Overview
This is a 24-month 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 at the enrollment visit and followed prospectively for 24 (+/- 3) months. 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.
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 pre and post pegcetacoplan treatment outcomes.
The study plans to include approximately 200 patients at 70 sites in Europe, Middle East, Canada and Australia. Patients meeting the eligibility criteria will be enrolled in the study at the enrollment visit and followed prospectively for 24 (+/- 3) months. 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.
Eligibility
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.