Researchers will conduct a real-world effectiveness study of pegcetacoplan in patients with paroxysmal nocturnal hemoglobinuria (PNH). This study is sponsored by Swedish Orphan Biovitrum. 

Pegcetacoplan is a relatively new drug used to treat patients with PNH; because of its novelty, there is a need for ongoing investigations to ensure it is as safe and effective as initial studies suggest. The aim of this new study is to enrich available medical literature regarding the use of pegcetacoplan in patients with PNH and assess how patients are affected pre- and post-treatment. 

This observational, single-arm study plans to recruit approximately 200 adult patients with PNH in Canada, Europe, the Middle East, and Australia. Participants must have been on pegcetacoplan for up to 12 months prior to enrollment or be prescribed pegcetacoplan upon enrollment. The exclusion criteria include enrollment in a concurrent clinical interventional study and the intake of an investigational medicinal product within 3 months of the start of pegcetacoplan treatment. 

The research team conducting this study plans to follow up on eligible candidates for around 24 months via in-person visits. In addition, they will collect retrospective data (up to 12 months) on the healthcare resource utilization of participants prior to being started on pegcetacoplan. Combined, the data collection period is approximately 48 months. 

Read more about PNH treatment 

The primary outcome of this study is the change in hemoglobin levels from treatment initiation to 6 months. Secondary outcomes include changes in absolute reticulocyte count and levels of lactate dehydrogenase, bilirubin, haptoglobin, and ferritin.

Researchers will also monitor participants for acute hemolytic events and the need for red blood cell transfusions during the study period. In addition, the research team will monitor reports of fatigue, quality of life, patient satisfaction with treatment, and healthcare resource utilization.

This study has an estimated start date of July 3, 2023, and is estimated to be completed on July 3, 2027. 


A real world effectiveness study of pegcetacoplan in patients with paroxysmal nocturnal hemoglobinuria (PNH) (COMPLETE). March 20, 2023. Accessed March 21, 2023.