Status:
RECRUITING
A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Lead Sponsor:
Swedish Orphan Biovitrum
Collaborating Sponsors:
Iqvia Pty Ltd
Conditions:
Paroxysmal Nocturnal Hemoglobinuria
Eligibility:
All Genders
18+ years
Brief 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 ...
Detailed Description
As pegcetacoplan is a new product on the market, with a new mechanism of action, there is an urgent need to provide data to treaters, payers and the PNH community on the real-world usage and effective...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
June 26 2023
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
August 15 2029
Estimated Enrollment :
200 Patients enrolled
Trial Details
Trial ID
NCT05776472
Start Date
June 26 2023
End Date
August 15 2029
Last Update
May 14 2025
Active Locations (75)
Enter a location and click search to find clinical trials sorted by distance.
1
Liverpool Hospital
Liverpool, New South Wales, Australia
2
Calvary Mater Newcastle
Waratah, New South Wales, Australia
3
Sunshine Coast University Hospital
Birtinya, Queensland, Australia
4
AZ Sint-Jan
Bruges, Belgium