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Evaluating the effectiveness of pegcetacoplan in patients with PNH

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

Observational Swedish Orphan Biovitrum · NCT05776472

This study is trying to see how well pegcetacoplan works for people with Paroxysmal Nocturnal Hemoglobinuria (PNH) over three years in everyday medical settings.

Quick facts

Study typeObservational
Enrollment200 (estimated)
Ages18 Years and up
SexAll
SponsorSwedish Orphan Biovitrum Industry-sponsored
Locations75 sites (Liverpool, New South Wales and 74 other locations)
Trial IDNCT05776472 on ClinicalTrials.gov

What this trial studies

This observational study aims to assess the real-world effectiveness of pegcetacoplan in patients diagnosed with Paroxysmal Nocturnal Hemoglobinuria (PNH) over a period of 36 months. Approximately 200 patients will be enrolled across 70 sites in Europe, the Middle East, Canada, and Australia, with data collected both retrospectively and prospectively. The study will track patient outcomes from the initiation of pegcetacoplan treatment, including healthcare resource utilization and RBC transfusions, to provide valuable insights into its effectiveness in routine medical practice.

Who should consider this trial

Good fit: Ideal candidates for this study are adults aged 18 and older with a documented diagnosis of PNH who have started pegcetacoplan treatment within the last 12 months or are prescribed pegcetacoplan at enrollment.

Not a fit: Patients currently enrolled in another clinical interventional study or those who initiated pegcetacoplan treatment as part of an interventional study may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide critical data on the real-world effectiveness of pegcetacoplan, potentially improving treatment strategies for patients with PNH.

How similar studies have performed: While pegcetacoplan is a new treatment, this study aims to fill a knowledge gap, and similar observational studies have shown promise in evaluating real-world treatment effectiveness.

Eligibility criteria

Show full inclusion / exclusion criteria 
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.

Where this trial is running

Liverpool, New South Wales and 74 other locations

+25 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Paroxysmal Nocturnal Hemoglobinuria
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.