AzTrials
AzTrials

Paroxysmal Nocturnal HemoglobinuriaCOMPLETENCT05776472

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

An observational study of Paroxysmal Nocturnal Hemoglobinuria, run by Swedish Orphan Biovitrum at 87 sites in 15 countries.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Paroxysmal Nocturnal Hemoglobinuria
What is being tested?
Pegcetacoplan
How many people
165 participants
Where
87 sitesin 15 countries
Study dates
26 Jun 2023to 15 Aug 2029
Phase
Observational
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

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

Who may be able to join

  • 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.

Who cannot join

  • 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.

Trial locations and contacts

87 sites in 15 countries.

87 sites in 15 countries

Italy14 sites

  • Azienda Ospedaliero Universitaria Consorziale Policlinico di Bari

    Bari

  • Ospedale San Bassiano

    Bassano del Grappa

  • Azienda Ospedaliera Universitaria Policlinico Sant'Orsola Malpighi IRCCS

    Bologna

  • Irccs Aou Di Bologna- Pol. Di S.Orsola

    Bologna

  • Ospedale Oncologico Armando Businco

    Cagliari

  • Azienda Ospedaliera Universitaria Careggi

    Florence

  • Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico

    Milan

  • Ospedale San Raffaele

    Milan

  • Ospedale Maggiore della Carità

    Novara

  • Grande Ospedale Metropolitano - Presidio Morelli

    Reggio Calabria

  • Fondazione Policlinico Universitario Agostino Gemelli IRCCS

    Roma

  • Università di Roma La Sapienza

    Rome

  • Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino - Onoclogy and Hematology Department

    Torino

  • Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino - UO Ematologia

    Torino

Other trials in Italy

Spain14 sites

  • ICO Badalona - Hospital Universitari Germans Trias i Pujol

    Badalona

  • Hospital de Cruces

    Barakaldo

  • Hospital Clinic de Barcelona

    Barcelona

  • Hospital Universitari Vall d'Hebron

    Barcelona

  • Hospital de Basurto

    Bilbao

  • Hospital Universitario Donostia

    Donostia / San Sebastian

  • Hospital Universitario Virgen de la Nieves

    El Palmar

  • Hospital Universitario Juan Ramon Jimenez

    Huelva

  • Hospital Universitario de Gran Canaria Dr. Negrin

    Las Palmas de Gran Canaria

  • Hospital Lucus Augusti

    Lugo

  • Hospital Regional Universitario de Malaga

    Málaga

  • Hospital Universitario Central de Asturias

    Oviedo

  • Hospital Universitari Son Espases

    Palma de Mallorca

  • Complejo Hospitalario Universitario de Santiago

    Santiago de Compostela

Other trials in Spain

France12 sites

  • CHU de Grenoble - Hôpital Albert Michallon

    Grenoble, Isere

  • Hôpital Saint-Louis

    La Defense, Paris

  • Hopital Claude Huriez - CHRU Lille

    Lille, Nord

  • CHU de Nantes - Hotel Dieu

    Nantes, Loire Atlantique

  • CHU Nice - Hôpital de l'Archet 1

    Nice, Alpes Maritimes

  • CHU de Bordeaux - Hôpital Haut-Lévêque

    Pessac, Gironde, 33604

  • Centre Hospitalier Lyon Sud

    Pierre-Bénite, Rhone

  • CHU Poitiers

    Poitiers

  • CHU Rennes - Hopital Pontchaillou

    Rennes

  • Centre Hospitalier Général de St Quentin

    Saint-Quentin, 02321

  • Hopital Foch

    Suresnes, Hauts de Seine

  • CHU de Nancy - Hôpital de Brabois Adultes

    Vandœuvre-lès-Nancy, Meurthe et Moselle

Other trials in France

Germany10 sites

  • Studienzentrum Aschaffenburg

    Aschaffenburg, Bavaria

  • MV Zentrum für Onkologie und Hämatologie

    Cologne

  • Universitaetsklinikum Essen

    Essen, North Rhine-Westphalia

  • Universitaetsklinikum Frankfurt Goethe-Universitaet

    Frankfurt am Main, Hesse

  • Universitaetsklinikum Giessen und Marburg GmbH Standort Giessen

    Giessen, Hesse

  • Alb-Fils-Kliniken GmbH

    Goettigen, Baden-Wurttemberg

  • Universitaetsklinikum Leipzig AoeR

    Leipzig, Saxony

  • Dres Ballo und Dr. Böck

    Offenbach, Hesse

  • Praxis Fenchel Saalfeld

    Saalfeld, Thuringia

  • Haematologisch-Onkologische Schwerpunktpraxis

    Troisdorf

Other trials in Germany

Poland7 sites

  • Uniwersytecki Szpital Kliniczny w Bialymstoku

    Bialystok

  • Szpital Uniwersytecki nr 2 im. dr J. Biziela w Bydgoszczy

    Bydgoszcz

  • Uniwersyteckie Centrum Kliniczne

    Gdansk

  • SPZOZ Szpital Uniwersytecki w Krakowie

    Krakow

  • WWCOiT in Lodz, Oddział Hematologii i Transplantologii

    Lodz

  • Uniwersytecki Szpital Kliniczny nr 1 w Lublinie

    Lublin

  • Warszawski Uniwersytet Medyczny

    Warsaw

Other trials in Poland

Greece6 sites

  • Evangelismos Hospital

    Athens

  • University General Hospital "Attikon"

    Athens

  • University General Hospital of Larissa

    Larissa, 41500

  • University General Hospital of Larissa

    Larissa

  • G Papanikolaou General Hospital

    Thessaloniki

  • General Hospital of Thessaloniki "IPPOKRATEIO"

    Thessaloniki

Other trials in Greece

Belgium5 sites

  • Institut Jules Bordet

    Anderlecht

  • AZ Sint-Jan

    Bruges

  • CHU Brugmann-Site Horta

    Brussels, 1020

  • Centre Hospitalier Universitaire de Liege

    Liège

  • AZ Herentals

    Turnhout

Other trials in Belgium

Australia4 sites

  • Sunshine Coast University Hospital

    Birtinya, Queensland

  • Canberra Hospital

    Garran, 2605

  • Liverpool Hospital

    Liverpool, New South Wales

  • Calvary Mater Newcastle

    Waratah, New South Wales

Other trials in Australia

Saudi Arabia4 sites

  • Prince Faisal Bin Bandar Cancer Center

    Buraidah

  • King Faisal Specialist Hospital & Research Center

    Jeddah

  • King Faisal Specialist Hospital and Research Center - Madinah

    Riyadh

  • King Faisal Specialist Hospital and Research Center - Riyadh

    Riyadh

Other trials in Saudi Arabia

Canada3 sites

  • London Health Sciences Centre (LHSC) - Victoria Hospital

    London, Ontario, N6A 5W9

  • Hopital Maisonneuve-Rosemont d/b/a CIUSSS de l'Est-de-l'Île-de-Montréal

    Montreal, Quebec

  • University Health Network

    Toronto, Ontario

Other trials in Canada

Hungary3 sites

  • DPC - Orszagos Hematologiai es Infektologiai Intezet

    Budapest

  • Semmelweis Egyetem

    Budapest

  • Semmelweis University

    Budapest

Other trials in Hungary

Czechia2 sites

  • Fakultni nemocnice Brno

    Brno

  • Ustav hematologie a krevni transfuze

    Prague

Other trials in Czechia

Croatia1 site

  • University hospital centre Zagreb

    Zagreb

Other trials in Croatia

Finland1 site

  • Helsingin yliopistollinen keskussairaala

    Helsinki

Other trials in Finland

United Kingdom1 site

  • King's College Hospital

    London

Other trials in United Kingdom

About this study

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)

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.

Read the 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.

Being tested
Pegcetacoplan
Medical subject headings
Hemoglobinuria, Paroxysmal

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • PegcetacoplanDrug

    Also called Aspaveli, Empaveli

    Pegcetacoplan will be prescribed according to the label in patients with PNH.

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Change in observed hemoglobin level from initiation of treatment with pegcetacoplan to 6 months

    Hemoglobin level in g/dL.

    Measured: 6 months

Secondary outcomes (135)

  1. Change of LDH values from initiation of pegcetacoplan treatment to 6 months

    Lactate Dehydrogenase (LDH) in U/L

    Measured: 6 months

  2. Change in Absolute Reticulocyte Count (ARC) from initiation of pegcetacoplan treatment to 6 months

    Absolute Reticulocyte Count (ARC) in 10^9/L

    Measured: 6 months

  3. Change in indirect/ total bilirubin from initiation of pegcetacoplan treatment to 6 months

    Indirect/ total bilirubin in umol/L

    Measured: 6 months

  4. Change in Haptoglobin from initiation of pegcetacoplan treatment to 6 months

    Haptoglobin in mg/dL

    Measured: 6 months

  5. Change in Ferritin from initiation of pegcetacoplan treatment to 6 months

    Ferritin in ug/L

    Measured: 6 months

  6. Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Hemoglobin in g/dL

    Measured: 6 months

  7. Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Hemoglobin in g/dL

    Measured: 12 months

  8. Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Hemoglobin in g/dL

    Measured: 18 months

  9. Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Hemoglobin in g/dL

    Measured: 24 months

  10. Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Hemoglobin in g/dL

    Measured: 30 months

  11. Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Hemoglobin in g/dL

    Measured: 36 months

  12. Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Lactate Dehydrogenase (LDH) in U/L

    Measured: 6 months

  13. Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Lactate Dehydrogenase (LDH) in U/L

    Measured: 12 months

  14. Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Lactate Dehydrogenase (LDH) in U/L

    Measured: 18 months

  15. Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Lactate Dehydrogenase (LDH) in U/L

    Measured: 24 months

  16. Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Lactate Dehydrogenase (LDH) in U/L

    Measured: 30 months

  17. Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Lactate Dehydrogenase (LDH) in U/L

    Measured: 36 months

  18. 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

    Measured: 6 months

  19. 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

    Measured: 12 months

  20. 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

    Measured: 18 months

  21. 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

    Measured: 24 months

  22. 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

    Measured: 30 months

  23. 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

    Measured: 36 months

  24. Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Indirect/ total bilirubin in umol/L

    Measured: 6 months

  25. Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Indirect/ total bilirubin in umol/L

    Measured: 12 months

  26. Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Indirect/ total bilirubin in umol/L

    Measured: 18 months

  27. Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Indirect/ total bilirubin in umol/L

    Measured: 24 months

  28. Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Indirect/ total bilirubin in umol/L

    Measured: 30 months

  29. Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Indirect/ total bilirubin in umol/L

    Measured: 36 months

  30. Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Haptoglobin in µmol/L

    Measured: 6 months

  31. Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Haptoglobin in µmol/L

    Measured: 12 months

  32. Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Haptoglobin in µmol/L

    Measured: 18 months

  33. Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Haptoglobin in µmol/L

    Measured: 24 months

  34. Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Haptoglobin in µmol/L

    Measured: 30 months

  35. Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Haptoglobin in mg/dL

    Measured: 36 months

  36. Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Ferritin in ug/L

    Measured: 6 months

  37. Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Ferritin in ug/L

    Measured: 12 months

  38. Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Ferritin in ug/L

    Measured: 18 months

  39. Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Ferritin in ug/L

    Measured: 24 months

  40. Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Ferritin in ug/L

    Measured: 30 months

  41. Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Ferritin in ug/L

    Measured: 36 months

  42. Hemoglobin ≥ 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Hemoglobin in g/dL

    Measured: 6 months

  43. Hemoglobin ≥ 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Hemoglobin in g/dL

    Measured: 12 months

  44. Hemoglobin ≥ 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Hemoglobin in g/dL

    Measured: 18 months

  45. Hemoglobin ≥ 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Hemoglobin in g/dL

    Measured: 24 months

  46. Hemoglobin ≥ 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Hemoglobin in g/dL

    Measured: 30 months

  47. Hemoglobin ≥ 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Hemoglobin in g/dL

    Measured: 36 months

  48. 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

    Measured: 6 months

  49. 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

    Measured: 12 months

  50. 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

    Measured: 18 months

  51. 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

    Measured: 24 months

  52. 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

    Measured: 30 months

  53. 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

    Measured: 36 months

  54. Hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Yes/No

    Measured: 6 months

  55. Hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Yes/No

    Measured: 12 months

  56. Hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Yes/No

    Measured: 18 months

  57. Hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Yes/No

    Measured: 24 months

  58. Hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Yes/No

    Measured: 30 months

  59. Hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Yes/No

    Measured: 36 months

  60. 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

    Measured: 12 months

  61. 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

    Measured: 12 months

  62. 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.

    Measured: 6 months

  63. 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.

    Measured: 12 months

  64. 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.

    Measured: 18 months

  65. 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.

    Measured: 24 months

  66. 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.

    Measured: 30 months

  67. 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.

    Measured: 36 months

  68. 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

    Measured: 12 months

  69. Patient treatment satisfaction every 6 months until end of follow-up

    Patient treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied

    Measured: 6 months

  70. Patient treatment satisfaction every 6 months until end of follow-up

    Patient treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied

    Measured: 12 months

  71. Patient treatment satisfaction every 6 months until end of follow-up

    Patient treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied

    Measured: 18 months

  72. Patient treatment satisfaction every 6 months until end of follow-up

    Patient treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied

    Measured: 24 months

  73. Patient treatment satisfaction every 6 months until end of follow-up

    Patient treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied

    Measured: 30 months

  74. Patient treatment satisfaction every 6 months until end of follow-up

    Patient treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied

    Measured: 36 months

  75. Physician treatment satisfaction every 6 months until end of follow-up

    Physician treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied

    Measured: 6 months

  76. Physician treatment satisfaction every 6 months until end of follow-up

    Physician treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied

    Measured: 12 months

  77. Physician treatment satisfaction every 6 months until end of follow-up

    Physician treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied

    Measured: 18 months

  78. Physician treatment satisfaction every 6 months until end of follow-up

    Physician treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied

    Measured: 24 months

  79. Physician treatment satisfaction every 6 months until end of follow-up

    Physician treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied

    Measured: 30 months

  80. Physician treatment satisfaction every 6 months until end of follow-up

    Physician treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied

    Measured: 36 months

  81. 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.

    Measured: 24 hours

  82. Alanine transaminase (ALT) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Alanine transaminase (ALT) in U/L

    Measured: 6 months

  83. Alanine transaminase (ALT) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Alanine transaminase (ALT) in U/L

    Measured: 12 months

  84. Alanine transaminase (ALT) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Alanine transaminase (ALT) in U/L

    Measured: 18 months

  85. Alanine transaminase (ALT) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Alanine transaminase (ALT) in U/L

    Measured: 24 months

  86. Alanine transaminase (ALT) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Alanine transaminase (ALT) in U/L

    Measured: 30 months

  87. Alanine transaminase (ALT) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Alanine transaminase (ALT) in U/L

    Measured: 36 months

  88. Aspartate aminotransferase (AST) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Aspartate aminotransferase (AST) in U/L

    Measured: 6 months

  89. Aspartate aminotransferase (AST) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Aspartate aminotransferase (AST) in U/L

    Measured: 12 months

  90. Aspartate aminotransferase (AST) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Aspartate aminotransferase (AST) in U/L

    Measured: 18 months

  91. Aspartate aminotransferase (AST) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Aspartate aminotransferase (AST) in U/L

    Measured: 24 months

  92. Aspartate aminotransferase (AST) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Aspartate aminotransferase (AST) in U/L

    Measured: 30 months

  93. Aspartate aminotransferase (AST) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Aspartate aminotransferase (AST) in U/L

    Measured: 36 months

  94. Alkaline Phosphatase at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Alkaline Phosphatase in µkat/L

    Measured: 6 months

  95. Alkaline Phosphatase at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Alkaline Phosphatase in µkat/L

    Measured: 12 months

  96. Alkaline Phosphatase at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Alkaline Phosphatase in µkat/L

    Measured: 18 months

  97. Alkaline Phosphatase at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Alkaline Phosphatase in µkat/L

    Measured: 24 months

  98. Alkaline Phosphatase at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Alkaline Phosphatase in µkat/L

    Measured: 30 months

  99. Alkaline Phosphatase at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Alkaline Phosphatase in µkat/L

    Measured: 36 months

  100. Bilirubin at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Indirect/ total bilirubin in umol/L

    Measured: 6 months

  101. Bilirubin at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Indirect/ total bilirubin in umol/L

    Measured: 12 months

  102. Bilirubin at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Indirect/ total bilirubin in umol/L

    Measured: 18 months

  103. Bilirubin at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Indirect/ total bilirubin in umol/L

    Measured: 24 months

  104. Bilirubin at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Indirect/ total bilirubin in umol/L

    Measured: 30 months

  105. Bilirubin at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Indirect/ total bilirubin in umol/L

    Measured: 36 months

  106. Creatinine at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Creatinine in mg/dL

    Measured: 6 months

  107. Creatinine at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Creatinine in mg/dL

    Measured: 12 months

  108. Creatinine at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Creatinine in mg/dL

    Measured: 18 months

  109. Creatinine at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Creatinine in mg/dL

    Measured: 24 months

  110. Creatinine at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Creatinine in mg/dL

    Measured: 30 months

  111. Creatinine at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Creatinine in mg/dL

    Measured: 36 months

  112. 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

    Measured: 6 months

  113. 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

    Measured: 12 months

  114. 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

    Measured: 18 months

  115. 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

    Measured: 24 months

  116. 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

    Measured: 30 months

  117. 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

    Measured: 36 months

  118. Urea at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Urea in mg/dL

    Measured: 6 months

  119. Urea at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Urea in mg/dL

    Measured: 12 months

  120. Urea at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Urea in mg/dL

    Measured: 18 months

  121. Urea at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Urea in mg/dL

    Measured: 24 months

  122. Urea at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Urea in mg/dL

    Measured: 30 months

  123. Urea at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Urea in mg/dL

    Measured: 36 months

  124. Potassium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Potassium in mmol/L

    Measured: 6 months

  125. Potassium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Potassium in mmol/L

    Measured: 12 months

  126. Potassium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Potassium in mmol/L

    Measured: 18 months

  127. Potassium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Potassium in mmol/L

    Measured: 24 months

  128. Potassium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Potassium in mmol/L

    Measured: 30 months

  129. Potassium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Potassium in mmol/L

    Measured: 36 months

  130. Sodium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Sodium in mmol/L

    Measured: 6 months

  131. Sodium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Sodium in mmol/L

    Measured: 12 months

  132. Sodium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Sodium in mmol/L

    Measured: 18 months

  133. Sodium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Sodium in mmol/L

    Measured: 24 months

  134. Sodium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Sodium in mmol/L

    Measured: 30 months

  135. Sodium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Sodium in mmol/L

    Measured: 36 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted20 Mar 2023
  2. Study start26 Jun 2023
  3. Primary completion15 Aug 2029 (estimated)
  4. Study completion15 Aug 2029 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Observational
Observational model
Cohort
Time perspective
Other
Enrolment
165 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05776472
Sponsor's protocol ID
Sobi.PEGCET-304
Acronym
COMPLETE
Status last verified
Jun 2026
First submitted
20 Feb 2023
Last updated
3 Sep 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

An observational study of Paroxysmal Nocturnal Hemoglobinuria, run by Swedish Orphan Biovitrum at 87 sites in 15 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 3 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 87 sites in Italy, Spain, France, Germany, Poland and 10 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change in observed hemoglobin level from initiation of treatment with pegcetacoplan to 6 months (time frame: 6 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05776472.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Sep 2026; AzTrials last refreshed its copy on 6 Nov 2026.

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