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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT04718961




Registration number
NCT04718961
Ethics application status
Date submitted
19/01/2021
Date registered
22/01/2021
Date last updated
8/02/2023

Titles & IDs
Public title
A Placebo-controlled Study of Volixibat in Subjects With Elevated Serum Bile Acids Associated With Intrahepatic Cholestasis of Pregnancy (OHANA)
Scientific 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).
Secondary ID [1] 0 0
2020-003448-96
Secondary ID [2] 0 0
VLX-401
Universal Trial Number (UTN)
Trial acronym
OHANA
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Intrahepatic Cholestasis of Pregnancy 0 0
Condition category
Condition code
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Reproductive Health and Childbirth 0 0 0 0
Fetal medicine and complications of pregnancy

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Volixibat
Treatment: Drugs - Placebo

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.

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.


Treatment: Drugs: Volixibat
Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.

Treatment: Drugs: Placebo
Capsules matched to study drug minus active substance.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Mean change in serum bile acids
Timepoint [1] 0 0
Through to end of treatment, up to 21 weeks
Secondary outcome [1] 0 0
Mean change in the weekly average worst daily itch score as measured by the Adult Itch Reported Outcome (ItchRO)
Timepoint [1] 0 0
Through to end of treatment, up to 21 weeks
Secondary outcome [2] 0 0
Proportion of participants experiencing one or more of adverse perinatal outcomes
Timepoint [2] 0 0
At least one month after delivery.
Secondary outcome [3] 0 0
Safety endpoint: The incidence of adverse events
Timepoint [3] 0 0
Through to end of treatment, up to 25 weeks

Eligibility
Key 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.
Minimum age
18 Years
Maximum age
45 Years
Sex
Females
Can healthy volunteers participate?
No
Key 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
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Terminated
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
4/01/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
7/12/2022
Sample size
Target
Accrual to date
Final
4
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
Connecticut
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Ohio
Country [5] 0 0
United States of America
State/province [5] 0 0
Texas
Country [6] 0 0
New Zealand
State/province [6] 0 0
Otago
Country [7] 0 0
New Zealand
State/province [7] 0 0
Christchurch
Country [8] 0 0
New Zealand
State/province [8] 0 0
Wellington
Country [9] 0 0
United Kingdom
State/province [9] 0 0
Kent
Country [10] 0 0
United Kingdom
State/province [10] 0 0
West Yorkshire
Country [11] 0 0
United Kingdom
State/province [11] 0 0
Birmingham
Country [12] 0 0
United Kingdom
State/province [12] 0 0
Cardiff
Country [13] 0 0
United Kingdom
State/province [13] 0 0
Chichester
Country [14] 0 0
United Kingdom
State/province [14] 0 0
London
Country [15] 0 0
United Kingdom
State/province [15] 0 0
Middlesex
Country [16] 0 0
United Kingdom
State/province [16] 0 0
Newcastle Upon Tyne

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Mirum Pharmaceuticals, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT04718961
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT04718961