A Study Observing Everyday Effectiveness and Safety of the Drug Elafibranor in Participants With Primary Biliary Cholangitis Who Are Receiving Ongoing Treatment

Официальное название

Prospective Non-interventional, Phase IV Multicentre Study to Assess the Effectiveness, Safety and Tolerability of Elafibranor 80 mg/Day in Participants With Primary Biliary Cholangitis Receiving Treatment in a Real-world Setting.

Обзор

This study will collect information from participants with Primary Biliary Cholangitis (PBC) as they use the drug elafibranor in real world setting. PBC is a progressive rare liver disease in which tubes in the liver called bile ducts are damaged. The liver damage in PBC may lead to scarring (cirrhosis). PBC may also be associated with multiple symptoms including pruritus (itching) and fatigue. Many patients with PBC may require liver transplant or may die if the disease progresses and a liver transplant is not done. In this study the main aim is to observe the effectiveness, safety and tolerability of elafibranor in participants with PBC who are receiving treatment in real world setting. The total study duration for each participants will be 60 months (approximately 5 years).

Первичные конечные точки

• Percentage of participants with response to treatment [Срок оценки: At month 6]

Вторичные конечные точки (12)

• Percentage of participants with normalization of ALP levels [Срок оценки: Up to 60 months following enrollment, based on routine physician follow-up visit]
• Percentage of participants with response to treatment [Срок оценки: Up to 60 months following enrollment, based on routine physician follow-up visits]
• Change from baseline in liver function parameters: Serum levels of alanine aminotransferase (ALT) [Срок оценки: Up to 60 months following enrollment, based on routine physician follow-up visit]
• Change from baseline in liver function parameters: Serum levels of Aspartate aminotransferase (AST) [Срок оценки: Up to 60 months following enrollment, based on routine physician follow-up visit]
• Change from baseline in liver function parameters: Serum levels of Gamma-glutamyl transferase (GGT) [Срок оценки: Up to 60 months following enrollment, based on routine physician follow-up visit]
• Change from baseline in liver function parameters: Serum levels of TB [Срок оценки: Up to 60 months following enrollment, based on routine physician follow-up visit]
• Change from baseline in liver function parameters: Conjugated (direct) bilirubin [Срок оценки: Up to 60 months following enrollment, based on routine physician follow-up visit]
• Change from baseline in liver function parameters: Serum levels of creatinine [Срок оценки: Up to 60 months following enrollment, based on routine physician follow-up visit]
• Change from baseline in liver function parameters: Serum levels of albumin [Срок оценки: Up to 60 months following enrollment, based on routine physician follow-up visit]
• Change from baseline in pruritus based on PBC Itch score [Срок оценки: Up to 60 months following enrollment, based on routine physician follow-up visit]
• Change from baseline in fatigue based on functional assessment of chronic illness therapy-fatigue (FACIT-Fatigue) scale [Срок оценки: Up to 60 months following enrollment, based on routine physician follow-up visit]
• Change from baseline in sleep based on Pittsburgh sleep quality index (PSQI) [Срок оценки: Up to 60 months following enrollment, based on routine physician follow-up visit]

Критерии участия

Критерии включения

• Participant has provided written informed consent and agrees to comply with the study protocol.
• Participant with PBC diagnosis.
• Participant for whom the treating physician has decided to start or participants who are currently receiving treatment with commercialized elafibranor.
• If a participant has a caregiver who agrees to complete the caregiver questionnaires, an informed consent should be collected from the caregiver before any data is collected.

Критерии исключения

• Participant is currently participating or, plans to participate in an investigational drug study or medical device study containing active substance.
• Participant with known hypersensitivity to the product or to any of its excipients.
• Participant with mental instability or incompetence, such that the validity of informed consent or ability to be compliant with the study is uncertain.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Когортное

Центры проведения

США · 20 центров

• Southern California Research Center — Coronado
• Cedars-Sinai Medical Center — Los Angeles
• University of California Davis Medical Center — Sacramento
• South Denver Gastroenterology,P.C. — Englewood
• Yale University School of Medicine — New Haven
• Schiff Center for Liver Diseases - University of Miami — Miami
• Beth Israel Deaconess Medical Center, Liver Research Center — Boston
• Virtua Center for Liver Disease - Cherry Hill — Cherry Hill
• … и ещё 12 центров

Великобритания · 9 центров

• Aberdeen Royal Infirmary NHS Grampian Grampian Health Board — Aberdeen
• Belfast Health and Social Care Trust - Royal Victoria Hospital — Belfast
• Queen Elizabeth Hospital Birmingham - University Hospitals Birmingham NHS Foundation Trust — Birmingham
• Bradford Royal Infirmary - Bradford Teaching Hospitals NHS Foundation — Bradford
• Hull Royal Infirmary - Hull University Teaching Hospitals NHS Trust — Hull
• King's College Hospital NHS Foundation Trust — London
• The Newcastle upon Tyne Hospitals NHS Foundation Trust - Freeman Hospital — Newcastle
• Queen's Medical Centre - Nottingham University Hospitals NHS Trust — Nottingham
• … и ещё 1 центр

Германия · 8 центров

• Charite Universitatsmedizin Berlin — Berlin
• DRK Kliniken Berlin Mitte — Berlin
• Klinikum der Johann Wolfgang Goethe-Universitaet — Frankfurt
• Studiengesellschaft BSF — Halle
• Gastroenterologsiche Studiengesellschaft Herne — Herne
• Universitaet des Saarlandes — Homburg
• Universitaetsklinikum Leipzig — Leipzig
• Hospital of the Merciful Brothers Trier — Trier

Италия · 7 центров

• ASST-Ospedale Papa Giovanni XXIII — Bergamo
• Ospedale Garibaldi Nesima — Catania
• Universita Degli Studi Di Firenze - Azienda Ospedaliero-Universitaria Careggi (AOUC) — Florence
• ASST Grande Ospedale Metropolitano Niguarda — Milan
• Ospedale Maggiore Della Carita — Novara
• A. Gemelli University Hospital, Catholic University of the Sacred Heart — Roma
• Fondazione Policlinico Universitario Campus Bio-Medico — Roma

Испания · 6 центров

• Hospital de Torrecardenas — Almería
• Hospital Clinic de Barcelona (Hospital Clinic i Provincial) — Barcelona
• Hospital Universitario Vall d'Hebron — Barcelona
• Hospital General Universitario Gregorio Maranon (HGUGM) — Madrid
• Complexo Hospitalario Universitario de Pontevedra — Pontevedra
• Instituto De Investigacion Marques De Valdecilla (IDIVAL) — Santander

Канада · 5 центров

• Centre de Recherche du Centre Hospitalier de l'Universite de Montreal (CRCHUM) — Montreal
• University of Calgary — Calgary
• London Health Sciences Centre (LHSC) - University Hospital — London
• The Ottawa Hospital - General Campus — Ottawa
• (G.I.R.I) GI Research Institute Foundation — Vancouver

Греция · 5 центров

• General Hospital of Athens Laiko — Athens
• University Hospital of Heraklion — Heraklion
• University General Hospital of Larissa — Larissa
• University General Hospital of Patras — Pátrai
• Ippokratio General Hospital of Thessaloniki — Thessaloniki

South Korea · 5 центров

• Pusan National University Hospital (PNUH) — Busan
• Seoul National University Bundang Hospital (SNUBH) — Seongnam-si
• Samsung Medical Center — Seoul
• Seoul National University Hospital — Seoul
• Severance Hospital, Yonsei University Health System — Seoul

ОАЭ · 4 центра

• Clemenceau Medical Center Hospital — Dubai
• Mediclinic Airport Road Hospital — Dubai
• Rashid Hospital — Dubai
• Sheikh Shakhbout Medical City — Dubai

Австрия · 3 центра

• Medizinische Universitaetsklinik Graz — Graz
• Medical University Innsbruck — Innsbruck
• Ordensklinikum Linz GmbH Barmherzige Schwestern — Linz

Австралия · 2 центра

• Royal Prince Alfred Hospital — Camperdown
• St Vincent's Hospital — Fitzroy

Идентификаторы

NCT: NCT06447168 · CLIN-60190-461

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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