Back to resultsA Placebo-controlled Study of Volixibat in Subjects With Elevated Serum Bile Acids Associated With Intrahepatic Cholestasis of Pregnancy (OHANA) (OHANA)
Primary Purpose
Intrahepatic Cholestasis of Pregnancy
Status
Terminated
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Volixibat
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Intrahepatic Cholestasis of Pregnancy focused on measuring Pruritus, Itch, Itching, Cholestasis, Bile Acid, Pregnancy, Pregnant, Intrahepatic, Liver, ICP
Eligibility Criteria
Inclusion Criteria:
- Female aged ≥18 and ≤45 years with a viable pregnancy.
- Provide signed informed consent as described in the protocol and willing to comply with all study visits and requirements.
- Diagnosis of ICP.
- (Part 2 only) Qualified level of pruritus associated with ICP, during screening.
Exclusion Criteria:
- 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.
- Known non-reassuring fetal status based upon antepartum testing (e.g., NST/CTG or BPP) at or within 7 days before the baseline visit.
- Known fetal anomaly likely to result in intrauterine fetal demise or neonatal death within the first 30 days of life.
- Participating in another ongoing interventional clinical study at screening or planning to participate in another contemporaneous interventional clinical study while participating in this study.
Sites / Locations
- University of Alabama at Birmingham
- Yale School of Medicine
- University of Miami
- The Ohio State University Wexner Medical Center
- The University of Texas Medical Branch - Galveston
- University of Texas Health Science Center
- University of Texas Health Science Center at San Antonio
- Dunedin Hospital
- Christchurch Women's Hospital
- Capital & Coast District Health Board, Wellington Regional Hospital
- Medway NHS Foundation Trust
- Bradford Royal Infirmary
- Birmingham Womens and Childrens NHS Foundation Trust
- University Hospital of Wales
- St Richard's Hospital
- Barts Health NHS Trust- Whipps Cross University Hospital
- Royal Free London Hospital NHS Foundation Trust
- Guy's and St Thomas' NHS Foundation Trust
- West Middlesex University Hospital
- The Newcastle upon Tyne Hospitals NHS Foundation Trust
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Experimental
Arm Label
Part 1 Arm 1 & Arm 2 - Volixibat 20mg/80mg
Part 2 Arm 1 - Volixibat Selected Dose mg
Arm Description
Part 1 Arm 1 - Volixibat 20mg (Experimental) Participants randomized to this arm will receive volixibat 20mg twice daily. Part 1 Arm 2 - Volixibat 80mg (Experimental) Participants randomized to this arm will receive volixibat 80mg twice daily.
Part 2 Arm 1 - Volixibat Selected Dose mg (experimental) Participants randomized to this arm will receive volixibat selected dose (mg) twice daily. 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.
Outcomes
Primary Outcome Measures
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
Secondary Outcome Measures
Mean change in the weekly average worst daily itch score as measured by the Adult Itch Reported Outcome (ItchRO)
Proportion of participants experiencing one or more of adverse perinatal outcomes
Safety endpoint: The incidence of adverse events
Full Information
NCT ID
NCT04718961
First Posted
January 19, 2021
Last Updated
February 3, 2023
Sponsor
Mirum Pharmaceuticals, Inc.
1. Study Identification
Unique Protocol Identification Number
NCT04718961
Brief Title
A Placebo-controlled Study of Volixibat in Subjects With Elevated Serum Bile Acids Associated With Intrahepatic Cholestasis of Pregnancy (OHANA)
Acronym
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).
Study Type
Interventional
2. Study Status
Record Verification Date
February 2023
Overall Recruitment Status
Terminated
Why Stopped
Due to company's assessment of enrollment feasibility.
Study Start Date
January 4, 2021 (Actual)
Primary Completion Date
December 7, 2022 (Actual)
Study Completion Date
December 7, 2022 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Mirum Pharmaceuticals, Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
5. Study Description
Brief 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Intrahepatic Cholestasis of Pregnancy
Keywords
Pruritus, Itch, Itching, Cholestasis, Bile Acid, Pregnancy, Pregnant, Intrahepatic, Liver, ICP
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Model Description
Randomized Open-label two arm study (Part 1) followed by a randomized double-blind, placebo controlled, study (Part 2)
Masking
ParticipantInvestigator
Masking Description
Masking in Part 2 Only
Allocation
Randomized
Enrollment
4 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Part 1 Arm 1 & Arm 2 - Volixibat 20mg/80mg
Arm Type
Experimental
Arm Description
Part 1 Arm 1 - Volixibat 20mg (Experimental) Participants randomized to this arm will receive volixibat 20mg twice daily.
Part 1 Arm 2 - Volixibat 80mg (Experimental) Participants randomized to this arm will receive volixibat 80mg twice daily.
Arm Title
Part 2 Arm 1 - Volixibat Selected Dose mg
Arm Type
Experimental
Arm Description
Part 2 Arm 1 - Volixibat Selected Dose mg (experimental) Participants randomized to this arm will receive volixibat selected dose (mg) twice daily.
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.
Intervention Type
Drug
Intervention Name(s)
Volixibat
Other Intervention Name(s)
SHP626
Intervention Description
Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Capsules matched to study drug minus active substance.
Primary Outcome Measure Information:
Title
Mean change in serum bile acids
Description
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
Time Frame
Through to end of treatment, up to 21 weeks
Secondary Outcome Measure Information:
Title
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
Title
Proportion of participants experiencing one or more of adverse perinatal outcomes
Time Frame
At least one month after delivery.
Title
Safety endpoint: The incidence of adverse events
Time Frame
Through to end of treatment, up to 25 weeks
10. Eligibility
Sex
Female
Gender Based
Yes
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
45 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Female aged ≥18 and ≤45 years with a viable pregnancy.
Provide signed informed consent as described in the protocol and willing to comply with all study visits and requirements.
Diagnosis of ICP.
(Part 2 only) Qualified level of pruritus associated with ICP, during screening.
Exclusion Criteria:
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.
Known non-reassuring fetal status based upon antepartum testing (e.g., NST/CTG or BPP) at or within 7 days before the baseline visit.
Known fetal anomaly likely to result in intrauterine fetal demise or neonatal death within the first 30 days of life.
Participating in another ongoing interventional clinical study at screening or planning to participate in another contemporaneous interventional clinical study while participating in this study.
Facility Information:
Facility Name
University of Alabama at Birmingham
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35294
Country
United States
Facility Name
Yale School of Medicine
City
New Haven
State/Province
Connecticut
ZIP/Postal Code
06511
Country
United States
Facility Name
University of Miami
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
The Ohio State University Wexner Medical Center
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43210
Country
United States
Facility Name
The University of Texas Medical Branch - Galveston
City
Galveston
State/Province
Texas
ZIP/Postal Code
77555
Country
United States
Facility Name
University of Texas Health Science Center
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
University of Texas Health Science Center at San Antonio
City
San Antonio
State/Province
Texas
ZIP/Postal Code
78229
Country
United States
Facility Name
Dunedin Hospital
City
Dunedin
State/Province
Otago
ZIP/Postal Code
6021
Country
New Zealand
Facility Name
Christchurch Women's Hospital
City
Christchurch
ZIP/Postal Code
8011
Country
New Zealand
Facility Name
Capital & Coast District Health Board, Wellington Regional Hospital
City
Wellington
ZIP/Postal Code
6021
Country
New Zealand
Facility Name
Medway NHS Foundation Trust
City
Gillingham
State/Province
Kent
ZIP/Postal Code
ME7 5NY
Country
United Kingdom
Facility Name
Bradford Royal Infirmary
City
Bradford
State/Province
West Yorkshire
ZIP/Postal Code
BD9 6RJ
Country
United Kingdom
Facility Name
Birmingham Womens and Childrens NHS Foundation Trust
City
Birmingham
ZIP/Postal Code
B15 2TG
Country
United Kingdom
Facility Name
University Hospital of Wales
City
Cardiff
ZIP/Postal Code
CF14 4XW
Country
United Kingdom
Facility Name
St Richard's Hospital
City
Chichester
ZIP/Postal Code
PO19 6SE
Country
United Kingdom
Facility Name
Barts Health NHS Trust- Whipps Cross University Hospital
City
London
ZIP/Postal Code
E11 1NR
Country
United Kingdom
Facility Name
Royal Free London Hospital NHS Foundation Trust
City
London
ZIP/Postal Code
NW3 2QG
Country
United Kingdom
Facility Name
Guy's and St Thomas' NHS Foundation Trust
City
London
ZIP/Postal Code
SE 1 7EH
Country
United Kingdom
Facility Name
West Middlesex University Hospital
City
Middlesex
ZIP/Postal Code
TW7 6AF
Country
United Kingdom
Facility Name
The Newcastle upon Tyne Hospitals NHS Foundation Trust
City
Newcastle Upon Tyne
Country
United Kingdom
12. IPD Sharing Statement
Learn more about this trial
A Placebo-controlled Study of Volixibat in Subjects With Elevated Serum Bile Acids Associated With Intrahepatic Cholestasis of Pregnancy (OHANA)
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