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| ID | Type | Description | Link |
|---|---|---|---|
| 2020-003448-96 | EudraCT Number |
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Due to company's assessment of enrollment feasibility.
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Part 1 Arm 1 - Volixibat 20mg | Experimental | Participants randomized to this arm will receive volixibat 20mg twice daily. |
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| Part 1 Arm 2 - Volixibat 80mg | Experimental | Participants randomized to this arm will receive volixibat 80mg twice daily. |
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| Part 2 Arm 1 - Volixibat Selected Dose mg | Experimental | Participants randomized to this arm will receive volixibat selected dose (mg) twice daily. |
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| 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. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Volixibat | Drug | Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor. |
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| Measure | Description | Time Frame |
|---|---|---|
| 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 | Through to end of treatment, up to 21 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| 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?" | Through to end of treatment, up to 21 weeks |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Alabama at Birmingham | Birmingham | Alabama | 35294 | United States | ||
| Yale School of Medicine |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 41603551 | Derived | 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. |
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After a screening period of up to 10 days during which all procedures listed for the screening visit are completed, eligible patients diagnosed with ICP with screening sBA ≥10 μmol/L during screening or at any time during the current pregnancy were randomized with stratification in a 2-arm (1:1), open-label fashion to receive volixibat 20 mg BID or volixibat 80 mg BID. A total of 26 ICP patients were screened for the study. 22 of these patients were screen failures.
A total of 26 participants were screened at 25 sites in 3 countries (New Zealand, United Kingdom, and United States). Of them, 4 were enrolled in the study.
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| ID | Title | Description |
|---|---|---|
| FG000 | Part 1 Arm 1 - Volixibat 20mg (Experimental) | Participants randomized to this arm will receive volixibat 20mg twice daily. Volixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor. |
| FG001 | Part 1 Arm 2 - Volixibat 80mg (Experimental) |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Apr 20, 2022 | Nov 30, 2023 |
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Randomized Open-label two arm study (Part 1) followed by a randomized double-blind, placebo controlled, study (Part 2)
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Masking in Part 2 Only
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| Placebo | Drug | Capsules matched to study drug minus active substance. |
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| Proportion of Participants Experiencing One or More of Adverse Perinatal Outcomes | At least one month after delivery. |
| New Haven |
| Connecticut |
| 06511 |
| United States |
| University of Miami | Miami | Florida | 33136 | United States |
| The Ohio State University Wexner Medical Center | Columbus | Ohio | 43210 | United States |
| The University of Texas Medical Branch - Galveston | Galveston | Texas | 77555 | United States |
| University of Texas Health Science Center | Houston | Texas | 77030 | United States |
| University of Texas Health Science Center at San Antonio | San Antonio | Texas | 78229 | United States |
| Dunedin Hospital | Dunedin | Otago | 6021 | New Zealand |
| Christchurch Women's Hospital | Christchurch | 8011 | New Zealand |
| Capital & Coast District Health Board, Wellington Regional Hospital | Wellington | 6021 | New Zealand |
| Medway NHS Foundation Trust | Gillingham | Kent | ME7 5NY | United Kingdom |
| Bradford Royal Infirmary | Bradford | West Yorkshire | BD9 6RJ | United Kingdom |
| Birmingham Womens and Childrens NHS Foundation Trust | Birmingham | B15 2TG | United Kingdom |
| University Hospital of Wales | Cardiff | CF14 4XW | United Kingdom |
| St Richard's Hospital | Chichester | PO19 6SE | United Kingdom |
| Barts Health NHS Trust- Whipps Cross University Hospital | London | E11 1NR | United Kingdom |
| Royal Free London Hospital NHS Foundation Trust | London | NW3 2QG | United Kingdom |
| Guy's and St Thomas' NHS Foundation Trust | London | SE 1 7EH | United Kingdom |
| West Middlesex University Hospital | Middlesex | TW7 6AF | United Kingdom |
| The Newcastle upon Tyne Hospitals NHS Foundation Trust | Newcastle upon Tyne | United Kingdom |
Participants randomized to this arm will receive volixibat 80mg twice daily. Volixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor. |
| FG002 | Part 2 Arm 1 - Volixibat Selected Dose mg (Experimental) | Participants randomized to this arm will receive volixibat selected dose (mg) twice daily. Volixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor. |
| FG003 | 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. Placebo: Oral capsules, administered twice daily. Capsules matched to study drug minus active substance. |
| COMPLETED |
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| NOT COMPLETED |
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Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.
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| ID | Title | Description |
|---|---|---|
| BG000 | Part 1 Arm 1 - Volixibat 20mg (Experimental) | Participants randomized to this arm will receive volixibat 20mg twice daily. Volixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor. |
| BG001 | Part 1 Arm 2 - Volixibat 80mg (Experimental) | Participants randomized to this arm will receive volixibat 80mg twice daily. Volixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor. |
| BG002 | Part 2 Arm 1 - Volixibat Selected Dose mg (Experimental) | Participants randomized to this arm will receive volixibat selected dose (mg) twice daily. Volixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor. |
| BG003 | 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. Placebo: Oral capsules, administered twice daily. Capsules matched to study drug minus active substance. |
| BG004 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Region of Enrollment | Number | participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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 | Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility. | Posted | Number | participants | Through to end of treatment, up to 21 weeks |
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| Secondary | 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?" | Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility. | Posted | Mean | Standard Deviation | units on a scale | Through to end of treatment, up to 21 weeks |
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| Secondary | Proportion of Participants Experiencing One or More of Adverse Perinatal Outcomes | Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility. | Posted | Count of Participants | Participants | At least one month after delivery. |
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All adverse event data will be collected from first dose of study drug at baseline until whichever of the following time points comes last: 28 days after delivery, or up to 30 days after date of discharge from hospital for mother or for baby, up to 25 weeks
Part 2 was early terminated due to lack of enrollment and the company's assessment of enrollment feasibility.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Part 1 Arm 1 - Volixibat 20mg (Experimental) | Participants randomized to this arm will receive volixibat 20mg twice daily. Volixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor. | 0 | 2 | 1 | 2 | 2 | 2 |
| EG001 | Part 1 Arm 2 - Volixibat 80mg (Experimental) | Participants randomized to this arm will receive volixibat 80mg twice daily. Volixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor. | 0 | 2 | 0 | 2 | 2 | 2 |
| EG002 | Part 2 Arm 1 - Volixibat Selected Dose mg (Experimental) | Participants randomized to this arm will receive volixibat selected dose (mg) twice daily. Volixibat: Oral capsules, administered twice daily. Volixibat is an ileal bile acid transporter (IBAT) inhibitor. | 0 | 0 | 0 | 0 | 0 | 0 |
| EG003 | 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. Placebo: Oral capsules, administered twice daily. Capsules matched to study drug minus active substance. | 0 | 0 | 0 | 0 | 0 | 0 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Diarrhoea | Gastrointestinal disorders | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anemia | Blood and lymphatic system disorders | Systematic Assessment |
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| Anxiety | Psychiatric disorders | Systematic Assessment |
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| Apnoic Episodes | Respiratory, thoracic and mediastinal disorders | Systematic Assessment | Newborn AE |
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| Diarrhoea | Gastrointestinal disorders | Systematic Assessment |
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| Gastroenteritis | Infections and infestations | Systematic Assessment |
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| Headache | Nervous system disorders | Systematic Assessment |
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| Heart Burn | Gastrointestinal disorders | Systematic Assessment |
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| Intermittent Abdominal Pain | Gastrointestinal disorders | Systematic Assessment |
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| Intermittent Diarrhoea | Gastrointestinal disorders | Systematic Assessment |
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| Intermittent Vomiting | Gastrointestinal disorders | Systematic Assessment |
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| Mild Diarrhoea | Gastrointestinal disorders | Systematic Assessment |
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| Postnatal Depression | Psychiatric disorders | Systematic Assessment |
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| Premature Labor | Pregnancy, puerperium and perinatal conditions | Systematic Assessment |
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| Raised Blood Pressure | Investigations | Systematic Assessment |
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| Threated Pre-Term Labor | Pregnancy, puerperium and perinatal conditions | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Mirum Clinical Trials | Mirum Pharmaceuticals | 650-667-4085 | medinfo@mirumpharma.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Nov 13, 2020 | Nov 30, 2023 | SAP_001.pdf |
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| ID | Term |
|---|---|
| C535932 | Intrahepatic Cholestasis of Pregnancy |
| D011537 | Pruritus |
| D002779 | Cholestasis |
| ID | Term |
|---|---|
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D012877 | Skin Manifestations |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D001649 | Bile Duct Diseases |
| D001660 | Biliary Tract Diseases |
| D004066 | Digestive System Diseases |
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| ID | Term |
|---|---|
| C000627853 | volixibat |
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| >=65 years |
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| Male |
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| Not Hispanic or Latino |
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| Unknown or Not Reported |
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| Asian |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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| United Kingdom |
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| OG003 | 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. Placebo: Oral capsules, administered twice daily. Capsules matched to study drug minus active substance. |
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Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily. Placebo: Oral capsules, administered twice daily. Capsules matched to study drug minus active substance. |
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