Global PNH Patient Registry

Recruiting now

Conditions studied: Paroxysmal Nocturnal Hemoglobinuria

In brief

The primary aim of the Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Patient Registry is to conduct a prospectively-planned and efficient natural history study that will result in a more comprehensive understanding of the disease and its course and pace over time. Other registry objectives include the following: * Provide a convenient online platform for participants (or caregivers) to self-report cases of PNH. * Develop a communications registry within the Global PNH Patient registry (e.g., to notify patients of research studies and clinical trials). * Characterize and describe the Global PNH population as a whole, enhancing the understanding of disease prevalence and phenotype as well as the rate of progression of disease characteristics. * Assist the PNH community with the development of recommendations and standards of care. * Be a case-finding resource to be used for researchers who seek to study the pathophysiology of PNH, retrospectively collate intervention outcomes, and design prospective trials of novel treatments.

Key facts

Study ID

NCT05755867

Run by

Aplastic Anemia and MDS International Foundation

People needed

500

Starts

2021-05-06

Expected to finish

2031-05-06

Last updated by the study team

2025-02-20

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• Individuals not able to read and understand English.

Where it is running

• Aplastic Anemia and MDS International Foundation — Bethesda, Maryland, United States (enrolling)

Full record on ClinicalTrials.gov

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