NCT04718961: OHANA

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

Terminated Phase 2 Results posted Last updated 6 August 2024

What this trial tests

Phase 2 trial testing Volixibat in Intrahepatic Cholestasis of Pregnancy in 4 participants. Terminated before completion.

Timeline

4 January 2021

Primary endpoint
7 December 2022

7 December 2022

Quick facts

Lead sponsor	Mirum Pharmaceuticals, Inc.
Phase	Phase 2
Status	Terminated
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	double
Primary purpose	treatment
Enrollment	4
Start date	4 January 2021
Primary completion	7 December 2022
Estimated completion	7 December 2022
Sites	20 locations across New Zealand, United Kingdom, United States

Drugs / interventions tested

• Volixibat — full drug profile →
• Placebo

Conditions studied

• Intrahepatic Cholestasis of Pregnancy — all drugs for Intrahepatic Cholestasis of Pregnancy →

Sponsor

Mirum Pharmaceuticals, Inc. — full company profile →

Who can join

Adults 18 to 45, female only, with Intrahepatic Cholestasis of Pregnancy. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Assess the Safety and Tolerability of Volixibat in Participants With ICP Primary · 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

Group	Value	95% CI
Part 1 Arm 1 - Volixibat 20mg (Experimental)	2
Part 1 Arm 2 - Volixibat 80mg (Experimental)	2

Mean Change in the Weekly Average Worst Daily Itch Score as Measured by the Adult Itch Reported Outcome (ItchRO) Secondary · 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?"

Group	Value	95% CI
Part 1 Arm 1 - Volixibat 20mg (Experimental)	-2.5	± 2.40
Part 1 Arm 2 - Volixibat 80mg (Experimental)	1.3	± 2.31

Proportion of Participants Experiencing One or More of Adverse Perinatal Outcomes Secondary · At least one month after delivery.

Group	Value	95% CI
Part 1 Arm 1 - Volixibat 20mg (Experimental)	0
Part 1 Arm 2 - Volixibat 80mg (Experimental)	2

Adverse events — posted to ClinicalTrials.gov

Time frame: All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, **up to 25 weeks**. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Part 1 Arm 1 - Volixibat 20mg (Experimental)

Serious: 1/2 (50%)

Deaths: 0/2

Part 1 Arm 2 - Volixibat 80mg (Experimental)

Serious: 0/2 (0%)

Deaths: 0/2

Part 2 Arm 1 - Volixibat Selected Dose mg (Experimental)

Serious: 0

Deaths: 0

Part 2 Arm 2 - Placebo (Placebo Comparator)

Serious: 0

Deaths: 0

Serious adverse events (1 terms)

Reaction	System	Part 1 Arm 1 - Volixibat 2…	Part 1 Arm 2 - Volixibat 8…	Part 2 Arm 1 - Volixibat S…	Part 2 Arm 2 - Placebo (Pl…
Diarrhoea	Gastrointestinal disorders	—	—	—	—

Other adverse events (16 terms — click to expand)

Reaction	System	Part 1 Arm 1 - Volixibat 2…	Part 1 Arm 2 - Volixibat 8…	Part 2 Arm 1 - Volixibat S…	Part 2 Arm 2 - Placebo (Pl…
Diarrhoea	Gastrointestinal disorders	—	—	—	—
Anemia	Blood and lymphatic system disorders	—	—	—	—
Anxiety	Psychiatric disorders	—	—	—	—
Apnoic Episodes	Respiratory, thoracic and mediastinal disorders	—	—	—	—
Gastroenteritis	Infections and infestations	—	—	—	—
Headache	Nervous system disorders	—	—	—	—
Heart Burn	Gastrointestinal disorders	—	—	—	—
Intermittent Abdominal Pain	Gastrointestinal disorders	—	—	—	—
Intermittent Diarrhoea	Gastrointestinal disorders	—	—	—	—
Intermittent Vomiting	Gastrointestinal disorders	—	—	—	—
Mild Diarrhoea	Gastrointestinal disorders	—	—	—	—
Postnatal Depression	Psychiatric disorders	—	—	—	—
Premature Labor	Pregnancy, puerperium and perinatal conditions	—	—	—	—
Raised Blood Pressure	Investigations	—	—	—	—
Threated Pre-Term Labor	Pregnancy, puerperium and perinatal conditions	—	—	—	—
Vomiting	Gastrointestinal disorders	—	—	—	—

Most-reported serious reactions: Diarrhoea.

Data from ClinicalTrials.gov NCT04718961 adverse events section.

Sponsor's own description

Part 1 is an open-label randomized study of volixibat in patients with Intrahepatic Cholestasis of Pregnancy (ICP) and elevated serum bile acid concentrations (sBA) to evaluate safety and tolerability of two doses of volixibat. Part 2 is a double-blind, placebo controlled, study designed to evaluate the safety and efficacy of a selected volixibat dose.

Publications & conference data

3 peer-reviewed publications reference this trial (live from Europe PMC):

1. Bile acid-mediated signaling in cholestatic liver diseases.
   Zeng J, Fan J, Zhou H. · · 2023 · cited 39× · PMID 37120573 · DOI 10.1186/s13578-023-01035-1
2. Efficacy, Safety and Tolerability of Volixibat, an IBAT Inhibitor, in Patients With Intrahepatic Cholestasis of Pregnancy.
   Ovadia C, Stone S, Sibai B, Nunes T, et al · · 2026 · cited 1× · PMID 41603551 · DOI 10.1111/liv.70523
3. Pediatric Cholestatic Diseases in the Era of Ileal Bile Acid Transporter (IBAT) Inhibitors.
   Sciveres M, Veraldi S, Cirillo F, Maggiore G. · · 2026 · PMID 41718432 · DOI 10.3390/pediatric18010019