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Global PNH Patient Registry

Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Patient Registry

Who can join

All ages · All sexes

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.

About the study

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.

Sponsor: Aplastic Anemia and MDS International Foundation · Participants: 500 · Started: May 6, 2021

Contact the study team

• Alice Houk, MS · Phone: 3012797202
• Elizabeth Kottke · Phone: 475.289.6855

Official record on ClinicalTrials.gov — NCT05755867

Locations in the U.S.

Conditions

• Paroxysmal Hemoglobinuria

From ClinicalTrials.gov, data retrieved Oct 1, 2026. Each study sets its own eligibility; the study team decides who can join.