A Single Arm, Long-term, Multicentre Observational Study to Evaluate Effectiveness of Pegcetacoplan Under Real World Conditions in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Brief Summary

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.

Primary Outcomes

Secondary Outcomes

• Change in Ferritin from initiation of pegcetacoplan treatment to 6 months(6 months)
• Change of LDH values from initiation of pegcetacoplan treatment to 6 months(6 months)
• Change in Absolute Reticulocyte Count (ARC) from initiation of pegcetacoplan treatment to 6 months(6 months)
• Change in indirect/ total bilirubin from initiation of pegcetacoplan treatment to 6 months(6 months)
• Change in Haptoglobin from initiation of pegcetacoplan treatment to 6 months(6 months)
• Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up(36 months)
• Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of follow-up(36 months)
• Absolute Reticulocyte Count at initiation of pegcetacoplan treatment and each 6 months until end of follow-up(36 months)
• Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up(36 months)
• Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up(36 months)
• Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up(36 months)
• Hemoglobin ≥ 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up(36 months)
• Increase in hemoglobin levels of ≥ 2 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up(36 months)
• Hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of follow-up(36 months)
• Annualized number of red blood cell (RBC) transfusions during pegcetacoplan treatment until end of follow-up compared to the 12 month period before pegcetacoplan treatment(12 months)
• Annualized number of red blood cell (RBC) units during pegcetacoplan treatment until end of follow-up compared to the 12 month period before pegcetacoplan treatment(12 months)
• Fatigue at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up(36 months)
• Health care resource use: Annualized number of hospitalizations and emergency room visits during pegcetacoplan treatment until end of follow-up compared to the 12-month period before pegcetacoplan treatment.(12 months)
• Patient treatment satisfaction every 6 months until end of follow-up(36 months)
• Physician treatment satisfaction every 6 months until end of follow-up(36 months)
• Adverse events (AE), including serious adverse events (SAE)(24 hours)
• Alanine transaminase (ALT) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up(36 months)
• Aspartate aminotransferase (AST) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up(36 months)
• Alkaline Phosphatase at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up(36 months)
• Bilirubin at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up(36 months)
• Creatinine at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up(36 months)
• estimated glomerular filtration rate (eGFR) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up(36 months)
• Urea at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up(36 months)
• Potassium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up(36 months)
• Sodium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up(36 months)