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| Name | Class |
|---|---|
| IQVIA Pty Ltd | INDUSTRY |
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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.
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.
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Pegcetacoplan | Drug | Pegcetacoplan will be prescribed according to the label in patients with PNH. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change in observed hemoglobin level from initiation of treatment with pegcetacoplan to 6 months | Hemoglobin level in g/dL. | 6 months |
| Measure | Description | Time Frame |
|---|---|---|
| Change of LDH values from initiation of pegcetacoplan treatment to 6 months | Lactate Dehydrogenase (LDH) in U/L | 6 months |
| Change in Absolute Reticulocyte Count (ARC) from initiation of pegcetacoplan treatment to 6 months |
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Inclusion Criteria:
Exclusion Criteria:
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Adult patients (≥18 years) with PNH, who started routine treatment with pegcetacoplan up to 12 months prior to enrollmentor at enrollment
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Clinical Program Lead | Contact | +46 (0)8 697 20 00 | medical.info@sobi.com |
| Name | Affiliation | Role |
|---|---|---|
| Clinical Program Lead +46 08-697-20 00 | Swedish Orphan Biovitrum | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Liverpool Hospital | Recruiting | Liverpool | New South Wales | Australia | ||
| Calvary Mater Newcastle |
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Absolute Reticulocyte Count (ARC) in 10^9/L
| 6 months |
| Change in indirect/ total bilirubin from initiation of pegcetacoplan treatment to 6 months | Indirect/ total bilirubin in umol/L | 6 months |
| Change in Haptoglobin from initiation of pegcetacoplan treatment to 6 months | Haptoglobin in mg/dL | 6 months |
| Change in Ferritin from initiation of pegcetacoplan treatment to 6 months | Ferritin in ug/L | 6 months |
| Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Hemoglobin in g/dL | 6 months |
| Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Hemoglobin in g/dL | 12 months |
| Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Hemoglobin in g/dL | 18 months |
| Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Hemoglobin in g/dL | 24 months |
| Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Hemoglobin in g/dL | 30 months |
| Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Hemoglobin in g/dL | 36 months |
| Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Lactate Dehydrogenase (LDH) in U/L | 6 months |
| Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Lactate Dehydrogenase (LDH) in U/L | 12 months |
| Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Lactate Dehydrogenase (LDH) in U/L | 18 months |
| Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Lactate Dehydrogenase (LDH) in U/L | 24 months |
| Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Lactate Dehydrogenase (LDH) in U/L | 30 months |
| Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Lactate Dehydrogenase (LDH) in U/L | 36 months |
| Absolute Reticulocyte Count at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Absolute Reticulocyte Count (ARC) in 10^9/L | 6 months |
| Absolute Reticulocyte Count at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Absolute Reticulocyte Count (ARC) in 10^9/L | 12 months |
| Absolute Reticulocyte Count at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Absolute Reticulocyte Count (ARC) in 10^9/L | 18 months |
| Absolute Reticulocyte Count at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Absolute Reticulocyte Count (ARC) in 10^9/L | 24 months |
| Absolute Reticulocyte Count at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Absolute Reticulocyte Count (ARC) in 10^9/L | 30 months |
| Absolute Reticulocyte Count at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Absolute Reticulocyte Count (ARC) in 10^9/L | 36 months |
| Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Indirect/ total bilirubin in umol/L | 6 months |
| Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Indirect/ total bilirubin in umol/L | 12 months |
| Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Indirect/ total bilirubin in umol/L | 18 months |
| Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Indirect/ total bilirubin in umol/L | 24 months |
| Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Indirect/ total bilirubin in umol/L | 30 months |
| Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Indirect/ total bilirubin in umol/L | 36 months |
| Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Haptoglobin in µmol/L | 6 months |
| Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Haptoglobin in µmol/L | 12 months |
| Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Haptoglobin in µmol/L | 18 months |
| Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Haptoglobin in µmol/L | 24 months |
| Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Haptoglobin in µmol/L | 30 months |
| Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Haptoglobin in mg/dL | 36 months |
| Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Ferritin in ug/L | 6 months |
| Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Ferritin in ug/L | 12 months |
| Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Ferritin in ug/L | 18 months |
| Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Ferritin in ug/L | 24 months |
| Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Ferritin in ug/L | 30 months |
| Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Ferritin in ug/L | 36 months |
| Hemoglobin ≥ 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Hemoglobin in g/dL | 6 months |
| Hemoglobin ≥ 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Hemoglobin in g/dL | 12 months |
| Hemoglobin ≥ 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Hemoglobin in g/dL | 18 months |
| Hemoglobin ≥ 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Hemoglobin in g/dL | 24 months |
| Hemoglobin ≥ 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Hemoglobin in g/dL | 30 months |
| Hemoglobin ≥ 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Hemoglobin in g/dL | 36 months |
| Increase in hemoglobin levels of ≥ 2 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Hemoglobin in g/dL | 6 months |
| Increase in hemoglobin levels of ≥ 2 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Hemoglobin in g/dL | 12 months |
| Increase in hemoglobin levels of ≥ 2 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Hemoglobin in g/dL | 18 months |
| Increase in hemoglobin levels of ≥ 2 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Hemoglobin in g/dL | 24 months |
| Increase in hemoglobin levels of ≥ 2 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Hemoglobin in g/dL | 30 months |
| Increase in hemoglobin levels of ≥ 2 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Hemoglobin in g/dL | 36 months |
| Hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Yes/No | 6 months |
| Hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Yes/No | 12 months |
| Hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Yes/No | 18 months |
| Hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Yes/No | 24 months |
| Hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Yes/No | 30 months |
| Hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of follow-up | Yes/No | 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 | Total number of RBC transfusions | 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 | Total number of RBC units | 12 months |
| Fatigue at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | 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. | 6 months |
| Fatigue at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | 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. | 12 months |
| Fatigue at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | 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. | 18 months |
| Fatigue at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | 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. | 24 months |
| Fatigue at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | 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. | 30 months |
| Fatigue at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | 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. | 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. | Number of hospitalizations and emergency room visits | 12 months |
| Patient treatment satisfaction every 6 months until end of follow-up | Patient treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied | 6 months |
| Patient treatment satisfaction every 6 months until end of follow-up | Patient treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied | 12 months |
| Patient treatment satisfaction every 6 months until end of follow-up | Patient treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied | 18 months |
| Patient treatment satisfaction every 6 months until end of follow-up | Patient treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied | 24 months |
| Patient treatment satisfaction every 6 months until end of follow-up | Patient treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied | 30 months |
| Patient treatment satisfaction every 6 months until end of follow-up | Patient treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied | 36 months |
| Physician treatment satisfaction every 6 months until end of follow-up | Physician treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied | 6 months |
| Physician treatment satisfaction every 6 months until end of follow-up | Physician treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied | 12 months |
| Physician treatment satisfaction every 6 months until end of follow-up | Physician treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied | 18 months |
| Physician treatment satisfaction every 6 months until end of follow-up | Physician treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied | 24 months |
| Physician treatment satisfaction every 6 months until end of follow-up | Physician treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied | 30 months |
| Physician treatment satisfaction every 6 months until end of follow-up | Physician treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied | 36 months |
| Adverse events (AE), including serious adverse events (SAE) | 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. | 24 hours |
| Alanine transaminase (ALT) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Alanine transaminase (ALT) in U/L | 6 months |
| Alanine transaminase (ALT) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Alanine transaminase (ALT) in U/L | 12 months |
| Alanine transaminase (ALT) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Alanine transaminase (ALT) in U/L | 18 months |
| Alanine transaminase (ALT) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Alanine transaminase (ALT) in U/L | 24 months |
| Alanine transaminase (ALT) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Alanine transaminase (ALT) in U/L | 30 months |
| Alanine transaminase (ALT) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Alanine transaminase (ALT) in U/L | 36 months |
| Aspartate aminotransferase (AST) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Aspartate aminotransferase (AST) in U/L | 6 months |
| Aspartate aminotransferase (AST) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Aspartate aminotransferase (AST) in U/L | 12 months |
| Aspartate aminotransferase (AST) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Aspartate aminotransferase (AST) in U/L | 18 months |
| Aspartate aminotransferase (AST) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Aspartate aminotransferase (AST) in U/L | 24 months |
| Aspartate aminotransferase (AST) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Aspartate aminotransferase (AST) in U/L | 30 months |
| Aspartate aminotransferase (AST) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Aspartate aminotransferase (AST) in U/L | 36 months |
| Alkaline Phosphatase at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Alkaline Phosphatase in µkat/L | 6 months |
| Alkaline Phosphatase at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Alkaline Phosphatase in µkat/L | 12 months |
| Alkaline Phosphatase at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Alkaline Phosphatase in µkat/L | 18 months |
| Alkaline Phosphatase at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Alkaline Phosphatase in µkat/L | 24 months |
| Alkaline Phosphatase at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Alkaline Phosphatase in µkat/L | 30 months |
| Alkaline Phosphatase at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Alkaline Phosphatase in µkat/L | 36 months |
| Bilirubin at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Indirect/ total bilirubin in umol/L | 6 months |
| Bilirubin at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Indirect/ total bilirubin in umol/L | 12 months |
| Bilirubin at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Indirect/ total bilirubin in umol/L | 18 months |
| Bilirubin at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Indirect/ total bilirubin in umol/L | 24 months |
| Bilirubin at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Indirect/ total bilirubin in umol/L | 30 months |
| Bilirubin at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Indirect/ total bilirubin in umol/L | 36 months |
| Creatinine at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Creatinine in mg/dL | 6 months |
| Creatinine at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Creatinine in mg/dL | 12 months |
| Creatinine at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Creatinine in mg/dL | 18 months |
| Creatinine at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Creatinine in mg/dL | 24 months |
| Creatinine at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Creatinine in mg/dL | 30 months |
| Creatinine at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Creatinine in mg/dL | 36 months |
| estimated glomerular filtration rate (eGFR) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | estimated glomerular filtration rate (eGFR) in mL/min/1.73 m² | 6 months |
| estimated glomerular filtration rate (eGFR) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | estimated glomerular filtration rate (eGFR) in mL/min/1.73 m² | 12 months |
| estimated glomerular filtration rate (eGFR) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | estimated glomerular filtration rate (eGFR) in mL/min/1.73 m² | 18 months |
| estimated glomerular filtration rate (eGFR) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | estimated glomerular filtration rate (eGFR) in mL/min/1.73 m² | 24 months |
| estimated glomerular filtration rate (eGFR) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | estimated glomerular filtration rate (eGFR) in mL/min/1.73 m² | 30 months |
| estimated glomerular filtration rate (eGFR) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | estimated glomerular filtration rate (eGFR) in mL/min/1.73 m² | 36 months |
| Urea at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Urea in mg/dL | 6 months |
| Urea at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Urea in mg/dL | 12 months |
| Urea at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Urea in mg/dL | 18 months |
| Urea at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Urea in mg/dL | 24 months |
| Urea at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Urea in mg/dL | 30 months |
| Urea at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Urea in mg/dL | 36 months |
| Potassium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Potassium in mmol/L | 6 months |
| Potassium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Potassium in mmol/L | 12 months |
| Potassium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Potassium in mmol/L | 18 months |
| Potassium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Potassium in mmol/L | 24 months |
| Potassium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Potassium in mmol/L | 30 months |
| Potassium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Potassium in mmol/L | 36 months |
| Sodium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Sodium in mmol/L | 6 months |
| Sodium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Sodium in mmol/L | 12 months |
| Sodium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Sodium in mmol/L | 18 months |
| Sodium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Sodium in mmol/L | 24 months |
| Sodium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Sodium in mmol/L | 30 months |
| Sodium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up | Sodium in mmol/L | 36 months |
| Recruiting |
| Waratah |
| New South Wales |
| Australia |
| Sunshine Coast University Hospital | Recruiting | Birtinya | Queensland | Australia |
| Canberra Hospital | Recruiting | Garran | 2605 | Australia |
| Institut Jules Bordet | Recruiting | Anderlecht | Belgium |
| AZ Sint-Jan | Recruiting | Bruges | Belgium |
| CHU Brugmann-Site Horta | Recruiting | Brussels | 1020 | Belgium |
| Centre Hospitalier Universitaire de Liege | Recruiting | Liège | Belgium |
| AZ Herentals | Recruiting | Turnhout | Belgium |
| London Health Sciences Centre (LHSC) - Victoria Hospital | Recruiting | London | Ontario | N6A 5W9 | Canada |
| University Health Network | Recruiting | Toronto | Ontario | Canada |
| Hopital Maisonneuve-Rosemont d/b/a CIUSSS de l'Est-de-l'Île-de-Montréal | Active, not recruiting | Montreal | Quebec | Canada |
| University hospital centre Zagreb | Recruiting | Zagreb | Croatia |
| Fakultni nemocnice Brno | Recruiting | Brno | Czechia |
| Ustav hematologie a krevni transfuze | Recruiting | Prague | Czechia |
| Helsingin yliopistollinen keskussairaala | Recruiting | Helsinki | Finland |
| CHU Nice - Hôpital de l'Archet 1 | Recruiting | Nice | Alpes Maritimes | France |
| CHU de Bordeaux - Hôpital Haut-Lévêque | Recruiting | Pessac | Gironde | 33604 | France |
| Hopital Foch | Recruiting | Suresnes | Hauts de Seine | France |
| CHU de Grenoble - Hôpital Albert Michallon | Recruiting | Grenoble | Isere | France |
| CHU de Nantes - Hotel Dieu | Recruiting | Nantes | Loire Atlantique | France |
| CHU de Nancy - Hôpital de Brabois Adultes | Recruiting | Vandœuvre-lès-Nancy | Meurthe et Moselle | France |
| Hopital Claude Huriez - CHRU Lille | Recruiting | Lille | Nord | France |
| Hôpital Saint-Louis | Recruiting | La Defense | Paris | France |
| Centre Hospitalier Lyon Sud | Recruiting | Pierre-Bénite | Rhone | France |
| CHU Poitiers | Not yet recruiting | Poitiers | France |
| CHU Rennes - Hopital Pontchaillou | Active, not recruiting | Rennes | France |
| Centre Hospitalier Général de St Quentin | Recruiting | Saint-Quentin | 02321 | France |
| Alb-Fils-Kliniken GmbH | Terminated | Goettigen | Baden-Wurttemberg | Germany |
| Studienzentrum Aschaffenburg | Terminated | Aschaffenburg | Bavaria | Germany |
| Universitaetsklinikum Frankfurt Goethe-Universitaet | Recruiting | Frankfurt am Main | Hesse | Germany |
| Universitaetsklinikum Giessen und Marburg GmbH Standort Giessen | Active, not recruiting | Giessen | Hesse | Germany |
| Dres Ballo und Dr. Böck | Recruiting | Offenbach | Hesse | Germany |
| Universitaetsklinikum Essen | Recruiting | Essen | North Rhine-Westphalia | Germany |
| Universitaetsklinikum Leipzig AoeR | Recruiting | Leipzig | Saxony | Germany |
| Praxis Fenchel Saalfeld | Recruiting | Saalfeld | Thuringia | Germany |
| MV Zentrum für Onkologie und Hämatologie | Not yet recruiting | Cologne | Germany |
| Haematologisch-Onkologische Schwerpunktpraxis | Terminated | Troisdorf | Germany |
| Evangelismos Hospital | Not yet recruiting | Athens | Greece |
| University General Hospital "Attikon" | Recruiting | Athens | Greece |
| University General Hospital of Larissa | Recruiting | Larissa | 41500 | Greece |
| University General Hospital of Larissa | Recruiting | Larissa | Greece |
| G Papanikolaou General Hospital | Recruiting | Thessaloniki | Greece |
| General Hospital of Thessaloniki "IPPOKRATEIO" | Recruiting | Thessaloniki | Greece |
| DPC - Orszagos Hematologiai es Infektologiai Intezet | Recruiting | Budapest | Hungary |
| Semmelweis Egyetem | Recruiting | Budapest | Hungary |
| Semmelweis University | Recruiting | Budapest | Hungary |
| Azienda Ospedaliero Universitaria Consorziale Policlinico di Bari | Recruiting | Bari | Italy |
| Ospedale San Bassiano | Recruiting | Bassano del Grappa | Italy |
| Azienda Ospedaliera Universitaria Policlinico Sant'Orsola Malpighi IRCCS | Active, not recruiting | Bologna | Italy |
| Irccs Aou Di Bologna- Pol. Di S.Orsola | Not yet recruiting | Bologna | Italy |
| Ospedale Oncologico Armando Businco | Not yet recruiting | Cagliari | Italy |
| Azienda Ospedaliera Universitaria Careggi | Not yet recruiting | Florence | Italy |
| Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico | Recruiting | Milan | Italy |
| Ospedale San Raffaele | Recruiting | Milan | Italy |
| Ospedale Maggiore della Carità | Recruiting | Novara | Italy |
| Grande Ospedale Metropolitano - Presidio Morelli | Recruiting | Reggio Calabria | Italy |
| Fondazione Policlinico Universitario Agostino Gemelli IRCCS | Recruiting | Roma | Italy |
| Università di Roma La Sapienza | Recruiting | Rome | Italy |
| Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino - Onoclogy and Hematology Department | Recruiting | Torino | Italy |
| Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino - UO Ematologia | Not yet recruiting | Torino | Italy |
| Uniwersytecki Szpital Kliniczny w Bialymstoku | Recruiting | Bialystok | Poland |
| Szpital Uniwersytecki nr 2 im. dr J. Biziela w Bydgoszczy | Recruiting | Bydgoszcz | Poland |
| Uniwersyteckie Centrum Kliniczne | Recruiting | Gdansk | Poland |
| SPZOZ Szpital Uniwersytecki w Krakowie | Recruiting | Krakow | Poland |
| WWCOiT in Lodz, Oddział Hematologii i Transplantologii | Recruiting | Lodz | Poland |
| Uniwersytecki Szpital Kliniczny nr 1 w Lublinie | Not yet recruiting | Lublin | Poland |
| Warszawski Uniwersytet Medyczny | Not yet recruiting | Warsaw | Poland |
| Prince Faisal Bin Bandar Cancer Center | Recruiting | Buraidah | Saudi Arabia |
| King Faisal Specialist Hospital & Research Center | Not yet recruiting | Jeddah | Saudi Arabia |
| King Faisal Specialist Hospital and Research Center - Madinah | Recruiting | Riyadh | Saudi Arabia |
| King Faisal Specialist Hospital and Research Center - Riyadh | Recruiting | Riyadh | Saudi Arabia |
| ICO Badalona - Hospital Universitari Germans Trias i Pujol | Recruiting | Badalona | Spain |
| Hospital de Cruces | Recruiting | Barakaldo | Spain |
| Hospital Clinic de Barcelona | Recruiting | Barcelona | Spain |
| Hospital Universitari Vall d'Hebron | Recruiting | Barcelona | Spain |
| Hospital de Basurto | Active, not recruiting | Bilbao | Spain |
| Hospital Universitario Donostia | Recruiting | Donostia / San Sebastian | Spain |
| Hospital Universitario Virgen de la Nieves | Recruiting | El Palmar | Spain |
| Hospital Universitario Juan Ramon Jimenez | Active, not recruiting | Huelva | Spain |
| Hospital Universitario de Gran Canaria Dr. Negrin | Recruiting | Las Palmas de Gran Canaria | Spain |
| Hospital Lucus Augusti | Recruiting | Lugo | Spain |
| Hospital Regional Universitario de Malaga | Recruiting | Málaga | Spain |
| Hospital Universitario Central de Asturias | Recruiting | Oviedo | Spain |
| Hospital Universitari Son Espases | Recruiting | Palma de Mallorca | Spain |
| Complejo Hospitalario Universitario de Santiago | Recruiting | Santiago de Compostela | Spain |
| King's College Hospital | Completed | London | United Kingdom |
| ID | Term |
|---|---|
| D006457 | Hemoglobinuria, Paroxysmal |
| ID | Term |
|---|---|
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D009190 | Myelodysplastic Syndromes |
| D001855 | Bone Marrow Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C000716074 | pegcetacoplan |
Not provided
Not provided
Not provided