Trial · NCT02160782
Safety and Efficacy Study of LUM001 (Maralixibat) With a Drug Withdrawal Period in Participants With Alagille Syndrome (ALGS)
NCT02160782, Phase 2, Completed, Mirum Pharmaceuticals, Inc.; results first posted 2021-07-14
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Safety and Efficacy Study of LUM001 (Maralixibat) With a Drug Withdrawal Period in Participants With Alagille Syndrome (ALGS). Trials, record NCT02160782. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02160782
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Safety and Efficacy Study of LUM001 (Maralixibat) With a Drug Withdrawal Period in Participants With Alagille Syndrome (ALGS)
- NCT number
- NCT02160782
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 31 participants, actual
- Lead sponsor
- Mirum Pharmaceuticals, Inc.
- Conditions
- Alagille Syndrome
- Interventions
- LUM001 (Maralixibat), Placebo
- First posted
- 2014-06-11
- Last updated
- 2021-07-14
- Other registries' IDs
- eudract:2013-005373-43
- Results first posted
- 2021-07-14
- 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 outcomes14
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Week 18 to Week 22 in Fasting sBA Levels in Participants Who Had a Reduction in sBA ≥50% From Baseline to Week 12 or Week 18 | Week 18 to Week 22 | The primary efficacy endpoint of this study was the mean change from Week 18 to Week 22 (the RWD period) of fasting sBA levels in participants who had a reduction in sBA ≥50% from baseline to Week 12 or Week 18 (Modified Intent-to-Treat \[MITT\] Population). Five participants in the MRX group and 10 participants in the placebo group met the prespecified sBA reduction criteria. |
| Secondary | Change From Baseline to Week 18 in Fasting sBA Levels | Baseline to Week 18 | This secondary efficacy endpoint is the mean change from baseline to Week 18 in fasting sBA levels |
| Secondary | Change From Baseline to Week 18 in Pruritus as Measured by ItchRO (Obs) | Baseline to Week 18 | This secondary efficacy endpoint is the change from baseline to Week 18 in pruritus as measured by ItchRO(Obs) weekly average morning score. ItchRO scores range from 0 to 4; the higher score indicates increasing itch severity (0 = none; 4 = very severe). |
| Secondary | Change From Baseline to Week 18 in Pruritus as Measured by ItchRO (Pt) | Baseline to Week 18 | This secondary efficacy endpoint is the change from baseline to Week 18 in pruritus as measured by ItchRO(Pt) weekly average morning score |
| Secondary | Change From Week 18 to Week 22 in Pruritus as Measured by ItchRO(Obs) | Week 18 to Week 22 | This secondary efficacy endpoint is the change from Week 18 to Week 22 in pruritus as measured by ItchRO(Obs) weekly average morning score. ItchRO scores range from 0 to 4; the higher score indicates increasing itch severity (0 = none; 4 = very severe). |
| Secondary | Change From Week 18 to Week 22 in Pruritus as Measured by ItchRO(Pt) | Week 18 to Week 22 | This secondary efficacy endpoint is the change from Week 18 to Week 22 in pruritus as measured by ItchRO(Pt) weekly average morning score. ItchRO scores range from 0 to 4; the higher score indicates increasing itch severity (0 = none; 4 = very severe). |
| Secondary | Change From Baseline to Week 18 in Alkaline Phosphatase | Baseline to Week 18 | This secondary efficacy endpoint is the mean change from baseline to Week 18 in ALP |
| Secondary | Change From Week 18 to Week 22 in Alkaline Phosphatase | Week 18 to Week 22 | This secondary efficacy endpoint is the mean change from Week 18 to Week 22 in ALP |
| Secondary | Change From Baseline to Week 18 in Alanine Aminotransferase | Baseline to Week 18 | This secondary efficacy endpoint is the mean change from baseline to Week 18 in ALT |
| Secondary | Change From Week 18 to Week 22 in Alanine Aminotransferase | Week 18 to Week 22 | This secondary efficacy endpoint is the mean change from Week 18 to Week 22 in alanine aminotransferase (ALT) |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 registered outcomesOutcomes14
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Week 18 to Week 22 in Fasting sBA Levels in Participants Who Had a Reduction in sBA ≥50% From Baseline to Week 12 or Week 18 | Week 18 to Week 22 | Primary outcome used the (Modified Intent-to-Treat \[MITT\]Population. The MITT population is defined as participants who had a reduction in sBA ≥50% from baseline to Week 12 or Week 18. Five participants in the MRX group and 10 participants in the placebo group met the prespecified sBA reduction criteria. | μmol/L | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline to Week 18 in Fasting sBA Levels | Baseline to Week 18 | Values were collected from 31 participants at baseline. Values were collected at Week 18 from 29 of the 31 participants who contributed values at baseline. | μmol/L | MEAN |
| SECONDARY | Change From Baseline to Week 18 in Pruritus as Measured by ItchRO (Obs) | Baseline to Week 18 | Values were collected from 31 participants at baseline. Values were collected at Week 18 from 29 of the 31 participants who contributed values at baseline. | Points | MEAN |
| SECONDARY | Change From Baseline to Week 18 in Pruritus as Measured by ItchRO (Pt) | Baseline to Week 18 | NOTE: 2 participants discontinued prior week 18 and did not provide data. | Points | MEAN |
| SECONDARY | Change From Week 18 to Week 22 in Pruritus as Measured by ItchRO(Obs) | Week 18 to Week 22 | For Maralixibat, n=12 for ItchRO(Obs) weekly average morning score For Placebo, n=16 for ItchRO(Obs) weekly average morning score | Points | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Week 18 to Week 22 in Pruritus as Measured by ItchRO(Pt) | Week 18 to Week 22 | ItchRO(Pt) was completed independently in participants 9 years old or older. Children between the ages of 5 and 8 years old completed the patient instrument with the assistance of their caregiver, if needed. | Points | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline to Week 18 in Alkaline Phosphatase | Baseline to Week 18 | Values were collected from 31 participants at baseline. Values were collected at Week 18 from 29 of the 31 participants who contributed values at baseline. | U/L | MEAN |
| SECONDARY | Change From Week 18 to Week 22 in Alkaline Phosphatase | Week 18 to Week 22 | U/L | LEAST_SQUARES_MEAN | |
| SECONDARY | Change From Baseline to Week 18 in Alanine Aminotransferase | Baseline to Week 18 | Values were collected from 31 participants at baseline. Values were collected at Week 18 from 29 of the 31 participants who contributed values at baseline. | U/L | MEAN |
| SECONDARY | Change From Week 18 to Week 22 in Alanine Aminotransferase | Week 18 to Week 22 | The difference between treatment groups in change from Week 18 to Week 22 in ALT levels was evaluated using an ANCOVA model with treatment group as a factor, and Week 18 ALT as a covariate. The analysis used a tabulation of fitted summary statistics from ANCOVA in the intent-to-treat population, which included all participants who were enrolled, and received at least one dose of study drug. | U/L | LEAST_SQUARES_MEAN |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 outcomesResults by group28
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Week 18 to Week 22 in Fasting sBA Levels in Participants Who Had a Reduction in sBA ≥50% From Baseline to Week 12 or Week 18 | Randomized Withdrawal Period: MRX | -21.73 | Standard Error 43.125 | μmol/L | |
| Change From Week 18 to Week 22 in Fasting sBA Levels in Participants Who Had a Reduction in sBA ≥50% From Baseline to Week 12 or Week 18 | Randomized Withdrawal Period: Placebo | 95.55 | Standard Error 30.488 | μmol/L | |
| Change From Baseline to Week 18 in Fasting sBA Levels | Open-label Period: MRX Baseline | 283.43 | Standard Deviation 210.569 | μmol/L | |
| Change From Baseline to Week 18 in Fasting sBA Levels | Open-label Period: MRX Week 18 | 192.50 | Standard Deviation 161.278 | μmol/L | |
| Change From Baseline to Week 18 in Pruritus as Measured by ItchRO (Obs) | Open-label Period: MRX Baseline | 2.909 | Standard Deviation 0.5480 | Points | |
| Change From Baseline to Week 18 in Pruritus as Measured by ItchRO (Obs) | Open-label Period: MRX Week 18 | 1.203 | Standard Deviation 0.8446 | Points | |
| Change From Baseline to Week 18 in Pruritus as Measured by ItchRO (Pt) | Open-label Period: MRX Baseline | 2.903 | Standard Deviation 0.6616 | Points | |
| Change From Baseline to Week 18 in Pruritus as Measured by ItchRO (Pt) | Open-label Period: MRX Week 18 | 0.831 | Standard Deviation 0.8122 | Points | |
| Change From Week 18 to Week 22 in Pruritus as Measured by ItchRO(Obs) | Randomized Withdrawal Period: MRX | 0.217 | Standard Error 0.2345 | Points | |
| Change From Week 18 to Week 22 in Pruritus as Measured by ItchRO(Obs) | Randomized Withdrawal Period: Placebo | 1.700 | Standard Error 0.2031 | Points |
1 to 10 of 28Source ClinicalTrials.gov
See all 28 results by groupParticipant flow60
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Open-label Period: Maralixibat | STARTED | Open-label Period: Maralixibat (LUM001) | 31 | |
| Open-label Period: Maralixibat | STARTED | Randomized Withdrawal Period: MRX | 0 | |
| Open-label Period: Maralixibat | STARTED | Randomized Withdrawal Period: Placebo | 0 | |
| Open-label Period: Maralixibat | STARTED | After Randomized Withdrawal Period: Maralixibat | 0 | |
| Open-label Period: Maralixibat | STARTED | Long-term Extension Period: Maralixibat | 0 | |
| Open-label Period: Maralixibat | COMPLETED | Open-label Period: Maralixibat (LUM001) | 29 | |
| Open-label Period: Maralixibat | COMPLETED | Randomized Withdrawal Period: MRX | 0 | |
| Open-label Period: Maralixibat | COMPLETED | Randomized Withdrawal Period: Placebo | 0 | |
| Open-label Period: Maralixibat | COMPLETED | After Randomized Withdrawal Period: Maralixibat | 0 | |
| Open-label Period: Maralixibat | COMPLETED | Long-term Extension Period: Maralixibat | 0 |
1 to 10 of 60Source ClinicalTrials.gov
See all 60 participant flowAdverse events1,026
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 | Hypertension | Vascular disorders | Open-label Period: Maralixibat (LUM001) | 1 | 31 | 1 |
| other | Hypertension | Vascular disorders | Randomized Withdrawal Period: MRX | 0 | 13 | 0 |
| other | Hypertension | Vascular disorders | Randomized Withdrawal Period: Placebo | 1 | 16 | 1 |
| other | Hypertension | Vascular disorders | After Randomized Withdrawal Period: Maralixibat | 0 | 29 | 0 |
| other | Hypertension | Vascular disorders | Long-term Extension Period: Maralixibat | 0 | 23 | 0 |
| other | Hypertension | Vascular disorders | Safety Population | 2 | 31 | 2 |
| other | Subclavian artery stenosis | Vascular disorders | Open-label Period: Maralixibat (LUM001) | 0 | 31 | 0 |
| other | Subclavian artery stenosis | Vascular disorders | Randomized Withdrawal Period: MRX | 0 | 13 | 0 |
| other | Subclavian artery stenosis | Vascular disorders | Randomized Withdrawal Period: Placebo | 0 | 16 | 0 |
| other | Subclavian artery stenosis | Vascular disorders | After Randomized Withdrawal Period: Maralixibat | 0 | 29 | 0 |
1 to 10 of 1,026Source ClinicalTrials.gov
See all 1,026 adverse eventsAdverse event totals18
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Open-label Period: Maralixibat (LUM001) | 0 | 31 |
| Total, all-cause mortality | Randomized Withdrawal Period: MRX | 0 | 13 |
| Total, all-cause mortality | Randomized Withdrawal Period: Placebo | 0 | 16 |
| Total, all-cause mortality | After Randomized Withdrawal Period: Maralixibat | 0 | 29 |
| Total, all-cause mortality | Long-term Extension Period: Maralixibat | 0 | 23 |
| Total, all-cause mortality | Safety Population | 0 | 31 |
| Total, other adverse events | Open-label Period: Maralixibat (LUM001) | 29 | 31 |
| Total, other adverse events | Randomized Withdrawal Period: MRX | 7 | 13 |
| Total, other adverse events | Randomized Withdrawal Period: Placebo | 12 | 16 |
| Total, other adverse events | After Randomized Withdrawal Period: Maralixibat | 25 | 29 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 adverse event totalsPapers3
The papers the trial lists on ClinicalTrials.gov.
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/NCT02160782
GET https://conductscience.org/api/trials/v1/NCT02160782/results?page=2