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

Mirum Pharmaceuticals

Status and phase: Terminated; Phase 2

Conditions: Intrahepatic Cholestasis of Pregnancy

Treatments: Drug: Placebo; Drug: Volixibat

Study type: Interventional

Funder types: Industry

Identifiers

NCT04718961

VLX-401

2020-003448-96 (EudraCT Number)

Details and patient eligibility

About

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.

Enrollment: 4 patients

Sex: Female

Ages: 18 to 45 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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4 participants in 4 patient groups, including a placebo group

Part 1 Arm 1 - Volixibat 20mg

Experimental group

Description:

Participants randomized to this arm will receive volixibat 20mg twice daily.

Treatment:

Drug: Volixibat

Part 1 Arm 2 - Volixibat 80mg

Experimental group

Description:

Participants randomized to this arm will receive volixibat 80mg twice daily.

Treatment:

Drug: Volixibat

Part 2 Arm 1 - Volixibat Selected Dose mg

Experimental group

Description:

Participants randomized to this arm will receive volixibat selected dose (mg) twice daily.

Treatment:

Drug: Volixibat

Part 2 Arm 2 - Placebo (Placebo Comparator)

Placebo Comparator group

Description:

Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily.

Treatment:

Drug: Placebo