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Study identifier: NCT05776472 Synced from ClinicalTrials.gov · July 28, 2026
● Recruiting

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

Condition: Paroxysmal Nocturnal Hemoglobinuria  ·  Sponsor: Swedish Orphan Biovitrum

PhaseN/A
Planned participants200
Who can joinAll sexes, 18 Years to no upper limit
Healthy volunteersNo

About this study

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.

This description comes directly from the study's public registry record.

Talk to the study team

Clinical Program Lead  ·  +46 (0)8 697 20 00  ·  medical.info@sobi.com

Always discuss trial participation with your own doctor first.

Locations (87)

Liverpool HospitalLiverpool, New South Wales, AustraliaRecruiting
Calvary Mater NewcastleWaratah, New South Wales, AustraliaRecruiting
Sunshine Coast University HospitalBirtinya, Queensland, AustraliaRecruiting
Canberra HospitalGarran, AustraliaRecruiting
Institut Jules BordetAnderlecht, BelgiumRecruiting
AZ Sint-JanBruges, BelgiumRecruiting
CHU Brugmann-Site HortaBrussels, BelgiumRecruiting
Centre Hospitalier Universitaire de LiegeLiège, BelgiumRecruiting
AZ HerentalsTurnhout, BelgiumRecruiting
London Health Sciences Centre (LHSC) - Victoria HospitalLondon, Ontario, CanadaRecruiting
University Health NetworkToronto, Ontario, CanadaRecruiting
Hopital Maisonneuve-Rosemont d/b/a CIUSSS de l'Est-de-l'Île-de-MontréalMontreal, Quebec, CanadaActive Not Recruiting

+ 75 more locations — full list on the registry record.

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Source record: clinicaltrials.gov/study/NCT05776472