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

Purpose

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.

Condition

  • Intrahepatic Cholestasis of Pregnancy

Eligibility

Eligible Ages
Between 18 Years and 45 Years
Eligible Genders
Female
Accepts Healthy Volunteers
No

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

Phase
Phase 2
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Intervention Model Description
Randomized Open-label two arm study (Part 1) followed by a randomized double-blind, placebo controlled, study (Part 2)
Primary Purpose
Treatment
Masking
Double (Participant, Investigator)
Masking Description
Masking in Part 2 Only

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Part 1 Arm 1 & Arm 2 - Volixibat 20mg/80mg
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.
  • 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
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.
  • Drug: Volixibat
    Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.
    Other names:
    • SHP626
  • Drug: Placebo
    Capsules matched to study drug minus active substance.

Recruiting Locations

University of Texas Health Science Center at San Antonio
San Antonio, Texas 78229
Contact:
Marisa Fernandez
210-450-8016
fernandezm10@uthscsa.edu

More Details

Status
Recruiting
Sponsor
Mirum Pharmaceuticals, Inc.

Study Contact

Clinical Trials Mirum
+16506674085
clinicaltrials@mirumpharma.com