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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT05776472
Registration number
NCT05776472
Ethics application status
Date submitted
20/02/2023
Date registered
20/03/2023
Date last updated
14/05/2025
Titles & IDs
Public title
A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
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Scientific 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)
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Secondary ID [1]
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0
Sobi.PEGCET-304
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Universal Trial Number (UTN)
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Trial acronym
COMPLETE
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Paroxysmal Nocturnal Hemoglobinuria
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0
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Condition category
Condition code
Blood
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0
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0
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Haematological diseases
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Intervention/exposure
Study type
Observational
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Patient registry
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Target follow-up duration
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Target follow-up type
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Description of intervention(s) / exposure
Treatment: Drugs - Pegcetacoplan
Treatment: Drugs: Pegcetacoplan
Pegcetacoplan will be prescribed according to the label in patients with PNH.
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Intervention code [1]
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0
Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Change in observed hemoglobin level from initiation of treatment with pegcetacoplan to 6 months
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Assessment method [1]
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Hemoglobin level in g/dL.
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Timepoint [1]
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0
6 months
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Secondary outcome [1]
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0
Change of LDH values from initiation of pegcetacoplan treatment to 6 months
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Assessment method [1]
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0
Lactate Dehydrogenase (LDH) in U/L
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Timepoint [1]
0
0
6 months
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Secondary outcome [2]
0
0
Change in Absolute Reticulocyte Count (ARC) from initiation of pegcetacoplan treatment to 6 months
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Assessment method [2]
0
0
Absolute Reticulocyte Count (ARC) in 10\^9/L
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Timepoint [2]
0
0
6 months
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Secondary outcome [3]
0
0
Change in indirect/ total bilirubin from initiation of pegcetacoplan treatment to 6 months
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Assessment method [3]
0
0
Indirect/ total bilirubin in umol/L
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Timepoint [3]
0
0
6 months
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Secondary outcome [4]
0
0
Change in Haptoglobin from initiation of pegcetacoplan treatment to 6 months
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Assessment method [4]
0
0
Haptoglobin in mg/dL
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Timepoint [4]
0
0
6 months
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Secondary outcome [5]
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0
Change in Ferritin from initiation of pegcetacoplan treatment to 6 months
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Assessment method [5]
0
0
Ferritin in ug/L
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Timepoint [5]
0
0
6 months
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Secondary outcome [6]
0
0
Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
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Assessment method [6]
0
0
Hemoglobin in g/dL
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Timepoint [6]
0
0
6 months
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Secondary outcome [7]
0
0
Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
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Assessment method [7]
0
0
Hemoglobin in g/dL
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Timepoint [7]
0
0
12 months
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Secondary outcome [8]
0
0
Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
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Assessment method [8]
0
0
Hemoglobin in g/dL
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Timepoint [8]
0
0
18 months
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Secondary outcome [9]
0
0
Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
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Assessment method [9]
0
0
Hemoglobin in g/dL
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Timepoint [9]
0
0
24 months
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Secondary outcome [10]
0
0
Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
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Assessment method [10]
0
0
Hemoglobin in g/dL
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Timepoint [10]
0
0
30 months
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Secondary outcome [11]
0
0
Hemoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
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Assessment method [11]
0
0
Hemoglobin in g/dL
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Timepoint [11]
0
0
36 months
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Secondary outcome [12]
0
0
Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
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Assessment method [12]
0
0
Lactate Dehydrogenase (LDH) in U/L
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Timepoint [12]
0
0
6 months
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Secondary outcome [13]
0
0
Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
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Assessment method [13]
0
0
Lactate Dehydrogenase (LDH) in U/L
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Timepoint [13]
0
0
12 months
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Secondary outcome [14]
0
0
Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
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Assessment method [14]
0
0
Lactate Dehydrogenase (LDH) in U/L
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Timepoint [14]
0
0
18 months
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Secondary outcome [15]
0
0
Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
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Assessment method [15]
0
0
Lactate Dehydrogenase (LDH) in U/L
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Timepoint [15]
0
0
24 months
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Secondary outcome [16]
0
0
Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
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Assessment method [16]
0
0
Lactate Dehydrogenase (LDH) in U/L
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Timepoint [16]
0
0
30 months
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Secondary outcome [17]
0
0
Lactate Dehydrogenase at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
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Assessment method [17]
0
0
Lactate Dehydrogenase (LDH) in U/L
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Timepoint [17]
0
0
36 months
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Secondary outcome [18]
0
0
Absolute Reticulocyte Count at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
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Assessment method [18]
0
0
Absolute Reticulocyte Count (ARC) in 10\^9/L
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Timepoint [18]
0
0
6 months
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Secondary outcome [19]
0
0
Absolute Reticulocyte Count at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
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Assessment method [19]
0
0
Absolute Reticulocyte Count (ARC) in 10\^9/L
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Timepoint [19]
0
0
12 months
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Secondary outcome [20]
0
0
Absolute Reticulocyte Count at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
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Assessment method [20]
0
0
Absolute Reticulocyte Count (ARC) in 10\^9/L
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Timepoint [20]
0
0
18 months
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Secondary outcome [21]
0
0
Absolute Reticulocyte Count at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
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Assessment method [21]
0
0
Absolute Reticulocyte Count (ARC) in 10\^9/L
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Timepoint [21]
0
0
24 months
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Secondary outcome [22]
0
0
Absolute Reticulocyte Count at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
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Assessment method [22]
0
0
Absolute Reticulocyte Count (ARC) in 10\^9/L
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Timepoint [22]
0
0
30 months
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Secondary outcome [23]
0
0
Absolute Reticulocyte Count at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
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Assessment method [23]
0
0
Absolute Reticulocyte Count (ARC) in 10\^9/L
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Timepoint [23]
0
0
36 months
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Secondary outcome [24]
0
0
Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
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Assessment method [24]
0
0
Indirect/ total bilirubin in umol/L
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Timepoint [24]
0
0
6 months
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Secondary outcome [25]
0
0
Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
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Assessment method [25]
0
0
Indirect/ total bilirubin in umol/L
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Timepoint [25]
0
0
12 months
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Secondary outcome [26]
0
0
Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
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Assessment method [26]
0
0
Indirect/ total bilirubin in umol/L
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Timepoint [26]
0
0
18 months
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Secondary outcome [27]
0
0
Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
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Assessment method [27]
0
0
Indirect/ total bilirubin in umol/L
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Timepoint [27]
0
0
24 months
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Secondary outcome [28]
0
0
Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
Query!
Assessment method [28]
0
0
Indirect/ total bilirubin in umol/L
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Timepoint [28]
0
0
30 months
Query!
Secondary outcome [29]
0
0
Indirect/ total bilirubin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
Query!
Assessment method [29]
0
0
Indirect/ total bilirubin in umol/L
Query!
Timepoint [29]
0
0
36 months
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Secondary outcome [30]
0
0
Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
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Assessment method [30]
0
0
Haptoglobin in µmol/L
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Timepoint [30]
0
0
6 months
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Secondary outcome [31]
0
0
Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
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Assessment method [31]
0
0
Haptoglobin in µmol/L
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Timepoint [31]
0
0
12 months
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Secondary outcome [32]
0
0
Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
Query!
Assessment method [32]
0
0
Haptoglobin in µmol/L
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Timepoint [32]
0
0
18 months
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Secondary outcome [33]
0
0
Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
Query!
Assessment method [33]
0
0
Haptoglobin in µmol/L
Query!
Timepoint [33]
0
0
24 months
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Secondary outcome [34]
0
0
Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
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Assessment method [34]
0
0
Haptoglobin in µmol/L
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Timepoint [34]
0
0
30 months
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Secondary outcome [35]
0
0
Haptoglobin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
Query!
Assessment method [35]
0
0
Haptoglobin in mg/dL
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Timepoint [35]
0
0
36 months
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Secondary outcome [36]
0
0
Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
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Assessment method [36]
0
0
Ferritin in ug/L
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Timepoint [36]
0
0
6 months
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Secondary outcome [37]
0
0
Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
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Assessment method [37]
0
0
Ferritin in ug/L
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Timepoint [37]
0
0
12 months
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Secondary outcome [38]
0
0
Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
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Assessment method [38]
0
0
Ferritin in ug/L
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Timepoint [38]
0
0
18 months
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Secondary outcome [39]
0
0
Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
Query!
Assessment method [39]
0
0
Ferritin in ug/L
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Timepoint [39]
0
0
24 months
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Secondary outcome [40]
0
0
Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
Query!
Assessment method [40]
0
0
Ferritin in ug/L
Query!
Timepoint [40]
0
0
30 months
Query!
Secondary outcome [41]
0
0
Ferritin at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
Query!
Assessment method [41]
0
0
Ferritin in ug/L
Query!
Timepoint [41]
0
0
36 months
Query!
Secondary outcome [42]
0
0
Hemoglobin = 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
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Assessment method [42]
0
0
Hemoglobin in g/dL
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Timepoint [42]
0
0
6 months
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Secondary outcome [43]
0
0
Hemoglobin = 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
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Assessment method [43]
0
0
Hemoglobin in g/dL
Query!
Timepoint [43]
0
0
12 months
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Secondary outcome [44]
0
0
Hemoglobin = 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
Query!
Assessment method [44]
0
0
Hemoglobin in g/dL
Query!
Timepoint [44]
0
0
18 months
Query!
Secondary outcome [45]
0
0
Hemoglobin = 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
Query!
Assessment method [45]
0
0
Hemoglobin in g/dL
Query!
Timepoint [45]
0
0
24 months
Query!
Secondary outcome [46]
0
0
Hemoglobin = 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
Query!
Assessment method [46]
0
0
Hemoglobin in g/dL
Query!
Timepoint [46]
0
0
30 months
Query!
Secondary outcome [47]
0
0
Hemoglobin = 12 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
Query!
Assessment method [47]
0
0
Hemoglobin in g/dL
Query!
Timepoint [47]
0
0
36 months
Query!
Secondary outcome [48]
0
0
Increase in hemoglobin levels of = 2 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
Query!
Assessment method [48]
0
0
Hemoglobin in g/dL
Query!
Timepoint [48]
0
0
6 months
Query!
Secondary outcome [49]
0
0
Increase in hemoglobin levels of = 2 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
Query!
Assessment method [49]
0
0
Hemoglobin in g/dL
Query!
Timepoint [49]
0
0
12 months
Query!
Secondary outcome [50]
0
0
Increase in hemoglobin levels of = 2 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
Query!
Assessment method [50]
0
0
Hemoglobin in g/dL
Query!
Timepoint [50]
0
0
18 months
Query!
Secondary outcome [51]
0
0
Increase in hemoglobin levels of = 2 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
Query!
Assessment method [51]
0
0
Hemoglobin in g/dL
Query!
Timepoint [51]
0
0
24 months
Query!
Secondary outcome [52]
0
0
Increase in hemoglobin levels of = 2 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
Query!
Assessment method [52]
0
0
Hemoglobin in g/dL
Query!
Timepoint [52]
0
0
30 months
Query!
Secondary outcome [53]
0
0
Increase in hemoglobin levels of = 2 g/dL at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
Query!
Assessment method [53]
0
0
Hemoglobin in g/dL
Query!
Timepoint [53]
0
0
36 months
Query!
Secondary outcome [54]
0
0
Hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
Query!
Assessment method [54]
0
0
Yes/No
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Timepoint [54]
0
0
6 months
Query!
Secondary outcome [55]
0
0
Hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
Query!
Assessment method [55]
0
0
Yes/No
Query!
Timepoint [55]
0
0
12 months
Query!
Secondary outcome [56]
0
0
Hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
Query!
Assessment method [56]
0
0
Yes/No
Query!
Timepoint [56]
0
0
18 months
Query!
Secondary outcome [57]
0
0
Hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
Query!
Assessment method [57]
0
0
Yes/No
Query!
Timepoint [57]
0
0
24 months
Query!
Secondary outcome [58]
0
0
Hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
Query!
Assessment method [58]
0
0
Yes/No
Query!
Timepoint [58]
0
0
30 months
Query!
Secondary outcome [59]
0
0
Hemolytic event requiring additional intervention at initiation of pegcetacoplan treatment and each 6 months until end of follow-up
Query!
Assessment method [59]
0
0
Yes/No
Query!
Timepoint [59]
0
0
36 months
Query!
Secondary outcome [60]
0
0
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
Query!
Assessment method [60]
0
0
Total number of RBC transfusions
Query!
Timepoint [60]
0
0
12 months
Query!
Secondary outcome [61]
0
0
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
Query!
Assessment method [61]
0
0
Total number of RBC units
Query!
Timepoint [61]
0
0
12 months
Query!
Secondary outcome [62]
0
0
Fatigue at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [62]
0
0
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.
Query!
Timepoint [62]
0
0
6 months
Query!
Secondary outcome [63]
0
0
Fatigue at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [63]
0
0
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.
Query!
Timepoint [63]
0
0
12 months
Query!
Secondary outcome [64]
0
0
Fatigue at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [64]
0
0
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.
Query!
Timepoint [64]
0
0
18 months
Query!
Secondary outcome [65]
0
0
Fatigue at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [65]
0
0
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.
Query!
Timepoint [65]
0
0
24 months
Query!
Secondary outcome [66]
0
0
Fatigue at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [66]
0
0
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.
Query!
Timepoint [66]
0
0
30 months
Query!
Secondary outcome [67]
0
0
Fatigue at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [67]
0
0
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.
Query!
Timepoint [67]
0
0
36 months
Query!
Secondary outcome [68]
0
0
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.
Query!
Assessment method [68]
0
0
Number of hospitalizations and emergency room visits
Query!
Timepoint [68]
0
0
12 months
Query!
Secondary outcome [69]
0
0
Patient treatment satisfaction every 6 months until end of follow-up
Query!
Assessment method [69]
0
0
Patient treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied
Query!
Timepoint [69]
0
0
6 months
Query!
Secondary outcome [70]
0
0
Patient treatment satisfaction every 6 months until end of follow-up
Query!
Assessment method [70]
0
0
Patient treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied
Query!
Timepoint [70]
0
0
12 months
Query!
Secondary outcome [71]
0
0
Patient treatment satisfaction every 6 months until end of follow-up
Query!
Assessment method [71]
0
0
Patient treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied
Query!
Timepoint [71]
0
0
18 months
Query!
Secondary outcome [72]
0
0
Patient treatment satisfaction every 6 months until end of follow-up
Query!
Assessment method [72]
0
0
Patient treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied
Query!
Timepoint [72]
0
0
24 months
Query!
Secondary outcome [73]
0
0
Patient treatment satisfaction every 6 months until end of follow-up
Query!
Assessment method [73]
0
0
Patient treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied
Query!
Timepoint [73]
0
0
30 months
Query!
Secondary outcome [74]
0
0
Patient treatment satisfaction every 6 months until end of follow-up
Query!
Assessment method [74]
0
0
Patient treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied
Query!
Timepoint [74]
0
0
36 months
Query!
Secondary outcome [75]
0
0
Physician treatment satisfaction every 6 months until end of follow-up
Query!
Assessment method [75]
0
0
Physician treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied
Query!
Timepoint [75]
0
0
6 months
Query!
Secondary outcome [76]
0
0
Physician treatment satisfaction every 6 months until end of follow-up
Query!
Assessment method [76]
0
0
Physician treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied
Query!
Timepoint [76]
0
0
12 months
Query!
Secondary outcome [77]
0
0
Physician treatment satisfaction every 6 months until end of follow-up
Query!
Assessment method [77]
0
0
Physician treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied
Query!
Timepoint [77]
0
0
18 months
Query!
Secondary outcome [78]
0
0
Physician treatment satisfaction every 6 months until end of follow-up
Query!
Assessment method [78]
0
0
Physician treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied
Query!
Timepoint [78]
0
0
24 months
Query!
Secondary outcome [79]
0
0
Physician treatment satisfaction every 6 months until end of follow-up
Query!
Assessment method [79]
0
0
Physician treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied
Query!
Timepoint [79]
0
0
30 months
Query!
Secondary outcome [80]
0
0
Physician treatment satisfaction every 6 months until end of follow-up
Query!
Assessment method [80]
0
0
Physician treatment satisfaction (1-5 point scale), 5=highly satisfied, 1= highly dissatisfied
Query!
Timepoint [80]
0
0
36 months
Query!
Secondary outcome [81]
0
0
Adverse events (AE), including serious adverse events (SAE)
Query!
Assessment method [81]
0
0
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.
Query!
Timepoint [81]
0
0
24 hours
Query!
Secondary outcome [82]
0
0
Alanine transaminase (ALT) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [82]
0
0
Alanine transaminase (ALT) in U/L
Query!
Timepoint [82]
0
0
6 months
Query!
Secondary outcome [83]
0
0
Alanine transaminase (ALT) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [83]
0
0
Alanine transaminase (ALT) in U/L
Query!
Timepoint [83]
0
0
12 months
Query!
Secondary outcome [84]
0
0
Alanine transaminase (ALT) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [84]
0
0
Alanine transaminase (ALT) in U/L
Query!
Timepoint [84]
0
0
18 months
Query!
Secondary outcome [85]
0
0
Alanine transaminase (ALT) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [85]
0
0
Alanine transaminase (ALT) in U/L
Query!
Timepoint [85]
0
0
24 months
Query!
Secondary outcome [86]
0
0
Alanine transaminase (ALT) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [86]
0
0
Alanine transaminase (ALT) in U/L
Query!
Timepoint [86]
0
0
30 months
Query!
Secondary outcome [87]
0
0
Alanine transaminase (ALT) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [87]
0
0
Alanine transaminase (ALT) in U/L
Query!
Timepoint [87]
0
0
36 months
Query!
Secondary outcome [88]
0
0
Aspartate aminotransferase (AST) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [88]
0
0
Aspartate aminotransferase (AST) in U/L
Query!
Timepoint [88]
0
0
6 months
Query!
Secondary outcome [89]
0
0
Aspartate aminotransferase (AST) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [89]
0
0
Aspartate aminotransferase (AST) in U/L
Query!
Timepoint [89]
0
0
12 months
Query!
Secondary outcome [90]
0
0
Aspartate aminotransferase (AST) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [90]
0
0
Aspartate aminotransferase (AST) in U/L
Query!
Timepoint [90]
0
0
18 months
Query!
Secondary outcome [91]
0
0
Aspartate aminotransferase (AST) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [91]
0
0
Aspartate aminotransferase (AST) in U/L
Query!
Timepoint [91]
0
0
24 months
Query!
Secondary outcome [92]
0
0
Aspartate aminotransferase (AST) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [92]
0
0
Aspartate aminotransferase (AST) in U/L
Query!
Timepoint [92]
0
0
30 months
Query!
Secondary outcome [93]
0
0
Aspartate aminotransferase (AST) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [93]
0
0
Aspartate aminotransferase (AST) in U/L
Query!
Timepoint [93]
0
0
36 months
Query!
Secondary outcome [94]
0
0
Alkaline Phosphatase at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [94]
0
0
Alkaline Phosphatase in µkat/L
Query!
Timepoint [94]
0
0
6 months
Query!
Secondary outcome [95]
0
0
Alkaline Phosphatase at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [95]
0
0
Alkaline Phosphatase in µkat/L
Query!
Timepoint [95]
0
0
12 months
Query!
Secondary outcome [96]
0
0
Alkaline Phosphatase at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [96]
0
0
Alkaline Phosphatase in µkat/L
Query!
Timepoint [96]
0
0
18 months
Query!
Secondary outcome [97]
0
0
Alkaline Phosphatase at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [97]
0
0
Alkaline Phosphatase in µkat/L
Query!
Timepoint [97]
0
0
24 months
Query!
Secondary outcome [98]
0
0
Alkaline Phosphatase at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [98]
0
0
Alkaline Phosphatase in µkat/L
Query!
Timepoint [98]
0
0
30 months
Query!
Secondary outcome [99]
0
0
Alkaline Phosphatase at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [99]
0
0
Alkaline Phosphatase in µkat/L
Query!
Timepoint [99]
0
0
36 months
Query!
Secondary outcome [100]
0
0
Bilirubin at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [100]
0
0
Indirect/ total bilirubin in umol/L
Query!
Timepoint [100]
0
0
6 months
Query!
Secondary outcome [101]
0
0
Bilirubin at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [101]
0
0
Indirect/ total bilirubin in umol/L
Query!
Timepoint [101]
0
0
12 months
Query!
Secondary outcome [102]
0
0
Bilirubin at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [102]
0
0
Indirect/ total bilirubin in umol/L
Query!
Timepoint [102]
0
0
18 months
Query!
Secondary outcome [103]
0
0
Bilirubin at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [103]
0
0
Indirect/ total bilirubin in umol/L
Query!
Timepoint [103]
0
0
24 months
Query!
Secondary outcome [104]
0
0
Bilirubin at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [104]
0
0
Indirect/ total bilirubin in umol/L
Query!
Timepoint [104]
0
0
30 months
Query!
Secondary outcome [105]
0
0
Bilirubin at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [105]
0
0
Indirect/ total bilirubin in umol/L
Query!
Timepoint [105]
0
0
36 months
Query!
Secondary outcome [106]
0
0
Creatinine at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [106]
0
0
Creatinine in mg/dL
Query!
Timepoint [106]
0
0
6 months
Query!
Secondary outcome [107]
0
0
Creatinine at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [107]
0
0
Creatinine in mg/dL
Query!
Timepoint [107]
0
0
12 months
Query!
Secondary outcome [108]
0
0
Creatinine at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [108]
0
0
Creatinine in mg/dL
Query!
Timepoint [108]
0
0
18 months
Query!
Secondary outcome [109]
0
0
Creatinine at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [109]
0
0
Creatinine in mg/dL
Query!
Timepoint [109]
0
0
24 months
Query!
Secondary outcome [110]
0
0
Creatinine at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [110]
0
0
Creatinine in mg/dL
Query!
Timepoint [110]
0
0
30 months
Query!
Secondary outcome [111]
0
0
Creatinine at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [111]
0
0
Creatinine in mg/dL
Query!
Timepoint [111]
0
0
36 months
Query!
Secondary outcome [112]
0
0
estimated glomerular filtration rate (eGFR) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [112]
0
0
estimated glomerular filtration rate (eGFR) in mL/min/1.73 m²
Query!
Timepoint [112]
0
0
6 months
Query!
Secondary outcome [113]
0
0
estimated glomerular filtration rate (eGFR) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [113]
0
0
estimated glomerular filtration rate (eGFR) in mL/min/1.73 m²
Query!
Timepoint [113]
0
0
12 months
Query!
Secondary outcome [114]
0
0
estimated glomerular filtration rate (eGFR) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [114]
0
0
estimated glomerular filtration rate (eGFR) in mL/min/1.73 m²
Query!
Timepoint [114]
0
0
18 months
Query!
Secondary outcome [115]
0
0
estimated glomerular filtration rate (eGFR) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [115]
0
0
estimated glomerular filtration rate (eGFR) in mL/min/1.73 m²
Query!
Timepoint [115]
0
0
24 months
Query!
Secondary outcome [116]
0
0
estimated glomerular filtration rate (eGFR) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [116]
0
0
estimated glomerular filtration rate (eGFR) in mL/min/1.73 m²
Query!
Timepoint [116]
0
0
30 months
Query!
Secondary outcome [117]
0
0
estimated glomerular filtration rate (eGFR) at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [117]
0
0
estimated glomerular filtration rate (eGFR) in mL/min/1.73 m²
Query!
Timepoint [117]
0
0
36 months
Query!
Secondary outcome [118]
0
0
Urea at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [118]
0
0
Urea in mg/dL
Query!
Timepoint [118]
0
0
6 months
Query!
Secondary outcome [119]
0
0
Urea at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [119]
0
0
Urea in mg/dL
Query!
Timepoint [119]
0
0
12 months
Query!
Secondary outcome [120]
0
0
Urea at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [120]
0
0
Urea in mg/dL
Query!
Timepoint [120]
0
0
18 months
Query!
Secondary outcome [121]
0
0
Urea at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [121]
0
0
Urea in mg/dL
Query!
Timepoint [121]
0
0
24 months
Query!
Secondary outcome [122]
0
0
Urea at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [122]
0
0
Urea in mg/dL
Query!
Timepoint [122]
0
0
30 months
Query!
Secondary outcome [123]
0
0
Urea at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [123]
0
0
Urea in mg/dL
Query!
Timepoint [123]
0
0
36 months
Query!
Secondary outcome [124]
0
0
Potassium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [124]
0
0
Potassium in mmol/L
Query!
Timepoint [124]
0
0
6 months
Query!
Secondary outcome [125]
0
0
Potassium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [125]
0
0
Potassium in mmol/L
Query!
Timepoint [125]
0
0
12 months
Query!
Secondary outcome [126]
0
0
Potassium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [126]
0
0
Potassium in mmol/L
Query!
Timepoint [126]
0
0
18 months
Query!
Secondary outcome [127]
0
0
Potassium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [127]
0
0
Potassium in mmol/L
Query!
Timepoint [127]
0
0
24 months
Query!
Secondary outcome [128]
0
0
Potassium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [128]
0
0
Potassium in mmol/L
Query!
Timepoint [128]
0
0
30 months
Query!
Secondary outcome [129]
0
0
Potassium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [129]
0
0
Potassium in mmol/L
Query!
Timepoint [129]
0
0
36 months
Query!
Secondary outcome [130]
0
0
Sodium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [130]
0
0
Sodium in mmol/L
Query!
Timepoint [130]
0
0
6 months
Query!
Secondary outcome [131]
0
0
Sodium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [131]
0
0
Sodium in mmol/L
Query!
Timepoint [131]
0
0
12 months
Query!
Secondary outcome [132]
0
0
Sodium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [132]
0
0
Sodium in mmol/L
Query!
Timepoint [132]
0
0
18 months
Query!
Secondary outcome [133]
0
0
Sodium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [133]
0
0
Sodium in mmol/L
Query!
Timepoint [133]
0
0
24 months
Query!
Secondary outcome [134]
0
0
Sodium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [134]
0
0
Sodium in mmol/L
Query!
Timepoint [134]
0
0
30 months
Query!
Secondary outcome [135]
0
0
Sodium at initiation of treatment with pegcetacoplan and every 6 months until end of follow-up
Query!
Assessment method [135]
0
0
Sodium in mmol/L
Query!
Timepoint [135]
0
0
36 months
Query!
Eligibility
Key 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.
Query!
Minimum age
18
Years
Query!
Query!
Maximum age
No limit
Query!
Query!
Sex
Both males and females
Query!
Can healthy volunteers participate?
No
Query!
Key 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.
Query!
Study design
Purpose
Query!
Duration
Query!
Selection
Query!
Timing
Query!
Statistical methods / analysis
Query!
Recruitment
Recruitment status
Recruiting
Query!
Data analysis
Query!
Reason for early stopping/withdrawal
Query!
Other reasons
Query!
Date of first participant enrolment
Anticipated
Query!
Actual
26/06/2023
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
15/08/2029
Query!
Actual
Query!
Sample size
Target
200
Query!
Accrual to date
Query!
Final
Query!
Recruitment in Australia
Recruitment state(s)
NSW,QLD
Query!
Recruitment hospital [1]
0
0
Liverpool Hospital - Liverpool
Query!
Recruitment hospital [2]
0
0
Calvary Mater Newcastle - Waratah
Query!
Recruitment hospital [3]
0
0
Sunshine Coast University Hospital - Birtinya
Query!
Recruitment postcode(s) [1]
0
0
- Liverpool
Query!
Recruitment postcode(s) [2]
0
0
- Waratah
Query!
Recruitment postcode(s) [3]
0
0
- Birtinya
Query!
Recruitment outside Australia
Country [1]
0
0
Belgium
Query!
State/province [1]
0
0
Brugge
Query!
Country [2]
0
0
Belgium
Query!
State/province [2]
0
0
Bruxelles
Query!
Country [3]
0
0
Belgium
Query!
State/province [3]
0
0
Liège
Query!
Country [4]
0
0
Belgium
Query!
State/province [4]
0
0
Turnhout
Query!
Country [5]
0
0
Canada
Query!
State/province [5]
0
0
Ontario
Query!
Country [6]
0
0
Canada
Query!
State/province [6]
0
0
Quebec
Query!
Country [7]
0
0
Czechia
Query!
State/province [7]
0
0
Brno
Query!
Country [8]
0
0
Czechia
Query!
State/province [8]
0
0
Praha 2
Query!
Country [9]
0
0
Finland
Query!
State/province [9]
0
0
Helsinki
Query!
Country [10]
0
0
France
Query!
State/province [10]
0
0
Alpes Maritimes
Query!
Country [11]
0
0
France
Query!
State/province [11]
0
0
Gironde
Query!
Country [12]
0
0
France
Query!
State/province [12]
0
0
Hauts De Seine
Query!
Country [13]
0
0
France
Query!
State/province [13]
0
0
Isere
Query!
Country [14]
0
0
France
Query!
State/province [14]
0
0
Loire Atlantique
Query!
Country [15]
0
0
France
Query!
State/province [15]
0
0
Meurthe Et Moselle
Query!
Country [16]
0
0
France
Query!
State/province [16]
0
0
Nord
Query!
Country [17]
0
0
France
Query!
State/province [17]
0
0
Paris
Query!
Country [18]
0
0
France
Query!
State/province [18]
0
0
Rhone
Query!
Country [19]
0
0
France
Query!
State/province [19]
0
0
Poitiers
Query!
Country [20]
0
0
Germany
Query!
State/province [20]
0
0
Baden Wuerttemberg
Query!
Country [21]
0
0
Germany
Query!
State/province [21]
0
0
Bayern
Query!
Country [22]
0
0
Germany
Query!
State/province [22]
0
0
Hessen
Query!
Country [23]
0
0
Germany
Query!
State/province [23]
0
0
Nordrhein Westfalen
Query!
Country [24]
0
0
Germany
Query!
State/province [24]
0
0
Sachsen
Query!
Country [25]
0
0
Germany
Query!
State/province [25]
0
0
Thueringen
Query!
Country [26]
0
0
Germany
Query!
State/province [26]
0
0
Koeln
Query!
Country [27]
0
0
Greece
Query!
State/province [27]
0
0
Athens
Query!
Country [28]
0
0
Greece
Query!
State/province [28]
0
0
Larissa
Query!
Country [29]
0
0
Greece
Query!
State/province [29]
0
0
Thessaloniki
Query!
Country [30]
0
0
Greece
Query!
State/province [30]
0
0
Thessaloníki
Query!
Country [31]
0
0
Hungary
Query!
State/province [31]
0
0
Budapest
Query!
Country [32]
0
0
Italy
Query!
State/province [32]
0
0
Bari
Query!
Country [33]
0
0
Italy
Query!
State/province [33]
0
0
Bassano Del Grappa
Query!
Country [34]
0
0
Italy
Query!
State/province [34]
0
0
Bologna
Query!
Country [35]
0
0
Italy
Query!
State/province [35]
0
0
Cagliari
Query!
Country [36]
0
0
Italy
Query!
State/province [36]
0
0
Firenze
Query!
Country [37]
0
0
Italy
Query!
State/province [37]
0
0
Milano
Query!
Country [38]
0
0
Italy
Query!
State/province [38]
0
0
Novara
Query!
Country [39]
0
0
Italy
Query!
State/province [39]
0
0
Reggio Calabria
Query!
Country [40]
0
0
Italy
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State/province [40]
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Torino
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Bialystok
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Bydgoszcz
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Lódz
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Buraydah
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Jeddah
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Riyadh
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Badalona
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Lugo
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Malaga
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Oviedo
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San Sebastian
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Santiago De Compostela
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United Kingdom
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London
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Swedish Orphan Biovitrum
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Commercial sector/industry
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Iqvia Pty Ltd
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Summary
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.
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Trial website
https://clinicaltrials.gov/study/NCT05776472
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Public notes
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Contacts
Principal investigator
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Study Physician +46 08-697-20 00,
[email protected]
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Swedish Orphan Biovitrum
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Contact person for public queries
Name
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Michael O'Malley
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Phone
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+41797977276
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[email protected]
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Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
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No/undecided IPD sharing reason/comment
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What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT05776472
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