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

NCT05776472 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 200

Last updated 2025-05-14

No results posted yet for this study

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.

Conditions

Interventions

DRUG

Pegcetacoplan

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

Sponsors & Collaborators

  • Iqvia Pty Ltd

    collaborator INDUSTRY

  • Swedish Orphan Biovitrum

    lead INDUSTRY

Principal Investigators

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-06-26
Primary Completion
2026-08-15
Completion
2029-08-15
FDA Drug
Yes

Countries

  • Australia
  • Belgium
  • Canada
  • Czechia
  • Finland
  • France
  • Germany
  • Greece
  • Hungary
  • Italy
  • Poland
  • Saudi Arabia
  • Spain
  • United Kingdom

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05776472 on ClinicalTrials.gov