Trial · NCT04718961

A Placebo-controlled Study of Volixibat in Subjects With Elevated Serum Bile Acids Associated With Intrahepatic Cholestasis of Pregnancy (OHANA)

NCT04718961, Phase 2, Terminated, Mirum Pharmaceuticals, Inc.; results first posted 2024-08-06

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. A Placebo-controlled Study of Volixibat in Subjects With Elevated Serum Bile Acids Associated With Intrahepatic Cholestasis of Pregnancy (OHANA). Trials, record NCT04718961. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04718961

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Trial

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

Title: A Placebo-controlled Study of Volixibat in Subjects With Elevated Serum Bile Acids Associated With Intrahepatic Cholestasis of Pregnancy (OHANA)
NCT number: NCT04718961
Status: Terminated
Phases: Phase 2
Enrollment: 4 participants, actual
Lead sponsor: Mirum Pharmaceuticals, Inc.
Conditions: Intrahepatic Cholestasis of Pregnancy
Interventions: Volixibat, Placebo
First posted: 2021-01-22
Last updated: 2024-08-06
Other registries' IDs: eudract:2020-003448-96
Results first posted: 2024-08-06

Source ClinicalTrials.gov

Registered outcomes3

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

List	Outcome	Time frame	Description
Primary	Assess the Safety and Tolerability of Volixibat in Participants With ICP	Through to end of treatment, up to 21 weeks	To assess the safety and tolerability of volixibat in participants with ICP on the basis of the following endpoints: Proportion of participants experiencing one or more of the following: Treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), adverse events of special interest (AESIs), events of clinical interest (ECIs), and adverse events (AEs) that lead to discontinuation of study drugs. Clinically significant laboratory abnormalities
Secondary	Mean Change in the Weekly Average Worst Daily Itch Score as Measured by the Adult Itch Reported Outcome (ItchRO)	Through to end of treatment, up to 21 weeks	Adult Itch Reported Outcome (ItchRO) is a 0 to 10 scale with 0 being "no itch" and 10 being "worst possible itch" where participants are responding to the following question "How would you rate the worst itch you experienced over the last 24hrs?"
Secondary	Proportion of Participants Experiencing One or More of Adverse Perinatal Outcomes	At least one month after delivery.

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	Assess the Safety and Tolerability of Volixibat in Participants With ICP	Through to end of treatment, up to 21 weeks	Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	participants	NUMBER
SECONDARY	Mean Change in the Weekly Average Worst Daily Itch Score as Measured by the Adult Itch Reported Outcome (ItchRO)	Through to end of treatment, up to 21 weeks	Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	units on a scale	MEAN
SECONDARY	Proportion of Participants Experiencing One or More of Adverse Perinatal Outcomes	At least one month after delivery.	Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.	Participants	COUNT_OF_PARTICIPANTS

Source ClinicalTrials.gov

Results by group6

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

Outcome	Group	Value	Spread	Units
Assess the Safety and Tolerability of Volixibat in Participants With ICP	Part 1 Arm 1 - Volixibat 20mg (Experimental)	2		participants
Assess the Safety and Tolerability of Volixibat in Participants With ICP	Part 1 Arm 2 - Volixibat 80mg (Experimental)	2		participants
Mean Change in the Weekly Average Worst Daily Itch Score as Measured by the Adult Itch Reported Outcome (ItchRO)	Part 1 Arm 1 - Volixibat 20mg (Experimental)	-2.5	Standard Deviation 2.40	units on a scale
Mean Change in the Weekly Average Worst Daily Itch Score as Measured by the Adult Itch Reported Outcome (ItchRO)	Part 1 Arm 2 - Volixibat 80mg (Experimental)	1.3	Standard Deviation 2.31	units on a scale
Proportion of Participants Experiencing One or More of Adverse Perinatal Outcomes	Part 1 Arm 1 - Volixibat 20mg (Experimental)	0		Participants
Proportion of Participants Experiencing One or More of Adverse Perinatal Outcomes	Part 1 Arm 2 - Volixibat 80mg (Experimental)	2		Participants

Source ClinicalTrials.gov

Participant flow12

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

Period	Milestone	Group	Participants
Overall Study	STARTED	Part 1 Arm 1 - Volixibat 20mg (Experimental)	2
Overall Study	STARTED	Part 1 Arm 2 - Volixibat 80mg (Experimental)	2
Overall Study	STARTED	Part 2 Arm 1 - Volixibat Selected Dose mg (Experimental)	0
Overall Study	STARTED	Part 2 Arm 2 - Placebo (Placebo Comparator)	0
Overall Study	COMPLETED	Part 1 Arm 1 - Volixibat 20mg (Experimental)	2
Overall Study	COMPLETED	Part 1 Arm 2 - Volixibat 80mg (Experimental)	2
Overall Study	COMPLETED	Part 2 Arm 1 - Volixibat Selected Dose mg (Experimental)	0
Overall Study	COMPLETED	Part 2 Arm 2 - Placebo (Placebo Comparator)	0
Overall Study	NOT COMPLETED	Part 1 Arm 1 - Volixibat 20mg (Experimental)	0
Overall Study	NOT COMPLETED	Part 1 Arm 2 - Volixibat 80mg (Experimental)	0

1 to 10 of 12Source ClinicalTrials.gov

See all 12 participant flow

Adverse events68

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	Anemia	Blood and lymphatic system disorders	Part 1 Arm 1 - Volixibat 20mg (Experimental)	1	2	1
other	Anemia	Blood and lymphatic system disorders	Part 1 Arm 2 - Volixibat 80mg (Experimental)	1	2	1
other	Anemia	Blood and lymphatic system disorders	Part 2 Arm 1 - Volixibat Selected Dose mg (Experimental)		0
other	Anemia	Blood and lymphatic system disorders	Part 2 Arm 2 - Placebo (Placebo Comparator)		0
other	Anxiety	Psychiatric disorders	Part 1 Arm 1 - Volixibat 20mg (Experimental)	0	2	0
other	Anxiety	Psychiatric disorders	Part 1 Arm 2 - Volixibat 80mg (Experimental)	1	2	1
other	Anxiety	Psychiatric disorders	Part 2 Arm 1 - Volixibat Selected Dose mg (Experimental)		0
other	Anxiety	Psychiatric disorders	Part 2 Arm 2 - Placebo (Placebo Comparator)		0
other	Apnoic Episodes	Respiratory, thoracic and mediastinal disorders	Part 1 Arm 1 - Volixibat 20mg (Experimental)	0	2	0
other	Apnoic Episodes	Respiratory, thoracic and mediastinal disorders	Part 1 Arm 2 - Volixibat 80mg (Experimental)	1	2	1

1 to 10 of 68Source ClinicalTrials.gov

See all 68 adverse events

Adverse event totals12

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Part 1 Arm 1 - Volixibat 20mg (Experimental)	0	2
Total, all-cause mortality	Part 1 Arm 2 - Volixibat 80mg (Experimental)	0	2
Total, all-cause mortality	Part 2 Arm 1 - Volixibat Selected Dose mg (Experimental)	0	0
Total, all-cause mortality	Part 2 Arm 2 - Placebo (Placebo Comparator)	0	0
Total, other adverse events	Part 1 Arm 1 - Volixibat 20mg (Experimental)	2	2
Total, other adverse events	Part 1 Arm 2 - Volixibat 80mg (Experimental)	2	2
Total, other adverse events	Part 2 Arm 1 - Volixibat Selected Dose mg (Experimental)	0	0
Total, other adverse events	Part 2 Arm 2 - Placebo (Placebo Comparator)	0	0
Total, serious adverse events	Part 1 Arm 1 - Volixibat 20mg (Experimental)	1	2
Total, serious adverse events	Part 1 Arm 2 - Volixibat 80mg (Experimental)	0	2

1 to 10 of 12Source ClinicalTrials.gov

See all 12 adverse event totals

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
41603551	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/NCT04718961

GET https://conductscience.org/api/trials/v1/NCT04718961/events?page=2

API reference For AI assistants