A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Pegcetacoplan.
- Who it may be relevant to
- Registry conditions: Paroxysmal Nocturnal Hemoglobinuria. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Australia, Belgium, Canada, Croatia, Czechia +10
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
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)
Overview
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.
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.
Interventions
- Drug Pegcetacoplan
Pegcetacoplan will be prescribed according to the label in patients with PNH.
Primary outcome measures
- Change in observed hemoglobin level from initiation of treatment with pegcetacoplan to 6 months [Time frame: 6 months]
Secondary outcome measures (12)
- Change of LDH values from initiation of pegcetacoplan treatment to 6 months [Time frame: 6 months]
- Change in Absolute Reticulocyte Count (ARC) from initiation of pegcetacoplan treatment to 6 months [Time frame: 6 months]
- Change in indirect/ total bilirubin from initiation of pegcetacoplan treatment to 6 months [Time frame: 6 months]
- Change in Haptoglobin from initiation of pegcetacoplan treatment to 6 months [Time frame: 6 months]
- Change in Ferritin from initiation of pegcetacoplan treatment to 6 months [Time frame: 6 months]
- Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up [Time frame: 6 months]
- Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up [Time frame: 12 months]
- Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up [Time frame: 18 months]
- Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up [Time frame: 24 months]
- Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up [Time frame: 30 months]
- Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up [Time frame: 36 months]
- Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of follow-up [Time frame: 6 months]
Eligibility criteria
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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
Italy · 14 centers
- 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
- … and 6 more centers
Spain · 14 centers
- 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
- … and 6 more centers
France · 12 centers
- CHU Nice - Hôpital de l'Archet 1 — Nice
- CHU de Bordeaux - Hôpital Haut-Lévêque — Pessac
- Hopital Foch — Suresnes
- CHU de Grenoble - Hôpital Albert Michallon — Grenoble
- CHU de Nantes - Hotel Dieu — Nantes
- CHU de Nancy - Hôpital de Brabois Adultes — Vandœuvre-lès-Nancy
- Hopital Claude Huriez - CHRU Lille — Lille
- Hôpital Saint-Louis — La Defense
- … and 4 more centers
Germany · 10 centers
- Alb-Fils-Kliniken GmbH — Goettigen
- Studienzentrum Aschaffenburg — Aschaffenburg
- Universitaetsklinikum Frankfurt Goethe-Universitaet — Frankfurt am Main
- Universitaetsklinikum Giessen und Marburg GmbH Standort Giessen — Giessen
- Dres Ballo und Dr. Böck — Offenbach
- Universitaetsklinikum Essen — Essen
- Universitaetsklinikum Leipzig AoeR — Leipzig
- Praxis Fenchel Saalfeld — Saalfeld
- … and 2 more centers
Poland · 7 centers
- 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
Greece · 6 centers
- Evangelismos Hospital — Athens
- University General Hospital "Attikon" — Athens
- University General Hospital of Larissa — Larissa
- University General Hospital of Larissa — Larissa
- G Papanikolaou General Hospital — Thessaloniki
- General Hospital of Thessaloniki "IPPOKRATEIO" — Thessaloniki
Belgium · 5 centers
- Institut Jules Bordet — Anderlecht
- AZ Sint-Jan — Bruges
- CHU Brugmann-Site Horta — Brussels
- Centre Hospitalier Universitaire de Liege — Liège
- AZ Herentals — Turnhout
Australia · 4 centers
- Liverpool Hospital — Liverpool
- Calvary Mater Newcastle — Waratah
- Sunshine Coast University Hospital — Birtinya
- Canberra Hospital — Garran
Saudi Arabia · 4 centers
- 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
Canada · 3 centers
- London Health Sciences Centre (LHSC) - Victoria Hospital — London
- University Health Network — Toronto
- Hopital Maisonneuve-Rosemont d/b/a CIUSSS de l'Est-de-l'Île-de-Montréal — Montreal
Hungary · 3 centers
- DPC - Orszagos Hematologiai es Infektologiai Intezet — Budapest
- Semmelweis Egyetem — Budapest
- Semmelweis University — Budapest
Czechia · 2 centers
- Fakultni nemocnice Brno — Brno
- Ustav hematologie a krevni transfuze — Prague
Croatia · 1 center
- University hospital centre Zagreb — Zagreb
Finland · 1 center
- Helsingin yliopistollinen keskussairaala — Helsinki
United Kingdom · 1 center
Center list to be confirmed — check the primary protocol.
Identifiers
NCT: NCT05776472 · Sobi.PEGCET-304