Trial · NCT02047318
An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Subjects With Alagille Syndrome (ALGS)
NCT02047318, Phase 2, Completed, Mirum Pharmaceuticals, Inc.; results first posted 2021-11-19
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Subjects With Alagille Syndrome (ALGS). Trials, record NCT02047318. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02047318
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Subjects With Alagille Syndrome (ALGS)
- NCT number
- NCT02047318
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 19 participants, actual
- Lead sponsor
- Mirum Pharmaceuticals, Inc.
- Conditions
- Alagille Syndrome
- Interventions
- LUM001 (Maralixibat)
- First posted
- 2014-01-28
- Last updated
- 2021-11-19
- Other registries' IDs
- eudract:2013-003832-54
- Results first posted
- 2021-11-19
- Limitations and caveats
- Limitations of the trial such as small numbers of subjects analyzed or technical problems leading to unreliable data.
Source ClinicalTrials.gov
Registered outcomes16
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From MRX Baseline to Week 48 in Fasting sBA Levels | MRX baseline to Week 48 | The primary endpoint of this study was the mean change from MRX baseline to Week 48 in fasting sBA level. |
| Secondary | Change From MRX Baseline Over Time in Fasting sBA Levels | MRX baseline to End of Treatment (maximum exposure was 336 weeks) | This secondary efficacy endpoint is the mean change from MRX baseline over time in fasting sBA levels. Results reported here are the long-term results. |
| Secondary | Change From MRX Baseline to Week 48 in Pruritus | MRX baseline to Week 48 | This secondary efficacy endpoint is the change from MRX baseline to Week 48 in pruritus as measured by ItchRO(Obs) weekly average morning severity score. ItchRO scores range from 0 to 4; the higher score indicates increasing itch severity (0 = none; 4 = very severe). |
| Secondary | Change From MRX Baseline Over Time in Pruritus | MRX baseline to End of Treatment (maximum exposure was 336 weeks) | This secondary efficacy endpoint is the change from MRX baseline over time in pruritus as measured by ItchRO(Obs) weekly average morning severity score. ItchRO scores range from 0 to 4; the higher score indicates increasing itch severity (0 = none; 4 = very severe). Results reported here are the long-term results. |
| Secondary | Change From MRX Baseline to Week 48 in Clinician Xanthoma Severity Score | MRX baseline to Week 48 | This secondary efficacy endpoint is the mean change from MRX baseline to Week 48 in clinician xanthoma severity scores. It is based on a 0-4 scale to rate the number of lesions present and the degree to which the participant's lesions interfere or limit his or her activities. Clinician xanthoma severity scores range from 0 to 4, with a xanthoma score of zero representing no evidence of xanthomatosis and a score of 4 representing xanthoma so severe that it is disabling. Clinician xanthoma severity scores were not assessed in Study LUM001-302 so mean clinician xanthoma severity score at MRX baseline was calculated from the 5 participants who were assigned to placebo in Study LUM001-302, and analysis of change from MRX baseline is not presented. |
| Secondary | Change From MRX Baseline Over Time in Clinician Xanthoma Severity Score | MRX baseline to End of Treatment (maximum exposure was 336 weeks) | This secondary efficacy endpoint is the mean change from MRX baseline over time (with Week 252 chosen as the end point, as the last analysis visit with at least 6 participants) in clinician xanthoma severity scores. It is based on a 0-4 scale to rate the number of lesions present and the degree to which the lesions interfere or limit activities. Clinician xanthoma severity scores range from 0 to 4, with a score of zero representing no evidence of xanthomatosis and a score of 4 representing xanthoma so severe that it is disabling. Clinician xanthoma severity scores were not assessed in Study LUM001-302 so mean clinician xanthoma severity score at MRX baseline was calculated from the 5 participants assigned to placebo in Study LUM001-302, and analysis of change from MRX baseline is not presented. Results reported here are the long-term results. |
| Secondary | Secondary: Change From MRX Baseline to Week 48 in Alkaline Phosphatase | MRX baseline to Week 48 | This secondary efficacy endpoint is the mean change from MRX baseline to Week 48 in ALP. |
| Secondary | Change From MRX Baseline Over Time in Alkaline Phosphatase | MRX baseline to end of treatment (maximum exposure was 336 weeks) | This secondary efficacy endpoint is the mean change from MRX baseline over time in ALP. Results reported here are the long-term results. |
| Secondary | Change From MRX Baseline to Week 48 in Alanine Aminotransferase | MRX baseline to Week 48 | This secondary efficacy endpoint is the mean change from MRX baseline to Week 48 in ALT. |
| Secondary | Change From MRX Baseline Over Time in Alanine Aminotransferase | MRX baseline to End of Treatment (maximum exposure was 336 weeks) | This secondary efficacy endpoint is the mean change from MRX baseline over time in ALT levels. Results reported here are the long-term results. |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 registered outcomesOutcomes16
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From MRX Baseline to Week 48 in Fasting sBA Levels | MRX baseline to Week 48 | Data collected from 19 participants at MRX baseline. Data collected at Week 48 from 17 of the 19 participants who contributed values at MRX baseline.~Since all participants escalated to MRX 280 ug/kg/day by Week 48, the summary is presented as a single arm. | μmol/L | MEAN |
| SECONDARY | Change From MRX Baseline Over Time in Fasting sBA Levels | MRX baseline to End of Treatment (maximum exposure was 336 weeks) | Data collected from 19 participants at MRX baseline. Data collected at Week 252 from all 6 participants who contributed values at MRX baseline. Week 252 was chosen as this was the latest timepoint with more than 5 participants reporting data and was considered representative of values near the end of treatment. Since all participants escalated to MRX 280 ug/kg/day, the summary is presented as a single arm. | μmol/L | MEAN |
| SECONDARY | Change From MRX Baseline to Week 48 in Pruritus | MRX baseline to Week 48 | Values were collected from 19 participants at MRX baseline. Data collected at Week 48 from 17 of the 19 participants who contributed values at MRX baseline.~Since all participants escalated to MRX 280 ug/kg/day by Week 48, the summary is presented as a single arm. | scores on a scale | MEAN |
| SECONDARY | Change From MRX Baseline Over Time in Pruritus | MRX baseline to End of Treatment (maximum exposure was 336 weeks) | Data collected from 19 participants at MRX baseline Data collected at Week 278 from all 6 participants who contributed values at MRX baseline. Week 278 was chosen as this was the latest timepoint with more than 5 participants reporting data and was considered representative of values near the end of treatment. Since all participants escalated to MRX 280 ug/kg/day, the summary is presented as a single arm. | scores on a scale | MEAN |
| SECONDARY | Change From MRX Baseline to Week 48 in Clinician Xanthoma Severity Score | MRX baseline to Week 48 | Mean MRX baseline scores were calculated from the 5 participants assigned placebo in LUM001-302 who had a baseline value in study LUM001-303. Those assigned to MRX within study LUM001-302 did not have this data collected at baseline.~Mean MRX Week 48 scores were calculated from 17 participants. Since all participants escalated to MRX 280 ug/kg/day by Week 48, the summary is presented as a single arm. | scores on a scale | MEAN |
| SECONDARY | Change From MRX Baseline Over Time in Clinician Xanthoma Severity Score | MRX baseline to End of Treatment (maximum exposure was 336 weeks) | Mean MRX baseline scores were calculated from the 5 participants assigned placebo in LUM001-302.Mean Week 252 scores were calculated from 6 participants. Week 252 was chosen as this was the latest timepoint with more than 5 participants reporting data and was considered representative of values near the end of treatment. Since all participants escalated to MRX 280 ug/kg/day, the summary is presented as a single arm. | scores on a scale | MEAN |
| SECONDARY | Secondary: Change From MRX Baseline to Week 48 in Alkaline Phosphatase | MRX baseline to Week 48 | Data collected from 19 participants at MRX baseline. \[Data collected at Week 48 from 17 of the 19 participants who contributed values at MRX baseline. Since all participants escalated to MRX 280 ug/kg/day by Week 48, the summary is presented as a single arm. | U/L | MEAN |
| SECONDARY | Change From MRX Baseline Over Time in Alkaline Phosphatase | MRX baseline to end of treatment (maximum exposure was 336 weeks) | Data collected from 19 participants at MRX baseline. Data collected at Week 252 from 6 participants who contributed values at MRX baseline. Week 252 was chosen as this was the latest timepoint with more than 5 participants reporting data and was considered representative of values near the end of treatment. Since all participants escalated to MRX 280 ug/kg/day, the summary is presented as a single arm. | U/L | MEAN |
| SECONDARY | Change From MRX Baseline to Week 48 in Alanine Aminotransferase | MRX baseline to Week 48 | Data collected from 19 participants at MRX baseline. Data collected at Week 48 from 17 of the 19 participants who contributed values at MRX baseline. Since all participants escalated to MRX 280 ug/kg/day by Week 48, the summary is presented as a single arm. | U/L | MEAN |
| SECONDARY | Change From MRX Baseline Over Time in Alanine Aminotransferase | MRX baseline to End of Treatment (maximum exposure was 336 weeks) | Data collected from 19 participants at MRX baseline. Data collected at Week 252 from all 6 participants who contributed values at MRX baseline. Week 252 was chosen as this was the latest timepoint with more than 5 participants reporting data and was considered representative of values near the end of treatment. Since all participants escalated to MRX 280 ug/kg/day, the summary is presented as a single arm. | U/L | MEAN |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 outcomesResults by group36
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From MRX Baseline to Week 48 in Fasting sBA Levels | Core Study Period: MRX Baseline Values | 261.96 | Standard Deviation 206.839 | μmol/L | |
| Change From MRX Baseline to Week 48 in Fasting sBA Levels | Core Study Period: Week 48 Values | 128.32 | Standard Deviation 101.742 | μmol/L | |
| Change From MRX Baseline Over Time in Fasting sBA Levels | Core Study Period: MRX Baseline Value | 261.96 | Standard Deviation 206.839 | μmol/L | |
| Change From MRX Baseline Over Time in Fasting sBA Levels | Week 252 Values | 118.32 | Standard Deviation 76.140 | μmol/L | |
| Change From MRX Baseline to Week 48 in Pruritus | Core Study Period: MRX Baseline Value | 2.435 | Standard Deviation 0.7952 | scores on a scale | |
| Change From MRX Baseline to Week 48 in Pruritus | Core Study Period: Week 48 Values | 1.307 | Standard Deviation 0.6995 | scores on a scale | |
| Change From MRX Baseline Over Time in Pruritus | Core Study Period: MRX Baseline Value | 2.435 | Standard Deviation 0.7952 | scores on a scale | |
| Change From MRX Baseline Over Time in Pruritus | Week 278 Values | 0.952 | Standard Deviation 0.4302 | scores on a scale | |
| Change From MRX Baseline to Week 48 in Clinician Xanthoma Severity Score | Core Study Period: MRX Baseline Values | 0.4 | Standard Deviation 0.55 | scores on a scale | |
| Change From MRX Baseline to Week 48 in Clinician Xanthoma Severity Score | Core Study Period: Week 48 Values | 0.2 | Standard Deviation 0.73 | scores on a scale |
1 to 10 of 36Source ClinicalTrials.gov
See all 36 results by groupParticipant flow17
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Core Study Period | STARTED | LUM001 (Maralixibat) | 19 | |
| Core Study Period | Received MRX 14 ug/kg/Day | LUM001 (Maralixibat) | 6 | |
| Core Study Period | Received MRX 35 ug/kg/Day | LUM001 (Maralixibat) | 6 | |
| Core Study Period | Received MRX 70 ug/kg/Day | LUM001 (Maralixibat) | 6 | |
| Core Study Period | Received MRX 140 ug/kg/Day | LUM001 (Maralixibat) | 11 | |
| Core Study Period | Received MRX 280 ug/kg/Day | LUM001 (Maralixibat) | 19 | |
| Core Study Period | COMPLETED | LUM001 (Maralixibat) | 7 | |
| Core Study Period | NOT COMPLETED | LUM001 (Maralixibat) | 12 | |
| 52-week Follow-up Treatment Period | STARTED | LUM001 (Maralixibat) | 6 | |
| 52-week Follow-up Treatment Period | COMPLETED | LUM001 (Maralixibat) | 6 |
1 to 10 of 17Source ClinicalTrials.gov
See all 17 participant flowAdverse events1,192
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 | Anaemia | Blood and lymphatic system disorders | 14 Microgram Per Kilogram Per Day (mcg/kg/Day) | 0 | 6 | 0 |
| other | Anaemia | Blood and lymphatic system disorders | 35 Microgram Per Kilogram Per Day (mcg/kg/Day) | 0 | 10 | 0 |
| other | Anaemia | Blood and lymphatic system disorders | 70 Microgram Per Kilogram Per Day (mcg/kg/Day) | 0 | 10 | 0 |
| other | Anaemia | Blood and lymphatic system disorders | 140 Microgram Per Kilogram Per Day (mcg/kg/Day) | 0 | 13 | 0 |
| other | Anaemia | Blood and lymphatic system disorders | 280 Microgram Per Kilogram Per Day (mcg/kg/Day) | 0 | 19 | 0 |
| other | Anaemia | Blood and lymphatic system disorders | 420 Microgram Per Kilogram Per Day (mcg/kg/Day) | 1 | 5 | 1 |
| other | Anaemia | Blood and lymphatic system disorders | 560 Microgram Per Kilogram Per Day (mcg/kg/Day) | 0 | 5 | 0 |
| other | Anaemia | Blood and lymphatic system disorders | LUM001 MRX Treatment | 1 | 19 | 1 |
| other | Lymphadenopathy | Blood and lymphatic system disorders | 14 Microgram Per Kilogram Per Day (mcg/kg/Day) | 0 | 6 | 0 |
| other | Lymphadenopathy | Blood and lymphatic system disorders | 35 Microgram Per Kilogram Per Day (mcg/kg/Day) | 0 | 10 | 0 |
1 to 10 of 1,192Source ClinicalTrials.gov
See all 1,192 adverse eventsAdverse event totals24
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | 14 Microgram Per Kilogram Per Day (mcg/kg/Day) | 0 | 6 |
| Total, all-cause mortality | 35 Microgram Per Kilogram Per Day (mcg/kg/Day) | 0 | 10 |
| Total, all-cause mortality | 70 Microgram Per Kilogram Per Day (mcg/kg/Day) | 0 | 10 |
| Total, all-cause mortality | 140 Microgram Per Kilogram Per Day (mcg/kg/Day) | 0 | 13 |
| Total, all-cause mortality | 280 Microgram Per Kilogram Per Day (mcg/kg/Day) | 0 | 19 |
| Total, all-cause mortality | 420 Microgram Per Kilogram Per Day (mcg/kg/Day) | 0 | 5 |
| Total, all-cause mortality | 560 Microgram Per Kilogram Per Day (mcg/kg/Day) | 0 | 5 |
| Total, all-cause mortality | LUM001 MRX Treatment | 0 | 19 |
| Total, other adverse events | 14 Microgram Per Kilogram Per Day (mcg/kg/Day) | 3 | 6 |
| Total, other adverse events | 35 Microgram Per Kilogram Per Day (mcg/kg/Day) | 6 | 10 |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 adverse event totalsPapers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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/NCT02047318
GET https://conductscience.org/api/trials/v1/NCT02047318/results?page=2