Global PNH Patient Registry

Launched by APLASTIC ANEMIA AND MDS INTERNATIONAL FOUNDATION · Feb 23, 2023

Keywords

ClinConnect Summary

The Global PNH Patient Registry is a clinical trial designed to gather important information about Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare blood disorder caused by a genetic mutation. The goal of this study is to better understand how PNH affects patients over time, including how common it is and how the disease progresses. Participants will have the opportunity to report their experiences online, which will help researchers learn more about the condition and improve care recommendations for patients.

Anyone with a confirmed diagnosis of PNH, regardless of age, can join the study, as long as they can provide informed consent or have a legal representative who can do so on their behalf. Participants should have access to the internet since the study involves online reporting and data collection. By joining this registry, patients will contribute to a valuable resource that not only aids ongoing research but also helps to connect them with information about new studies and treatments for PNH.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • - Individuals of any age with a confirmed diagnosis of PNH or diagnosis consistent with PNH are eligible for inclusion. PNH is defined as a genetic mutation in the PIG-A gene.
• Individuals must be willing to provide informed consent. Participants can be:
• • legal adult participants who are able to provide their own consent;
• • children and adults unable to provide their own consent, for whom consent must be provided by a Legally Authorized Representative (LAR) who is a legal adult.
• • Individuals must have at least periodic access to the internet and be able to comply with web-based study procedures and data collections
• Exclusion Criteria:
• • Individuals not able to read and understand English.