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

July 11, 2024 updated by: Mirum Pharmaceuticals, Inc.

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).

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.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

4

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • New Zealand
      • Christchurch, New Zealand, 8011
        • Christchurch Women's Hospital
      • Wellington, New Zealand, 6021
        • Capital & Coast District Health Board, Wellington Regional Hospital
    • Otago
      • Dunedin, Otago, New Zealand, 6021
        • Dunedin Hospital
  • United Kingdom
      • Birmingham, United Kingdom, B15 2TG
        • Birmingham Womens and Childrens NHS Foundation Trust
      • Cardiff, United Kingdom, CF14 4XW
        • University Hospital of Wales
      • Chichester, United Kingdom, PO19 6SE
        • St Richard's Hospital
      • London, United Kingdom, E11 1NR
        • Barts Health NHS Trust- Whipps Cross University Hospital
      • London, United Kingdom, NW3 2QG
        • Royal Free London Hospital NHS Foundation Trust
      • London, United Kingdom, SE 1 7EH
        • Guy's and St Thomas' NHS Foundation Trust
      • Middlesex, United Kingdom, TW7 6AF
        • West Middlesex University Hospital
      • Newcastle Upon Tyne, United Kingdom
        • The Newcastle upon Tyne Hospitals NHS Foundation Trust
    • Kent
      • Gillingham, Kent, United Kingdom, ME7 5NY
        • Medway NHS Foundation Trust
    • West Yorkshire
      • Bradford, West Yorkshire, United Kingdom, BD9 6RJ
        • Bradford Royal Infirmary
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35294
        • University of Alabama at Birmingham
    • Connecticut
      • New Haven, Connecticut, United States, 06511
        • Yale School of Medicine
    • Florida
      • Miami, Florida, United States, 33136
        • University of Miami
    • Ohio
      • Columbus, Ohio, United States, 43210
        • The Ohio State University Wexner Medical Center
    • Texas
      • Galveston, Texas, United States, 77555
        • The University of Texas Medical Branch - Galveston
      • Houston, Texas, United States, 77030
        • University of Texas Health Science Center
      • San Antonio, Texas, United States, 78229
        • University of Texas Health Science Center at San Antonio

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 45 years (Adult)

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Double

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Part 1 Arm 1 - Volixibat 20mg
Participants randomized to this arm will receive volixibat 20mg twice daily.
Drug: Volixibat
Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.
Other Names:
  • SHP626
Experimental: Part 1 Arm 2 - Volixibat 80mg
Participants randomized to this arm will receive volixibat 80mg twice daily.
Drug: Volixibat
Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.
Other Names:
  • SHP626
Experimental: Part 2 Arm 1 - Volixibat Selected Dose mg
Participants randomized to this arm will receive volixibat selected dose (mg) twice daily.
Drug: Volixibat
Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.
Other Names:
  • SHP626
Placebo Comparator: Part 2 Arm 2 - Placebo (Placebo Comparator)
Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily.
Drug: Placebo
Capsules matched to study drug minus active substance.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Assess the Safety and Tolerability of Volixibat in Participants With ICP
Time Frame: 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
Through to end of treatment, up to 21 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Mean Change in the Weekly Average Worst Daily Itch Score as Measured by the Adult Itch Reported Outcome (ItchRO)
Time Frame: 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?"
Through to end of treatment, up to 21 weeks
Proportion of Participants Experiencing One or More of Adverse Perinatal Outcomes
Time Frame: At least one month after delivery.
At least one month after delivery.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Mirum Pharmaceuticals, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 4, 2021

Primary Completion (Actual)

December 7, 2022

Study Completion (Actual)

December 7, 2022

Study Registration Dates

First Submitted

January 19, 2021

First Submitted That Met QC Criteria

January 19, 2021

First Posted (Actual)

January 22, 2021

Study Record Updates

Last Update Posted (Actual)

August 6, 2024

Last Update Submitted That Met QC Criteria

July 11, 2024

Last Verified

July 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • VLX-401
  • 2020-003448-96 (EudraCT Number)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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