En langtidsundersøgelse af Elafibranor hos voksne deltagere med primær biliær kolangitis (ELFIDENCE)
En fase III randomiseret, parallel-gruppe, dobbeltblind, placebokontrolleret, to-arm undersøgelse til evaluering af effektiviteten og sikkerheden af Elafibranor 80 mg på langsigtede kliniske resultater hos voksne deltagere med primær biliær kolangitis (PBC)
The participants of this study will have confirmed Primary Biliary Cholangitis (PBC) and cirrhosis (scarring of the liver).
PBC is a slowly progressive disease, characterised by damage to the bile ducts in the liver, leading to a build-up of bile acids which causes further damage.
The liver damage in PBC may lead to cirrhosis. PBC may also be associated with multiple symptoms. Many patients with PBC may require liver transplant or may die if the disease progresses and a liver transplant is not done.
This study will compare a daily dose of elafibranor (the study drug) to a daily dose of placebo (a dummy treatment) and will last up to 3.5 years for each participant.
The main aim of this study is to determine if elafibranor is better than placebo in preventing clinical outcome events showing disease worsening (including progression of disease leading to liver transplant or death).
This study will also study the safety of long-term treatment with elafibranor, as well as the impact on symptoms such as itching and tiredness.
Studieoversigt
Status
Betingelser
Intervention / Behandling
Undersøgelsestype
Tilmelding (Anslået)
Fase
- Fase 3
Kontakter og lokationer
Studiekontakt
- Navn: Ipsen Clinical Study Enquiries
- Telefonnummer: See e mail
- E-mail: clinical.trials@ipsen.com
Studiesteder
-
-
-
Buenos Aires, Argentina
- Rekruttering
- Hospital Italiano de Buenos Aires
-
Buenos Aires, Argentina
- Rekruttering
- Fundacion Respirar (Centro Medico Dra. De Salvo) - Instituto Argentino de Investigaciones Clinicas (IAIC) S.R.L
-
Buenos Aires, Argentina
- Rekruttering
- DIM Centro Medico
-
Buenos Aires, Argentina
- Rekruttering
- Hospital Britanico de Buenos Aires
-
Córdoba, Argentina
- Rekruttering
- Centro Medico Colon
-
Mendoza, Argentina
- Trukket tilbage
- Hospital Espanol de Mendoza
-
Pilar, Argentina
- Rekruttering
- Hospital Universitario Austral
-
San Juan Bautista, Argentina
- Rekruttering
- Hospital El Cruce
-
-
-
-
-
Birtinya, Australien
- Rekruttering
- Sunshine Coast University Hospital
-
Footscray, Australien
- Rekruttering
- Footscray Hospital, Western Health
-
Kingswood, Australien
- Trukket tilbage
- Nepean Clinical School
-
Melbourne, Australien
- Trukket tilbage
- Monash University - Monash Health -Monash Medical Centre
-
Sydney, Australien
- Trukket tilbage
- St George Hospital
-
Westmead, Australien
- Rekruttering
- Westmead Hospital
-
-
-
-
-
Edegem, Belgien
- Trukket tilbage
- Universitair Ziekenhuis Antwerpen
-
Genk, Belgien
- Trukket tilbage
- Ziekenhuis Oost-Limburg
-
Ghent, Belgien
- Rekruttering
- Gent University Hospital
-
Roeselare, Belgien
- Trukket tilbage
- Algemeen Ziekenhuis Delta
-
-
-
-
-
Belo Horizonte, Brasilien
- Rekruttering
- NUPEC Cardio
-
Goiânia, Brasilien
- Rekruttering
- Universidade Federal de Goias
-
Porto Alegre, Brasilien
- Rekruttering
- Hospital de Clinicas de Porto Alegre (HCPA)
-
Salvador, Brasilien
- Rekruttering
- Instituto D'Or de Pesquisa e Ensino - Hospital Aliança
-
São Paulo, Brasilien
- Rekruttering
- Hospital de Base - Centro Integrado de Pesquisa ( CIP )
-
São Paulo, Brasilien
- Rekruttering
- Universidade Estadual Paulista Julio De Mesquita Filho (UNESP) Hospital das Clinicas Faculdade de Medicina de Botucatu (HCFMB)
-
São Paulo, Brasilien
- Rekruttering
- Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo
-
-
-
-
-
Sofia, Bulgarien
- Rekruttering
- Tokuda Hospital, Dept. of Internal Medicine
-
Sofia, Bulgarien
- Rekruttering
- University Hospital City Clinic Cancer Center
-
Sofia, Bulgarien
- Aktiv, ikke rekrutterende
- Diagnostic-Consultative Center Aleksandrovska EOOD
-
-
-
-
-
Edmonton, Canada
- Rekruttering
- University of Alberta - Faculty of Medicine & Dentistry - The Centre of Excellence for Gastrointestinal Inflammation and Immunity Research (CEGIIR)
-
Toronto, Canada
- Rekruttering
- University Health Network (UHN) - Toronto General Hospital (TGH) - Toronto General Research Institute (TGRI)
-
-
-
-
-
Las Condes, Chile
- Rekruttering
- Universidad De Los Andes
-
Santiago, Chile
- Rekruttering
- Enroll SpA
-
Santiago, Chile
- Rekruttering
- Centro de Estudios Clinicos e Investigaciones Medicas (CECIM)
-
Santiago, Chile
- Rekruttering
- Hospital Clinico de la Pontificia Universidad Catolica de Chile
-
Santiago, Chile
- Rekruttering
- University of Chile Clinical Hospital (Hospital Clinico de la Universidad de Chile)
-
Viña del Mar, Chile
- Rekruttering
- Centro Medico Cedid - Centro de Diagnostico Digestivo
-
-
-
-
-
Barranquilla, Colombia
- Rekruttering
- Clinica de la Costa
-
Cali, Colombia
- Rekruttering
- Clinica Imbanaco de S.A.S.
-
Cali, Colombia
- Rekruttering
- Salud SURA Chipichape
-
Floridablanca, Colombia
- Rekruttering
- Fundacion Oftalmologica de Santander-Clinica Ardila Lulle La FOSCAL
-
Medellín, Colombia
- Rekruttering
- Hospital Pablo Tobon Uribe
-
Medellín, Colombia
- Rekruttering
- Servicios de Salud IPS Suramericana S.A.S. Industriales
-
-
-
-
-
Copenhagen, Danmark
- Rekruttering
- Bispebjerg Hospital
-
-
-
-
Arizona
-
Tucson, Arizona, Forenede Stater, 85641
- Rekruttering
- Arizona Liver Health
-
-
Arkansas
-
Little Rock, Arkansas, Forenede Stater, 55130
- Afsluttet
- Arkansas Diagnostic Center, PA
-
-
California
-
Coronado, California, Forenede Stater, 92118
- Rekruttering
- Southern California Research Center
-
Los Angeles, California, Forenede Stater, 90048
- Trukket tilbage
- Cedars-Sinai Medical Center
-
Los Angeles, California, Forenede Stater, 90404
- Trukket tilbage
- University of California Los Angeles
-
Los Angeles, California, Forenede Stater, 90067
- Aktiv, ikke rekrutterende
- GastroIntestinal BioSciences
-
Sacramento, California, Forenede Stater, 95817
- Rekruttering
- University of California Davis Medical Center
-
-
Colorado
-
Aurora, Colorado, Forenede Stater, 80045
- Rekruttering
- University of Colorado
-
Colorado Springs, Colorado, Forenede Stater, 80829
- Rekruttering
- Peak Gastroenterology Associates
-
Englewood, Colorado, Forenede Stater, 80113
- Aktiv, ikke rekrutterende
- South Denver Gastroenterology, P.C.
-
Littleton, Colorado, Forenede Stater, 80120
- Rekruttering
- Rocky Mountain Gastroenterology
-
-
Florida
-
Miami, Florida, Forenede Stater, 33136
- Rekruttering
- University Of Miami School Of Medicine, Center For Liver Diseases
-
Pembroke Pines, Florida, Forenede Stater, 12105
- Rekruttering
- Bolanos Clinical Research
-
Tampa, Florida, Forenede Stater, 33614
- Rekruttering
- International Center for Research
-
-
Kansas
-
Kansas City, Kansas, Forenede Stater, 66160
- Ikke rekrutterer endnu
- University of Kansas Medical Center (KUMC) - University of Kansas Liver Center - Hepatology Clinic
-
-
Louisiana
-
Shreveport, Louisiana, Forenede Stater, 71103
- Rekruttering
- Louisiana Research Center, LLC
-
-
Michigan
-
Ann Arbor, Michigan, Forenede Stater, 48109
- Rekruttering
- University of Michigan Health System
-
Ypsilanti, Michigan, Forenede Stater, 48197
- Trukket tilbage
- Huron Gastroenterology Associates - Center for Digestive Care
-
-
Minnesota
-
Rochester, Minnesota, Forenede Stater, 55905
- Rekruttering
- Mayo Clinic
-
-
New Mexico
-
Albuquerque, New Mexico, Forenede Stater, 87109
- Afsluttet
- Southwest Gastroenterology Associates, PC (SWGA)
-
-
New York
-
New York, New York, Forenede Stater, 10016
- Rekruttering
- NYU Langone Gastroenterology and Hepatology Associates
-
-
Pennsylvania
-
Pittsburgh, Pennsylvania, Forenede Stater, 15213
- Rekruttering
- University of Pittsburgh
-
-
South Carolina
-
Charleston, South Carolina, Forenede Stater, 29425
- Rekruttering
- Medical University of South Carolina
-
-
Tennessee
-
Cordova, Tennessee, Forenede Stater, 38138
- Rekruttering
- Gastroenterology Center of the Midsouth
-
-
Texas
-
Arlington, Texas, Forenede Stater, 22201
- Rekruttering
- Texas Clinical Research Institute
-
Austin, Texas, Forenede Stater, 78757
- Rekruttering
- American Research Corporation
-
Dallas, Texas, Forenede Stater, 75390
- Rekruttering
- University of Texas Southwestern Medical Center at Dallas
-
Dallas, Texas, Forenede Stater, 75203
- Rekruttering
- Methodist Transplant Physicians
-
Dallas, Texas, Forenede Stater, 60612
- Trukket tilbage
- Rush University Medical Center - University Cardiovascular Surgeons
-
Dallas, Texas, Forenede Stater, 75203
- Afsluttet
- Liver Center of Texas
-
Fort Worth, Texas, Forenede Stater, 76104
- Trukket tilbage
- Baylor Scott & White All Saints Medical Center - Forth Worth
-
Houston, Texas, Forenede Stater, 77030
- Rekruttering
- Liver Associates of Texas
-
Houston, Texas, Forenede Stater, 77584
- Aktiv, ikke rekrutterende
- Houston Methodist Cancer Center
-
Katy, Texas, Forenede Stater, 77904
- Afsluttet
- Gastro health & Nutrition
-
San Antonio, Texas, Forenede Stater, 78015
- Rekruttering
- American Research Corporation at The Texas Liver Institute
-
San Antonio, Texas, Forenede Stater, 78251
- Rekruttering
- Starhope Liver Center, PA - Starhope Clinical Research, LLC
-
Waco, Texas, Forenede Stater, 76710
- Trukket tilbage
- Impact Research Tx
-
-
Virginia
-
Charlottesville, Virginia, Forenede Stater, 22903
- Rekruttering
- University of Virginia Medical Center
-
Richmond, Virginia, Forenede Stater, 23298
- Ikke rekrutterer endnu
- Virginia Commonwealth University Medical Center - West Hospital
-
Richmond, Virginia, Forenede Stater, 23226
- Rekruttering
- Bon Secours St. Mary's Hospital of Richmond, Inc
-
-
Washington
-
Columbia, Washington, Forenede Stater, 20007
- Trukket tilbage
- Medstar Georgetown Transplant Institute University Hospital (MGUH)
-
Seattle, Washington, Forenede Stater, 98105
- Rekruttering
- Velocity Clinical Research at Liver Institute Northwest
-
-
West Virginia
-
Huntington, West Virginia, Forenede Stater, 25701
- Rekruttering
- University Physicians and Surgeons Inc, dba Marshall Health
-
-
-
-
-
Clichy, Frankrig
- Rekruttering
- Hopital Beaujon
-
Créteil, Frankrig, 94000
- Rekruttering
- Hospital Henri Mondor
-
Lille, Frankrig
- Trukket tilbage
- Centre Hospitalier Regional Universitaire de Lille - Hopital Claude Huriez
-
Lyon, Frankrig
- Rekruttering
- Hospices Civils de Lyon (HCL) - Hopital de la Croix-Rousse
-
Marseille, Frankrig
- Trukket tilbage
- Hopital Saint Joseph - Marseille
-
Nice, Frankrig
- Rekruttering
- CHU de Nice, Hopital de l'Archet
-
Paris, Frankrig, 75013
- Rekruttering
- Hopital Pitie-Salpetriere - APHP
-
Poitiers, Frankrig
- Trukket tilbage
- CHU Poitiers
-
Toulouse, Frankrig
- Trukket tilbage
- Clinique Pasteur
-
Villejuif, Frankrig
- Trukket tilbage
- Hopital Paul-Brousse - APHP
-
-
-
-
-
Athens, Grækenland
- Rekruttering
- General Hospital of Athens Laiko
-
Athens, Grækenland
- Rekruttering
- General Oncological Hospital of Kifisia Oi Agioi Anargyroi
-
Athens, Grækenland
- Rekruttering
- Agios Savvas Regional Cancer Hospital
-
Thessaloniki, Grækenland
- Rekruttering
- GeneIppokratio General Hospital of Thessaloniki
-
-
-
-
-
Beersheba, Israel
- Trukket tilbage
- Institute of Gastroenterology and Liver Diseases - Soroka University Medical Center
-
Haifa, Israel
- Trukket tilbage
- Rambam Health Care Campus (RHCC)
-
Jerusalem, Israel
- Afsluttet
- Hadassah University Hospital (HUH) - Ein-Kerem
-
Jerusalem, Israel
- Afsluttet
- Shaare Zedek Medical Center
-
Nahariya, Israel
- Afsluttet
- Galilee Medical Center
-
Nahariya, Israel
- Trukket tilbage
- Galilee Medical Center, ZIV Medical Center
-
Petah Tikva, Israel
- Trukket tilbage
- Rabin Medical Center - Beilinson Hospital - Liver Institute
-
-
-
-
-
Modena, Italien
- Trukket tilbage
- Azienda Ospedaliero Universitaria Modena
-
Monza, Italien
- Aktiv, ikke rekrutterende
- Ospedale San Gerardo
-
Naples, Italien
- Afsluttet
- Azienda Ospedaliero Universitaria Federico II di Napoli
-
Padova, Italien
- Trukket tilbage
- Azienda Ospedaliera di Padova - U.O.C. di Gastroenterologia
-
Palermo, Italien
- Afsluttet
- Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone
-
Pavia, Italien
- Trukket tilbage
- Fondazione I.R.C.C.S. Policlinico San Matteo
-
Pisa, Italien
- Trukket tilbage
- Azienda Ospedaliero Universitaria Pisana
-
San Giovanni Rotondo, Italien
- Trukket tilbage
- Ospedale Casa Sollievo della Sofferenza
-
-
-
-
-
Kaunas, Litauen
- Rekruttering
- Hospital of Lithuanian University of Health Sciences Kaunas
-
Vilnius, Litauen
- Rekruttering
- Vilnius University Hospital Santaros Klinikos
-
-
-
-
-
Johor Bahru, Malaysia
- Rekruttering
- Hospital Sultanah Aminah
-
Kota Kinabalu, Malaysia
- Rekruttering
- Hospital Queen Elizabeth II
-
Kuala Lumpur, Malaysia
- Rekruttering
- University of Malaya Medical Centre
-
-
-
-
-
Guadalajara, Mexico
- Rekruttering
- Centro de Investigacion Clinica y Medicina Traslacional
-
Jalisco, Mexico
- Rekruttering
- Centro de Investigacion Medico Biologica y Terapia Avanzada
-
Mexico City, Mexico
- Rekruttering
- Centro de Investigacion y Gastroenterologia SC
-
Mexico City, Mexico
- Rekruttering
- Instituto Nacional de Ciencias Medicas y Nutricion Salvador Zubiran (INCMNSZ)
-
Monterrey, Mexico
- Rekruttering
- Hospital Angeles Valle Oriente
-
Yucatán, Mexico
- Rekruttering
- Medical Care & Research
-
-
-
-
-
Christchurch, New Zealand
- Rekruttering
- Canterbury District Health Board
-
Hamilton, New Zealand
- Rekruttering
- Waikato Hospital
-
-
-
-
-
Lima, Peru
- Rekruttering
- Clinica Delgado
-
-
-
-
-
Elblag, Polen
- Rekruttering
- NZOZ Twoje Zdrowie EL Sp. z o.o.
-
Katowice, Polen
- Rekruttering
- Uniwersyteckie Centrum Kliniczne im. Prof. K. Gibinskiego Slaskiego Uniwersytetu Medycznego
-
Krakow, Polen
- Rekruttering
- Krakowskie Centrum Medyczne Sp.z.o.o - FutureMeds
-
Krakow, Polen
- Trukket tilbage
- Szpital Uniwersytecki w Krakowie
-
Lodz, Polen, 90-153
- Rekruttering
- Oddzial Kliniczny Gastroenterologii Ogolnej i Onkologicznej SPZOZ, Uniwersytecki Szpital Kliniczny im. Barlickiego w Lodzi
-
Lublin, Polen
- Rekruttering
- Medrise Sp. z o.o.
-
Małogoskie, Polen
- Ikke rekrutterer endnu
- Krakowskie Centrum Medyczne Sp.z.o.o. - FutureMeds
-
Rzeszów, Polen
- Rekruttering
- Centrum Medyczne Medyk Sp. z o.o. Sp. K.
-
Sosnowiec, Polen
- Trukket tilbage
- Niepubliczny Zaklad Opieki Zdrowotnej (NZOZ) - Poliklinika Doktora Bessera
-
Warsaw, Polen
- Rekruttering
- FutureMeds Warszawa Centrum
-
Wroclaw, Polen
- Rekruttering
- FutureMeds sp. z o. o
-
Wroclaw, Polen
- Rekruttering
- Nzoz Centrum BadanKlinicznych
-
Wroclaw, Polen
- Rekruttering
- PlanetMed Sp. z o.o.
-
-
-
-
-
Funchal, Portugal, 9004-514
- Rekruttering
- Hospital Dr. Nelio Mendonca
-
Vila Real, Portugal, 5000-508
- Rekruttering
- Unidade Local de Saúde de Trás-os-Montes e Alto Douro, E. P. E
-
-
Praceta Mota Pinto
-
Coimbra, Praceta Mota Pinto, Portugal
- Rekruttering
- Hospitais da Universidade de Coimbra (H.U.C) - Universidade de Coimbra
-
-
-
-
-
Bucharest, Rumænien
- Rekruttering
- Centrul Pentru Studiul Metabolismului
-
Bucharest, Rumænien
- Rekruttering
- Sana S.R.L
-
Bucharest, Rumænien
- Rekruttering
- Spital Clinic Dr I Cantacuzino
-
Cluj-Napoca, Rumænien
- Rekruttering
- Cluj County Clinical Emergency Hospital
-
Cluj-Napoca, Rumænien
- Rekruttering
- Spitalul Clinic De Urgenta Prof. Dr. Octavian Fodor
-
Iași, Rumænien, 700111
- Rekruttering
- Gastromedica Srl
-
-
-
-
-
Bratislava, Slovakiet
- Rekruttering
- Gastroenterological Centre Thalion
-
Košice, Slovakiet
- Rekruttering
- Univerzitna nemocnica L. Pasteura Kosice
-
Nitra, Slovakiet
- Rekruttering
- Fakultna nemocnica Nitra
-
-
-
-
-
Barcelona, Spanien
- Rekruttering
- Hospital Universitario Vall d'Hebron
-
Barcelona, Spanien
- Rekruttering
- Hospital Clinic i Provincial de Barcelona
-
Madrid, Spanien
- Trukket tilbage
- Hospital Universitario La Paz
-
Madrid, Spanien
- Rekruttering
- Clinica Universidad Navarra-Sede Madrid
-
Madrid, Spanien
- Aktiv, ikke rekrutterende
- Hospital General Universitario Gregorio Maranon (HGUGM)
-
Majadahonda, Spanien
- Rekruttering
- Hospital Universitario Puerta de Hierro de Majadahonda
-
Málaga, Spanien
- Rekruttering
- Hospital Universitario Virgen de la Victoria
-
Pontevedra, Spanien
- Rekruttering
- Hospital De Montecelo
-
Sabadell, Spanien
- Aktiv, ikke rekrutterende
- Institut d Investigacio i Innovacio Parc Tauli, Hospital Universitari Parc Tauli
-
Seville, Spanien
- Rekruttering
- Hospital Universitario Virgen del Rocio
-
Valencia, Spanien, 46026
- Afsluttet
- Hospital Universitario y Politecnico La Fe
-
Valladolid, Spanien
- Trukket tilbage
- Hospital Universitario Rio Hortega
-
Zaragoza, Spanien
- Rekruttering
- Hospital Universitario Miguel Servet
-
-
-
-
-
Bucheon-si, Sydkorea
- Rekruttering
- Soon Chun Hyang University Bucheon Hospital
-
Busan, Sydkorea
- Rekruttering
- Pusan National University Hospital (PNUH)
-
Daegu, Sydkorea
- Rekruttering
- Keimyung University Dongsan Hospital
-
Daejeon, Sydkorea
- Trukket tilbage
- The Catholic University of Korea Daejeon St.Mary's Hospital
-
Goyang-si, Sydkorea
- Rekruttering
- Inje University Ilsan Paik Hospital
-
Gyeonggi-do, Sydkorea
- Trukket tilbage
- Korea University Ansan Hospital
-
Incheon, Sydkorea
- Rekruttering
- Inha University Hospital
-
Junggu, Sydkorea
- Rekruttering
- Kyungpook National University Hospital (KNUH)
-
Seongnam-si, Sydkorea
- Rekruttering
- CHA Bundang Medical Center, CHA University
-
Seoul, Sydkorea
- Rekruttering
- Samsung Medical Center
-
Seoul, Sydkorea
- Rekruttering
- Seoul National University Hospital
-
Seoul, Sydkorea
- Rekruttering
- Korea University Guro Hospital
-
Seoul, Sydkorea
- Trukket tilbage
- Hallym University Kangnam Sacred Heart Hospital
-
Seoul, Sydkorea
- Rekruttering
- Seoul National University Bundang Hospital (SNUBH)
-
Seoul, Sydkorea
- Rekruttering
- The Catholic University of Korea, Eunpyeong St. Mary's Hospital
-
Seoul, Sydkorea
- Rekruttering
- The Catholic University of Korea, Seoul St. Mary's Hospital
-
Seoul, Sydkorea
- Rekruttering
- Hanyang University Seoul Hospital
-
Seoul, Sydkorea
- Trukket tilbage
- Chung-Ang University Hospital
-
Seoul, Sydkorea
- Afsluttet
- Severance Hospital, Yonsei University Health System
-
Uijeongbu-si, Sydkorea
- Rekruttering
- The Catholic University of Korea, Uijeongbu St. Mary's Hospital
-
-
-
-
-
Chiang Mai, Thailand
- Rekruttering
- Chiang Mai University
-
Hat Yai, Thailand
- Rekruttering
- Prince of Songkla University (PSU) - Songklanagarind Hospital
-
Khon Kaen, Thailand
- Rekruttering
- Srinagarind Hospital - Khon Kaen University
-
-
-
-
-
Hradec Králové, Tjekkiet
- Rekruttering
- Hepato-Gastroenterologie HK, s.r.o.
-
Liberec, Tjekkiet
- Trukket tilbage
- Krajska Nemocnice Liberec
-
Ostrava, Tjekkiet
- Rekruttering
- Artroscan
-
Pilsen, Tjekkiet
- Rekruttering
- Research Site s.r.o.
-
Prague, Tjekkiet
- Rekruttering
- Institute for Clinical and Experimental Medicine - IKEM
-
-
-
-
-
Budapest, Ungarn
- Rekruttering
- Central Hospital of Northern Pest - Military Hospital
-
Debrecen, Ungarn
- Rekruttering
- Kenezy County Hospital
-
Szeged, Ungarn
- Rekruttering
- Szegedi Tudomanyegyetem AOK, I. sz Belgyogyaszati Klinika
-
-
Deltagelseskriterier
Berettigelseskriterier
Aldre berettiget til at studere
Tager imod sunde frivillige
Køn, der er berettiget til at studere
Beskrivelse
Inclusion Criteria :
- Male or female participants must be ≥18 years of age at the time of signing the informed consent.
- Participants with a definite or probable diagnosis of primary biliary cholangitis (PBC)
- Participants with cirrhosis at SV1. • Participants must be Child Pugh A or Child Pugh B.
- Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
- Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
Exclusion Criteria :
-
History or presence of other concomitant liver disease including but not limited to:
- i) Primary sclerosing cholangitis (PSC).
- ii) Autoimmune hepatitis (AIH) by simplified Diagnostic Criteria of the International Autoimmune Hepatitis Group (IAIHG) ≥6, or if treated for an overlap of PBC with AIH, or if there is clinical suspicion and evidence of overlap AIH features, that cannot be explained alone by insufficient response to UDCA.
- iii) Positive hepatitis B surface antigen (HBsAg). Participants with negative HBsAg and positive hepatitis B core antibody (HBcAb) may be eligible if hepatitis B virus deoxyribonucleic acid (HBV DNA) is negative.
- iv) Hepatitis C virus (HCV) infection defined by positive anti-HCV antibody and positive HCV ribonucleic acid (RNA) (Note: Participants with positive anti-HCV antibody due to previously treated HCV infection, may be enrolled if a confirmatory HCV RNA is undetectable and sustained viral response has been documented).
- v) Alcohol-associated liver disease (ALD).
- vi) Nonalcoholic steatohepatitis (NASH).
- vii) Other chronic liver diseases, such as alpha-1 antitrypsin deficiency.
-
History or presence of clinically significant hepatic decompensation, including:
- i) History of liver transplantation, current placement on a liver transplant list, current model for end-stage liver disease including (MELD) 3.0 score >12 due to hepatic impairment.
- ii) Evidence of complications of cirrhosis, including hepatic decompensation or evidence of significant portal hypertension complications including presence of uncontrolled ascites; history of variceal bleeding or related interventions (e.g. variceal banding, or transjugular intrahepatic portosystemic shunt placement); presence of hepatic encephalopathy Grade 2 or higher per West-Haven criteria; history or presence of spontaneous bacterial peritonitis. Note: participants with low-risk varices (Grade I) without history of bleeding or other treatment may be eligible to enrol.
- iii) Hepatorenal syndrome (HRS) (type I or II ). • vi) Hospitalisation for liver-related complication within 12 weeks prior to SV1.
-
Known history of human immunodeficiency virus (HIV) infection or having a positive confirmatory test for HIV type 1 or 2.
-
Medical conditions that may cause non-hepatic increases in ALP (e.g. Paget's disease).
-
Evidence of any other unstable or untreated clinically significant immunological, endocrine, hematologic, gastrointestinal, neurological, or psychiatric disease as evaluated by the investigator; other clinically significant conditions that are not well controlled.
-
Non-hepatic medical conditions that may diminish life expectancy to <2 years, including known cancers.
-
History of hepatocellular carcinoma.
-
Alpha-fetoprotein (AFP) >20 ng/mL with 4-phase liver computerised tomography (CT) or magnetic resonance imaging (MRI) imaging suggesting presence of hepatocellular carcinoma.
-
Known malignancy or history of malignancy within the last 5 years. Participants with non-melanoma skin cancer, or carcinoma in situ (e.g. breast carcinoma, cervical cancer in situ) that have undergone potentially curative therapy are not excluded.
-
Administration of the following medications is prohibited during the study, and prior to the study as per the timelines specified below: • i) 3 months prior to baseline: fibrates, seladelpar, glitazones, obeticholic acid, azathioprine, cyclosporine, methotrexate, mycophenolate, pentoxifylline, budesonide and other systemic corticosteroids (parenteral and oral chronic administration only); potentially hepatotoxic drugs (including α-methyl-dopa, sodium valproic acid, isoniazid or nitrofurantoin).
-
Participants who are currently participating in, plan to participate in, or have participated in an investigational drug study or medical device study containing active substance within 30 days or 5 half-lives, whichever is longer, prior to the screening period.
i) If the previous study was for an experimental therapy being studied for potential benefit in PBC, and the potential therapeutic agent was proven to have no beneficial effect in PBC and there are no safety concerns, the participant may enrol after 30 days or 5 half-lives from the last dose of the therapeutic agent, whichever is longer.ii) For therapeutic agents being studied for potential benefit in PBC for which it is still unclear if there may be a potential benefit, participants may enrol after 6 months from the last dose of the therapeutic agent.
-
Electrocardiogram (ECG) with QT interval corrected by Fridericia's formula (QTcF) >450 msec in males or QTcF >470 msec in females for participants without bundle branch block. For participants with bundle branch block or other intraventricular conduction delay, a longer QTcF >480 msec would be exclusionary.
-
Total bilirubin (TB) >5x ULN
-
Alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) >5x ULN at SV1
-
Creatinine phosphokinase (CPK) >2x ULN.
-
Platelet count <50,000/μL
-
International normalised ratio (INR) >1.8 in the absence of anticoagulant therapy.
-
Estimated glomerular filtration rate (eGFR) <45 mL/min/1.73m2 per the Modification of Diet in Renal Disease (MDRD)-6 Study formula at SV1.
-
Significant renal disease, including nephritic syndrome, chronic kidney disease (CKD) (defined as participants with evidence of significantly impaired kidney function or underlying kidney injury).
-
For female participants: known current pregnancy, or has a positive serum pregnancy test, or is breastfeeding.
-
Participants unwilling or unable to be abstinent from alcohol during the study.
-
History of alcohol abuse, or other substance abuse within 1 year prior to SV1.
-
Known hypersensitivity to elafibranor or to any of the excipients of the investigational product(s).
-
Mental instability or incompetence, such that the validity of informed consent or ability to be compliant with the study is uncertain.
-
Any other condition that, in the opinion of the investigator, would interfere with study participation or completion, or would put the participant at risk, including a potential participant assessed as being at high risk of noncompliance with the study.
-
Alkaline phosphatase (ALP) ≥10x ULN.
-
Albumin <2.8 g/dL due to impaired hepatic function.
Studieplan
Hvordan er undersøgelsen tilrettelagt?
Design detaljer
- Primært formål: Behandling
- Tildeling: Randomiseret
- Interventionel model: Parallel tildeling
- Maskning: Tredobbelt
Våben og indgreb
Deltagergruppe / Arm |
Intervention / Behandling |
|---|---|
|
Eksperimentel: Elafibranor 80 mg
Participants will take 1 tablet of elafibranor 80 mg per day orally with a glass of water at approximately the same time each morning, with or without food. |
Duration: up to an estimated 42-month (3.5-year) double-blind treatment period during which elafibranor 80 mg tablet will be administered once daily |
|
Placebo komparator: Placebo
Participants will take 1 placebo tablet per day orally (matching the 80 mg elafibranor sized tablet) with a glass of water at approximately the same time each morning, with or without food. |
Duration: up to an estimated 42-month (3.5-year) double-blind treatment period during which matching placebo tablet will be administered once daily |
Hvad måler undersøgelsen?
Primære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
|
Event-free survival
Tidsramme: From baseline until 4 weeks after the last dose of study intervention (maximum duration of 3.5 years)
|
Event-free survival is defined as the time from randomisation to either adjudicated disease progression or death, whichever occurs first. |
From baseline until 4 weeks after the last dose of study intervention (maximum duration of 3.5 years)
|
Sekundære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
|
Areal under plasmakoncentration-tid-kurven fra tiden 0 til 24 timer: AUC0-24
Tidsramme: På dag 1/basislinje, måned 6 og måned 12 (i en doseringsperiode på 24 timer)
|
På dag 1/basislinje, måned 6 og måned 12 (i en doseringsperiode på 24 timer)
|
|
|
Maksimal (peak) plasma-lægemiddelkoncentration: Cmax
Tidsramme: På dag 1/basislinje, måned 6 og måned 12 (i en doseringsperiode på 24 timer)
|
På dag 1/basislinje, måned 6 og måned 12 (i en doseringsperiode på 24 timer)
|
|
|
Tid til at nå maksimal (peak) plasmakoncentration efter lægemiddeladministration): Tmax
Tidsramme: På dag 1/basislinje, måned 6 og måned 12 (i en doseringsperiode på 24 timer)
|
På dag 1/basislinje, måned 6 og måned 12 (i en doseringsperiode på 24 timer)
|
|
|
Tilsyneladende clearance af lægemiddel fra plasma (CL)
Tidsramme: På dag 1/basislinje, måned 6 og måned 12 (i en doseringsperiode på 24 timer)
|
På dag 1/basislinje, måned 6 og måned 12 (i en doseringsperiode på 24 timer)
|
|
|
Tilsyneladende distributionsvolumen (VZ)
Tidsramme: På dag 1/basislinje, måned 6 og måned 12 (i en doseringsperiode på 24 timer)
|
På dag 1/basislinje, måned 6 og måned 12 (i en doseringsperiode på 24 timer)
|
|
|
Ændring fra baseline i alkalisk fosfatase (ALP)
Tidsramme: Ved 6. måned, 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
Ved 6. måned, 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
|
|
Ændring fra baseline i total bilirubin (TB)
Tidsramme: Ved 6. måned, 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
Ved 6. måned, 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
|
|
Procentdel af deltagere med ALP≤ 1,67x ULN og TB≤ ULN
Tidsramme: Ved 6. måned, 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
Ved 6. måned, 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
|
|
Procentdel af deltagere med et svar baseret på albuminnormalisering
Tidsramme: Ved 6. måned, 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
Ved 6. måned, 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
|
|
Ændring i serumniveauer af aspartataminotransferase (AST) sammenlignet med baseline
Tidsramme: Ved 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
Ved 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
|
|
Ændring i serumniveauer af ALAT sammenlignet med baseline
Tidsramme: Ved 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
Ved 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
|
|
Ændring i serumniveauer af gamma-glutamyltransferase (GGT) sammenlignet med baseline
Tidsramme: Ved 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
Ved 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
|
|
Ændring i serumniveauer af Albumin sammenlignet med baseline
Tidsramme: Ved 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
Ved 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
|
|
Ændring fra baseline i leverfunktion: international normaliseret ratio (INR)
Tidsramme: Ved 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
Ved 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
|
|
Ændring fra baseline i leverfunktion: fraktioneret ALP
Tidsramme: Ved 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
Ved 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
|
|
Procentdel af deltagere med ALP-reduktion på 40 %
Tidsramme: Ved 6. måned, 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
Ved 6. måned, 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
|
|
Procentdel af deltagere med ALP <1,5x ULN, ALP-fald ≥15 % og TB ≤ULN
Tidsramme: Ved 6. måned, 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
Ved 6. måned, 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
|
|
Procentdel af deltagere med ALP <1,5x ULN, ALP-fald ≥40 % og TB ≤ULN
Tidsramme: Ved 6. måned, 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
Ved 6. måned, 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
|
|
Procentdel af deltagere med ALP <1,67x ULN, ALP-fald ≥15 % og TB ≤ULN
Tidsramme: Ved 6. måned, 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
Ved 6. måned, 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
|
|
Procentdel af deltagere med ALP <3x ULN, AST <2x ULN og TB ≤1 mg/dL (Paris I)
Tidsramme: Ved 6. måned, 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
Ved 6. måned, 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
|
|
Procentdel af deltagere med ALP ≤1,5x ULN, AST ≤1,5x ULN og TB ≤1 mg/dL (Paris II-kriterier)
Tidsramme: Ved 6. måned, 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
Ved 6. måned, 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
|
|
Procentdel af deltagere med normalisering af unormal TB
Tidsramme: Vurderet ved 6. måned, 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
Vurderet ved 6. måned, 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
|
|
Procentdel af deltagere med normalisering af unormal TB og albumin (Rotterdam-kriterier)
Tidsramme: Ved 6. måned, 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
Ved 6. måned, 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
|
|
Ændring fra baseline i lipidparametre: total kolesterol (TC)
Tidsramme: Ved 6. måned, 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
Ved 6. måned, 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
|
|
Ændring fra baseline i lipidparametre: højdensitetslipoproteinkolesterol (HDL-C)
Tidsramme: Ved 6. måned, 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
Ved 6. måned, 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
|
|
Ændring fra baseline i lipidparametre: lavdensitetslipoproteinkolesterol (LDL-C)
Tidsramme: Ved 6. måned, 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
Ved 6. måned, 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
|
|
Ændring fra baseline i lipidparametre: triglycerider (TG)
Tidsramme: Ved 6. måned, 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
Ved 6. måned, 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
|
|
Ændring fra baseline i lipidparametre: beregnet meget lavdensitetslipoproteinkolesterol (VLDL-C)
Tidsramme: Ved 6. måned, 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
Ved 6. måned, 12. måned og hver 12. måned indtil behandlingens afslutning (maksimal varighed på 3,5 år)
|
|
|
Change from baseline in hepatic function: Conjugated (direct) bilirubin
Tidsramme: At Month 12, and every 12 months up to end of treatment (maximum duration of 3.5 years)
|
At Month 12, and every 12 months up to end of treatment (maximum duration of 3.5 years)
|
|
|
Percentage of participants with ALP reduction >40% or ALP normalisation (Barcelona criteria)
Tidsramme: At Month 6, Month 12, and every 12 months up to end of treatment (maximum duration of 3.5 years)
|
At Month 6, Month 12, and every 12 months up to end of treatment (maximum duration of 3.5 years)
|
|
|
Percentage of participants with TB ≤0.6× ULN in participants with TB >0.6× ULN at baseline
Tidsramme: At Month 6, Month 12, and every 12 months up to end of treatment (maximum duration of 3.5 years)
|
At Month 6, Month 12, and every 12 months up to end of treatment (maximum duration of 3.5 years)
|
|
|
Percentage of participants experiencing Treatment Emergent Adverse Events (TEAEs), treatment-related TEAEs, Serious Adverse Events (SAEs), and Adverse Events of Special Interests (AESIs)
Tidsramme: From baseline until 4 weeks after the last dose of study intervention (maximum duration of 3.5 years)
|
An adverse event (AE) is any unfavourable medical occurrence in a trial participant administered the investigational product. The AE does not necessarily have a causal relationship with the treatment. AESIs are AEs that may or may not be serious but are of special importance to a particular drug or class of drugs. |
From baseline until 4 weeks after the last dose of study intervention (maximum duration of 3.5 years)
|
|
Percentage of participants developing clinically significant changes in physical examination findings
Tidsramme: From baseline until 4 weeks after the last dose of study intervention (maximum duration of 3.5 years)
|
Complete physical examination at screening and targeted examination at all other clinical visit timepoints. |
From baseline until 4 weeks after the last dose of study intervention (maximum duration of 3.5 years)
|
|
Percentage of participants developing clinically significant changes in vital signs
Tidsramme: From baseline until 4 weeks after the last dose of study intervention (maximum duration of 3.5 years)
|
Percentage of participants with clinically significant changes in Vital Signs will be reported. The clinical significance will be graded by the investigator. |
From baseline until 4 weeks after the last dose of study intervention (maximum duration of 3.5 years)
|
|
Percentage of participants developing clinically significant changes in Electrocardiogram (ECG) readings.
Tidsramme: From baseline until 4 weeks after the last dose of study intervention (maximum duration of 3.5 years)
|
Percentage of participants with clinically significant changes in ECG readings will be reported. The clinical significance will be graded by the investigator. |
From baseline until 4 weeks after the last dose of study intervention (maximum duration of 3.5 years)
|
|
Percentage of participants with clinically significant changes in laboratory parameters (blood chemistry, hematology, coagulation and urinalysis)
Tidsramme: From baseline until 4 weeks after the last dose of study intervention (maximum duration of 3.5 years)
|
Percentage of participants with clinically significant change in laboratory parameters (blood chemistry, hematology and coagulation) will be reported. The clinical significance will be graded by the investigator. |
From baseline until 4 weeks after the last dose of study intervention (maximum duration of 3.5 years)
|
|
Percentage of participants with complete biochemical response
Tidsramme: At Month 6, Month 12, and every 12 months up to end of treatment (maximum duration of 3.5 years)
|
Defined as normal levels of TB, ALP, transaminases, albumin, and International normalised ratio (INR) |
At Month 6, Month 12, and every 12 months up to end of treatment (maximum duration of 3.5 years)
|
|
Percentage of participants with normalisation of TB and ALP
Tidsramme: At Month 6, Month 12, and every 12 months up to end of treatment (maximum duration of 3.5 years)
|
Defined as TB< Upper Limit Normal (ULN) and ALP< ULN |
At Month 6, Month 12, and every 12 months up to end of treatment (maximum duration of 3.5 years)
|
|
Percentage of participants with stabilisation in TB (i.e. no increase)
Tidsramme: At Month 6, Month 12, and every 12 months up to end of treatment (maximum duration of 3.5 years)
|
Defined as TB< 1x ULN or increase from baseline <0.1x ULN |
At Month 6, Month 12, and every 12 months up to end of treatment (maximum duration of 3.5 years)
|
|
Change from baseline in liver stiffness measurement (LSM)
Tidsramme: At Month 12, and every 12 months up to end of treatment (maximum duration of 3.5 years)
|
Assessed by vibration-controlled transient elastography (VCTE) using Fibroscan® on the day of the visit. |
At Month 12, and every 12 months up to end of treatment (maximum duration of 3.5 years)
|
|
Change from baseline in PBC risk scores based in Global PBC Study Group (GLOBE) score
Tidsramme: At Month 12, and every 12 months up to end of treatment (maximum duration of 3.5 years)
|
The GLOBE score is a validated risk assessment tool providing an estimate of transplant-free survival for patients with PBC. It was developed by the Global PBC Study Group using Cox regression model on over 4,000 patients with PBC. Lower GLOBE score predicts lower risk. It is calculated from the following equation: GLOBE score = (0.044378 * age + 0.93982 * LN(total bilirubin/ULN) +(0.335648 * LN(alkaline phosphatase/ULN)) - 2.266708 * albumin /LLN -0.002581 * platelet count per 109/L) + 1.216865 GLOBE scoring system, which calculation is based on serum values of bilirubin, ALP, albumin and platelet count after 1 year of treatment and age at baseline. A high number is indicative of a worse score. |
At Month 12, and every 12 months up to end of treatment (maximum duration of 3.5 years)
|
|
Change from baseline in PBC risk scores based on United Kingdom (UK)-PBC score.
Tidsramme: At Month 12, and every 12 months up to end of treatment (maximum duration of 3.5 years)
|
PBC risk score developed by United Kingdom (UK)-PBC Consortium is a scoring system and the calculation is based on laboratory test measurements and upper limits of normal (ULN) for the total bilirubin (BIL12); alanine transaminase or aspartate transaminase (TA12), and alkaline phosphatase (ALP12) after at least 12 months of UDCA, and the laboratory test measurements and lower limits of normal (LLN) for the serum albumin and platelet count in the same timeframe. A high number is indicative of a worse score. |
At Month 12, and every 12 months up to end of treatment (maximum duration of 3.5 years)
|
|
Percentage of participants with LSM ≥15 kPa
Tidsramme: At Month 12, and every 12 months up to end of treatment (maximum duration of 3.5 years)
|
Assessed by VCTE using Fibroscan® |
At Month 12, and every 12 months up to end of treatment (maximum duration of 3.5 years)
|
|
Percentage of participants with no worsening of LSM
Tidsramme: At Month 12, and every 12 months up to end of treatment (maximum duration of 3.5 years)
|
Assessed by VCTE using Fibroscan® defined as no increase >2kPa from baseline |
At Month 12, and every 12 months up to end of treatment (maximum duration of 3.5 years)
|
|
Percentage of participants with a response in PBC Worst Itch NRS score
Tidsramme: Through 6 months up to end of treatment (maximum duration of 3.5 years)
|
Defined as ≥2-point reduction from baseline NRS in participants with a baseline NRS ≥4 |
Through 6 months up to end of treatment (maximum duration of 3.5 years)
|
|
Percentage of participants with a response in PBC Worst Itch NRS
Tidsramme: Assessed through 6 months up to end of treatment (maximum duration of 3.5 years)
|
Defined as ≥3-point reduction from baseline NRS in participants with a baseline NRS ≥4 |
Assessed through 6 months up to end of treatment (maximum duration of 3.5 years)
|
|
Change from baseline in 5D-Itch scale
Tidsramme: Assessed at every 6 months up to end of treatment (maximum duration of 3.5 years)
|
Questionnaire that assesses symptoms in terms of 5 domains: degree, duration, direction, disability and distribution. Participants rate their symptoms over the preceding 2-week period on a 1 to 5 scale, with 5 being the most affected. |
Assessed at every 6 months up to end of treatment (maximum duration of 3.5 years)
|
|
Change from baseline in Patient Global Impression of Severity (PGI-S)
Tidsramme: Assessed at every 6 months up to end of treatment (maximum duration of 3.5 years)
|
A 1-item, 5-point scale designed to assess the participant's impression of disease severity |
Assessed at every 6 months up to end of treatment (maximum duration of 3.5 years)
|
|
Patient Global Impression of Change (PGI-C)
Tidsramme: Assessed at every 6 months up to end of treatment (maximum duration of 3.5 years)
|
A 1-item, 5-point scale designed to assess the participant's impression of change in disease severity since the baseline visit |
Assessed at every 6 months up to end of treatment (maximum duration of 3.5 years)
|
|
Change from baseline in Patient Reported Outcome Measurement Information System (PROMIS) Fatigue Short Form 7a
Tidsramme: Assessed at every 6 months up to end of treatment (maximum duration of 3.5 years)
|
Consists of 7 items that measure both the experience of fatigue and the interference of fatigue on daily activities over the past week. Response options are on a 5-point Likert scale, ranging from 1 to 5. Scores can range from 7 to 35, with higher scores indicating greater fatigue. |
Assessed at every 6 months up to end of treatment (maximum duration of 3.5 years)
|
|
Change from baseline in the Epworth Sleepiness Scale (ESS)
Tidsramme: Assessed at every 6 months up to end of treatment (maximum duration of 3.5 years)
|
Self-administered questionnaire that consists of 8 questions asking to rate how likely it is to fall asleep in different situations commonly encountered in daily life (each question can be scored from 0 to 3 points; '0' indicates no sleepiness, '3' indicates significant sleepiness). It provides a total score which has been shown to relate to the participant's level of daytime sleepiness (total score range 0-24 points). |
Assessed at every 6 months up to end of treatment (maximum duration of 3.5 years)
|
|
Change from baseline in PBC-40 score
Tidsramme: Assessed at every 6 months up to end of treatment (maximum duration of 3.5 years)
|
40-item questionnaire that assesses symptoms across 6 domains: fatigue, emotional and social, cognitive function, general symptoms and itch. Participants respond on a verbal response scale, depending on the section options range from 'never' / 'not at all' / 'strongly disagree' to 'always' / 'very much' / 'strongly agree'. Six items (3/3 in the itch domain, 2/10 in the social domain, and 1/7 in the general symptoms domain) also include a 'does not apply' option. A score for each domain is provided (but a total score is not calculated), with each verbal response scale correlating to a score of 1 to 5 per item (0 to 5 on items with a 'does not apply' option) with 5 being the most affected (greatest burden). The PBC-40 has a 4-week recall period. |
Assessed at every 6 months up to end of treatment (maximum duration of 3.5 years)
|
|
Change from baseline in PBC Worst Itch Numeric Rating Scale (NRS) score
Tidsramme: Assessed through 6 months up to end of treatment (maximum duration of 3.5 years)
|
Self-administered patient-reported outcome questionnaire that measures itch intensity. It asks participants to rate the intensity of their Worst Itch on an 11-point scale ranging from 0 (no itch) to 10 (Worst Itch imaginable): - once daily (24-hour recall period) using the eDiary during the screening and initial 2 years of the study, - at the clinic visits (7-day recall period), from Year 3 onwards |
Assessed through 6 months up to end of treatment (maximum duration of 3.5 years)
|
|
Change from baseline in EuroQol 5-dimensional 5-level questionnaire (EQ-5D-5L)
Tidsramme: Assessed at every 6 months up to end of treatment (maximum duration of 3.5 years)
|
Self-administered standardised questionnaire that assesses the 5-dimensions of mobility, self-care, usual activities, pain/discomfort, anxiety/depression descriptively (each dimension has 5 levels) and the overall health state via an EQ Visual Analogue Scale (VAS). |
Assessed at every 6 months up to end of treatment (maximum duration of 3.5 years)
|
|
Change from baseline in Work Productivity and Activity Impairment General Health (WPAI-GH)
Tidsramme: Assessed at every 6 months up to end of treatment (maximum duration of 3.5 years)
|
Questionnaire that measures absenteeism, presenteeism as well as the impairments in unpaid activity because of health problem during the past seven days. It consists of 6 questions: 1=currently employed; 2=hours missed because of health problems; 3=hours missed because of other reasons; 4=hours actually worked; 5=degree health affected productivity while working (using a 0 to 10 Visual Analogue Scale (VAS)); 6=degree health affected productivity in regular unpaid activities (VAS). |
Assessed at every 6 months up to end of treatment (maximum duration of 3.5 years)
|
|
Time to the first occurrence of each of individual adjudicated clinical outcome events
Tidsramme: From baseline until 4 weeks after the last dose of study intervention
|
Among: • All-cause mortality • Liver-related mortality • Liver transplant • MELD-3.0 score ≥15 in participants with baseline MELD score ≤12 • Liver decompensation |
From baseline until 4 weeks after the last dose of study intervention
|
|
Percentage of participants who experience any subsequent adjudicated clinical outcome events after the first occurrence of such an event
Tidsramme: From baseline until 4 weeks after the last dose of study intervention
|
Among: • All-cause mortality • Liver-related mortality • Liver transplant • MELD-3.0 score ≥15 in participants with baseline MELD score ≤12 • Liver decompensation |
From baseline until 4 weeks after the last dose of study intervention
|
|
Change from baseline in model for end-stage liver disease (MELD) 3.0 score
Tidsramme: From screening until end of treatment (maximum duration of 3.5 years)
|
The MELD 3.0 score is calculated from parameters (sex, creatinine, bilirubin, INR, sodium and albumin) where a high score indicates a greater risk of needing a liver transplant. |
From screening until end of treatment (maximum duration of 3.5 years)
|
|
Change from baseline in Child Pugh grade
Tidsramme: From screening until end of treatment (maximum duration of 3.5 years)
|
Child Pugh grade is calculated using bilirubin, albumin, INR, ascites and encephalopathy. A high grade is indicative of worse liver disease. |
From screening until end of treatment (maximum duration of 3.5 years)
|
Samarbejdspartnere og efterforskere
Sponsor
Efterforskere
- Studieleder: Ipsen Medical Director, Ipsen
Datoer for undersøgelser
Studer store datoer
Studiestart (Faktiske)
Primær færdiggørelse (Anslået)
Studieafslutning (Anslået)
Datoer for studieregistrering
Først indsendt
Først indsendt, der opfyldte QC-kriterier
Først opslået (Faktiske)
Opdateringer af undersøgelsesjournaler
Sidste opdatering sendt (Faktiske)
Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier
Sidst verificeret
Mere information
Begreber relateret til denne undersøgelse
Yderligere relevante MeSH-vilkår
- Patologiske processer
- Sygdomme i fordøjelsessystemet
- Galdevejssygdomme
- Leversygdomme
- Galdevejssygdomme
- Fibrose
- Kolestase, intrahepatisk
- Kolestase
- Levercirrhose
- Patologiske tilstande, tegn og symptomer
- Levercirrhose, galdevejr
- 2- (2,6-dimethyl-4- (3- (4- (methylthio) phenyl) -3-oxo-1-propenyl) phenoxyl) -2-methylpropansyre
Andre undersøgelses-id-numre
- CLIN-60190-454
- 2023 (U.S. NIH-bevilling/kontrakt)
- 2023-505251-43-00 (Ctis)
Plan for individuelle deltagerdata (IPD)
Planlægger du at dele individuelle deltagerdata (IPD)?
IPD-planbeskrivelse
Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, annotated case report form, statistical analysis plan, and dataset specifications.
Patient level data will be anonymized and study documents will be redacted to protect the privacy of study participants.
IPD-delingstidsramme
Where applicable, data from eligible studies are available 6 months after the studied medicine and indication have been approved in the US and/or EU.
IPD-delingsadgangskriterier
Further details on Ipsen's sharing criteria and process for sharing are available here (https://www.ipsen.com/science/clinical-trials/clinical-data-transparency/).
Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter
Studerer et amerikansk FDA-reguleret lægemiddelprodukt
Studerer et amerikansk FDA-reguleret enhedsprodukt
Disse oplysninger blev hentet direkte fra webstedet clinicaltrials.gov uden ændringer. Hvis du har nogen anmodninger om at ændre, fjerne eller opdatere dine undersøgelsesoplysninger, bedes du kontakte register@clinicaltrials.gov. Så snart en ændring er implementeret på clinicaltrials.gov, vil denne også blive opdateret automatisk på vores hjemmeside .