Trial · NCT01903460

Safety and Efficacy Study of LUM001 in the Treatment of Cholestatic Liver Disease in Patients With Alagille Syndrome

NCT01903460, Phase 2, Completed, Mirum Pharmaceuticals, Inc.; results first posted 2015-12-07

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Safety and Efficacy Study of LUM001 in the Treatment of Cholestatic Liver Disease in Patients With Alagille Syndrome. Trials, record NCT01903460. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01903460

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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 in the Treatment of Cholestatic Liver Disease in Patients With Alagille Syndrome
NCT number: NCT01903460
Status: Completed
Phases: Phase 2
Enrollment: 20 participants, actual
Lead sponsor: Mirum Pharmaceuticals, Inc.
Conditions: Alagille Syndrome
Interventions: LUM001, Placebo
First posted: 2013-07-19
Last updated: 2019-03-28
Other registries' IDs: eudract:2012-005346-38
Results first posted: 2015-12-07

Source ClinicalTrials.gov

Registered outcomes3

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Change From Baseline to Week 13 (End of Treatment) in Fasting Serum Bile Acid Level	Baseline to 13 weeks or end of treatment	Participants were required to fast for at least 4 hours; only water was permitted prior to collection. A negative change from baseline indicates that the level of bile acid decreased.
Secondary	Change From Baseline to Week 13 (End of Treatment) in Liver Enzymes	Baseline to 13 weeks or end of treatment	Analysis of liver enzymes included alanine aminotransferase (ALT), aspartate aminotransferase (AST), and alkaline phosphatase (ALP). A negative change from baseline indicates that the level of that enzyme decreased.
Secondary	Change From Baseline to Week 13 (End of Treatment) in Pruritus as Measured by The Patient And Observer Itch Reported Outcome (ItchRO) Average Daily Scores	Baseline to 13 weeks or end of treatment	The ItchRO was administered as a twice daily electronic diary (eDiary). Children ≥9 years of age completed the patient ItchRO; those between the ages of 5 and 8 completed the patient ItchRO with the assistance of their caregiver. There was no patient report for subjects under the age of 5. ItchRO scores range from 0 to 4, with the higher score indicating increasing itch severity. ItchRO average daily scores were calculated as the sum of daily scores (ie, the maximum of morning and evening scores) divided by the number of days. The average daily score was calculated by using the 7 days pre-treatment for baseline, and the last 7 days of treatment for Week 13. A negative change from Baseline indicates that itch severity decreased.

Source ClinicalTrials.gov

Outcomes3

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Change From Baseline to Week 13 (End of Treatment) in Fasting Serum Bile Acid Level	Baseline to 13 weeks or end of treatment	The modified Intent-to-Treat (mITT) population, defined as all participants in the Safety population who had at least 1 post-baseline efficacy assessment. The Safety population was defined as all participants who were randomly assigned to study treatment and who received any amount of study drug.	umol/L	LEAST_SQUARES_MEAN
SECONDARY	Change From Baseline to Week 13 (End of Treatment) in Liver Enzymes	Baseline to 13 weeks or end of treatment	The mITT population, defined as all participants in the Safety population who had at least 1 post-baseline efficacy assessment. The Safety population was defined as all participants who were randomly assigned to study treatment and who received any amount of study drug.	U/L	LEAST_SQUARES_MEAN
SECONDARY	Change From Baseline to Week 13 (End of Treatment) in Pruritus as Measured by The Patient And Observer Itch Reported Outcome (ItchRO) Average Daily Scores	Baseline to 13 weeks or end of treatment	The mITT population, defined as all participants in the Safety population who had at least 1 post-baseline efficacy assessment. The Safety population was defined as all participants who were randomly assigned to study treatment and who received any amount of study drug.	units on a scale	LEAST_SQUARES_MEAN

Source ClinicalTrials.gov

Results by group24

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
Change From Baseline to Week 13 (End of Treatment) in Fasting Serum Bile Acid Level	LUM001 140ug/kg/Day		-82.864	Standard Error 50.1513	umol/L
Change From Baseline to Week 13 (End of Treatment) in Fasting Serum Bile Acid Level	LUM001 280ug/kg/Day		-49.388	Standard Error 43.4732	umol/L
Change From Baseline to Week 13 (End of Treatment) in Fasting Serum Bile Acid Level	LUM001 Overall		-66.126	Standard Error 33.1208	umol/L
Change From Baseline to Week 13 (End of Treatment) in Fasting Serum Bile Acid Level	Placebo Overall		-42.157	Standard Error 50.0903	umol/L
Change From Baseline to Week 13 (End of Treatment) in Liver Enzymes	LUM001 140ug/kg/Day	ALT	59.3	Standard Error 20.99	U/L
Change From Baseline to Week 13 (End of Treatment) in Liver Enzymes	LUM001 280ug/kg/Day	ALT	10.5	Standard Error 18.06	U/L
Change From Baseline to Week 13 (End of Treatment) in Liver Enzymes	LUM001 Overall	ALT	34.9	Standard Error 13.86	U/L
Change From Baseline to Week 13 (End of Treatment) in Liver Enzymes	Placebo Overall	ALT	2.7	Standard Error 21.06	U/L
Change From Baseline to Week 13 (End of Treatment) in Liver Enzymes	LUM001 140ug/kg/Day	AST	37.2	Standard Error 13.64	U/L
Change From Baseline to Week 13 (End of Treatment) in Liver Enzymes	LUM001 280ug/kg/Day	AST	-2.7	Standard Error 11.70	U/L

1 to 10 of 24Source ClinicalTrials.gov

See all 24 results by group

Participant flow12

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	LUM001 140ug/kg/Day	6
Overall Study	STARTED	LUM001 280ug/kg/Day	8
Overall Study	STARTED	Placebo Cohort A	3
Overall Study	STARTED	Placebo Cohort B	3
Overall Study	COMPLETED	LUM001 140ug/kg/Day	6
Overall Study	COMPLETED	LUM001 280ug/kg/Day	8
Overall Study	COMPLETED	Placebo Cohort A	3
Overall Study	COMPLETED	Placebo Cohort B	2
Overall Study	NOT COMPLETED	LUM001 140ug/kg/Day	0
Overall Study	NOT COMPLETED	LUM001 280ug/kg/Day	0

1 to 10 of 12Source ClinicalTrials.gov

See all 12 participant flow

Adverse events126

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	Bradycardia	Cardiac disorders	LUM001 140ug/kg/Day	0	6	0
other	Bradycardia	Cardiac disorders	LUM001 280ug/kg/Day	1	8	1
other	Bradycardia	Cardiac disorders	Placebo Overall	0	6	0
other	Deafness unilateral	Ear and labyrinth disorders	LUM001 140ug/kg/Day	1	6	1
other	Deafness unilateral	Ear and labyrinth disorders	LUM001 280ug/kg/Day	0	8	0
other	Deafness unilateral	Ear and labyrinth disorders	Placebo Overall	0	6	0
other	Ear pain	Ear and labyrinth disorders	LUM001 140ug/kg/Day	1	6	1
other	Ear pain	Ear and labyrinth disorders	LUM001 280ug/kg/Day	0	8	0
other	Ear pain	Ear and labyrinth disorders	Placebo Overall	0	6	0
other	Middle ear inflammation	Ear and labyrinth disorders	LUM001 140ug/kg/Day	1	6	1

1 to 10 of 126Source ClinicalTrials.gov

See all 126 adverse events

Adverse event totals9

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	LUM001 140ug/kg/Day
Total, all-cause mortality	LUM001 280ug/kg/Day
Total, all-cause mortality	Placebo Overall
Total, other adverse events	LUM001 140ug/kg/Day	6	6
Total, other adverse events	LUM001 280ug/kg/Day	7	8
Total, other adverse events	Placebo Overall	4	6
Total, serious adverse events	LUM001 140ug/kg/Day	0	6
Total, serious adverse events	LUM001 280ug/kg/Day	0	8
Total, serious adverse events	Placebo Overall	0	6

Source ClinicalTrials.gov

Papers0

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.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

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.

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