Clinical trial NCT04718961: A Placebo-controlled Study of Volixibat in Subjects With Elevated Serum Bile Acids Associated With Intrahepatic Cholestasis of Pregnancy (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
Overall status: TERMINATED
Phase: PHASE2
Conditions: Intrahepatic Cholestasis of Pregnancy
Keywords: Pruritus
Itch
Itching
Cholestasis
Bile Acid
Pregnancy
Pregnant
Intrahepatic
Liver
ICP
Lead sponsor: Mirum Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2021-01-22
Enrollment: 4
Sex: FEMALE
Minimum age (years): 18
Maximum age (years): 45
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
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

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Assess the Safety and Tolerability of Volixibat in Participants With ICP
timeFrame: Through to end of treatment, up to 21 weeks
Secondary outcomes:
measure: Mean Change in the Weekly Average Worst Daily Itch Score as Measured by the Adult Itch Reported Outcome (ItchRO)
timeFrame: Through to end of treatment, up to 21 weeks
measure: Proportion of Participants Experiencing One or More of Adverse Perinatal Outcomes
timeFrame: At least one month after delivery.
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama at Birmingham
state: Alabama
city: New Haven
country: United States
facility: Yale School of Medicine
state: Connecticut
city: Miami
country: United States
facility: University of Miami
state: Florida
city: Columbus
country: United States
facility: The Ohio State University Wexner Medical Center
state: Ohio
city: Galveston
country: United States
facility: The University of Texas Medical Branch - Galveston
state: Texas
city: Houston
country: United States
facility: University of Texas Health Science Center
state: Texas
city: San Antonio
country: United States
facility: University of Texas Health Science Center at San Antonio
state: Texas
city: Dunedin
country: New Zealand
facility: Dunedin Hospital
state: Otago
city: Christchurch
country: New Zealand
facility: Christchurch Women's Hospital
city: Wellington
country: New Zealand
facility: Capital & Coast District Health Board, Wellington Regional Hospital
city: Gillingham
country: United Kingdom
facility: Medway NHS Foundation Trust
state: Kent
city: Bradford
country: United Kingdom
facility: Bradford Royal Infirmary
state: West Yorkshire
city: Birmingham
country: United Kingdom
facility: Birmingham Womens and Childrens NHS Foundation Trust
city: Cardiff
country: United Kingdom
facility: University Hospital of Wales
city: Chichester
country: United Kingdom
facility: St Richard's Hospital
city: London
country: United Kingdom
facility: Barts Health NHS Trust- Whipps Cross University Hospital
city: London
country: United Kingdom
facility: Royal Free London Hospital NHS Foundation Trust
city: London
country: United Kingdom
facility: Guy's and St Thomas' NHS Foundation Trust
city: Middlesex
country: United Kingdom
facility: West Middlesex University Hospital
city: Newcastle Upon Tyne
country: United Kingdom
facility: The Newcastle upon Tyne Hospitals NHS Foundation Trust
Study officials:
References: