A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Registration Number
- NCT05776472
- Lead Sponsor
- Swedish Orphan Biovitrum
- Brief 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.
- Detailed Description
As pegcetacoplan is a new product on the market, with a new mechanism of action, there is an urgent need to provide data to treaters, payers and the PNH community on the real-world usage and effectiveness of pegcetacoplan. This study aims to fill part of that knowledge gap and to add to the knowledge base regarding the use of pegcetacoplan in routine medical practice. Another important rationale for this study is to provide information on RBC transfusions and health care resource utilization pre and post pegcetacoplan treatment initiation.
The study plans to include approximately 200 patients at 70 sites in Europe, Middle East, Canada and Australia. Additional countries may be added in the study if necessary. 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. Patients will come to their routine visits and the available data from each visit will be collected.
The scope of the study is to collect both retrospective and prospective data. The main part of the study will be prospective, collecting data on effectiveness, safety, patient- and clinician-reported outcomes and health care resource use. The study will also collect retrospective data before pegcetacoplan treatment start, which will consist of information on PNH treatment, blood transfusions and healthcare resource use. Data will be collected for up to 12 months prior to pegcetacoplan treatment start. As patients may have been treated with pegcetacoplan for up to 12 months prior to enrollment, retrospective data may be collected for up to 24 months. This means that the total data collection period including both the retrospective and the prospective part is up to 48 (+/- 3) months.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 200
- Patients ≥18 years of age with a documented PNH diagnosis.
- Patient started routine treatment with pegcetacoplan for PNH up to 12 months before enrollment or prescribed pegcetacoplan at enrollment. Decision to initiate treatment shall be made by the treating physician and independently from the decision to include the patient in the study.
- Patient is willing and able to provide written informed consent to participate in the study in a manner approved by the Institutional Review Board/Independent Ethics Committee and local regulations.
- Enrollment in a concurrent clinical interventional study, or intake of an Investigational Medicinal Product (IMP), within three months prior to the start of the current pegcetacoplan treatment.
- Initiated current treatment with pegcetacoplan in an interventional study.
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Change in observed hemoglobin level from initiation of treatment with pegcetacoplan to 6 months 6 months Hemoglobin level in g/dL.
- Secondary Outcome Measures
Name Time Method Hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of follow-up 36 months Yes/No
Annualized number of red blood cell (RBC) transfusions during pegcetacoplan treatment until end of follow-up compared to the 12 month period before pegcetacoplan treatment 12 months Total number of RBC transfusions
Annualized number of red blood cell (RBC) units during pegcetacoplan treatment until end of follow-up compared to the 12 month period before pegcetacoplan treatment 12 months Total number of RBC units
Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up 36 months Ferritin in ug/L
Hemoglobin ≥ 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up 36 months Hemoglobin in g/dL
Increase in hemoglobin levels of ≥ 2 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up 36 months Hemoglobin in g/dL
Change of LDH values from initiation of pegcetacoplan treatment to 6 months 6 months Lactate Dehydrogenase (LDH) in U/L
Change in Absolute Reticulocyte Count (ARC) from initiation of pegcetacoplan treatment to 6 months 6 months Absolute Reticulocyte Count (ARC) in 10\^9/L
Change in indirect/ total bilirubin from initiation of pegcetacoplan treatment to 6 months 6 months Indirect/ total bilirubin in umol/L
Change in Haptoglobin from initiation of pegcetacoplan treatment to 6 months 6 months Haptoglobin in mg/dL
Change in Ferritin from initiation of pegcetacoplan treatment to 6 months 6 months Ferritin in ug/L
Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up 36 months Hemoglobin in g/dL
Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of follow-up 36 months Lactate Dehydrogenase (LDH) in U/L
Absolute Reticulocyte Count at initiation of pegcetacoplan treatment and each 6 months until end of follow-up 36 months Absolute Reticulocyte Count (ARC) in 10\^9/L
Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up 36 months Indirect/ total bilirubin in umol/L
Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up 36 months Haptoglobin in mg/dL
Fatigue at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up 36 months Patient reported outcome scale scores for the Functional assessment of chronic illness therapy (FACIT) - Fatigue. It is a 13-item scale and each item is scored on a 5-point Likert Scale ranging from "0-Not at all" to "4-Very much". The FACIT-fatigue score is obtained by summing all item scores. The score range from 0 to 52, higher score indicating less fatigue and lower score indicating more fatigue.
Health care resource use: Annualized number of hospitalizations and emergency room visits during pegcetacoplan treatment until end of follow-up compared to the 12-month period before pegcetacoplan treatment. 12 months Number of hospitalizations and emergency room visits
Patient treatment satisfaction every 6 months until end of follow-up 36 months Patient treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied
Physician treatment satisfaction every 6 months until end of follow-up 36 months Physician treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied
Adverse events (AE), including serious adverse events (SAE) 24 hours Adverse events (AE) and serious adverse events (SAE) are to be reported on the AE page of the eCRF as specified in the eCRF data entry guidelines.
Alanine transaminase (ALT) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up 36 months Alanine transaminase (ALT) in U/L
Aspartate aminotransferase (AST) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up 36 months Aspartate aminotransferase (AST) in U/L
Alkaline Phosphatase at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up 36 months Alkaline Phosphatase in µkat/L
Bilirubin at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up 36 months Indirect/ total bilirubin in umol/L
Creatinine at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up 36 months Creatinine in mg/dL
estimated glomerular filtration rate (eGFR) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up 36 months estimated glomerular filtration rate (eGFR) in mL/min/1.73 m²
Urea at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up 36 months Urea in mg/dL
Potassium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up 36 months Potassium in mmol/L
Sodium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up 36 months Sodium in mmol/L
Trial Locations
- 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
🇧🇪Brugge, Belgium
CHU Brugmann-Site Horta
🇧🇪Bruxelles, 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
🇨🇦Montréal, Quebec, Canada
Fakultni nemocnice Brno
🇨🇿Brno, Czechia
Ustav hematologie a krevni transfuze
🇨🇿Praha 2, Czechia
Helsingin yliopistollinen keskussairaala
🇫🇮Helsinki, Finland
CHU Nice - Hôpital de l'Archet 1
🇫🇷Nice, Alpes Maritimes, France
CHU de Bordeaux - Hôpital Haut-Lévêque
🇫🇷Pessac cedex, Gironde, France
Hopital Foch
🇫🇷Suresnes Cedex, Hauts De Seine, France
CHU de Grenoble - Hôpital Albert Michallon
🇫🇷Grenoble, Isere, France
CHU de Nantes - Hotel Dieu
🇫🇷Nantes cedex 1, Loire Atlantique, France
CHU de Nancy - Hôpital de Brabois Adultes
🇫🇷Vandœuvre-lès-Nancy, Meurthe Et Moselle, France
Hopital Claude Huriez - CHRU Lille
🇫🇷Lille cedex, Nord, France
Hôpital Saint-Louis
🇫🇷Paris La Defense, Paris, France
Centre Hospitalier Lyon Sud
🇫🇷Pierre-Bénite, Rhone, France
CHU Poitiers
🇫🇷Poitiers, France
Alb-Fils-Kliniken GmbH
🇩🇪Goettigen, Baden Wuerttemberg, Germany
Studienzentrum Aschaffenburg
🇩🇪Aschaffenburg, Bayern, Germany
Universitaetsklinikum Frankfurt Goethe-Universitaet
🇩🇪Frankfurt, Hessen, Germany
Universitaetsklinikum Giessen und Marburg GmbH Standort Giessen
🇩🇪Gießen, Hessen, Germany
Dres Ballo und Dr. Böck
🇩🇪Offenbach, Hessen, Germany
Universitaetsklinikum Essen
🇩🇪Essen, Nordrhein Westfalen, Germany
Universitaetsklinikum Leipzig AoeR
🇩🇪Leipzig, Sachsen, Germany
Praxis Fenchel Saalfeld
🇩🇪Saalfeld, Thueringen, Germany
MV Zentrum für Onkologie und Hämatologie
🇩🇪Koeln, Germany
Evangelismos Hospital
🇬🇷Athens, Greece
University General Hospital "Attikon"
🇬🇷Athens, Greece
University General Hospital of Larissa
🇬🇷Larissa, Greece
General Hospital of Thessaloniki "IPPOKRATEIO"
🇬🇷Thessaloniki, Greece
G Papanikolaou General Hospital
🇬🇷Thessaloníki, 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
🇮🇹Firenze, Italy
Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico
🇮🇹Milano, Italy
Ospedale San Raffaele
🇮🇹Milano, 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
🇵🇱Białystok, Poland
Szpital Uniwersytecki nr 2 im. dr J. Biziela w Bydgoszczy
🇵🇱Bydgoszcz, Poland
Uniwersyteckie Centrum Kliniczne
🇵🇱Gdańsk, Poland
SPZOZ Szpital Uniwersytecki w Krakowie
🇵🇱Kraków, Poland
Uniwersytecki Szpital Kliniczny nr 1 w Lublinie
🇵🇱Lublin, Poland
Warszawski Uniwersytet Medyczny
🇵🇱Warszawa, Poland
WWCOiT in Lodz, Oddział Hematologii i Transplantologii
🇵🇱Łódź, Poland
Prince Faisal Bin Bandar Cancer Center
🇸🇦Buraydah, 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 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
🇪🇸Malaga, Spain
Hospital Universitario Central de Asturias
🇪🇸Oviedo, Spain
Hospital Universitario Donostia
🇪🇸San Sebastian, Spain
Complejo Hospitalario Universitario de Santiago
🇪🇸Santiago De Compostela, Spain
King's College Hospital
🇬🇧London, United Kingdom