Trial · NCT04168385

MRX-800: A Long-Term Safety Study of Maralixibat in the Treatment of Cholestatic Liver Disease in Subjects Who Previously Participated in a Maralixibat Study

NCT04168385, Phase 2, Completed, Mirum Pharmaceuticals, Inc.; results first posted 2025-12-08

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. MRX-800: A Long-Term Safety Study of Maralixibat in the Treatment of Cholestatic Liver Disease in Subjects Who Previously Participated in a Maralixibat Study. Trials, record NCT04168385. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04168385

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Trial

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

Title: MRX-800: A Long-Term Safety Study of Maralixibat in the Treatment of Cholestatic Liver Disease in Subjects Who Previously Participated in a Maralixibat Study
NCT number: NCT04168385
Status: Completed
Phases: Phase 2
Enrollment: 52 participants, actual
Lead sponsor: Mirum Pharmaceuticals, Inc.
Conditions: Cholestatic Liver Disease
Interventions: Maralixibat
First posted: 2019-11-19
Last updated: 2025-12-08
Results first posted: 2025-12-08

Source ClinicalTrials.gov

Registered outcomes3

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

List	Outcome	Time frame	Description
Primary	Incidence of Treatment-Emergent Adverse Events	From informed consent through approximately 4.5 years, including 30 days after last dose.	TEAE = Treatment-emergent Adverse Event; AESI = Adverse Event of Special Interest..
Secondary	Long-Term Effect on Pruritus	From Baseline through Week 160, including Change from Baseline values.	Change from Baseline in Pruritus Severity assessed using the Clinician Scratch Score (CSS), a 5-point scale where 0 indicates no evidence of scratching and 4 indicates cutaneous mutilation with bleeding, hemorrhage, and scarring.
Secondary	Long-Term Effect on Serum Bile Acid Levels	From Baseline through Week 160, including Change from Baseline values.

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	Incidence of Treatment-Emergent Adverse Events	From informed consent through approximately 4.5 years, including 30 days after last dose.	Includes all participants with Alagille Syndrome (ALGS) or Progressive Familial Intrahepatic Cholestasis (PFIC) who received at least one dose of maralixibat.	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Long-Term Effect on Pruritus	From Baseline through Week 160, including Change from Baseline values.	Includes all participants with Alagille Syndrome (ALGS) or Progressive Familial Intrahepatic Cholestasis (PFIC) who received at least one dose of maralixibat.	score on a scale	MEAN
SECONDARY	Long-Term Effect on Serum Bile Acid Levels	From Baseline through Week 160, including Change from Baseline values.	Includes all participants with Alagille Syndrome (ALGS) or Progressive Familial Intrahepatic Cholestasis (PFIC) who received at least one dose of maralixibat.	µmol/L	MEAN

Source ClinicalTrials.gov

Results by group21

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

Outcome	Group	Category	Value	Units
Incidence of Treatment-Emergent Adverse Events	Maralixibat: ALGS	At least one TEAE	36	Participants
Incidence of Treatment-Emergent Adverse Events	Maralixibat: PFIC	At least one TEAE	12	Participants
Incidence of Treatment-Emergent Adverse Events	Maralixibat: ALGS	TEAE ≥ 3	9	Participants
Incidence of Treatment-Emergent Adverse Events	Maralixibat: PFIC	TEAE ≥ 3	4	Participants
Incidence of Treatment-Emergent Adverse Events	Maralixibat: ALGS	Serious TEAE	10	Participants
Incidence of Treatment-Emergent Adverse Events	Maralixibat: PFIC	Serious TEAE	3	Participants
Incidence of Treatment-Emergent Adverse Events	Maralixibat: ALGS	Serious Treatment-Related Adverse Event	0	Participants
Incidence of Treatment-Emergent Adverse Events	Maralixibat: PFIC	Serious Treatment-Related Adverse Event	0	Participants
Incidence of Treatment-Emergent Adverse Events	Maralixibat: ALGS	Serious Treatment-Related TEAE	0	Participants
Incidence of Treatment-Emergent Adverse Events	Maralixibat: PFIC	Serious Treatment-Related TEAE	0	Participants

1 to 10 of 21Source ClinicalTrials.gov

See all 21 results by group

Participant flow6

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

Period	Milestone	Group	Participants
Overall Study	STARTED	Maralixibat: ALGS	40
Overall Study	STARTED	Maralixibat: PFIC	12
Overall Study	COMPLETED	Maralixibat: ALGS	33
Overall Study	COMPLETED	Maralixibat: PFIC	10
Overall Study	NOT COMPLETED	Maralixibat: ALGS	7
Overall Study	NOT COMPLETED	Maralixibat: PFIC	2

Source ClinicalTrials.gov

Adverse events226

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	Splenomegaly	Blood and lymphatic system disorders	Maralixibat: ALGS	3	40	4
other	Splenomegaly	Blood and lymphatic system disorders	Maralixibat: PFIC	0	12	0
other	Ear pain	Ear and labyrinth disorders	Maralixibat: ALGS	2	40	3
other	Ear pain	Ear and labyrinth disorders	Maralixibat: PFIC	0	12	0
other	Abdominal pain	Gastrointestinal disorders	Maralixibat: ALGS	6	40	10
other	Abdominal pain	Gastrointestinal disorders	Maralixibat: PFIC	3	12	7
other	Abdominal pain upper	Gastrointestinal disorders	Maralixibat: ALGS	2	40	2
other	Abdominal pain upper	Gastrointestinal disorders	Maralixibat: PFIC	1	12	1
other	Aphthous ulcer	Gastrointestinal disorders	Maralixibat: ALGS	0	40	0
other	Aphthous ulcer	Gastrointestinal disorders	Maralixibat: PFIC	1	12	1

1 to 10 of 226Source ClinicalTrials.gov

See all 226 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Maralixibat: ALGS	1	40
Total, all-cause mortality	Maralixibat: PFIC	0	12
Total, other adverse events	Maralixibat: ALGS	36	40
Total, other adverse events	Maralixibat: PFIC	12	12
Total, serious adverse events	Maralixibat: ALGS	10	40
Total, serious adverse events	Maralixibat: PFIC	3	12

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 comparisons2

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
Maralixibat: ALGS vs Maralixibat: PFIC: deaths from any cause	Trial comparisons
Maralixibat: ALGS vs Maralixibat: PFIC: serious adverse events	Trial comparisons

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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