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

Launched by SWEDISH ORPHAN BIOVITRUM · Mar 17, 2023

Keywords

ClinConnect Summary

This clinical trial is studying the effectiveness of a medication called pegcetacoplan in treating patients with a rare blood condition known as Paroxysmal Nocturnal Hemoglobinuria (PNH). The trial will last for 24 months and involves collecting information about how well the medication works, its safety, and how it impacts patients' lives. Researchers will follow patients who are already taking pegcetacoplan or who start taking it during the study to see how they respond to the treatment in real-world settings.

To participate in this trial, individuals must be at least 18 years old and have a confirmed diagnosis of PNH. They should either have started treatment with pegcetacoplan within the last year or be starting it at the time of enrollment. Participants will need to give their consent to take part in the study. It's important to note that individuals who are currently involved in another clinical trial or who recently participated in one may not be eligible. Throughout the study, participants can expect regular check-ins to monitor their health and experiences while on the medication.

Gender

ALL

Eligibility 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.