Global Patient Registry for Paroxysmal Nocturnal Hemoglobinuria PNH A Web-Based Study to Understand PNH Over Time and Support Research

What this Trial Is About

Researchers are conducting the Global Paroxysmal Nocturnal Hemoglobinuria PNH Patient Registry to better understand the natural history, progression, and characteristics of PNH over time. This observational study aims to gather comprehensive data on the disease to support recommendations, standards of care, and future research, including clinical trials of new treatments. The registry also provides a platform for participants or caregivers to self-report PNH cases and facilitates communication within the PNH community. Participants with PNH will be followed prospectively through a web-based platform that allows them or authorized respondents to contribute information at varying intervals, at least once per year or as needed. Data collected includes demographics, quality of life, medical history, disease phenotypes, disease-related events, medications, and general health status. The study is overseen by a Registry Advisory Board to ensure proper conduct and data use. During the study, participants will provide data online, which will be stored indefinitely unless consent is withdrawn. Researchers will use this information to characterize the global PNH population and support further research and advocacy efforts. There is no experimental treatment involved, and participant data may be shared in de-identified form with related rare disease databases for cross-disease research. The study will continue for several years, with ongoing data collection and communication development.

Global PNH Patient Registry