Trial · NCT02057718

Open Label Study to Evaluate Efficacy and Long Term Safety of LUM001 (Maralixibat) in the Treatment of Cholestatic Liver Disease in Patients With Progressive Familial Intrahepatic Cholestasis

NCT02057718, Phase 2, Completed, Mirum Pharmaceuticals, Inc.; results first posted 2021-07-12

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Open Label Study to Evaluate Efficacy and Long Term Safety of LUM001 (Maralixibat) in the Treatment of Cholestatic Liver Disease in Patients With Progressive Familial Intrahepatic Cholestasis. Trials, record NCT02057718. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02057718

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Trial

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

Title: Open Label Study to Evaluate Efficacy and Long Term Safety of LUM001 (Maralixibat) in the Treatment of Cholestatic Liver Disease in Patients With Progressive Familial Intrahepatic Cholestasis
NCT number: NCT02057718
Status: Completed
Phases: Phase 2
Enrollment: 33 participants, actual
Lead sponsor: Mirum Pharmaceuticals, Inc.
Conditions: Progressive Familial Intrahepatic Cholestasis (PFIC)
Interventions: LUM001 (Maralixibat)
First posted: 2014-02-07
Last updated: 2023-10-23
Other registries' IDs: eudract:2013-003833-14
Results first posted: 2021-07-12
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 outcomes6

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) in Fasting sBA Level	Baseline (Day 0) to Week 13
Secondary	Change From Baseline to Week 13/ET in Pruritus as Measured by ItchRO(Obs)	Baseline (Day 0) to Week 13	This secondary efficacy endpoint is the change from baseline to Week 13 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 13/ET in Pruritus as Measured by ItchRO(Pt)	Baseline (Day 0) to Week 13	This secondary efficacy endpoint is the change from baseline to Week 13 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 13/ET in ALT	Baseline (Day 0) to Week 13
Secondary	Change From Baseline to Week 13/ET in Total Bilirubin	Baseline (Day 0) to Week 13
Secondary	Change From Baseline to Week 13/ET in Direct Bilirubin	Baseline (Day 0) to Week 13

Source ClinicalTrials.gov

Outcomes6

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

Type	Outcome	Time frame	Units	Measured as
PRIMARY	Change From Baseline to Endpoint (Week 13) in Fasting sBA Level	Baseline (Day 0) to Week 13	umol/L	MEAN
SECONDARY	Change From Baseline to Week 13/ET in Pruritus as Measured by ItchRO(Obs)	Baseline (Day 0) to Week 13	scores on a scale	MEAN
SECONDARY	Change From Baseline to Week 13/ET in Pruritus as Measured by ItchRO(Pt)	Baseline (Day 0) to Week 13	scores on a scale	MEAN
SECONDARY	Change From Baseline to Week 13/ET in ALT	Baseline (Day 0) to Week 13	U/L	MEAN
SECONDARY	Change From Baseline to Week 13/ET in Total Bilirubin	Baseline (Day 0) to Week 13	mg/dL	MEAN
SECONDARY	Change From Baseline to Week 13/ET in Direct Bilirubin	Baseline (Day 0) to Week 13	mg/dL	MEAN

Source ClinicalTrials.gov

Results by group18

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

Outcome	Group	Value	Spread	Units
Change From Baseline to Endpoint (Week 13) in Fasting sBA Level	Maralixibat	-23	Full Range -463 to 279	umol/L
Change From Baseline to Endpoint (Week 13) in Fasting sBA Level	PFIC1	18	Full Range -117 to 149	umol/L
Change From Baseline to Endpoint (Week 13) in Fasting sBA Level	PFIC2 (Overall)	-38	Full Range -463 to 279	umol/L
Change From Baseline to Week 13/ET in Pruritus as Measured by ItchRO(Obs)	Maralixibat	-0.7	Standard Deviation 0.65	scores on a scale
Change From Baseline to Week 13/ET in Pruritus as Measured by ItchRO(Obs)	PFIC1	-0.8	Standard Deviation 0.83	scores on a scale
Change From Baseline to Week 13/ET in Pruritus as Measured by ItchRO(Obs)	PFIC2 (Overall)	-0.7	Standard Deviation 0.59	scores on a scale
Change From Baseline to Week 13/ET in Pruritus as Measured by ItchRO(Pt)	Maralixibat	-0.6	Standard Deviation 0.57	scores on a scale
Change From Baseline to Week 13/ET in Pruritus as Measured by ItchRO(Pt)	PFIC1	-0.4	Standard Deviation 0.65	scores on a scale
Change From Baseline to Week 13/ET in Pruritus as Measured by ItchRO(Pt)	PFIC2 (Overall)	-0.7	Standard Deviation 0.57	scores on a scale
Change From Baseline to Week 13/ET in ALT	Maralixibat	-9	Standard Deviation 61.8	U/L

1 to 10 of 18Source ClinicalTrials.gov

See all 18 results by group

Participant flow14

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

Period	Milestone	Group	Participants
Completed Study Treatment Through Wk. 72	STARTED	Progressive Familial Intrahepatic Cholestasis 1 (PFIC1)	8
Completed Study Treatment Through Wk. 72	STARTED	Progressive Familial Intrahepatic Cholestasis 2 (PFIC2) (Overall)	25
Completed Study Treatment Through Wk. 72	Completed Study Treatment Through Week 72	Progressive Familial Intrahepatic Cholestasis 1 (PFIC1)	6
Completed Study Treatment Through Wk. 72	Completed Study Treatment Through Week 72	Progressive Familial Intrahepatic Cholestasis 2 (PFIC2) (Overall)	16
Completed Study Treatment Through Wk. 72	COMPLETED	Progressive Familial Intrahepatic Cholestasis 1 (PFIC1)	6
Completed Study Treatment Through Wk. 72	COMPLETED	Progressive Familial Intrahepatic Cholestasis 2 (PFIC2) (Overall)	16
Completed Study Treatment Through Wk. 72	NOT COMPLETED	Progressive Familial Intrahepatic Cholestasis 1 (PFIC1)	2
Completed Study Treatment Through Wk. 72	NOT COMPLETED	Progressive Familial Intrahepatic Cholestasis 2 (PFIC2) (Overall)	9
Optional Follow-up Treatment Period	STARTED	Progressive Familial Intrahepatic Cholestasis 1 (PFIC1)	6
Optional Follow-up Treatment Period	STARTED	Progressive Familial Intrahepatic Cholestasis 2 (PFIC2) (Overall)	16

1 to 10 of 14Source ClinicalTrials.gov

See all 14 participant flow

Adverse events236

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	Safety Population	1	33	1
other	Spider vein	Vascular disorders	Safety Population	1	33	1
other	Biliary-tract operation	Surgical and medical procedures	Safety Population	1	33	1
other	Seasonal allergy	Immune system disorders	Safety Population	3	33	7
other	Pyrexia	General disorders	Safety Population	20	33	83
other	Fatigue	General disorders	Safety Population	4	33	4
other	Influenza like illness	General disorders	Safety Population	3	33	3
other	Malaise	General disorders	Safety Population	3	33	4
other	Chest pain	General disorders	Safety Population	2	33	4
other	Disease progression	General disorders	Safety Population	2	33	2

1 to 10 of 236Source ClinicalTrials.gov

See all 236 adverse events

Adverse event totals3

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Safety Population	0	33
Total, other adverse events	Safety Population	33	33
Total, serious adverse events	Safety Population	15	33

Source ClinicalTrials.gov

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
35507739	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.

GET https://conductscience.org/api/trials/v1/NCT02057718

GET https://conductscience.org/api/trials/v1/NCT02057718/events?page=2

API reference For AI assistants