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The purpose of this clinical research study is to learn more about the use of the study medicine, volixibat, for the treatment of pruritus (itching) associated with Primary Biliary Cholangitis (PBC), and to assess the possible impact on the disease progression of PBC.
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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. |
|
| Part 1 Arm 2: Volixibat 80mg | Experimental | Participants randomized to this arm will receive volixibat 80mg twice daily. |
|
| Part 1 Arm 3: Placebo | Placebo Comparator | Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily. |
|
| Part 2 Arm 1: Volixibat Selected Dose 20mg | Experimental | Participants randomized to this arm will receive volixibat 20mg twice daily. |
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| 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Volixibat | Drug | Oral capsules, administered twice daily. Volixibat is an Ileal Bile Acid Transporter (IBAT) inhibitor. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Mean change in the daily itch scores using the Adult Itch Reported Outcome (Adult ItchRO) questionnaire | The Adult ItchRO is an 11-point NRS measurement of itch severity ranging from 0=no itch to 10=worst possible itch. | Baseline to week 28 |
| Measure | Description | Time Frame |
|---|---|---|
| Proportion of participants with itch response using the Adult ItchRO | Baseline to week 28 | |
| Incidence of adverse events | Baseline to week 28 | |
| Changes in alkaline phosphatase |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Southern California Research Center | Terminated | Coronado | California | 92218 | United States | |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 40439725 | Derived | Klatzkow H, Bhavsar-Burke I, Pearson M, Wentworth BJ. Primary Biliary Cholangitis in 2025: A New Frontier. Am J Gastroenterol. 2025 Dec 1;120(12):2746-2749. doi: 10.14309/ajg.0000000000003559. Epub 2025 May 29. No abstract available. |
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| Placebo | Drug | Capsules matched to study drug without the active pharmaceutical ingredient |
|
| Baseline to week 28 |
| Changes in total bilirubin | Baseline to week 28 |
| Changes in serum bile acid levels | Baseline to week 28 |
| Change in Primary Biliary Cholangitis Quality of Life Measure (PBC-40) | PBC-40 questionnaire is scored on a scale of 40 to 200, with higher scores indicating poorer quality of life | Baseline to week 28 |
| Change in Patient-Reported Outcomes Measurement Information System (PROMIS®) fatigue questionnaire | The PROMIS® Fatigue questionnaire is a 5-point response scale. Respondents endorse each item using a 5-point response scale: Never, Rarely, Sometimes, Often, and Always. | Baseline to week 28 |
| Change in Patient-Reported Outcomes Measurement Information System (PROMIS®) sleep disturbance questionnaire | The PROMIS® Sleep Disturbance in an 8-item measure that assesses quality of sleep, sleep depth, and restoration associated with sleep. It is a 5-point response scale, the specific response options vary by items. The first 4 items use response options that range from Not at all to Very much. The next 3 items use response options that range from Never to Always. The final item assesses sleep quality ranging from Very poor to Very good. | Baseline to week 28 |
| Cedars Sinai Medical Center |
| Recruiting |
| Los Angeles |
| California |
| 90048 |
| United States |
|
| University of California Los Angeles | Recruiting | Los Angeles | California | 90095 | United States |
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| UC Davis | Recruiting | Sacramento | California | 95817 | United States |
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| University of Colorado | Recruiting | Aurora | Colorado | 80045 | United States |
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| Yale University Digestive Diseases | Recruiting | New Haven | Connecticut | 06510 | United States |
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| Covenant Metabolic Specialists | Terminated | Fort Myers | Florida | 33912 | United States |
| UF Hepatology Research at CTRB | Recruiting | Gainesville | Florida | 32610 | United States |
|
| UF Health Gastroenterology- Emerson | Terminated | Jacksonville | Florida | 32209 | United States |
| IHS Health | Terminated | Kissimmee | Florida | 34741 | United States |
| Schiff Center for Liver Diseases | Terminated | Miami | Florida | 33136 | United States |
| Advanced Research Institute, Inc | Recruiting | New Port Richey | Florida | 34653 | United States |
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| Covenant Research and Clinics, LLC | Terminated | Sarasota | Florida | 34240 | United States |
| Tampa General Hospital | Recruiting | Tampa | Florida | 33606 | United States |
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| Cleveland Clinic Florida | Recruiting | Weston | Florida | 33331 | United States |
|
| Southeast Clinical Research Center | Terminated | Dalton | Georgia | 30720 | United States |
| Northwestern University | Terminated | Chicago | Illinois | 60611 | United States |
| Loyola University Health System - Loyola Outpatient Center | Recruiting | Maywood | Illinois | 60153 | United States |
|
| University of Iowa Hospitals & Clinics | Recruiting | Iowa City | Iowa | 52242 | United States |
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| Ochsner Clinic Foundation-Baton Rouge | Recruiting | Baton Rouge | Louisiana | 70836 | United States |
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| LSU Health Science Center Shreveport | Recruiting | Shreveport | Louisiana | 71103 | United States |
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| Massachusetts General Hospital | Recruiting | Boston | Massachusetts | 02114 | United States |
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| Beth Israel Deaconess | Recruiting | Boston | Massachusetts | 02215 | United States |
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| Mayo Clinic | Recruiting | Rochester | Minnesota | 55905 | United States |
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| Jackson Liver and GI Specialists | Terminated | Jackson | Mississippi | 39216 | United States |
| CHI Health Clinic (Alegent Creighton Clinic)- Gastroenterology-Bergan | Recruiting | Omaha | Nebraska | 68124 | United States |
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| Virtua Center for Liver Disease-Camden | Recruiting | Camden | New Jersey | 08103 | United States |
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| Montefiore Medical Center | Recruiting | The Bronx | New York | 10467 | United States |
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| Science 37, Inc (Remote-homebased Telemedicine) | Recruiting | Morrisville | North Carolina | 27560 | United States |
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| Cleveland Clinic - Main Campus | Recruiting | Cleveland | Ohio | 44195 | United States |
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| Einstein Healthcare Network - Einstein Medical Center | Recruiting | Philadelphia | Pennsylvania | 19141 | United States |
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| Methodist Le Bonheur Healthcare | Terminated | Memphis | Tennessee | 38104 | United States |
| Galen Medical Group, P.C | Recruiting | Tennessee City | Tennessee | 37343 | United States |
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| Amel Med LLC | Terminated | Austin | Texas | 78750 | United States |
| The Liver Institute at Methodist Dallas Medical Center | Recruiting | Dallas | Texas | 75203 | United States |
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| Soma Clinical Trials | Terminated | Denison | Texas | 75020 | United States |
| Baylor College of Medicine | Recruiting | Houston | Texas | 77030 | United States |
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| Liver Associates of Texas | Recruiting | Houston | Texas | 77030 | United States |
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| Impact Research | Terminated | Waco | Texas | 76710 | United States |
| University of Utah Health Care | Recruiting | Salt Lake City | Utah | 84132 | United States |
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| MedStar Health Research Institute | Recruiting | Fairfax | Virginia | 22031 | United States |
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| Bon Secours Liver Institute of Hampton Roads Mary Immaculate Hospital | Terminated | Newport News | Virginia | 23602 | United States |
| Bon Secours Richmond Community Hospital LLC d/b/a Bon Secours Liver Institute of Richmond | Recruiting | Richmond | Virginia | 23226 | United States |
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| Liver Institute Northwest | Recruiting | Seattle | Washington | 98105 | United States |
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| Hospital de Alta Complejidad En Red El Cruce | Recruiting | San Juan Bautista | Buenos Aires | 1888 | Argentina |
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| Hospital Provincial Del Centenario | Recruiting | Rosario | Santa Fe Province | 2000 | Argentina |
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| Glenny Corp S.A | Recruiting | CABA | 1430 | Argentina |
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| Clínica de Nefrología, Urología y Enfermedades Cardiovasculares | Recruiting | Santa Fe | 3000 | Argentina |
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| Universitair Ziekenhuis Antwerpe UZA | Recruiting | Antwerp | 2650 | Belgium |
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| Cliniques Universitaires Saint-Luc | Recruiting | Brussels | 1200 | Belgium |
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| UZ Gent | Recruiting | Ghent | 9000 | Belgium |
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| Centro de Pesquisa Clínica da Bahiana Saúde | Recruiting | Salvador | Estado de Bahia | 40290-000 | Brazil |
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| Hospital Das Clínicas Da Ufg | Recruiting | Goiânia | Goiás | 74605-050 | Brazil |
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| Centro de Pesquisas Clínicas do HC/UFMG-EBSERH | Recruiting | Belo Horizonte | Minas Gerais | 30130-100 | Brazil |
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| Hospital de Clínicas de Porto Alegre | Recruiting | Porto Alegre | Rio Grande do Sul | 90035903 | Brazil |
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| Hospital Universitário Clementino Fraga Filho - Secretaria de Hepatologia | Recruiting | Rio de Janeiro | 21941-617 | Brazil |
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| Instituto de Educação, Pesquisa e Gestão em Saúde | Recruiting | Rio de Janeiro | 22775-001 | Brazil |
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| Fundação Oswaldo Ramos | Recruiting | São Paulo | 04038-002 | Brazil |
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| Gordon and Leslie Diamond Health Care Centre, Division of Gastroenterology | Recruiting | Vancouver | British Columbia | V5Z 1M9 | Canada |
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| (G.I.R.I.) GI Research Institute Foundation | Recruiting | Vancouver | British Columbia | V6Z 2K5 | Canada |
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| McMaster University Medical Center | Recruiting | Hamilton | Ontario | L8S 4K1 | Canada |
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| University Hospital, LHSC | Recruiting | London | Ontario | N6A5A5 | Canada |
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| Toronto General Hospital | Recruiting | Toronto | Ontario | M5G 1Z5 | Canada |
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| McGill University Health Centre | Recruiting | Montreal | Quebec | H4A 3J1 | Canada |
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| University of Alberta | Recruiting | Edmonton | T6G 2X8 | Canada |
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| CRCHUM (Centre de Recherche du Centre Hospitalier de l'Université de Montréal) | Recruiting | Montreal | H2X 0A9 | Canada |
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| Mengchao Hepatobiliary Hospital of Fujian Medical University | Recruiting | Fuzhou | Fujian | 350028 | China |
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| Union Hospital Tongji Medical College Huazhong University of Science and Technology | Recruiting | Wuhan | Hubei | 430022 | China |
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| The First Hospital of Jilin University | Recruiting | Changchun | Jilin | 130031 | China |
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| The First Affiliated Hospital, Zhejiang University School of Medicine | Recruiting | Hangzhou | Zhejiang | 310003 | China |
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| Beijing Youan Hospital, Capital Medical University | Recruiting | Beijing | 100069 | China |
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| Beijing Friendship Hospital, Capital Medical University | Recruiting | Beijing | 10050 | China |
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| Sir Run Run Shaw Hospital -Zhejiang University School of Medicine | Recruiting | Hangzhou | 310009 | China |
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| CHU Grenoble - Hôpital MICHALLON | Recruiting | Grenoble | 38043 | France |
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| Hopital Claude Huriez-Digestive system disease clinic | Recruiting | Lille | 59037 | France |
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| CHU de NICE- Hospital L'Archet 2 | Recruiting | Nice | 06202 | France |
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| Hospital Saint Antoine | Recruiting | Paris | 75012 | France |
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| Nouvel Hospital Civil | Recruiting | Strasbourg | 67091 | France |
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| Hospital Paul Brousse- APHP | Recruiting | Villejuif | 94800 | France |
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| Klinikum Chemnitz gGmbH | Recruiting | Chemnitz | 09116 | Germany |
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| Universitätsklinikum Frankfurt am Main | Recruiting | Frankfurt | Germany |
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| Medizinische Hochschule Hannover (MHH) | Recruiting | Hanover | 30625 | Germany |
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| Universitätsklinikum Schleswig-Holstein | Recruiting | Kiel | 24105 | Germany |
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| Universitätsklinikum Leipzig, Klinik und Poliklinik für Onkologie, Gastroenterologie, Hepatologie und Pneumologie | Recruiting | Leipzig | 04103 | Germany |
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| Otto-von-Guericke-Universität Klinik für Gastroenterologie, Hepatologie und Infektiologie Universitätsklinikum Magdeburg A.ö.R. | Recruiting | Magdeburg | 39120 | Germany |
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| University Hospital Munster | Recruiting | Münster | 48149 | Germany |
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| University Hospital Tübingen Medical clinic | Recruiting | Tübingen | 72076 | Germany |
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| St. Josefs-Hospital Wiesbaden | Active, not recruiting | Wiesbaden | 65189 | Germany |
| Hillel Yaffe Medical Center | Terminated | Hadera | 3810101 | Israel |
| Rambam Medical Center | Recruiting | Haifa | 3109601 | Israel |
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| Carmel Medical Center | Recruiting | Haifa | 3434104 | Israel |
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| Shaare Zedek Medical Center | Terminated | Jerusalem | 9103102 | Israel |
| Hadassah Medical Center - Ein Karem, Liver Institute Kiryat Hadassah POB 12000 | Recruiting | Jerusalem | 9112001 | Israel |
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| Galilee Medical Center | Recruiting | Nahariya | 2210001 | Israel |
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| Rabin Medical Center | Recruiting | Petah Tikva | 4941492 | Israel |
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| Sheba Medical Center | Recruiting | Ramat Gan | 52621 | Israel |
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| The Tel Aviv Sourasky Medical Center | Terminated | Tel Aviv | 6423906 | Israel |
| Universita Politecnica delle Marche- Ospedali Riuniti | Recruiting | Ancona | Italy |
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| ASST Papa Giovanni XXIII | Recruiting | Bergamo | 24127 | Italy |
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| Fondazione Ca Granda | Terminated | Milan | 20122 | Italy |
| ASST Grande Ospedale Metropolitano Niguarda | Recruiting | Milan | 20162 | Italy |
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| Azienda Ospedaliero Universitaria Di Modena | Recruiting | Modena | 41126 | Italy |
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| Fondazione IRCCS San Gerardo dei Tintori | Recruiting | Monza | 20900 | Italy |
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| Azienda Ospedale-Università di Padova | Recruiting | Padova | 35128 | Italy |
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| Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone | Recruiting | Palermo | 90127 | Italy |
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| Azienda Ospedaliero Universitaria Pisana (AOUP) | Recruiting | Pisa | 56124 | Italy |
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| Istituto Clinico Humanitas | Recruiting | Rozzano | Italy |
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| National Hospital Organization Nagasaki Medical Center | Recruiting | Ōmura | Nagasaki | 856-8562 | Japan |
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| Ehime University Hospital | Recruiting | Ehime | 791-0295 | Japan |
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| Teine Keijinkai Hospital | Recruiting | Hokkaido | 006-8555 | Japan |
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| Osaka Rosai Hospital | Recruiting | Osaka | 591-8025 | Japan |
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| Toranomon Hospital | Recruiting | Tokyo | 105-8470 | Japan |
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| Amstedam University Medical Centers | Recruiting | Amsterdam | 1081 HV | Netherlands |
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| Radboudumc | Recruiting | Nijmegen | 6525 GA | Netherlands |
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| Erasmus medical center | Recruiting | Rotterdam | Netherlands |
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| Hospital Universitari Vall d' Hebron | Recruiting | Barcelona | 08035 | Spain |
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| Hospital Clínic de Barcelona | Recruiting | Barcelona | 08036 | Spain |
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| Hospital Universitari Parc Tauli | Recruiting | Barcelona | 08208 | Spain |
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| Hospital General Universitario Gregorio Maranon | Recruiting | Madrid | 28007 | Spain |
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| Hospital Universitario Virgen del Rocio | Recruiting | Seville | 41013 | Spain |
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| Hospital Universitario Miguel Servet | Recruiting | Zaragoza | 50009 | Spain |
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| Ente Ospedaliero Cantonale | Recruiting | Lugano | 6900 | Switzerland |
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| University Hospital Zurich(USZ) | Recruiting | Zurich | 8091 | Switzerland |
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| University Hospital Southampton NHS Foundation Trust | Recruiting | Southampton | Hampshire | S016 6YD | United Kingdom |
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| University Hospitals Birmingham NHS Foundation Trust of Trust Headquarters | Recruiting | Birmingham | West Midlands | B15 2GW | United Kingdom |
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| Belfast Health & Social Care Trust (BHSCT) Royal Victoria Hospital | Recruiting | Belfast | BT12 6BA | United Kingdom |
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| Royal Free Hospital NHS Foundation Trust | Recruiting | London | NW3 2QZ | United Kingdom |
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| King's College Hospital NHS Foundation Trust | Recruiting | London | SE5 9RS | United Kingdom |
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| Freeman Hospital | Recruiting | Newcastle upon Tyne | NE7 7DN | United Kingdom |
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| Nottingham University Hospitals NHS Trust | Recruiting | Nottingham | United Kingdom |
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| ID | Term |
|---|---|
| D008105 | Liver Cirrhosis, Biliary |
| D011537 | Pruritus |
| D002761 | Cholangitis |
| D004066 | Digestive System Diseases |
| ID | Term |
|---|---|
| D002780 | Cholestasis, Intrahepatic |
| D002779 | Cholestasis |
| D001649 | Bile Duct Diseases |
| D001660 | Biliary Tract Diseases |
| D008107 | Liver Diseases |
| D008103 | Liver Cirrhosis |
| D005355 | Fibrosis |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D012877 | Skin Manifestations |
| D012816 | Signs and Symptoms |
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| ID | Term |
|---|---|
| C000627853 | volixibat |
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