Trial · NCT02061540
Open Label Study to Evaluate Safety and Efficacy of LUM001 in Patients With Primary Sclerosing Cholangitis
NCT02061540, Phase 2, Completed, Mirum Pharmaceuticals, Inc.; results first posted 2017-04-12
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Open Label Study to Evaluate Safety and Efficacy of LUM001 in Patients With Primary Sclerosing Cholangitis. Trials, record NCT02061540. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02061540
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Open Label Study to Evaluate Safety and Efficacy of LUM001 in Patients With Primary Sclerosing Cholangitis
- NCT number
- NCT02061540
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 27 participants, actual
- Lead sponsor
- Mirum Pharmaceuticals, Inc.
- Conditions
- Primary Sclerosing Cholangitis (PSC)
- Interventions
- LUM001
- First posted
- 2014-02-13
- Last updated
- 2019-03-29
- Other registries' IDs
- eudract:2014-005558-21
- Results first posted
- 2017-04-12
Source ClinicalTrials.gov
Registered outcomes6
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | 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. |
| Primary | Change From Baseline in Fasting Serum Bile Acid Level at Week 14 | Baseline, Week 14 | Serum bile acid levels were evaluated using blood samples collected. |
| Secondary | Change From Baseline in Liver Enzyme Levels in Serum | Baseline, Week 14 | Levels of liver enzymes such as Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST), Alkaline Phosphatase (ALP) in serum were evaluated. |
| Secondary | Change From Baseline in Bilirubin Levels at Week 14 | Baseline, Week 14 | Total Bilirubin and Direct (Conjugated) Bilirubin levels were evaluated. |
| Secondary | Change From Baseline in Pruritus as Measured by Adult Itch Reported Outcome (ItchRO) Weekly Sum Score | Baseline, Week 14 | 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. |
| Other | Change From Baseline for Other Biochemical Markers of Cholestasis: Total Cholesterol, Low Density Lipoprotein Cholesterol | Baseline, Week 14 | Total cholesterol (TC) level and low density lipoprotein cholesterol (LDLC) level were considered as biochemical markers of cholestasis. |
Source ClinicalTrials.gov
Outcomes6
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | From start of study drug administration until Week 18 | Safety population included all participants who received at least 1 dose of the investigational product. | participant | NUMBER |
| PRIMARY | Change From Baseline in Fasting Serum Bile Acid Level at Week 14 | Baseline, Week 14 | Modified intent-to-treat (mITT) population included all participants who received at least 1 dose of investigational product and had at least 1 post baseline serum bile acid laboratory assessment. | micromoles per liter | MEAN |
| SECONDARY | Change From Baseline in Liver Enzyme Levels in Serum | Baseline, Week 14 | mITT population included all participants who received at least 1 dose of investigational product and had at least 1 post baseline serum bile acid laboratory assessment. | units per liter (U/L) | MEAN |
| SECONDARY | Change From Baseline in Bilirubin Levels at Week 14 | Baseline, Week 14 | mITT population included all participants who received at least 1 dose of investigational product and had at least 1 post baseline serum bile acid laboratory assessment. | milligram per deciliter (mg/dL) | MEAN |
| SECONDARY | Change From Baseline in Pruritus as Measured by Adult Itch Reported Outcome (ItchRO) Weekly Sum Score | Baseline, Week 14 | mITT population included all participants who received at least 1 dose of investigational product and had at least 1 post baseline serum bile acid laboratory assessment. | units on scale | MEAN |
| OTHER_PRE_SPECIFIED | Change From Baseline for Other Biochemical Markers of Cholestasis: Total Cholesterol, Low Density Lipoprotein Cholesterol | Baseline, Week 14 | mITT population included all participants who received at least 1 dose of investigational product and had at least 1 post baseline serum bile acid laboratory assessment. | mg/dL | MEAN |
Source ClinicalTrials.gov
Results by group23
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Maralixibat (LUM001) 1 mg | 1 | participant | ||
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Maralixibat (LUM001) 2.5 mg | 2 | participant | ||
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Maralixibat (LUM001) 5 mg | 2 | participant | ||
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Maralixibat (LUM001) 7.5 mg | 1 | participant | ||
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Maralixibat (LUM001) 10 mg | 19 | participant | ||
| Change From Baseline in Fasting Serum Bile Acid Level at Week 14 | Maralixibat (LUM001) | Baseline | 38.941 | Standard Deviation 38.6614 | micromoles per liter |
| Change From Baseline in Fasting Serum Bile Acid Level at Week 14 | Maralixibat (LUM001) | Change From Baseline | -14.841 | Standard Deviation 31.3709 | micromoles per liter |
| Change From Baseline in Liver Enzyme Levels in Serum | Maralixibat (LUM001) | ALT: Baseline | 108.5 | Standard Deviation 78.95 | units per liter (U/L) |
| Change From Baseline in Liver Enzyme Levels in Serum | Maralixibat (LUM001) | ALT: Change From Baseline | 10.5 | Standard Deviation 63.07 | units per liter (U/L) |
| Change From Baseline in Liver Enzyme Levels in Serum | Maralixibat (LUM001) | AST: Baseline | 88.3 | Standard Deviation 43.70 | units per liter (U/L) |
1 to 10 of 23Source ClinicalTrials.gov
See all 23 results by groupParticipant flow4
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Maralixibat (LUM001) | 27 | |
| Overall Study | Down-Titrated on LUM001 During the Study | Maralixibat (LUM001) | 5 | |
| Overall Study | COMPLETED | Maralixibat (LUM001) | 23 | |
| Overall Study | NOT COMPLETED | Maralixibat (LUM001) | 4 |
Source ClinicalTrials.gov
Adverse events22
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Arthralgia | Musculoskeletal and connective tissue disorders | Maralixibat (LUM001) | 3 | 27 | 9 |
| other | Muscle spasms | Musculoskeletal and connective tissue disorders | Maralixibat (LUM001) | 2 | 27 | 2 |
| other | Neck pain | Musculoskeletal and connective tissue disorders | Maralixibat (LUM001) | 2 | 27 | 2 |
| other | Abdominal discomfort | Gastrointestinal disorders | Maralixibat (LUM001) | 3 | 27 | 4 |
| other | Abdominal distension | Gastrointestinal disorders | Maralixibat (LUM001) | 4 | 27 | 5 |
| other | Abdominal pain | Gastrointestinal disorders | Maralixibat (LUM001) | 8 | 27 | 8 |
| other | Abdominal pain upper | Gastrointestinal disorders | Maralixibat (LUM001) | 2 | 27 | 4 |
| other | Diarrhoea | Gastrointestinal disorders | Maralixibat (LUM001) | 14 | 27 | 27 |
| other | Frequent bowel movements | Gastrointestinal disorders | Maralixibat (LUM001) | 2 | 27 | 2 |
| other | Nausea | Gastrointestinal disorders | Maralixibat (LUM001) | 9 | 27 | 12 |
1 to 10 of 22Source ClinicalTrials.gov
See all 22 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Maralixibat (LUM001) | ||
| Total, other adverse events | Maralixibat (LUM001) | 25 | 27 |
| Total, serious adverse events | Maralixibat (LUM001) | 4 | 27 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 37184523 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02061540
GET https://conductscience.org/api/trials/v1/NCT02061540/results?page=2