Paroxysmal Nocturnal Hemoglobinuria
Conditions
Brief summary
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
DRUGPegcetacoplan
Pegcetacoplan will be prescribed according to the label in patients with PNH.
Sponsors
Swedish Orphan Biovitrum
IQVIA Pty Ltd
Study design
Observational model
COHORT
Time perspective
OTHER
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in observed hemoglobin level from initiation of treatment with pegcetacoplan to 6 months | 6 months | Hemoglobin level in g/dL. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | 6 months | Yes/No |
| 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 | Total number of RBC transfusions |
| Change of LDH values from initiation of pegcetacoplan treatment to 6 months | 6 months | Lactate Dehydrogenase (LDH) in U/L |
| Change in Absolute Reticulocyte Count (ARC) from initiation of pegcetacoplan treatment to 6 months | 6 months | Absolute Reticulocyte Count (ARC) in 10\^9/L |
| Change in indirect/ total bilirubin from initiation of pegcetacoplan treatment to 6 months | 6 months | Indirect/ total bilirubin in umol/L |
| Change in Haptoglobin from initiation of pegcetacoplan treatment to 6 months | 6 months | Haptoglobin in mg/dL |
| Change in Ferritin from initiation of pegcetacoplan treatment to 6 months | 6 months | Ferritin in ug/L |
| Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | 6 months | Hemoglobin in g/dL |
| Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | 6 months | Lactate Dehydrogenase (LDH) in U/L |
| Absolute Reticulocyte Count at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | 6 months | Absolute Reticulocyte Count (ARC) in 10\^9/L |
| Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | 6 months | Indirect/ total bilirubin in umol/L |
| Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | 6 months | Haptoglobin in µmol/L |
| Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | 6 months | Ferritin in ug/L |
| Hemoglobin ≥ 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | 6 months | Hemoglobin in g/dL |
| Increase in hemoglobin levels of ≥ 2 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | 6 months | Hemoglobin in g/dL |
| Fatigue at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | 6 months | Patient reported outcome scale scores for the Functional assessment of chronic illness therapy (FACIT) - Fatigue. It is a 13-item scale and each item is scored on a 5-point Likert Scale ranging from "0-Not at all" to "4-Very much". The FACIT-fatigue score is obtained by summing all item scores. The score range from 0 to 52, higher score indicating less fatigue and lower score indicating more fatigue. |
| 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 | Number of hospitalizations and emergency room visits |
| Patient treatment satisfaction every 6 months until end of follow-up | 6 months | Patient treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied |
| Physician treatment satisfaction every 6 months until end of follow-up | 6 months | Physician treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied |
| Adverse events (AE), including serious adverse events (SAE) | 24 hours | Adverse events (AE) and serious adverse events (SAE) are to be reported on the AE page of the eCRF as specified in the eCRF data entry guidelines. |
| Alanine transaminase (ALT) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | 6 months | Alanine transaminase (ALT) in U/L |
| Aspartate aminotransferase (AST) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | 6 months | Aspartate aminotransferase (AST) in U/L |
| Alkaline Phosphatase at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | 6 months | Alkaline Phosphatase in µkat/L |
| Bilirubin at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | 6 months | Indirect/ total bilirubin in umol/L |
| Creatinine at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | 6 months | Creatinine in mg/dL |
| estimated glomerular filtration rate (eGFR) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | 6 months | estimated glomerular filtration rate (eGFR) in mL/min/1.73 m² |
| Urea at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | 6 months | Urea in mg/dL |
| Potassium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | 6 months | Potassium in mmol/L |
| 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 | Total number of RBC units |
| Sodium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | 6 months | Sodium in mmol/L |
Countries
Australia, Belgium, Canada, Croatia, Czechia, Finland, France, Germany, Greece, Hungary, Italy, Poland, Saudi Arabia, Spain, United Kingdom
Contacts
STUDY_DIRECTORClinical Program Lead +46 08-697-20 00
Swedish Orphan Biovitrum
Outcome results
None listed