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

Sponsor
Swedish Orphan Biovitrum
Study ID
NCT05776472
Status
Recruiting
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Conditions

  • Paroxysmal Nocturnal Hemoglobinuria

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

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

Study Details

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.

Key Dates

Start date
Jun 26, 2023
Status verified
May 2025
Primary completion
Aug 15, 2026
Completion
Aug 15, 2029

Study Design

Enrollment
200 participants (estimated)

Primary Outcome Measure

Change in observed hemoglobin level from initiation of treatment with pegcetacoplan to 6 months [ Time Frame: 6 months ]

Central Contacts

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