Intrahepatic Cholestasis of PregnancyPhase 2OHANANCT04718961
A Placebo-controlled Study of Volixibat in Subjects With Elevated Serum Bile Acids Associated With Intrahepatic Cholestasis of Pregnancy (OHANA)
A Phase 2 study testing Volixibat in people with Intrahepatic Cholestasis of Pregnancy, run by Mirum Pharmaceuticals, Inc. at 20 sites in 3 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 45 years, women only
- Conditions the study is for
- Intrahepatic Cholestasis of Pregnancy
- What is being tested?
- Volixibat
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 4 participants
- Where
- 20 sitesin 3 countries
- Study dates
- 4 Jan 2021to 7 Dec 2022
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 45 years
- Women only
- No healthy volunteers
- Adult
Who may be able to join
- Female aged ≥18 and ≤45 years with a viable pregnancy.
- Provide signed informed consent as described in the protocol and willing to comply with all study visits and requirements.
- Diagnosis of ICP.
- (Part 2 only) Qualified level of pruritus associated with ICP, during screening.
Who cannot join
- 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.
- Known non-reassuring fetal status based upon antepartum testing (e.g., NST/CTG or BPP) at or within 7 days before the baseline visit.
- Known fetal anomaly likely to result in intrauterine fetal demise or neonatal death within the first 30 days of life.
- 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 locations and contacts
20 sites in 3 countries.
20 sites in 3 countries
United Kingdom10 sites
Birmingham Womens and Childrens NHS Foundation Trust
Birmingham, B15 2TG
Bradford Royal Infirmary
Bradford, West Yorkshire, BD9 6RJ
University Hospital of Wales
Cardiff, CF14 4XW
St Richard's Hospital
Chichester, PO19 6SE
Medway NHS Foundation Trust
Gillingham, Kent, ME7 5NY
Barts Health NHS Trust- Whipps Cross University Hospital
London, E11 1NR
Royal Free London Hospital NHS Foundation Trust
London, NW3 2QG
Guy's and St Thomas' NHS Foundation Trust
London, SE 1 7EH
West Middlesex University Hospital
Middlesex, TW7 6AF
The Newcastle upon Tyne Hospitals NHS Foundation Trust
Newcastle upon Tyne
United States7 sites
University of Alabama at Birmingham
Birmingham, Alabama, 35294
The Ohio State University Wexner Medical Center
Columbus, Ohio, 43210
The University of Texas Medical Branch - Galveston
Galveston, Texas, 77555
University of Texas Health Science Center
Houston, Texas, 77030
University of Miami
Miami, Florida, 33136
Yale School of Medicine
New Haven, Connecticut, 06511
University of Texas Health Science Center at San Antonio
San Antonio, Texas, 78229
New Zealand3 sites
Christchurch Women's Hospital
Christchurch, 8011
Dunedin Hospital
Dunedin, Otago, 6021
Capital & Coast District Health Board, Wellington Regional Hospital
Wellington, 6021
About this study
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).
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.
- Conditions
- Intrahepatic Cholestasis of Pregnancy
- Being tested
- Volixibat, Placebo
- Keywords
- Pruritus, Itch, Itching, Cholestasis, Bile Acid, Pregnancy, Pregnant, Intrahepatic, Liver, ICP
- Medical subject headings
- Intrahepatic Cholestasis of Pregnancy, Pruritus, Cholestasis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VolixibatDrug
Also called SHP626
Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor.
PlaceboDrug
Capsules matched to study drug minus active substance.
| Study group | Role | What they receive |
|---|---|---|
| Part 1 Arm 1 - Volixibat 20mg | Experimental | Participants randomized to this arm will receive volixibat 20mg twice daily. Receives: Drug: Volixibat |
| Part 1 Arm 2 - Volixibat 80mg | Experimental | Participants randomized to this arm will receive volixibat 80mg twice daily. Receives: Drug: Volixibat |
| Part 2 Arm 1 - Volixibat Selected Dose mg | Experimental | Participants randomized to this arm will receive volixibat selected dose (mg) twice daily. Receives: Drug: Volixibat |
| Part 2 Arm 2 - Placebo (Placebo Comparator) | Placebo comparator | Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Assess the Safety and Tolerability of Volixibat in Participants With ICP
To assess the safety and tolerability of volixibat in participants with ICP on the basis of the following endpoints:
Proportion of participants experiencing one or more of the following:
Treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), adverse events of special interest (AESIs), events of clinical interest (ECIs), and adverse events (AEs) that lead to discontinuation of study drugs.
Clinically significant laboratory abnormalities
Measured: Through to end of treatment, up to 21 weeks
Secondary outcomes (2)
Mean Change in the Weekly Average Worst Daily Itch Score as Measured by the Adult Itch Reported Outcome (ItchRO)
Adult Itch Reported Outcome (ItchRO) is a 0 to 10 scale with 0 being "no itch" and 10 being "worst possible itch" where participants are responding to the following question "How would you rate the worst itch you experienced over the last 24hrs?"
Measured: Through to end of treatment, up to 21 weeks
Proportion of Participants Experiencing One or More of Adverse Perinatal Outcomes
Measured: At least one month after delivery.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jan 2021
- Study start4 Jan 2021
- Primary completion7 Dec 2022
- Study completion7 Dec 2022
Why it stopped: Due to company's assessment of enrollment feasibility.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Intrahepatic Cholestasis of PregnancyStudies of the same condition, open and under way.
- More trials run by Mirum Pharmaceuticals, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Randomized Open-label two arm study (Part 1) followed by a randomized double-blind, placebo controlled, study (Part 2)
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mirum Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04718961
- Sponsor's protocol ID
- VLX-401
- Other IDs
- 2020-003448-96
- Acronym
- OHANA
- Status last verified
- Jul 2024
- First submitted
- 19 Jan 2021
- Last updated
- 6 Aug 2024
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Ovadia C, Stone S, Sibai B, Nunes T, Mogul DB, Krishnaswami J, Kahng J, Li F, Warsi QA, Chien E, Vig P, Williamson C. Efficacy, Safety and Tolerability of Volixibat, an IBAT Inhibitor, in Patients With Intrahepatic Cholestasis of Pregnancy. Liver Int. 2026 Mar;46(3):e70523. doi: 10.1111/liv.70523.
PubMed 41603551
Study at a glance
From the registry record, last updated by the sponsor on 6 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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).
- ClinicalTrials.gov ID
- NCT04718961
- Acronym / sponsor ID
- OHANA / VLX-401
- Conditions
- Intrahepatic Cholestasis of Pregnancy
- Keywords
- Pruritus, Itch, Itching, Cholestasis, Bile Acid, Pregnancy, Pregnant, Intrahepatic
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Due to company's assessment of enrollment feasibility.
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Mirum Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 4 Jan 2021 (actual)
- Primary completion
- 7 Dec 2022 (actual)
- Study completion
- 7 Dec 2022 (actual)
- First posted
- 22 Jan 2021 (actual)
- Results
- Posted 6 Aug 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 4
- Model description
- Randomized Open-label two arm study (Part 1) followed by a randomized double-blind, placebo controlled, study (Part 2)
| Arm | Type | Receives |
|---|---|---|
| Part 1 Arm 1 - Volixibat 20mg | Experimental | Drug: VolixibatParticipants randomized to this arm will receive volixibat 20mg twice daily. |
| Part 1 Arm 2 - Volixibat 80mg | Experimental | Drug: VolixibatParticipants randomized to this arm will receive volixibat 80mg twice daily. |
| Part 2 Arm 1 - Volixibat Selected Dose mg | Experimental | Drug: VolixibatParticipants randomized to this arm will receive volixibat selected dose (mg) twice daily. |
| Part 2 Arm 2 - Placebo (Placebo Comparator) | Placebo comparator | Drug: PlaceboParticipants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily. |
Who may qualify
- Ages
- 18 to 45 years
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (4 listed)
- Female aged ≥18 and ≤45 years with a viable pregnancy.
- Provide signed informed consent as described in the protocol and willing to comply with all study visits and requirements.
- Diagnosis of ICP.
- (Part 2 only) Qualified level of pruritus associated with ICP, during screening.
Key exclusion criteria (4 listed)
- 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.
- Known non-reassuring fetal status based upon antepartum testing (e.g., NST/CTG or BPP) at or within 7 days before the baseline visit.
- Known fetal anomaly likely to result in intrauterine fetal demise or neonatal death within the first 30 days of life.
- Participating in another ongoing interventional clinical study at screening or planning to participate in another contemporaneous interventional clinical study while participating in this study.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VolixibatAlso: SHP626Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor. | Drug | Part 1 Arm 1 - Volixibat 20mg, Part 1 Arm 2 - Volixibat 80mg, Part 2 Arm 1 - Volixibat Selected Dose mg |
| PlaceboCapsules matched to study drug minus active substance. | Drug | Part 2 Arm 2 - Placebo (Placebo Comparator) |
Primary outcome
Assess the Safety and Tolerability of Volixibat in Participants With ICP
Time frame: Through to end of treatment, up to 21 weeks
To assess the safety and tolerability of volixibat in participants with ICP on the basis of the following endpoints: Proportion of participants experiencing one or more of the following: Treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), adverse events of special interest (AESIs), events of clinical interest (ECIs), and adverse events (AEs) that lead to discontinuation of study drugs. Clinically significant laboratory abnormalities
2 secondary outcomes
Mean Change in the Weekly Average Worst Daily Itch Score as Measured by the Adult Itch Reported Outcome (ItchRO)
Time frame: Through to end of treatment, up to 21 weeks
Proportion of Participants Experiencing One or More of Adverse Perinatal Outcomes
Time frame: At least one month after delivery.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Aug 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 41603551Ovadia C, Stone S, Sibai B, Nunes T, Mogul DB, Krishnaswami J, Kahng J, Li F, Warsi QA, Chien E, Vig P, Williamson C. Efficacy, Safety and Tolerability of Volixibat, an IBAT Inhibitor, in Patients With Intrahepatic Cholestasis of Pregnancy. Liver Int. 2026 Mar;46(3):e70523. doi: 10.1111/liv.70523.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
20 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Volixibat in people with Intrahepatic Cholestasis of Pregnancy, run by Mirum Pharmaceuticals, Inc. at 20 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 6 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 20 sites in United Kingdom, United States, New Zealand. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 6 Aug 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Assess the Safety and Tolerability of Volixibat in Participants With ICP (time frame: Through to end of treatment, up to 21 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04718961, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Aug 2024.