Global registry for patients with Paroxysmal Nocturnal Hemoglobinuria
Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Patient Registry
This study is collecting information from people with Paroxysmal Nocturnal Hemoglobinuria to better understand the condition and improve care for patients.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 500 (estimated) |
| Sex | All |
| Sponsor | Aplastic Anemia and MDS International Foundation Academic / other |
| Locations | 1 site (Bethesda, Maryland) |
| Trial ID | NCT05755867 on ClinicalTrials.gov |
What this trial studies
The Global PNH Patient Registry aims to create a comprehensive natural history study of Paroxysmal Nocturnal Hemoglobinuria (PNH) by collecting data from participants over time. This observational registry allows individuals with PNH to self-report their experiences and health status through a convenient online platform. The study will gather information on demographics, quality of life, medical history, and disease progression, which will help enhance understanding of PNH and inform standards of care. An Advisory Board will oversee the study to ensure proper data use and participant confidentiality.
Who should consider this trial
Good fit: Ideal candidates include individuals of any age with a confirmed diagnosis of PNH or a diagnosis consistent with PNH.
Not a fit: Patients who cannot read and understand English may not benefit from this study.
Why it matters
Potential benefit: If successful, this registry could significantly improve the understanding of PNH and lead to better patient care and treatment recommendations.
How similar studies have performed: Other patient registries have shown success in enhancing understanding of rare diseases, making this approach promising.
Eligibility criteria
Show full inclusion / exclusion 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.
Where this trial is running
Bethesda, Maryland
- Aplastic Anemia and MDS International Foundation — Bethesda, Maryland, United States (Recruiting)
Study contacts
- Principal investigator: Alice Houk, MS — Aplastic Anemia and MDS International Foundation
- Study coordinator: Alice Houk, MS
- Email: houk@aamds.org
- Phone: 3012797202
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.