Trial · NCT02117713
An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Pediatric Subjects With Alagille Syndrome
NCT02117713, Phase 2, Completed, Mirum Pharmaceuticals, Inc.; results first posted 2021-06-22
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 Pediatric Subjects With Alagille Syndrome. Trials, record NCT02117713. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02117713
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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 Pediatric Subjects With Alagille Syndrome
- NCT number
- NCT02117713
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 34 participants, actual
- Lead sponsor
- Mirum Pharmaceuticals, Inc.
- Conditions
- Alagille Syndrome
- Interventions
- LUM001 (Maralixibat)
- First posted
- 2014-04-21
- Last updated
- 2021-07-01
- Results first posted
- 2021-06-22
- Limitations and caveats
- Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
Source ClinicalTrials.gov
Registered outcomes11
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 Serum Bile Acid (sBA) | Baseline to Week 48 | This primacy efficacy endpoint is the mean change from MRX baseline to week 48 in fasting sBA levels. |
| Secondary | Change From MRX Baseline to Week 216 in Fasting Serum Bile Acid (sBA) | Baseline to week 216 | The secondary endpoint of this study was the mean change from MRX baseline to week 216 fasting in sBA levels. |
| Secondary | Change From Baseline to Week 218 in Pruritus | Baseline to Week 218 | This secondary efficacy endpoint is the mean change from MRX baseline over time to week 218 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 Baseline to Week 216 in Alanine Aminotransferase | Baseline to week 216 | This secondary efficacy endpoint is the mean change from MRX baseline to week 216 in ALT levels. |
| Secondary | Change From Baseline to End of Treatment in Alkaline Phosphatase | Baseline to Week 216 | This secondary efficacy endpoint is the mean change from MRX baseline to week 216 in ALP levels. |
| Secondary | Change From MRX Baseline to Week 216 in Aspartate Aminotransferase | Baseline to week 216 | This secondary efficacy endpoint is the mean change from MRX baseline to week 216 in AST levels. |
| Secondary | Change From MRX Baseline to Week 216 in Clinician Xanthoma Severity Score | Baseline to week 216 | This secondary efficacy endpoint is the mean change from MRX baseline to week 216 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. |
| Secondary | Change From Baseline to Week 216/LOFC Clinician Scratch Scale (CSS) Score | Baseline to Week 216 | This secondary efficacy endpoint is the mean change from MRX baseline over time to week 216/LOCF in pruritus as measured by the Clinician Scratch Scale (CSS). The Clinician Scratch Scale uses a 5-point scale, where 0 = none; 1 = rubbing or mild scratching when undistracted; 2 = active scratching without evident skin abrasions; 3 = abrasion evident; 4 = cutaneous mutilation, haemorrhage and scarring evident. |
| Secondary | Change From MRX Baseline to Week 216 in Gamma Glutamyltransferase | Baseline to Week 216 | This secondary efficacy endpoint is the mean change from MRX baseline to week 216 in GGT. |
| Secondary | Mean Change From MRX Baseline to Week 216 in Total Bilirubin | Baseline to week 216 | This secondary efficacy endpoint is the mean change from MRX baseline to week 216 in total bilirubin. |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 registered outcomesOutcomes11
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 Serum Bile Acid (sBA) | Baseline to Week 48 | Since most 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 216 in Fasting Serum Bile Acid (sBA) | Baseline to week 216 | Since most participants escalated to MRX 280 ug/kg/day, the summary is presented as a single arm. | μmol/L | MEAN |
| SECONDARY | Change From Baseline to Week 218 in Pruritus | Baseline to Week 218 | Since most participants escalated to MRX 280 ug/kg/day, the summary is presented as a single arm. | score on a scale | MEAN |
| SECONDARY | Change From Baseline to Week 216 in Alanine Aminotransferase | Baseline to week 216 | Since most participants escalated to MRX 280 ug/kg/day, the summary is presented as a single arm. | U/L | MEAN |
| SECONDARY | Change From Baseline to End of Treatment in Alkaline Phosphatase | Baseline to Week 216 | Since most 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 216 in Aspartate Aminotransferase | Baseline to week 216 | Since most 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 216 in Clinician Xanthoma Severity Score | Baseline to week 216 | Since most participants escalated to MRX 280 ug/kg/day, the summary is presented as a single arm. | Points | MEAN |
| SECONDARY | Change From Baseline to Week 216/LOFC Clinician Scratch Scale (CSS) Score | Baseline to Week 216 | Since most participants escalated to MRX 280 ug/kg/day, the summary is presented as a single arm. | Points | MEAN |
| SECONDARY | Change From MRX Baseline to Week 216 in Gamma Glutamyltransferase | Baseline to Week 216 | Since most participants escalated to MRX 280 ug/kg/day, the summary is presented as a single arm. | U/L | MEAN |
| SECONDARY | Mean Change From MRX Baseline to Week 216 in Total Bilirubin | Baseline to week 216 | Since most participants escalated to MRX 280 ug/kg/day, the summary is presented as a single arm. | mg/dL | MEAN |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 outcomesResults by group22
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 Serum Bile Acid (sBA) | MRX Baseline Value | 223.62 | Standard Deviation 205.041 | μmol/L | |
| Change From MRX Baseline to Week 48 in Fasting Serum Bile Acid (sBA) | Maralixibat Week 48 Values | 157.35 | Standard Deviation 178.538 | μmol/L | |
| Change From MRX Baseline to Week 216 in Fasting Serum Bile Acid (sBA) | Maralixibat Baseline Values | 223.62 | Standard Deviation 205.041 | μmol/L | |
| Change From MRX Baseline to Week 216 in Fasting Serum Bile Acid (sBA) | Week 216 | 118.81 | Standard Deviation 126.878 | μmol/L | |
| Change From Baseline to Week 218 in Pruritus | Maralixibat Baseline Values | 2.533 | Standard Deviation 0.8423 | score on a scale | |
| Change From Baseline to Week 218 in Pruritus | Week 218 | 0.429 | Standard Deviation 0.8571 | score on a scale | |
| Change From Baseline to Week 216 in Alanine Aminotransferase | MRX Baseline Value | 152.9 | Standard Deviation 82.26 | U/L | |
| Change From Baseline to Week 216 in Alanine Aminotransferase | Week 216 Values | 222.4 | Standard Deviation 126.53 | U/L | |
| Change From Baseline to End of Treatment in Alkaline Phosphatase | MRX Baseline Value | 598.8 | Standard Deviation 199.89 | U/L | |
| Change From Baseline to End of Treatment in Alkaline Phosphatase | Week 216 Values | 528.3 | Standard Deviation 221.93 | U/L |
1 to 10 of 22Source ClinicalTrials.gov
See all 22 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Week 48 | STARTED | LUM001 (Maralixibat) | 34 | |
| Week 48 | COMPLETED | LUM001 (Maralixibat) | 26 | |
| Week 48 | NOT COMPLETED | LUM001 (Maralixibat) | 8 | |
| Long Term Follow-up Treatment | STARTED | LUM001 (Maralixibat) | 26 | |
| Long Term Follow-up Treatment | COMPLETED | LUM001 (Maralixibat) | 20 | |
| Long Term Follow-up Treatment | NOT COMPLETED | LUM001 (Maralixibat) | 6 |
Source ClinicalTrials.gov
Adverse events184
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 | Lymphadenopathy | Blood and lymphatic system disorders | LUM001 (Maralixibat) | 2 | 34 | 2 |
| other | Splenic infarction | Blood and lymphatic system disorders | LUM001 (Maralixibat) | 1 | 34 | 1 |
| other | Bicuspid aortic valve | Congenital, familial and genetic disorders | LUM001 (Maralixibat) | 1 | 34 | 1 |
| other | Non-compaction cardiomyopathy | Congenital, familial and genetic disorders | LUM001 (Maralixibat) | 1 | 34 | 1 |
| other | Ear pain | Ear and labyrinth disorders | LUM001 (Maralixibat) | 3 | 34 | 5 |
| other | Eustachian tube dysfunction | Ear and labyrinth disorders | LUM001 (Maralixibat) | 1 | 34 | 2 |
| other | Excessive cerumen production | Ear and labyrinth disorders | LUM001 (Maralixibat) | 1 | 34 | 1 |
| other | Tympanic membrane perforation | Ear and labyrinth disorders | LUM001 (Maralixibat) | 1 | 34 | 1 |
| other | Vertigo | Ear and labyrinth disorders | LUM001 (Maralixibat) | 1 | 34 | 1 |
| other | Asthenopia | Eye disorders | LUM001 (Maralixibat) | 1 | 34 | 1 |
1 to 10 of 184Source ClinicalTrials.gov
See all 184 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | LUM001 (Maralixibat) | 0 | 34 |
| Total, other adverse events | LUM001 (Maralixibat) | 34 | 34 |
| Total, serious adverse events | LUM001 (Maralixibat) | 6 | 34 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 42002216 | 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/NCT02117713
GET https://conductscience.org/api/trials/v1/NCT02117713/results?page=2