sclerosing cholangitis: Open-label pilot study. Hepatol Commun. 2023 May 15;7(6):e0153. doi: 10.1097/HC9.0000000000000153. eCollection 2023 Jun 1.
PubMed 37184523
Study at a glance
From the registry record, last updated by the sponsor on 29 Mar 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot, Open-Label Study to Evaluate the Safety, Tolerability and Efficacy of LUM001, an Apical Sodium-dependent Bile Acid Transporter Inhibitor (ASBTi), in Patients With Primary Sclerosing Cholangitis
- ClinicalTrials.gov ID
- NCT02061540
- Acronym / sponsor ID
- CAMEO / LUM001-401
- Conditions
- Primary Sclerosing Cholangitis (PSC)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 27 participants (actual)
- Lead sponsor
- Mirum Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- Mar 2014
- Primary completion
- Feb 2016 (actual)
- Study completion
- Feb 2016 (actual)
- First posted
- 13 Feb 2014 (estimated)
- Results
- Posted 12 Apr 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The study is an open-label study in adults with primary sclerosing cholangitis to evaluate the safety, tolerability, and effect of 14-weeks of daily dosing of LUM001.
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 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- Male or female subjects between the ages of 18-80 years, inclusive.
- Diagnosis of PSC
- If inflammatory bowel disease (IBD) is present, disease activity ≤ 2 (normal to moderate), using the physician assessment on the Mayo ulcerative colitis (UC) disease activity score.
- Patients receiving azathioprine for intestinal bowel disease are eligible to participate in the study provided that they have had no IBD exacerbations for at least 6 months.
- Females of childbearing potential must have a negative serum pregnancy test [β human chorionic gonadotropin (β-hCG)] during screening and negative urine pregnancy test at the baseline/Day 0 visit.
Key exclusion criteria (17 listed)
- Small duct PSC (clinical biochemical and histological features compatible with PSC, but having a normal cholangiogram).
- Presence of a dominant stricture unless brushings and/or biopsies of the stricture are negative for dysplasia or malignancy within 6 months of screening.
- Surgical or endoscopic biliary tree interventions for treatment of clinically significant strictures within 6 months of screening.
- IBD flare (Mayo UC disease activity score > 5 including endoscopic evaluation) within 3 months prior to screening.
- Secondary cause of sclerosing cholangitis (e.g., choledocholithiasis, post-surgical biliary stricture, intra-arterial chemotherapy, recurrent pancreatitis, IgG4 associated cholangiopathy, AIDS cholangiopathy).
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 oral dose | Drug | LUM001 |
Primary outcome
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Time frame: From start of study drug administration until Week 18
An Adverse Event (AE) was defined as any unfavorable and unintended sign (including a clinically significant abnormal laboratory finding, for example), symptom, or disease temporally associated with the study or use of investigational drug product, whether or not the AE was considered to be related to the investigational drug product. TEAEs were AEs with a start date on or after the first dose of investigational product and started prior to the last dose of investigational product plus 14 days.
Change From Baseline in Fasting Serum Bile Acid Level at Week 14
Time frame: Baseline, Week 14
Serum bile acid levels were evaluated using blood samples collected.
3 secondary outcomes
Change From Baseline in Liver Enzyme Levels in Serum
Time frame: Baseline, Week 14
Change From Baseline in Bilirubin Levels at Week 14
Time frame: Baseline, Week 14
Change From Baseline in Pruritus as Measured by Adult Itch Reported Outcome (ItchRO) Weekly Sum Score
Time frame: Baseline, Week 14
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Mar 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 37184523Bowlus CL, Eksteen B, Cheung AC, Thorburn D, Moylan CA, Pockros PJ, Forman LM, Dorenbaum A, Hirschfield GM, Kennedy C, Jaecklin T, McKibben A, Chien E, Baek M, Vig P, Levy C. Safety, tolerability, and efficacy of maralixibat in adults with primary sclerosing cholangitis: Open-label pilot study. Hepatol Commun. 2023 May 15;7(6):e0153. doi: 10.1097/HC9.0000000000000153. eCollection 2023 Jun 1.
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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
9 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 in people with Primary Sclerosing Cholangitis (PSC), run by Mirum Pharmaceuticals, Inc. at 9 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 12 inclusion and 17 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 29 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 9 sites in United States, Canada, 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?
Yes. Results are posted in the registry (first posted 12 Apr 2017). 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?
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) (time frame: From start of study drug administration until Week 18). The study lists 2 primary outcomes in all. 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 NCT02061540, which also lists 1 linked publication.
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 29 Mar 2019.