A Placebo-controlled Study of Volixibat in Subjects With Elevated Serum Bile Acids Associated With Intrahepatic Cholestasis of Pregnancy (OHANA)
- Conditions
- Intrahepatic Cholestasis of Pregnancy
- Interventions
- Drug: Placebo
- Registration Number
- NCT04718961
- Lead Sponsor
- Mirum Pharmaceuticals, Inc.
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- Female
- Target Recruitment
- 4
- 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.
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Part 1 Arm 2 - Volixibat 80mg Volixibat Participants randomized to this arm will receive volixibat 80mg twice daily. Part 2 Arm 2 - Placebo (Placebo Comparator) Placebo Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily. Part 1 Arm 1 - Volixibat 20mg Volixibat Participants randomized to this arm will receive volixibat 20mg twice daily. Part 2 Arm 1 - Volixibat Selected Dose mg Volixibat Participants randomized to this arm will receive volixibat selected dose (mg) twice daily.
- Primary Outcome Measures
Name Time Method 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 Outcome Measures
Name Time Method 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?"
Proportion of Participants Experiencing One or More of Adverse Perinatal Outcomes At least one month after delivery.
Trial Locations
- Locations (20)
The University of Texas Medical Branch - Galveston
🇺🇸Galveston, Texas, United States
University of Miami
🇺🇸Miami, Florida, United States
University of Alabama at Birmingham
🇺🇸Birmingham, Alabama, United States
University of Texas Health Science Center at San Antonio
🇺🇸San Antonio, Texas, United States
Christchurch Women's Hospital
🇳🇿Christchurch, New Zealand
Medway NHS Foundation Trust
🇬🇧Gillingham, Kent, United Kingdom
Yale School of Medicine
🇺🇸New Haven, Connecticut, United States
The Ohio State University Wexner Medical Center
🇺🇸Columbus, Ohio, United States
University of Texas Health Science Center
🇺🇸Houston, Texas, United States
Dunedin Hospital
🇳🇿Dunedin, Otago, New Zealand
Bradford Royal Infirmary
🇬🇧Bradford, West Yorkshire, United Kingdom
University Hospital of Wales
🇬🇧Cardiff, United Kingdom
Birmingham Womens and Childrens NHS Foundation Trust
🇬🇧Birmingham, United Kingdom
St Richard's Hospital
🇬🇧Chichester, United Kingdom
Barts Health NHS Trust- Whipps Cross University Hospital
🇬🇧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 Kingdom
Capital & Coast District Health Board, Wellington Regional Hospital
🇳🇿Wellington, New Zealand
Guy's and St Thomas' NHS Foundation Trust
🇬🇧London, United Kingdom