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

Phase 2

Terminated

• Conditions: Intrahepatic Cholestasis of Pregnancy
• Interventions: Drug: Volixibat; 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

Study Timeline

• Study Start: 2021-01-04
• Study First Submitted: 2021-01-19
• Study First Submitted QC: 2021-01-19
• Study First Posted: 2021-01-22
• Primary Completion: 2022-12-07
• Study Completion: 2022-12-07
• Results First Submitted: 2023-11-30
• Status Verified: 2024-07
• Results First Submitted QC: 2024-07-11
• Last Update Submitted: 2024-07-11
• Results First Posted: 2024-08-06
• Last Update Posted: 2024-08-06

Recruitment & Eligibility

• Status: TERMINATED
• Sex: Female
• Target Recruitment: 4

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 & 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)

University of Alabama at Birmingham; 🇺🇸 Birmingham, Alabama, United States

Yale School of Medicine; 🇺🇸 New Haven, Connecticut, United States

University of Miami; 🇺🇸 Miami, Florida, 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 Texas Health Science Center at San Antonio; 🇺🇸 San Antonio, Texas, United States

Dunedin Hospital; 🇳🇿 Dunedin, Otago, New Zealand

Christchurch Women's Hospital; 🇳🇿 Christchurch, New Zealand

Capital & Coast District Health Board, Wellington Regional Hospital; 🇳🇿 Wellington, New Zealand

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