Trial · NCT02057692

Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome

NCT02057692, Phase 2, Completed, Mirum Pharmaceuticals, Inc.; results first posted 2018-01-23

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome. Trials, record NCT02057692. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02057692

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome
NCT number: NCT02057692
Status: Completed
Phases: Phase 2
Enrollment: 37 participants, actual
Lead sponsor: Mirum Pharmaceuticals, Inc.
Conditions: Alagille Syndrome
Interventions: LUM001, Placebo
First posted: 2014-02-07
Last updated: 2019-03-26
Results first posted: 2018-01-23

Source ClinicalTrials.gov

Registered outcomes5

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

List	Outcome	Time frame	Description
Primary	Change From Baseline to Endpoint (Week 13/Early Termination) in Pruritus	Baseline, Week 13/Early Termination	Pruritus was assessed using Itch report outcome measure (ItchRO\[Obs\]), administered as an electronic diary (eDiary) which was completed by the participants twice daily (morning and evening). ItchRO(Obs) score ranged from 0 to 4, with the higher score indicating increasing itch severity. The highest score between the morning and evening ItchRO(Obs) reports represented the daily score: a measure of the worst itching over the previous 24-hour period.
Secondary	Change From Baseline to Endpoint (Week 13/Early Termination) in Fasting Serum Bile Acid (sBA) Level	Baseline, Week 13/Early Termination	Fasting sBA level was measured by using a liquid chromatography mass spectrometry method.
Secondary	Change From Baseline to Endpoint (Week 13/Early Termination) in Liver Enzyme Levels	Baseline, Week 13/Early Termination	Liver enzyme levels of alanine aminotransferase, alkaline phosphatase, aspartate aminotransferase and gamma glutamyl transferase were reported here.
Secondary	Change From Baseline to Endpoint (Week 13/Early Termination) in Total and Direct Bilirubin Concentrations	Baseline, Week 13/Early Termination	Liver enzyme levels of total bilirubin and direct bilirubin were reported here.
Other	Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)	From the start of study drug administration up to Week 17	An AE was 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 related to the investigational drug product. A serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged in-patient hospitalization; life -threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly.TEAEs were defined as AEs/SAEs that started or worsened after the study drug treatment.

Source ClinicalTrials.gov

Outcomes5

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 Endpoint (Week 13/Early Termination) in Pruritus	Baseline, Week 13/Early Termination	Modified Intent-to-Treat Population (mITT) population included all participants randomly assigned to study treatment, receiving at least 1 dose of treatment, and having at least 1 post baseline observer itch reported outcome (ItchRO\[Obs\]) average daily score.	Score on a scale	LEAST_SQUARES_MEAN
SECONDARY	Change From Baseline to Endpoint (Week 13/Early Termination) in Fasting Serum Bile Acid (sBA) Level	Baseline, Week 13/Early Termination	mITT population included all participants randomly assigned to study treatment, receiving at least 1 dose of treatment, and having at least 1 post baseline observer itch reported outcome (ItchRO\[Obs\]) average daily score.	Micro moles per liter (mcmol/L)	LEAST_SQUARES_MEAN
SECONDARY	Change From Baseline to Endpoint (Week 13/Early Termination) in Liver Enzyme Levels	Baseline, Week 13/Early Termination	mITT population included all participants randomly assigned to study treatment, receiving at least 1 dose of treatment, and having at least 1 post baseline observer itch reported outcome (ItchRO\[Obs\]) average daily score.	Unit per liter (U/L)	LEAST_SQUARES_MEAN
SECONDARY	Change From Baseline to Endpoint (Week 13/Early Termination) in Total and Direct Bilirubin Concentrations	Baseline, Week 13/Early Termination	mITT population included all participants randomly assigned to study treatment, receiving at least 1 dose of treatment, and having at least 1 post baseline observer itch reported outcome (ItchRO\[Obs\]) average daily score.	Milligrams per deciliter (mg/dL)	LEAST_SQUARES_MEAN
OTHER_PRE_SPECIFIED	Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)	From the start of study drug administration up to Week 17	The Safety Population included all participants who were randomly assigned to study treatment and received at least one dose of the study drug.	Participants	COUNT_OF_PARTICIPANTS

Source ClinicalTrials.gov

Results by group42

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 Endpoint (Week 13/Early Termination) in Pruritus	Maralixibat 70 mcg/kg/Day		-1.470	Standard Error 0.3034	Score on a scale
Change From Baseline to Endpoint (Week 13/Early Termination) in Pruritus	Maralixibat 140 mcg/kg/Day		-1.486	Standard Error 0.2597	Score on a scale
Change From Baseline to Endpoint (Week 13/Early Termination) in Pruritus	Maralixibat 280 mcg/kg/Day		-0.620	Standard Error 0.3575	Score on a scale
Change From Baseline to Endpoint (Week 13/Early Termination) in Pruritus	Placebo		-0.580	Standard Error 0.2453	Score on a scale
Change From Baseline to Endpoint (Week 13/Early Termination) in Fasting Serum Bile Acid (sBA) Level	Maralixibat 70 mcg/kg/Day		-117.401	Standard Error 46.2353	Micro moles per liter (mcmol/L)
Change From Baseline to Endpoint (Week 13/Early Termination) in Fasting Serum Bile Acid (sBA) Level	Maralixibat 140 mcg/kg/Day		-40.358	Standard Error 34.8650	Micro moles per liter (mcmol/L)
Change From Baseline to Endpoint (Week 13/Early Termination) in Fasting Serum Bile Acid (sBA) Level	Maralixibat 280 mcg/kg/Day		-27.437	Standard Error 46.2823	Micro moles per liter (mcmol/L)
Change From Baseline to Endpoint (Week 13/Early Termination) in Fasting Serum Bile Acid (sBA) Level	Placebo		-10.442	Standard Error 32.6776	Micro moles per liter (mcmol/L)
Change From Baseline to Endpoint (Week 13/Early Termination) in Liver Enzyme Levels	Maralixibat 70 mcg/kg/Day	Alanine Aminotransferase	14.616	Standard Error 17.9442	Unit per liter (U/L)
Change From Baseline to Endpoint (Week 13/Early Termination) in Liver Enzyme Levels	Maralixibat 140 mcg/kg/Day	Alanine Aminotransferase	13.236	Standard Error 14.9424	Unit per liter (U/L)

1 to 10 of 42Source ClinicalTrials.gov

See all 42 results by group

Participant flow12

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

Period	Milestone	Group	Participants
Overall Study	STARTED	Maralixibat 70 mcg/kg/Day	8
Overall Study	STARTED	Maralixibat 140 mcg/kg/Day	11
Overall Study	STARTED	Maralixibat 280 mcg/kg/Day	6
Overall Study	STARTED	Placebo	12
Overall Study	COMPLETED	Maralixibat 70 mcg/kg/Day	7
Overall Study	COMPLETED	Maralixibat 140 mcg/kg/Day	11
Overall Study	COMPLETED	Maralixibat 280 mcg/kg/Day	6
Overall Study	COMPLETED	Placebo	11
Overall Study	NOT COMPLETED	Maralixibat 70 mcg/kg/Day	1
Overall Study	NOT COMPLETED	Maralixibat 140 mcg/kg/Day	0

1 to 10 of 12Source ClinicalTrials.gov

See all 12 participant flow

Adverse events320

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 14 mcg/kg/Day	0	1	0
other	Arthralgia	Musculoskeletal and connective tissue disorders	Maralixibat 70 mcg/kg/Day	0	7	0
other	Arthralgia	Musculoskeletal and connective tissue disorders	Maralixibat 140 mcg/kg/Day	0	11	0
other	Arthralgia	Musculoskeletal and connective tissue disorders	Maralixibat 280 mcg/kg/Day	0	6	0
other	Arthralgia	Musculoskeletal and connective tissue disorders	Placebo	1	12	1
other	Growing pains	Musculoskeletal and connective tissue disorders	Maralixibat 14 mcg/kg/Day	0	1	0
other	Growing pains	Musculoskeletal and connective tissue disorders	Maralixibat 70 mcg/kg/Day	0	7	0
other	Growing pains	Musculoskeletal and connective tissue disorders	Maralixibat 140 mcg/kg/Day	0	11	0
other	Growing pains	Musculoskeletal and connective tissue disorders	Maralixibat 280 mcg/kg/Day	0	6	0
other	Growing pains	Musculoskeletal and connective tissue disorders	Placebo	1	12	4

1 to 10 of 320Source ClinicalTrials.gov

See all 320 adverse events

Adverse event totals15

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Maralixibat 14 mcg/kg/Day	0	1
Total, all-cause mortality	Maralixibat 70 mcg/kg/Day	0	7
Total, all-cause mortality	Maralixibat 140 mcg/kg/Day	0	11
Total, all-cause mortality	Maralixibat 280 mcg/kg/Day	0	6
Total, all-cause mortality	Placebo	0	12
Total, other adverse events	Maralixibat 14 mcg/kg/Day	1	1
Total, other adverse events	Maralixibat 70 mcg/kg/Day	7	7
Total, other adverse events	Maralixibat 140 mcg/kg/Day	9	11
Total, other adverse events	Maralixibat 280 mcg/kg/Day	5	6
Total, other adverse events	Placebo	12	12

1 to 10 of 15Source ClinicalTrials.gov

See all 15 adverse event totals

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.

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.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

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