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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)
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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).
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Secondary ID [1]
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2020-003448-96
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Secondary ID [2]
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VLX-401
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Universal Trial Number (UTN)
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Trial acronym
OHANA
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Intrahepatic Cholestasis of Pregnancy
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Condition category
Condition code
Oral and Gastrointestinal
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Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
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Reproductive Health and Childbirth
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Fetal medicine and complications of pregnancy
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Intervention/exposure
Study type
Interventional
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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.
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Mean change in serum bile acids
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Assessment method [1]
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Part 1: To assess the safety and tolerability of volixibat in participants with ICP on the basis of the following endpoints
Part 2: To assess the efficacy of volixibat on the reduction of elevated sBA concentrations in participants with ICP
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Timepoint [1]
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Through to end of treatment, up to 21 weeks
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Secondary outcome [1]
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Mean change in the weekly average worst daily itch score as measured by the Adult Itch Reported Outcome (ItchRO)
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Assessment method [1]
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Timepoint [1]
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Through to end of treatment, up to 21 weeks
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Secondary outcome [2]
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Proportion of participants experiencing one or more of adverse perinatal outcomes
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Assessment method [2]
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Timepoint [2]
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At least one month after delivery.
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Secondary outcome [3]
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Safety endpoint: The incidence of adverse events
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Assessment method [3]
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Timepoint [3]
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Through to end of treatment, up to 25 weeks
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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.
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Minimum age
18
Years
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Maximum age
45
Years
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Sex
Females
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Can healthy volunteers participate?
No
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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.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Terminated
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
4/01/2021
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
7/12/2022
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Sample size
Target
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Accrual to date
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Final
4
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Recruitment in Australia
Recruitment state(s)
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Recruitment outside Australia
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United States of America
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State/province [1]
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Alabama
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United States of America
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Connecticut
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United States of America
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Florida
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United States of America
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Ohio
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United States of America
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Texas
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New Zealand
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Otago
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New Zealand
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Christchurch
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New Zealand
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Wellington
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United Kingdom
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Kent
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United Kingdom
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West Yorkshire
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United Kingdom
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Birmingham
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United Kingdom
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Cardiff
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United Kingdom
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Chichester
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United Kingdom
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London
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Country [15]
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United Kingdom
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Middlesex
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Country [16]
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United Kingdom
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State/province [16]
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Newcastle Upon Tyne
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Mirum Pharmaceuticals, Inc.
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Address
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Ethics approval
Ethics application status
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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.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT04718961
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Address
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Fax
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT04718961
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