Real-World test: can a targeted drug keep PNH in check for years?

NCT ID NCT05776472

NEW Recruiting now Disease control Sponsor: Swedish Orphan Biovitrum Source: ClinicalTrials.gov ↗

First seen Jul 01, 2026 · Last updated Jul 01, 2026

Summary

This study follows about 200 adults with paroxysmal nocturnal hemoglobinuria (PNH) who are taking pegcetacoplan (Aspaveli/Empaveli) as part of their routine care. Researchers will track blood markers, symptoms, and side effects for up to 3 years to see how well the drug works outside of a controlled clinical trial. The goal is to understand the real-world effectiveness and safety of pegcetacoplan over the long term.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance

pegcetacoplan

What this could lead to

If results are positive, this could confirm pegcetacoplan as a reliable long-term treatment option for people with PNH in real-world settings.

What could go wrong

This is an observational study, not a controlled trial, so it cannot prove cause and effect. Results may be influenced by patient selection or other treatments.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

Hemoglobinuria, Paroxysmal paroxysmal nocturnal hemoglobinuria

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

PAROXYSMAL NOCTURNAL HEMOGLOBINURIA

Contacts and locations

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Study contacts

Contact

Phone: •••-•••-•••• Email: •••••@•••••

Locations

AZ Herentals
RECRUITING

Turnhout, Belgium
AZ Sint-Jan
RECRUITING

Bruges, Belgium
Alb-Fils-Kliniken GmbH
TERMINATED

Goettigen, Baden-Wurttemberg, Germany
Azienda Ospedaliera Universitaria Careggi
NOT_YET_RECRUITING

Florence, Italy
Azienda Ospedaliera Universitaria Policlinico Sant'Orsola Malpighi IRCCS
ACTIVE_NOT_RECRUITING

Bologna, Italy
Azienda Ospedaliero Universitaria Consorziale Policlinico di Bari
RECRUITING

Bari, Italy
Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino - Onoclogy and Hematology Department
RECRUITING

Torino, Italy
Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino - UO Ematologia
NOT_YET_RECRUITING

Torino, Italy
CHU Brugmann-Site Horta
RECRUITING

Brussels, 1020, Belgium
CHU Nice - Hôpital de l'Archet 1
RECRUITING

Nice, Alpes Maritimes, France
CHU Poitiers
NOT_YET_RECRUITING

Poitiers, France
CHU Rennes - Hopital Pontchaillou
ACTIVE_NOT_RECRUITING

Rennes, France
CHU de Bordeaux - Hôpital Haut-Lévêque
RECRUITING

Pessac, Gironde, 33604, France
CHU de Grenoble - Hôpital Albert Michallon
RECRUITING

Grenoble, Isere, France
CHU de Nancy - Hôpital de Brabois Adultes
RECRUITING

Vandœuvre-lès-Nancy, Meurthe et Moselle, France
CHU de Nantes - Hotel Dieu
RECRUITING

Nantes, Loire Atlantique, France
Calvary Mater Newcastle
RECRUITING

Waratah, New South Wales, Australia
Canberra Hospital
RECRUITING

Garran, 2605, Australia
Centre Hospitalier Général de St Quentin
RECRUITING

Saint-Quentin, 02321, France
Centre Hospitalier Lyon Sud
RECRUITING

Pierre-Bénite, Rhone, France
Centre Hospitalier Universitaire de Liege
RECRUITING

Liège, Belgium
Complejo Hospitalario Universitario de Santiago
RECRUITING

Santiago de Compostela, Spain
DPC - Orszagos Hematologiai es Infektologiai Intezet
RECRUITING

Budapest, Hungary
Dres Ballo und Dr. Böck
RECRUITING

Offenbach, Hesse, Germany
Evangelismos Hospital
NOT_YET_RECRUITING

Athens, Greece
Fakultni nemocnice Brno
RECRUITING

Brno, Czechia
Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico
RECRUITING

Milan, Italy
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
RECRUITING

Roma, Italy
G Papanikolaou General Hospital
RECRUITING

Thessaloniki, Greece
General Hospital of Thessaloniki "IPPOKRATEIO"
RECRUITING

Thessaloniki, Greece
Grande Ospedale Metropolitano - Presidio Morelli
RECRUITING

Reggio Calabria, Italy
Haematologisch-Onkologische Schwerpunktpraxis
TERMINATED

Troisdorf, Germany
Helsingin yliopistollinen keskussairaala
RECRUITING

Helsinki, Finland
Hopital Claude Huriez - CHRU Lille
RECRUITING

Lille, Nord, France
Hopital Foch
RECRUITING

Suresnes, Hauts de Seine, France
Hopital Maisonneuve-Rosemont d/b/a CIUSSS de l'Est-de-l'Île-de-Montréal
ACTIVE_NOT_RECRUITING

Montreal, Quebec, Canada
Hospital Clinic de Barcelona
RECRUITING

Barcelona, Spain
Hospital Lucus Augusti
RECRUITING

Lugo, Spain
Hospital Regional Universitario de Malaga
RECRUITING

Málaga, Spain
Hospital Universitari Son Espases
RECRUITING

Palma de Mallorca, Spain
Hospital Universitari Vall d'Hebron
RECRUITING

Barcelona, Spain
Hospital Universitario Central de Asturias
RECRUITING

Oviedo, Spain
Hospital Universitario Donostia
RECRUITING

Donostia / San Sebastian, Spain
Hospital Universitario Juan Ramon Jimenez
ACTIVE_NOT_RECRUITING

Huelva, Spain
Hospital Universitario Virgen de la Nieves
RECRUITING

El Palmar, Spain
Hospital Universitario de Gran Canaria Dr. Negrin
RECRUITING

Las Palmas de Gran Canaria, Spain
Hospital de Basurto
ACTIVE_NOT_RECRUITING

Bilbao, Spain
Hospital de Cruces
RECRUITING

Barakaldo, Spain
Hôpital Saint-Louis
RECRUITING

La Defense, Paris, France
ICO Badalona - Hospital Universitari Germans Trias i Pujol
RECRUITING

Badalona, Spain
Institut Jules Bordet
RECRUITING

Anderlecht, Belgium
Irccs Aou Di Bologna- Pol. Di S.Orsola
NOT_YET_RECRUITING

Bologna, Italy
King Faisal Specialist Hospital & Research Center
NOT_YET_RECRUITING

Jeddah, Saudi Arabia
King Faisal Specialist Hospital and Research Center - Madinah
RECRUITING

Riyadh, Saudi Arabia
King Faisal Specialist Hospital and Research Center - Riyadh
RECRUITING

Riyadh, Saudi Arabia
King's College Hospital
COMPLETED

London, United Kingdom
Liverpool Hospital
RECRUITING

Liverpool, New South Wales, Australia
London Health Sciences Centre (LHSC) - Victoria Hospital
RECRUITING

London, Ontario, N6A 5W9, Canada
MV Zentrum für Onkologie und Hämatologie
NOT_YET_RECRUITING

Cologne, Germany
Ospedale Maggiore della Carità
RECRUITING

Novara, Italy
Ospedale Oncologico Armando Businco
NOT_YET_RECRUITING

Cagliari, Italy
Ospedale San Bassiano
RECRUITING

Bassano del Grappa, Italy
Ospedale San Raffaele
RECRUITING

Milan, Italy
Praxis Fenchel Saalfeld
RECRUITING

Saalfeld, Thuringia, Germany
Prince Faisal Bin Bandar Cancer Center
RECRUITING

Buraidah, Saudi Arabia
SPZOZ Szpital Uniwersytecki w Krakowie
RECRUITING

Krakow, Poland
Semmelweis Egyetem
RECRUITING

Budapest, Hungary
Semmelweis University
RECRUITING

Budapest, Hungary
Studienzentrum Aschaffenburg
TERMINATED

Aschaffenburg, Bavaria, Germany
Sunshine Coast University Hospital
RECRUITING

Birtinya, Queensland, Australia
Szpital Uniwersytecki nr 2 im. dr J. Biziela w Bydgoszczy
RECRUITING

Bydgoszcz, Poland
Universitaetsklinikum Essen
RECRUITING

Essen, North Rhine-Westphalia, Germany
Universitaetsklinikum Frankfurt Goethe-Universitaet
RECRUITING

Frankfurt am Main, Hesse, Germany
Universitaetsklinikum Giessen und Marburg GmbH Standort Giessen
ACTIVE_NOT_RECRUITING

Giessen, Hesse, Germany
Universitaetsklinikum Leipzig AoeR
RECRUITING

Leipzig, Saxony, Germany
University General Hospital "Attikon"
RECRUITING

Athens, Greece
University General Hospital of Larissa
RECRUITING

Larissa, 41500, Greece
University General Hospital of Larissa
RECRUITING

Larissa, Greece
University Health Network
RECRUITING

Toronto, Ontario, Canada
University hospital centre Zagreb
RECRUITING

Zagreb, Croatia
Università di Roma La Sapienza
RECRUITING

Rome, Italy
Uniwersytecki Szpital Kliniczny nr 1 w Lublinie
NOT_YET_RECRUITING

Lublin, Poland
Uniwersytecki Szpital Kliniczny w Bialymstoku
RECRUITING

Bialystok, Poland
Uniwersyteckie Centrum Kliniczne
RECRUITING

Gdansk, Poland
Ustav hematologie a krevni transfuze
RECRUITING

Prague, Czechia
WWCOiT in Lodz, Oddział Hematologii i Transplantologii
RECRUITING

Lodz, Poland
Warszawski Uniwersytet Medyczny
NOT_YET_RECRUITING

Warsaw, Poland