A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) (COMPLETE)

A Single Arm, Long-term, Multicentre Observational Study to Evaluate Effectiveness of Pegcetacoplan Under Real World Conditions in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

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.

Study Overview

• Status: Recruiting
• Conditions: Paroxysmal Nocturnal Hemoglobinuria
• Intervention / Treatment: Drug: Pegcetacoplan

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.

• Study Type: Observational
• Enrollment (Estimated): 200

Contacts and Locations

• Study Contact: Name: Clinical Program Lead; Phone Number: +46 (0)8 697 20 00; Email: medical.info@sobi.com

Study Locations

• Australia
  • Garran, Australia, 2605
    • Recruiting
    • Canberra Hospital
  • New South Wales
    • Liverpool, New South Wales, Australia
      • Recruiting
      • Liverpool Hospital
    • Waratah, New South Wales, Australia
      • Recruiting
      • Calvary Mater Newcastle
  • Queensland
    • Birtinya, Queensland, Australia
      • Recruiting
      • Sunshine Coast University Hospital
• Belgium
  • Anderlecht, Belgium
    • Recruiting
    • Institut Jules Bordet
  • Bruges, Belgium
    • Recruiting
    • AZ Sint-Jan
  • Brussels, Belgium, 1020
    • Recruiting
    • CHU Brugmann-Site Horta
  • Liège, Belgium
    • Recruiting
    • Centre Hospitalier Universitaire de Liege
  • Turnhout, Belgium
    • Recruiting
    • AZ Herentals
• Canada
  • Ontario
    • London, Ontario, Canada, N6A 5W9
      • Recruiting
      • London Health Sciences Centre (LHSC) - Victoria Hospital
    • Toronto, Ontario, Canada
      • Recruiting
      • University Health Network
  • Quebec
    • Montreal, Quebec, Canada
      • Active, not recruiting
      • Hopital Maisonneuve-Rosemont d/b/a CIUSSS de l'Est-de-l'Île-de-Montréal
• Croatia
  • Zagreb, Croatia
    • Recruiting
    • University hospital centre Zagreb
• Czechia
  • Brno, Czechia
    • Recruiting
    • Fakultni Nemocnice Brno
  • Prague, Czechia
    • Recruiting
    • Ustav Hematologie A Krevni Transfuze
• Finland
  • Helsinki, Finland
    • Recruiting
    • Helsingin yliopistollinen keskussairaala
• France
  • Poitiers, France
    • Not yet recruiting
    • CHU Poitiers
  • Rennes, France
    • Active, not recruiting
    • Chu Rennes - Hopital Pontchaillou
  • Saint-Quentin, France, 02321
    • Recruiting
    • Centre Hospitalier Général de St Quentin
  • Alpes Maritimes
    • Nice, Alpes Maritimes, France
      • Recruiting
      • CHU Nice - Hôpital de l'Archet 1
  • Gironde
    • Pessac, Gironde, France, 33604
      • Recruiting
      • CHU de Bordeaux - Hôpital Haut-Lévêque
  • Hauts de Seine
    • Suresnes, Hauts de Seine, France
      • Recruiting
      • Hopital Foch
  • Isere
    • Grenoble, Isere, France
      • Recruiting
      • CHU de Grenoble - Hôpital Albert Michallon
  • Loire Atlantique
    • Nantes, Loire Atlantique, France
      • Recruiting
      • CHU de Nantes - Hotel Dieu
  • Meurthe et Moselle
    • Vandœuvre-lès-Nancy, Meurthe et Moselle, France
      • Recruiting
      • CHU de Nancy - Hôpital de Brabois Adultes
  • Nord
    • Lille, Nord, France
      • Recruiting
      • Hopital Claude Huriez - CHRU Lille
  • Paris
    • La Defense, Paris, France
      • Recruiting
      • Hôpital Saint-Louis
  • Rhone
    • Pierre-Bénite, Rhone, France
      • Recruiting
      • Centre Hospitalier Lyon Sud
• Germany
  • Cologne, Germany
    • Not yet recruiting
    • MV Zentrum für Onkologie und Hämatologie
  • Troisdorf, Germany
    • Terminated
    • Haematologisch-Onkologische Schwerpunktpraxis
  • Baden-Wurttemberg
    • Goettigen, Baden-Wurttemberg, Germany
      • Terminated
      • Alb-Fils-Kliniken GmbH
  • Bavaria
    • Aschaffenburg, Bavaria, Germany
      • Terminated
      • Studienzentrum Aschaffenburg
  • Hesse
    • Frankfurt am Main, Hesse, Germany
      • Recruiting
      • Universitaetsklinikum Frankfurt Goethe-Universitaet
    • Giessen, Hesse, Germany
      • Active, not recruiting
      • Universitaetsklinikum Giessen und Marburg GmbH Standort Giessen
    • Offenbach, Hesse, Germany
      • Recruiting
      • Dres Ballo und Dr. Böck
  • North Rhine-Westphalia
    • Essen, North Rhine-Westphalia, Germany
      • Recruiting
      • Universitaetsklinikum Essen
  • Saxony
    • Leipzig, Saxony, Germany
      • Recruiting
      • Universitaetsklinikum Leipzig AoeR
  • Thuringia
    • Saalfeld, Thuringia, Germany
      • Recruiting
      • Praxis Fenchel Saalfeld
• Greece
  • Athens, Greece
    • Recruiting
    • University General Hospital "ATTIKON"
  • Athens, Greece
    • Not yet recruiting
    • Evangelismos Hospital
  • Larissa, Greece
    • Recruiting
    • University General Hospital of Larissa
  • Larissa, Greece, 41500
    • Recruiting
    • University General Hospital of Larissa
  • Thessaloniki, Greece
    • Recruiting
    • General Hospital of Thessaloniki "IPPOKRATEIO"
  • Thessaloniki, Greece
    • Recruiting
    • G Papanikolaou General Hospital
• Hungary
  • Budapest, Hungary
    • Recruiting
    • Semmelweis Egyetem
  • Budapest, Hungary
    • Recruiting
    • Semmelweis University
  • Budapest, Hungary
    • Recruiting
    • DPC - Orszagos Hematologiai es Infektologiai Intezet
• Italy
  • Bari, Italy
    • Recruiting
    • Azienda Ospedaliero Universitaria Consorziale Policlinico Di Bari
  • Bassano del Grappa, Italy
    • Recruiting
    • Ospedale San Bassiano
  • Bologna, Italy
    • Not yet recruiting
    • Irccs Aou Di Bologna- Pol. Di S.Orsola
  • Bologna, Italy
    • Active, not recruiting
    • Azienda Ospedaliera Universitaria Policlinico Sant'Orsola Malpighi IRCCS
  • Cagliari, Italy
    • Not yet recruiting
    • Ospedale Oncologico Armando Businco
  • Florence, Italy
    • Not yet recruiting
    • Azienda Ospedaliera Universitaria Careggi
  • Milan, Italy
    • Recruiting
    • Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
  • Milan, Italy
    • Recruiting
    • Ospedale San Raffaele
  • Novara, Italy
    • Recruiting
    • Ospedale Maggiore Della Carita
  • Reggio Calabria, Italy
    • Recruiting
    • Grande Ospedale Metropolitano - Presidio Morelli
  • Roma, Italy
    • Recruiting
    • Fondazione Policlinico Universitario Agostino Gemelli IRCCS
  • Rome, Italy
    • Recruiting
    • Università di Roma La Sapienza
  • Torino, Italy
    • Recruiting
    • Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino - Onoclogy and Hematology Department
  • Torino, Italy
    • Not yet recruiting
    • Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino - UO Ematologia
• Poland
  • Bialystok, Poland
    • Recruiting
    • Uniwersytecki Szpital Kliniczny w Bialymstoku
  • Bydgoszcz, Poland
    • Recruiting
    • Szpital Uniwersytecki nr 2 im. dr J. Biziela w Bydgoszczy
  • Gdansk, Poland
    • Recruiting
    • Uniwersyteckie Centrum Kliniczne
  • Krakow, Poland
    • Recruiting
    • SPZOZ Szpital Uniwersytecki w Krakowie
  • Lodz, Poland
    • Recruiting
    • WWCOiT in Lodz, Oddział Hematologii i Transplantologii
  • Lublin, Poland
    • Not yet recruiting
    • Uniwersytecki Szpital Kliniczny nr 1 w Lublinie
  • Warsaw, Poland
    • Not yet recruiting
    • Warszawski Uniwersytet Medyczny
• Saudi Arabia
  • Buraidah, Saudi Arabia
    • Recruiting
    • Prince Faisal Bin Bandar Cancer Center
  • Jeddah, Saudi Arabia
    • Not yet recruiting
    • King Faisal Specialist Hospital & Research Center
  • Riyadh, Saudi Arabia
    • Recruiting
    • King Faisal Specialist Hospital and Research Center - Madinah
  • Riyadh, Saudi Arabia
    • Recruiting
    • King Faisal Specialist Hospital and Research Center - Riyadh
• Spain
  • Badalona, Spain
    • Recruiting
    • ICO Badalona - Hospital Universitari Germans Trias i Pujol
  • Barakaldo, Spain
    • Recruiting
    • Hospital de Cruces
  • Barcelona, Spain
    • Recruiting
    • Hospital Universitari Vall d'Hebron
  • Barcelona, Spain
    • Recruiting
    • Hospital Clinic de Barcelona
  • Bilbao, Spain
    • Active, not recruiting
    • Hospital de Basurto
  • Donostia / San Sebastian, Spain
    • Recruiting
    • Hospital Universitario Donostia
  • El Palmar, Spain
    • Recruiting
    • Hospital Universitario Virgen de la Nieves
  • Huelva, Spain
    • Active, not recruiting
    • Hospital Universitario Juan Ramon Jimenez
  • Las Palmas de Gran Canaria, Spain
    • Recruiting
    • Hospital Universitario de Gran Canaria Dr. Negrin
  • Lugo, Spain
    • Recruiting
    • Hospital Lucus Augusti
    • Principal Investigator: Esperanza Lavilla, MD
  • Málaga, Spain
    • Recruiting
    • Hospital Regional Universitario de Malaga
  • Oviedo, Spain
    • Recruiting
    • Hospital Universitario Central de Asturias
  • Palma de Mallorca, Spain
    • Recruiting
    • Hospital Universitari Son Espases
  • Santiago de Compostela, Spain
    • Recruiting
    • Complejo Hospitalario Universitario de Santiago
• United Kingdom
  • London, United Kingdom
    • Completed
    • King's College Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Adult patients (≥18 years) with PNH, who started routine treatment with pegcetacoplan up to 12 months prior to enrollmentor at enrollment

Description

Inclusion Criteria:

• 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.

Exclusion Criteria:

• 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 Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in observed hemoglobin level from initiation of treatment with pegcetacoplan to 6 months	Hemoglobin level in g/dL.	6 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in Absolute Reticulocyte Count (ARC) from initiation of pegcetacoplan treatment to 6 months	Absolute Reticulocyte Count (ARC) in 10^9/L	6 months
Change in indirect/ total bilirubin from initiation of pegcetacoplan treatment to 6 months	Indirect/ total bilirubin in umol/L	6 months
Change in Haptoglobin from initiation of pegcetacoplan treatment to 6 months	Haptoglobin in mg/dL	6 months
Change in Ferritin from initiation of pegcetacoplan treatment to 6 months	Ferritin in ug/L	6 months
Change of LDH values from initiation of pegcetacoplan treatment to 6 months	Lactate Dehydrogenase (LDH) in U/L	6 months
Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Hemoglobin in g/dL	6 months
Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Hemoglobin in g/dL	12 months
Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Hemoglobin in g/dL	18 months
Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Hemoglobin in g/dL	24 months
Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Hemoglobin in g/dL	30 months
Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Hemoglobin in g/dL	36 months
Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Lactate Dehydrogenase (LDH) in U/L	6 months
Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Lactate Dehydrogenase (LDH) in U/L	12 months
Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Lactate Dehydrogenase (LDH) in U/L	18 months
Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Lactate Dehydrogenase (LDH) in U/L	24 months
Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Lactate Dehydrogenase (LDH) in U/L	30 months
Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Lactate Dehydrogenase (LDH) in U/L	36 months
Absolute Reticulocyte Count at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Absolute Reticulocyte Count (ARC) in 10^9/L	6 months
Absolute Reticulocyte Count at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Absolute Reticulocyte Count (ARC) in 10^9/L	12 months
Absolute Reticulocyte Count at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Absolute Reticulocyte Count (ARC) in 10^9/L	18 months
Absolute Reticulocyte Count at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Absolute Reticulocyte Count (ARC) in 10^9/L	24 months
Absolute Reticulocyte Count at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Absolute Reticulocyte Count (ARC) in 10^9/L	30 months
Absolute Reticulocyte Count at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Absolute Reticulocyte Count (ARC) in 10^9/L	36 months
Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Indirect/ total bilirubin in umol/L	6 months
Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Indirect/ total bilirubin in umol/L	12 months
Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Indirect/ total bilirubin in umol/L	18 months
Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Indirect/ total bilirubin in umol/L	24 months
Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Indirect/ total bilirubin in umol/L	30 months
Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Indirect/ total bilirubin in umol/L	36 months
Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Haptoglobin in µmol/L	6 months
Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Haptoglobin in µmol/L	12 months
Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Haptoglobin in µmol/L	18 months
Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Haptoglobin in µmol/L	24 months
Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Haptoglobin in µmol/L	30 months
Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Haptoglobin in mg/dL	36 months
Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Ferritin in ug/L	6 months
Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Ferritin in ug/L	12 months
Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Ferritin in ug/L	18 months
Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Ferritin in ug/L	24 months
Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Ferritin in ug/L	30 months
Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Ferritin in ug/L	36 months
Hemoglobin ≥ 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Hemoglobin in g/dL	6 months
Hemoglobin ≥ 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Hemoglobin in g/dL	12 months
Hemoglobin ≥ 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Hemoglobin in g/dL	18 months
Hemoglobin ≥ 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Hemoglobin in g/dL	24 months
Hemoglobin ≥ 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Hemoglobin in g/dL	30 months
Hemoglobin ≥ 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Hemoglobin in g/dL	36 months
Increase in hemoglobin levels of ≥ 2 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Hemoglobin in g/dL	6 months
Increase in hemoglobin levels of ≥ 2 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Hemoglobin in g/dL	12 months
Increase in hemoglobin levels of ≥ 2 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Hemoglobin in g/dL	18 months
Increase in hemoglobin levels of ≥ 2 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Hemoglobin in g/dL	24 months
Increase in hemoglobin levels of ≥ 2 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Hemoglobin in g/dL	30 months
Increase in hemoglobin levels of ≥ 2 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Hemoglobin in g/dL	36 months
Hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Yes/No	6 months
Hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Yes/No	12 months
Hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Yes/No	18 months
Hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Yes/No	24 months
Hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Yes/No	30 months
Hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of follow-up	Yes/No	36 months
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	Total number of RBC transfusions	12 months
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	Total number of RBC units	12 months
Fatigue at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	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.	6 months
Fatigue at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	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.	12 months
Fatigue at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	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.	18 months
Fatigue at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	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.	24 months
Fatigue at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	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.	30 months
Fatigue at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	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.	36 months
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.	Number of hospitalizations and emergency room visits	12 months
Patient treatment satisfaction every 6 months until end of follow-up	Patient treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied	6 months
Patient treatment satisfaction every 6 months until end of follow-up	Patient treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied	12 months
Patient treatment satisfaction every 6 months until end of follow-up	Patient treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied	18 months
Patient treatment satisfaction every 6 months until end of follow-up	Patient treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied	24 months
Patient treatment satisfaction every 6 months until end of follow-up	Patient treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied	30 months
Patient treatment satisfaction every 6 months until end of follow-up	Patient treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied	36 months
Physician treatment satisfaction every 6 months until end of follow-up	Physician treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied	6 months
Physician treatment satisfaction every 6 months until end of follow-up	Physician treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied	12 months
Physician treatment satisfaction every 6 months until end of follow-up	Physician treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied	18 months
Physician treatment satisfaction every 6 months until end of follow-up	Physician treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied	24 months
Physician treatment satisfaction every 6 months until end of follow-up	Physician treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied	30 months
Physician treatment satisfaction every 6 months until end of follow-up	Physician treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied	36 months
Adverse events (AE), including serious adverse events (SAE)	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.	24 hours
Alanine transaminase (ALT) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Alanine transaminase (ALT) in U/L	6 months
Alanine transaminase (ALT) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Alanine transaminase (ALT) in U/L	12 months
Alanine transaminase (ALT) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Alanine transaminase (ALT) in U/L	18 months
Alanine transaminase (ALT) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Alanine transaminase (ALT) in U/L	24 months
Alanine transaminase (ALT) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Alanine transaminase (ALT) in U/L	30 months
Alanine transaminase (ALT) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Alanine transaminase (ALT) in U/L	36 months
Aspartate aminotransferase (AST) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Aspartate aminotransferase (AST) in U/L	6 months
Aspartate aminotransferase (AST) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Aspartate aminotransferase (AST) in U/L	12 months
Aspartate aminotransferase (AST) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Aspartate aminotransferase (AST) in U/L	18 months
Aspartate aminotransferase (AST) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Aspartate aminotransferase (AST) in U/L	24 months
Aspartate aminotransferase (AST) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Aspartate aminotransferase (AST) in U/L	30 months
Aspartate aminotransferase (AST) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Aspartate aminotransferase (AST) in U/L	36 months
Alkaline Phosphatase at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Alkaline Phosphatase in µkat/L	6 months
Alkaline Phosphatase at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Alkaline Phosphatase in µkat/L	12 months
Alkaline Phosphatase at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Alkaline Phosphatase in µkat/L	18 months
Alkaline Phosphatase at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Alkaline Phosphatase in µkat/L	24 months
Alkaline Phosphatase at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Alkaline Phosphatase in µkat/L	30 months
Alkaline Phosphatase at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Alkaline Phosphatase in µkat/L	36 months
Bilirubin at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Indirect/ total bilirubin in umol/L	6 months
Bilirubin at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Indirect/ total bilirubin in umol/L	12 months
Bilirubin at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Indirect/ total bilirubin in umol/L	18 months
Bilirubin at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Indirect/ total bilirubin in umol/L	24 months
Bilirubin at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Indirect/ total bilirubin in umol/L	30 months
Bilirubin at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Indirect/ total bilirubin in umol/L	36 months
Creatinine at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Creatinine in mg/dL	6 months
Creatinine at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Creatinine in mg/dL	12 months
Creatinine at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Creatinine in mg/dL	18 months
Creatinine at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Creatinine in mg/dL	24 months
Creatinine at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Creatinine in mg/dL	30 months
Creatinine at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Creatinine in mg/dL	36 months
estimated glomerular filtration rate (eGFR) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	estimated glomerular filtration rate (eGFR) in mL/min/1.73 m²	6 months
estimated glomerular filtration rate (eGFR) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	estimated glomerular filtration rate (eGFR) in mL/min/1.73 m²	12 months
estimated glomerular filtration rate (eGFR) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	estimated glomerular filtration rate (eGFR) in mL/min/1.73 m²	18 months
estimated glomerular filtration rate (eGFR) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	estimated glomerular filtration rate (eGFR) in mL/min/1.73 m²	24 months
estimated glomerular filtration rate (eGFR) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	estimated glomerular filtration rate (eGFR) in mL/min/1.73 m²	30 months
estimated glomerular filtration rate (eGFR) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	estimated glomerular filtration rate (eGFR) in mL/min/1.73 m²	36 months
Urea at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Urea in mg/dL	6 months
Urea at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Urea in mg/dL	12 months
Urea at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Urea in mg/dL	18 months
Urea at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Urea in mg/dL	24 months
Urea at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Urea in mg/dL	30 months
Urea at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Urea in mg/dL	36 months
Potassium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Potassium in mmol/L	6 months
Potassium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Potassium in mmol/L	12 months
Potassium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Potassium in mmol/L	18 months
Potassium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Potassium in mmol/L	24 months
Potassium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Potassium in mmol/L	30 months
Potassium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Potassium in mmol/L	36 months
Sodium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Sodium in mmol/L	6 months
Sodium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Sodium in mmol/L	12 months
Sodium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Sodium in mmol/L	18 months
Sodium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Sodium in mmol/L	24 months
Sodium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Sodium in mmol/L	30 months
Sodium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up	Sodium in mmol/L	36 months

Collaborators and Investigators

• Sponsor: Swedish Orphan Biovitrum
• Collaborators: IQVIA Pty Ltd
• Investigators: Study Director: Clinical Program Lead +46 08-697-20 00, Swedish Orphan Biovitrum

Study record dates

Study Major Dates

• Study Start (Actual): June 26, 2023
• Primary Completion (Estimated): August 15, 2029
• Study Completion (Estimated): August 21, 2029

Study Registration Dates

• First Submitted: February 20, 2023
• First Submitted That Met QC Criteria: March 17, 2023
• First Posted (Actual): March 20, 2023

Study Record Updates

• Last Update Posted (Actual): July 1, 2026
• Last Update Submitted That Met QC Criteria: June 30, 2026
• Last Verified: June 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Bone Marrow Diseases; Anemia, Hemolytic; Anemia; Myelodysplastic Syndromes; Hemic and Lymphatic Diseases; Hemoglobinuria, Paroxysmal; pegcetacoplan
• Other Study ID Numbers: Sobi.PEGCET-304

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No