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A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Sponsor: Swedish Orphan Biovitrum
Summary
This is a 36-month, long-term, multicenter, observational study designed to describe the real world effectiveness of pegcetacoplan in patients with PNH. Patients meeting the eligibility criteria will be enrolled in the study and followed prospectively for approximately 36 months. Patient data will be collected from start of pegcetacoplan treatment to end of follow-up. Retrospective data on pegcetacoplan will be captured from the time of pegcetacoplan treatment initiation. Pegcetacoplan treatment data will be collected for a minimum of approximately 36 months and up to a maximum of approximately 72 months, including retrospective period depending on when the patient started pegcetacoplan treatment. After pegcetacoplan treatment discontinuation, patients will remain in the study for 8 weeks to capture any AEs. The scope of the study is to collect both retrospective and prospective data. Baseline is defined as start of pegcetacoplan treatment. The main part of the study will be prospective,collecting data on effectiveness, safety (all AEs), patient- and clinician-reported outcomes and health care resource use.
Official title: A Single Arm, Long-term, Multicentre Observational Study to Evaluate Effectiveness of Pegcetacoplan Under Real World Conditions in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Key Details
Gender
All
Age Range
18 Years - Any
Study Type
OBSERVATIONAL
Enrollment
200
Start Date
2023-06-26
Completion Date
2029-08-15
Last Updated
2025-05-14
Healthy Volunteers
No
Conditions
Interventions
Pegcetacoplan
Pegcetacoplan will be prescribed according to the label in patients with PNH.
Locations (75)
Liverpool Hospital
Liverpool, New South Wales, Australia
Calvary Mater Newcastle
Waratah, New South Wales, Australia
Sunshine Coast University Hospital
Birtinya, Queensland, Australia
AZ Sint-Jan
Bruges, Belgium
CHU Brugmann-Site Horta
Brussels, Belgium
Centre Hospitalier Universitaire de Liege
Liège, Belgium
AZ Turnhout - Campus Sint-Elisabeth
Turnhout, Belgium
University Health Network
Toronto, Ontario, Canada
Hopital Maisonneuve-Rosemont d/b/a CIUSSS de l'Est-de-l'Île-de-Montréal
Montreal, Quebec, Canada
Fakultni nemocnice Brno
Brno, Czechia
Ustav hematologie a krevni transfuze
Prague, Czechia
Helsingin yliopistollinen keskussairaala
Helsinki, Finland
CHU Nice - Hôpital de l'Archet 1
Nice, Alpes Maritimes, France
CHU de Bordeaux - Hôpital Haut-Lévêque
Pessac, Gironde, France
Hopital Foch
Suresnes, Hauts De Seine, France
CHU de Grenoble - Hôpital Albert Michallon
Grenoble, Isere, France
CHU de Nantes - Hotel Dieu
Nantes, Loire Atlantique, France
CHU de Nancy - Hôpital de Brabois Adultes
Vandœuvre-lès-Nancy, Meurthe Et Moselle, France
Hopital Claude Huriez - CHRU Lille
Lille, Nord, France
Hôpital Saint-Louis
La Defense, Paris, France
Centre Hospitalier Lyon Sud
Pierre-Bénite, Rhone, France
CHU Poitiers
Poitiers, France
Alb-Fils-Kliniken GmbH
Goettigen, Baden-Wurttemberg, Germany
Studienzentrum Aschaffenburg
Aschaffenburg, Bavaria, Germany
Universitaetsklinikum Frankfurt Goethe-Universitaet
Frankfurt am Main, Hesse, Germany
Universitaetsklinikum Giessen und Marburg GmbH Standort Giessen
Giessen, Hesse, Germany
Dres Ballo und Dr. Böck
Offenbach, Hesse, Germany
Universitaetsklinikum Essen
Essen, North Rhine-Westphalia, Germany
Universitaetsklinikum Leipzig AoeR
Leipzig, Saxony, Germany
Praxis Fenchel Saalfeld
Saalfeld, Thuringia, Germany
MV Zentrum für Onkologie und Hämatologie
Cologne, Germany
Evangelismos Hospital
Athens, Greece
University General Hospital "Attikon"
Athens, Greece
University General Hospital of Larissa
Larissa, Greece
G Papanikolaou General Hospital
Thessaloniki, Greece
General Hospital of Thessaloniki "IPPOKRATEIO"
Thessaloniki, Greece
DPC - Orszagos Hematologiai es Infektologiai Intezet
Budapest, Hungary
Semmelweis Egyetem
Budapest, Hungary
Semmelweis University
Budapest, Hungary
Azienda Ospedaliero Universitaria Consorziale Policlinico di Bari
Bari, Italy
Ospedale San Bassiano
Bassano del Grappa, Italy
Irccs Aou Di Bologna- Pol. Di S.Orsola
Bologna, Italy
Ospedale Oncologico Armando Businco
Cagliari, Italy
Azienda Ospedaliera Universitaria Careggi
Florence, Italy
Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico
Milan, Italy
Ospedale San Raffaele
Milan, Italy
Ospedale Maggiore della Carità
Novara, Italy
Grande Ospedale Metropolitano - Presidio Morelli
Reggio Calabria, Italy
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Roma, Italy
Università di Roma La Sapienza
Rome, Italy
Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino
Torino, Italy
Uniwersytecki Szpital Kliniczny w Bialymstoku
Bialystok, Poland
Szpital Uniwersytecki nr 2 im. dr J. Biziela w Bydgoszczy
Bydgoszcz, Poland
Uniwersyteckie Centrum Kliniczne
Gdansk, Poland
SPZOZ Szpital Uniwersytecki w Krakowie
Krakow, Poland
WWCOiT in Lodz, Oddział Hematologii i Transplantologii
Lodz, Poland
Uniwersytecki Szpital Kliniczny nr 1 w Lublinie
Lublin, Poland
Warszawski Uniwersytet Medyczny
Warsaw, Poland
Prince Faisal Bin Bandar Cancer Center
Buraidah, Saudi Arabia
King Faisal Specialist Hospital & Research Center
Jeddah, Saudi Arabia
King Faisal Specialist Hospital and Research Center - Madinah
Riyadh, Saudi Arabia
King Faisal Specialist Hospital and Research Center - Riyadh
Riyadh, Saudi Arabia
ICO Badalona - Hospital Universitari Germans Trias i Pujol
Badalona, Spain
Hospital de Cruces
Barakaldo, Spain
Hospital Clinic de Barcelona
Barcelona, Spain
Hospital Universitari Vall d'Hebron
Barcelona, Spain
Hospital de Basurto
Bilbao, Spain
Hospital Universitario Donostia
Donostia / San Sebastian, Spain
Hospital Universitario Virgen de la Nieves
El Palmar, Spain
Hospital Universitario de Gran Canaria Dr. Negrin
Las Palmas de Gran Canaria, Spain
Hospital Lucus Augusti
Lugo, Spain
Hospital Regional Universitario de Malaga
Málaga, Spain
Hospital Universitario Central de Asturias
Oviedo, Spain
Complejo Hospitalario Universitario de Santiago
Santiago de Compostela, Spain
King's College Hospital
London, United Kingdom