Informations générales (source: ClinicalTrials.gov)
A Single Arm, Long-term, Multicentre Observational Study to Evaluate Effectiveness of Pegcetacoplan Under Real World Conditions in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) (COMPLETE)
Observational
Swedish Orphan Biovitrum (Voir sur ClinicalTrials)
juin 2023
août 2029
23 avril 2025
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.
Etablissements
| Les établissements d'Île-de-France ayant mis à jour leurs données Origine et niveau de fiabilité des données | |||||
|---|---|---|---|---|---|
| HOPITAL FOCH | YOLAND SCHOINDRE | 05/05/2025 07:12:14 | Contacter | ||
| Les établissements sans correspondance certaine dans le répertoire FINESS dont les données sont issues de ClinicalTrials.gov Origine et niveau de fiabilité des données | |||||
| Centre Hospitalier Lyon Sud - 69495 - Pierre-Bénite - Rhone - France | Contact (sur clinicalTrials) | ||||
| CHU de Bordeaux - Hôpital Haut-Lévêque - 33604 - Pessac cedex - Gironde - France | Contact (sur clinicalTrials) | ||||
| CHU de Grenoble - Hôpital Albert Michallon - 38043 - Grenoble - Isere - France | Contact (sur clinicalTrials) | ||||
| CHU de Nancy - Hôpital de Brabois Adultes - 54511 - Vandœuvre-lès-Nancy - Meurthe Et Moselle - France | Contact (sur clinicalTrials) | ||||
| CHU de Nantes - Hotel Dieu - 44093 - Nantes cedex 1 - Loire Atlantique - France | Contact (sur clinicalTrials) | ||||
| CHU Nice - Hôpital de l'Archet 1 - 06200 - Nice - Alpes Maritimes - France | Contact (sur clinicalTrials) | ||||
| Hopital Claude Huriez - CHRU Lille - 59037 - Lille cedex - Nord - France | Contact (sur clinicalTrials) | ||||
| Hôpital Saint-Louis - 75475 - Paris La Defense - Paris - France | Contact (sur clinicalTrials) | ||||
Critères
Tous
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.
- 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.
- 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.