Actively Recruiting
A Single Arm, Long-term, Multicentre Observational Study to Evaluate Effectiveness of Pegcetacoplan Under Real World Conditions in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Led by Swedish Orphan Biovitrum · Updated on 2025-05-14
200
Participants Needed
75
Research Sites
156 weeks
Total Duration
On this page
Sponsors
S
Swedish Orphan Biovitrum
Lead Sponsor
I
IQVIA Pty Ltd
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are observing the real-world use and effects of pegcetacoplan in adults with Paroxysmal Nocturnal Hemoglobinuria (PNH). This long-term study aims to provide important information on how pegcetacoplan is used in routine medical practice and to understand its impact on blood transfusions and healthcare resource use. Approximately 200 patients will participate at various sites across Europe, the Middle East, Canada, and Australia. Pegcetacoplan treatment data will be collected both retrospectively and prospectively, covering up to 72 months depending on when treatment began. After patients stop pegcetacoplan, they will be followed for an additional 8 weeks to monitor any adverse events. Patient data includes effectiveness, safety, patient- and clinician-reported outcomes, and healthcare resource use before and after treatment. Participants will attend their regular medical visits where data will be collected. Researchers will monitor changes in hemoglobin levels and other blood markers every 6 months. They will also track adverse events and patient satisfaction. The total participation may last up to 48 months, including both retrospective and prospective data collection periods.
CONDITIONS
Brief Title
A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients 18 years of age or older with a documented diagnosis of Paroxysmal Nocturnal Hemoglobinuria (PNH)
- Patient started routine treatment with pegcetacoplan for PNH up to 12 months before enrollment or is prescribed pegcetacoplan at enrollment
- Patient is willing and able to provide written informed consent to participate as approved by ethics committees and local regulations
You will not qualify if you...
- Enrollment in a concurrent clinical interventional study or use of an investigational drug within three months prior to starting current pegcetacoplan treatment
- Started current pegcetacoplan treatment as part of an interventional study
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 72 months including retrospective and prospective data
Participants who undergo routine treatment with pegcetacoplan for PNH are observed. Data on treatment effectiveness, safety, patient- and clinician-reported outcomes, and healthcare resource use are collected from retrospective and prospective routine visits.
Routine visits as per usual care, with data collected approximately every 6 months
Duration - 8 weeks
After pegcetacoplan treatment discontinuation, participants remain in the study for safety monitoring to capture any adverse events.
Visits as needed during 8-week safety monitoring period
Trial Site Locations
Total: 75 locations
1
Liverpool Hospital
Liverpool, New South Wales, Australia
Actively Recruiting
2
Calvary Mater Newcastle
Waratah, New South Wales, Australia
Actively Recruiting
3
Sunshine Coast University Hospital
Birtinya, Queensland, Australia
Actively Recruiting
4
AZ Sint-Jan
Bruges, Belgium
Active, Not Recruiting
5
CHU Brugmann-Site Horta
Brussels, Belgium, 1020
Active, Not Recruiting
6
Centre Hospitalier Universitaire de Liege
Liège, Belgium
Actively Recruiting
7
AZ Turnhout - Campus Sint-Elisabeth
Turnhout, Belgium
Active, Not Recruiting
8
University Health Network
Toronto, Ontario, Canada
Actively Recruiting
9
Hopital Maisonneuve-Rosemont d/b/a CIUSSS de l'Est-de-l'Île-de-Montréal
Montreal, Quebec, Canada
Active, Not Recruiting
10
Fakultni nemocnice Brno
Brno, Czechia
Actively Recruiting
11
Ustav hematologie a krevni transfuze
Prague, Czechia
Actively Recruiting
12
Helsingin yliopistollinen keskussairaala
Helsinki, Finland
Actively Recruiting
13
CHU Nice - Hôpital de l'Archet 1
Nice, Alpes Maritimes, France
Actively Recruiting
14
CHU de Bordeaux - Hôpital Haut-Lévêque
Pessac, Gironde, France, 33604
Actively Recruiting
15
Hopital Foch
Suresnes, Hauts De Seine, France
Actively Recruiting
16
CHU de Grenoble - Hôpital Albert Michallon
Grenoble, Isere, France
Actively Recruiting
17
CHU de Nantes - Hotel Dieu
Nantes, Loire Atlantique, France
Actively Recruiting
18
CHU de Nancy - Hôpital de Brabois Adultes
Vandœuvre-lès-Nancy, Meurthe Et Moselle, France
Actively Recruiting
19
Hopital Claude Huriez - CHRU Lille
Lille, Nord, France
Actively Recruiting
20
Hôpital Saint-Louis
La Defense, Paris, France
Actively Recruiting
21
Centre Hospitalier Lyon Sud
Pierre-Bénite, Rhone, France
Actively Recruiting
22
CHU Poitiers
Poitiers, France
Not Yet Recruiting
23
Alb-Fils-Kliniken GmbH
Goettigen, Baden-Wurttemberg, Germany
Active, Not Recruiting
24
Studienzentrum Aschaffenburg
Aschaffenburg, Bavaria, Germany
Active, Not Recruiting
25
Universitaetsklinikum Frankfurt Goethe-Universitaet
Frankfurt am Main, Hesse, Germany
Actively Recruiting
26
Universitaetsklinikum Giessen und Marburg GmbH Standort Giessen
Giessen, Hesse, Germany
Active, Not Recruiting
27
Dres Ballo und Dr. Böck
Offenbach, Hesse, Germany
Actively Recruiting
28
Universitaetsklinikum Essen
Essen, North Rhine-Westphalia, Germany
Actively Recruiting
29
Universitaetsklinikum Leipzig AoeR
Leipzig, Saxony, Germany
Active, Not Recruiting
30
Praxis Fenchel Saalfeld
Saalfeld, Thuringia, Germany
Actively Recruiting
31
MV Zentrum für Onkologie und Hämatologie
Cologne, Germany
Not Yet Recruiting
32
Evangelismos Hospital
Athens, Greece
Not Yet Recruiting
33
University General Hospital "Attikon"
Athens, Greece
Actively Recruiting
34
University General Hospital of Larissa
Larissa, Greece
Actively Recruiting
35
G Papanikolaou General Hospital
Thessaloniki, Greece
Not Yet Recruiting
36
General Hospital of Thessaloniki "IPPOKRATEIO"
Thessaloniki, Greece
Actively Recruiting
37
DPC - Orszagos Hematologiai es Infektologiai Intezet
Budapest, Hungary
Actively Recruiting
38
Semmelweis Egyetem
Budapest, Hungary
Actively Recruiting
39
Semmelweis University
Budapest, Hungary
Actively Recruiting
40
Azienda Ospedaliero Universitaria Consorziale Policlinico di Bari
Bari, Italy
Actively Recruiting
41
Ospedale San Bassiano
Bassano del Grappa, Italy
Actively Recruiting
42
Irccs Aou Di Bologna- Pol. Di S.Orsola
Bologna, Italy
Not Yet Recruiting
43
Ospedale Oncologico Armando Businco
Cagliari, Italy
Not Yet Recruiting
44
Azienda Ospedaliera Universitaria Careggi
Florence, Italy
Not Yet Recruiting
45
Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico
Milan, Italy
Actively Recruiting
46
Ospedale San Raffaele
Milan, Italy
Actively Recruiting
47
Ospedale Maggiore della Carità
Novara, Italy
Actively Recruiting
48
Grande Ospedale Metropolitano - Presidio Morelli
Reggio Calabria, Italy
Actively Recruiting
49
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Roma, Italy
Actively Recruiting
50
Università di Roma La Sapienza
Rome, Italy
Actively Recruiting
51
Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino
Torino, Italy
Not Yet Recruiting
52
Uniwersytecki Szpital Kliniczny w Bialymstoku
Bialystok, Poland
Not Yet Recruiting
53
Szpital Uniwersytecki nr 2 im. dr J. Biziela w Bydgoszczy
Bydgoszcz, Poland
Not Yet Recruiting
54
Uniwersyteckie Centrum Kliniczne
Gdansk, Poland
Not Yet Recruiting
55
SPZOZ Szpital Uniwersytecki w Krakowie
Krakow, Poland
Not Yet Recruiting
56
WWCOiT in Lodz, Oddział Hematologii i Transplantologii
Lodz, Poland
Actively Recruiting
57
Uniwersytecki Szpital Kliniczny nr 1 w Lublinie
Lublin, Poland
Not Yet Recruiting
58
Warszawski Uniwersytet Medyczny
Warsaw, Poland
Not Yet Recruiting
59
Prince Faisal Bin Bandar Cancer Center
Buraidah, Saudi Arabia
Not Yet Recruiting
60
King Faisal Specialist Hospital & Research Center
Jeddah, Saudi Arabia
Not Yet Recruiting
61
King Faisal Specialist Hospital and Research Center - Madinah
Riyadh, Saudi Arabia
Actively Recruiting
62
King Faisal Specialist Hospital and Research Center - Riyadh
Riyadh, Saudi Arabia
Actively Recruiting
63
ICO Badalona - Hospital Universitari Germans Trias i Pujol
Badalona, Spain
Actively Recruiting
64
Hospital de Cruces
Barakaldo, Spain
Actively Recruiting
65
Hospital Clinic de Barcelona
Barcelona, Spain
Actively Recruiting
66
Hospital Universitari Vall d'Hebron
Barcelona, Spain
Actively Recruiting
67
Hospital de Basurto
Bilbao, Spain
Active, Not Recruiting
68
Hospital Universitario Donostia
Donostia / San Sebastian, Spain
Actively Recruiting
69
Hospital Universitario Virgen de la Nieves
El Palmar, Spain
Actively Recruiting
70
Hospital Universitario de Gran Canaria Dr. Negrin
Las Palmas de Gran Canaria, Spain
Actively Recruiting
71
Hospital Lucus Augusti
Lugo, Spain
Actively Recruiting
72
Hospital Regional Universitario de Malaga
Málaga, Spain
Actively Recruiting
73
Hospital Universitario Central de Asturias
Oviedo, Spain
Actively Recruiting
74
Complejo Hospitalario Universitario de Santiago
Santiago de Compostela, Spain
Actively Recruiting
75
King's College Hospital
London, United Kingdom
Actively Recruiting
Research Team
M
Michael O'Malley
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
0
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