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

NCT ID NCT05776472

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jul 01, 2026 · Last updated Sep 04, 2026 · Updated 3 times

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

165 people

The number who actually took part.

Started

Jun 2023

Expected to finish

Aug 2029

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

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

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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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

Locations

  • AZ Herentals

    Turnhout, Belgium

  • AZ Sint-Jan

    Bruges, Belgium

  • Alb-Fils-Kliniken GmbH

    Goettigen, Baden-Wurttemberg, Germany

  • Azienda Ospedaliera Universitaria Careggi

    Florence, Italy

  • Azienda Ospedaliera Universitaria Policlinico Sant'Orsola Malpighi IRCCS

    Bologna, Italy

  • Azienda Ospedaliero Universitaria Consorziale Policlinico di Bari

    Bari, Italy

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

    Torino, Italy

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

    Torino, Italy

  • CHU Brugmann-Site Horta

    Brussels, 1020, Belgium

  • CHU Nice - Hôpital de l'Archet 1

    Nice, Alpes Maritimes, France

  • CHU Poitiers

    Poitiers, France

  • CHU Rennes - Hopital Pontchaillou

    Rennes, France

  • CHU de Bordeaux - Hôpital Haut-Lévêque

    Pessac, Gironde, 33604, France

  • CHU de Grenoble - Hôpital Albert Michallon

    Grenoble, Isere, France

  • CHU de Nancy - Hôpital de Brabois Adultes

    Vandœuvre-lès-Nancy, Meurthe et Moselle, France

  • CHU de Nantes - Hotel Dieu

    Nantes, Loire Atlantique, France

  • Calvary Mater Newcastle

    Waratah, New South Wales, Australia

  • Canberra Hospital

    Garran, 2605, Australia

  • Centre Hospitalier Général de St Quentin

    Saint-Quentin, 02321, France

  • Centre Hospitalier Lyon Sud

    Pierre-Bénite, Rhone, France

  • Centre Hospitalier Universitaire de Liege

    Liège, Belgium

  • Complejo Hospitalario Universitario de Santiago

    Santiago de Compostela, Spain

  • DPC - Orszagos Hematologiai es Infektologiai Intezet

    Budapest, Hungary

  • Dres Ballo und Dr. Böck

    Offenbach, Hesse, Germany

  • Evangelismos Hospital

    Athens, Greece

  • Fakultni nemocnice Brno

    Brno, Czechia

  • Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico

    Milan, Italy

  • Fondazione Policlinico Universitario Agostino Gemelli IRCCS

    Roma, Italy

  • G Papanikolaou General Hospital

    Thessaloniki, Greece

  • General Hospital of Thessaloniki "IPPOKRATEIO"

    Thessaloniki, Greece

  • Grande Ospedale Metropolitano - Presidio Morelli

    Reggio Calabria, Italy

  • Haematologisch-Onkologische Schwerpunktpraxis

    Troisdorf, Germany

  • Helsingin yliopistollinen keskussairaala

    Helsinki, Finland

  • Hopital Claude Huriez - CHRU Lille

    Lille, Nord, France

  • Hopital Foch

    Suresnes, Hauts de Seine, France

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

    Montreal, Quebec, Canada

  • Hospital Clinic de Barcelona

    Barcelona, Spain

  • Hospital Lucus Augusti

    Lugo, Spain

  • Hospital Regional Universitario de Malaga

    Málaga, Spain

  • Hospital Universitari Son Espases

    Palma de Mallorca, Spain

  • Hospital Universitari Vall d'Hebron

    Barcelona, Spain

  • Hospital Universitario Central de Asturias

    Oviedo, Spain

  • Hospital Universitario Donostia

    Donostia / San Sebastian, Spain

  • Hospital Universitario Juan Ramon Jimenez

    Huelva, Spain

  • Hospital Universitario Virgen de la Nieves

    El Palmar, Spain

  • Hospital Universitario de Gran Canaria Dr. Negrin

    Las Palmas de Gran Canaria, Spain

  • Hospital de Basurto

    Bilbao, Spain

  • Hospital de Cruces

    Barakaldo, Spain

  • Hôpital Saint-Louis

    La Defense, Paris, France

  • ICO Badalona - Hospital Universitari Germans Trias i Pujol

    Badalona, Spain

  • Institut Jules Bordet

    Anderlecht, Belgium

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

    Bologna, Italy

  • King Faisal Specialist Hospital & Research Center

    Jeddah, Saudi Arabia

  • King Faisal Specialist Hospital and Research Center - Madinah

    Riyadh, Saudi Arabia

  • King Faisal Specialist Hospital and Research Center - Riyadh

    Riyadh, Saudi Arabia

  • King's College Hospital

    London, United Kingdom

  • Liverpool Hospital

    Liverpool, New South Wales, Australia

  • London Health Sciences Centre (LHSC) - Victoria Hospital

    London, Ontario, N6A 5W9, Canada

  • MV Zentrum für Onkologie und Hämatologie

    Cologne, Germany

  • Ospedale Maggiore della Carità

    Novara, Italy

  • Ospedale Oncologico Armando Businco

    Cagliari, Italy

  • Ospedale San Bassiano

    Bassano del Grappa, Italy

  • Ospedale San Raffaele

    Milan, Italy

  • Praxis Fenchel Saalfeld

    Saalfeld, Thuringia, Germany

  • Prince Faisal Bin Bandar Cancer Center

    Buraidah, Saudi Arabia

  • SPZOZ Szpital Uniwersytecki w Krakowie

    Krakow, Poland

  • Semmelweis Egyetem

    Budapest, Hungary

  • Semmelweis University

    Budapest, Hungary

  • Studienzentrum Aschaffenburg

    Aschaffenburg, Bavaria, Germany

  • Sunshine Coast University Hospital

    Birtinya, Queensland, Australia

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

    Bydgoszcz, Poland

  • Universitaetsklinikum Essen

    Essen, North Rhine-Westphalia, Germany

  • Universitaetsklinikum Frankfurt Goethe-Universitaet

    Frankfurt am Main, Hesse, Germany

  • Universitaetsklinikum Giessen und Marburg GmbH Standort Giessen

    Giessen, Hesse, Germany

  • Universitaetsklinikum Leipzig AoeR

    Leipzig, Saxony, Germany

  • University General Hospital "Attikon"

    Athens, Greece

  • University General Hospital of Larissa

    Larissa, 41500, Greece

  • University General Hospital of Larissa

    Larissa, Greece

  • University Health Network

    Toronto, Ontario, Canada

  • University hospital centre Zagreb

    Zagreb, Croatia

  • Università di Roma La Sapienza

    Rome, Italy

  • Uniwersytecki Szpital Kliniczny nr 1 w Lublinie

    Lublin, Poland

  • Uniwersytecki Szpital Kliniczny w Bialymstoku

    Bialystok, Poland

  • Uniwersyteckie Centrum Kliniczne

    Gdansk, Poland

  • Ustav hematologie a krevni transfuze

    Prague, Czechia

  • WWCOiT in Lodz, Oddział Hematologii i Transplantologii

    Lodz, Poland

  • Warszawski Uniwersytet Medyczny

    Warsaw, Poland

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