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

Intrahepatic Cholestasis of Pregnancy Clinical Trial

— OHANA  

Official title:

A Phase 2a/2b Randomized Double-Blind Placebo-Controlled Study to Evaluate the Efficacy and Safety of Volixibat in Adult Women With Intrahepatic Cholestasis of Pregnancy and Elevated Serum Bile Acid Concentrations (OHANA).

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT04718961
• Other study ID #: VLX-401
• Secondary ID: 2020-003448-96
• Status: Terminated
• Phase: Phase 2
• Start date: January 4, 2021
• Est. completion date: December 7, 2022

Study information

• Verified date: February 2023
• Source: Mirum Pharmaceuticals, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This is a two-part randomized study of volixibat in patients with Intrahepatic Cholestasis of Pregnancy (ICP) and elevated serum bile acid concentrations (sBA). Part 1 is an open-label study 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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 4
• Est. completion date: December 7, 2022
• Est. primary completion date: December 7, 2022
• Accepts healthy volunteers: No
• Gender: Female
• Age group: 18 Years to 45 Years

Eligibility

Inclusion Criteria:

1. Female aged =18 and =45 years with a viable pregnancy.

2. Provide signed informed consent as described in the protocol and willing to comply with all study visits and requirements.

3. Diagnosis of ICP.

4. (Part 2 only) Qualified level of pruritus associated with ICP, during screening.

Exclusion Criteria:

1. At the time of either the screening or baseline visit, decision has already been made to deliver within the next 7 days, for any indication.

2. Known non-reassuring fetal status based upon antepartum testing (e.g., NST/CTG or BPP) at or within 7 days before the baseline visit.

3. Known fetal anomaly likely to result in intrauterine fetal demise or neonatal death within the first 30 days of life.

4. Participating in another ongoing interventional clinical study at screening or planning to participate in another contemporaneous interventional clinical study while participating in this study.

Related Conditions & MeSH terms

• Cholestasis
• Cholestasis, Intrahepatic
• Intrahepatic Cholestasis of Pregnancy
• Pregnancy Complications

Intervention

Drug:
• Volixibat
Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.
• Placebo
Capsules matched to study drug minus active substance.

Locations

Country	Name	City	State
New Zealand	Christchurch Women's Hospital	Christchurch
New Zealand	Dunedin Hospital	Dunedin	Otago
New Zealand	Capital & Coast District Health Board, Wellington Regional Hospital	Wellington
United Kingdom	Birmingham Womens and Childrens NHS Foundation Trust	Birmingham
United Kingdom	Bradford Royal Infirmary	Bradford	West Yorkshire
United Kingdom	University Hospital of Wales	Cardiff
United Kingdom	St Richard's Hospital	Chichester
United Kingdom	Medway NHS Foundation Trust	Gillingham	Kent
United Kingdom	Barts Health NHS Trust- Whipps Cross University Hospital	London
United Kingdom	Guy's and St Thomas' NHS Foundation Trust	London
United Kingdom	Royal Free London Hospital NHS Foundation Trust	London
United Kingdom	West Middlesex University Hospital	Middlesex
United Kingdom	The Newcastle upon Tyne Hospitals NHS Foundation Trust	Newcastle Upon Tyne
United States	University of Alabama at Birmingham	Birmingham	Alabama
United States	The Ohio State University Wexner Medical Center	Columbus	Ohio
United States	The University of Texas Medical Branch - Galveston	Galveston	Texas
United States	University of Texas Health Science Center	Houston	Texas
United States	University of Miami	Miami	Florida
United States	Yale School of Medicine	New Haven	Connecticut
United States	University of Texas Health Science Center at San Antonio	San Antonio	Texas

Sponsors (1)

Lead Sponsor	Collaborator
Mirum Pharmaceuticals, Inc.

Countries where clinical trial is conducted

United States,  New Zealand,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Mean change in serum bile acids	Part 1: To assess the safety and tolerability of volixibat in participants with ICP on the basis of the following endpoints; Part 2: To assess the efficacy of volixibat on the reduction of elevated sBA concentrations in participants with ICP	Through to end of treatment, up to 21 weeks
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
Secondary	Proportion of participants experiencing one or more of adverse perinatal outcomes		At least one month after delivery.
Secondary	Safety endpoint: The incidence of adverse events		Through to end of treatment, up to 25 weeks