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NCT Number: NCT05776472

A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

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.

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Key information

Conditions

Paroxysmal Nocturnal Hemoglobinuria Anemia Anemia, Hemolytic Bone Marrow Diseases Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinuria, Paroxysmal Myelodysplastic Syndromes

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Liverpool Hospital, Liverpool, New South Wales, Australia

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Pegcetacoplan

Drug

Pegcetacoplan will be prescribed according to the label in patients with PNH.

Other names: Aspaveli, Empaveli

Primary outcomes

  1. Change in observed hemoglobin level from initiation of treatment with pegcetacoplan to 6 months

    Time frame: 6 months

    Hemoglobin level in g/dL.

Secondary outcomes

  1. Change of LDH values from initiation of pegcetacoplan treatment to 6 months

    Time frame: 6 months

    Lactate Dehydrogenase (LDH) in U/L

  2. Change in Absolute Reticulocyte Count (ARC) from initiation of pegcetacoplan treatment to 6 months

    Time frame: 6 months

    Absolute Reticulocyte Count (ARC) in 10^9/L

  3. Change in indirect/ total bilirubin from initiation of pegcetacoplan treatment to 6 months

    Time frame: 6 months

    Indirect/ total bilirubin in umol/L

  4. Change in Haptoglobin from initiation of pegcetacoplan treatment to 6 months

    Time frame: 6 months

    Haptoglobin in mg/dL

  5. Change in Ferritin from initiation of pegcetacoplan treatment to 6 months

    Time frame: 6 months

    Ferritin in ug/L

  6. Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 6 months

    Hemoglobin in g/dL

  7. Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 12 months

    Hemoglobin in g/dL

  8. Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 18 months

    Hemoglobin in g/dL

  9. Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 24 months

    Hemoglobin in g/dL

  10. Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 30 months

    Hemoglobin in g/dL

  11. Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 36 months

    Hemoglobin in g/dL

  12. Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 6 months

    Lactate Dehydrogenase (LDH) in U/L

  13. Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 12 months

    Lactate Dehydrogenase (LDH) in U/L

  14. Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 18 months

    Lactate Dehydrogenase (LDH) in U/L

  15. Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 24 months

    Lactate Dehydrogenase (LDH) in U/L

  16. Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 30 months

    Lactate Dehydrogenase (LDH) in U/L

  17. Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 36 months

    Lactate Dehydrogenase (LDH) in U/L

  18. Absolute Reticulocyte Count at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 6 months

    Absolute Reticulocyte Count (ARC) in 10^9/L

  19. Absolute Reticulocyte Count at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 12 months

    Absolute Reticulocyte Count (ARC) in 10^9/L

  20. Absolute Reticulocyte Count at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 18 months

    Absolute Reticulocyte Count (ARC) in 10^9/L

  21. Absolute Reticulocyte Count at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 24 months

    Absolute Reticulocyte Count (ARC) in 10^9/L

  22. Absolute Reticulocyte Count at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 30 months

    Absolute Reticulocyte Count (ARC) in 10^9/L

  23. Absolute Reticulocyte Count at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 36 months

    Absolute Reticulocyte Count (ARC) in 10^9/L

  24. Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 6 months

    Indirect/ total bilirubin in umol/L

  25. Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 12 months

    Indirect/ total bilirubin in umol/L

  26. Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 18 months

    Indirect/ total bilirubin in umol/L

  27. Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 24 months

    Indirect/ total bilirubin in umol/L

  28. Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 30 months

    Indirect/ total bilirubin in umol/L

  29. Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 36 months

    Indirect/ total bilirubin in umol/L

  30. Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 6 months

    Haptoglobin in µmol/L

  31. Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 12 months

    Haptoglobin in µmol/L

  32. Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 18 months

    Haptoglobin in µmol/L

  33. Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 24 months

    Haptoglobin in µmol/L

  34. Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 30 months

    Haptoglobin in µmol/L

  35. Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 36 months

    Haptoglobin in mg/dL

  36. Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 6 months

    Ferritin in ug/L

  37. Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 12 months

    Ferritin in ug/L

  38. Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 18 months

    Ferritin in ug/L

  39. Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 24 months

    Ferritin in ug/L

  40. Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 30 months

    Ferritin in ug/L

  41. Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 36 months

    Ferritin in ug/L

  42. Hemoglobin ≥ 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 6 months

    Hemoglobin in g/dL

  43. Hemoglobin ≥ 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 12 months

    Hemoglobin in g/dL

  44. Hemoglobin ≥ 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 18 months

    Hemoglobin in g/dL

  45. Hemoglobin ≥ 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 24 months

    Hemoglobin in g/dL

  46. Hemoglobin ≥ 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 30 months

    Hemoglobin in g/dL

  47. Hemoglobin ≥ 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 36 months

    Hemoglobin in g/dL

  48. Increase in hemoglobin levels of ≥ 2 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 6 months

    Hemoglobin in g/dL

  49. Increase in hemoglobin levels of ≥ 2 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 12 months

    Hemoglobin in g/dL

  50. Increase in hemoglobin levels of ≥ 2 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 18 months

    Hemoglobin in g/dL

  51. Increase in hemoglobin levels of ≥ 2 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 24 months

    Hemoglobin in g/dL

  52. Increase in hemoglobin levels of ≥ 2 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 30 months

    Hemoglobin in g/dL

  53. Increase in hemoglobin levels of ≥ 2 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 36 months

    Hemoglobin in g/dL

  54. Hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 6 months

    Yes/No

  55. Hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 12 months

    Yes/No

  56. Hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 18 months

    Yes/No

  57. Hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 24 months

    Yes/No

  58. Hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 30 months

    Yes/No

  59. Hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of follow-up

    Time frame: 36 months

    Yes/No

  60. 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

    Time frame: 12 months

    Total number of RBC transfusions

  61. 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

    Time frame: 12 months

    Total number of RBC units

  62. Fatigue at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 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.

  63. Fatigue at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 12 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.

  64. Fatigue at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 18 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.

  65. Fatigue at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 24 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.

  66. Fatigue at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 30 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.

  67. Fatigue at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 36 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.

  68. 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.

    Time frame: 12 months

    Number of hospitalizations and emergency room visits

  69. Patient treatment satisfaction every 6 months until end of follow-up

    Time frame: 6 months

    Patient treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied

  70. Patient treatment satisfaction every 6 months until end of follow-up

    Time frame: 12 months

    Patient treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied

  71. Patient treatment satisfaction every 6 months until end of follow-up

    Time frame: 18 months

    Patient treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied

  72. Patient treatment satisfaction every 6 months until end of follow-up

    Time frame: 24 months

    Patient treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied

  73. Patient treatment satisfaction every 6 months until end of follow-up

    Time frame: 30 months

    Patient treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied

  74. Patient treatment satisfaction every 6 months until end of follow-up

    Time frame: 36 months

    Patient treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied

  75. Physician treatment satisfaction every 6 months until end of follow-up

    Time frame: 6 months

    Physician treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied

  76. Physician treatment satisfaction every 6 months until end of follow-up

    Time frame: 12 months

    Physician treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied

  77. Physician treatment satisfaction every 6 months until end of follow-up

    Time frame: 18 months

    Physician treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied

  78. Physician treatment satisfaction every 6 months until end of follow-up

    Time frame: 24 months

    Physician treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied

  79. Physician treatment satisfaction every 6 months until end of follow-up

    Time frame: 30 months

    Physician treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied

  80. Physician treatment satisfaction every 6 months until end of follow-up

    Time frame: 36 months

    Physician treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied

  81. Adverse events (AE), including serious adverse events (SAE)

    Time frame: 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.

  82. Alanine transaminase (ALT) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 6 months

    Alanine transaminase (ALT) in U/L

  83. Alanine transaminase (ALT) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 12 months

    Alanine transaminase (ALT) in U/L

  84. Alanine transaminase (ALT) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 18 months

    Alanine transaminase (ALT) in U/L

  85. Alanine transaminase (ALT) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 24 months

    Alanine transaminase (ALT) in U/L

  86. Alanine transaminase (ALT) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 30 months

    Alanine transaminase (ALT) in U/L

  87. Alanine transaminase (ALT) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 36 months

    Alanine transaminase (ALT) in U/L

  88. Aspartate aminotransferase (AST) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 6 months

    Aspartate aminotransferase (AST) in U/L

  89. Aspartate aminotransferase (AST) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 12 months

    Aspartate aminotransferase (AST) in U/L

  90. Aspartate aminotransferase (AST) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 18 months

    Aspartate aminotransferase (AST) in U/L

  91. Aspartate aminotransferase (AST) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 24 months

    Aspartate aminotransferase (AST) in U/L

  92. Aspartate aminotransferase (AST) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 30 months

    Aspartate aminotransferase (AST) in U/L

  93. Aspartate aminotransferase (AST) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 36 months

    Aspartate aminotransferase (AST) in U/L

  94. Alkaline Phosphatase at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 6 months

    Alkaline Phosphatase in µkat/L

  95. Alkaline Phosphatase at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 12 months

    Alkaline Phosphatase in µkat/L

  96. Alkaline Phosphatase at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 18 months

    Alkaline Phosphatase in µkat/L

  97. Alkaline Phosphatase at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 24 months

    Alkaline Phosphatase in µkat/L

  98. Alkaline Phosphatase at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 30 months

    Alkaline Phosphatase in µkat/L

  99. Alkaline Phosphatase at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 36 months

    Alkaline Phosphatase in µkat/L

  100. Bilirubin at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 6 months

    Indirect/ total bilirubin in umol/L

  101. Bilirubin at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 12 months

    Indirect/ total bilirubin in umol/L

  102. Bilirubin at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 18 months

    Indirect/ total bilirubin in umol/L

  103. Bilirubin at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 24 months

    Indirect/ total bilirubin in umol/L

  104. Bilirubin at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 30 months

    Indirect/ total bilirubin in umol/L

  105. Bilirubin at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 36 months

    Indirect/ total bilirubin in umol/L

  106. Creatinine at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 6 months

    Creatinine in mg/dL

  107. Creatinine at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 12 months

    Creatinine in mg/dL

  108. Creatinine at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 18 months

    Creatinine in mg/dL

  109. Creatinine at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 24 months

    Creatinine in mg/dL

  110. Creatinine at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 30 months

    Creatinine in mg/dL

  111. Creatinine at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 36 months

    Creatinine in mg/dL

  112. estimated glomerular filtration rate (eGFR) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 6 months

    estimated glomerular filtration rate (eGFR) in mL/min/1.73 m²

  113. estimated glomerular filtration rate (eGFR) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 12 months

    estimated glomerular filtration rate (eGFR) in mL/min/1.73 m²

  114. estimated glomerular filtration rate (eGFR) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 18 months

    estimated glomerular filtration rate (eGFR) in mL/min/1.73 m²

  115. estimated glomerular filtration rate (eGFR) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 24 months

    estimated glomerular filtration rate (eGFR) in mL/min/1.73 m²

  116. estimated glomerular filtration rate (eGFR) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 30 months

    estimated glomerular filtration rate (eGFR) in mL/min/1.73 m²

  117. estimated glomerular filtration rate (eGFR) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 36 months

    estimated glomerular filtration rate (eGFR) in mL/min/1.73 m²

  118. Urea at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 6 months

    Urea in mg/dL

  119. Urea at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 12 months

    Urea in mg/dL

  120. Urea at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 18 months

    Urea in mg/dL

  121. Urea at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 24 months

    Urea in mg/dL

  122. Urea at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 30 months

    Urea in mg/dL

  123. Urea at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 36 months

    Urea in mg/dL

  124. Potassium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 6 months

    Potassium in mmol/L

  125. Potassium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 12 months

    Potassium in mmol/L

  126. Potassium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 18 months

    Potassium in mmol/L

  127. Potassium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 24 months

    Potassium in mmol/L

  128. Potassium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 30 months

    Potassium in mmol/L

  129. Potassium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 36 months

    Potassium in mmol/L

  130. Sodium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 6 months

    Sodium in mmol/L

  131. Sodium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 12 months

    Sodium in mmol/L

  132. Sodium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 18 months

    Sodium in mmol/L

  133. Sodium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 24 months

    Sodium in mmol/L

  134. Sodium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 30 months

    Sodium in mmol/L

  135. Sodium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up

    Time frame: 36 months

    Sodium in mmol/L

Study contacts

Contact information is provided by the study sponsor or research team.

Clinical Program Lead

CONTACT

[email protected]

+46 (0)8 697 20 00

Sponsors and collaborators

Lead sponsor

Swedish Orphan Biovitrum

Industry

Collaborators

  • IQVIA Pty Ltd

Registry information

Official study title

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

Acronym: COMPLETE

Important dates

Study start
2023
Primary completion
2029
Study completion
2029
First posted
Mar 20, 2023
Registry last updated
Jul 1, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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