Global registry for patients with Paroxysmal Nocturnal Hemoglobinuria
Official title: Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Patient Registry
Run by Aplastic Anemia and MDS International Foundation · NCT05755867 · Data updated 2026-11-12
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 |
|---|---|
| Sex | All |
| Enrollment | 500 (estimated) |
| Sponsor | Aplastic Anemia and MDS International Foundation Academic / other |
| Locations | 1 site (Bethesda, Maryland) |
| Status | Recruiting |
| Started | May 6, 2021 |
| Main results expected | May 6, 2026 |
| Study ends | May 6, 2031 |
| Registry record updated | February 20, 2025 |
| Trial ID | NCT05755867 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates include individuals of any age with a confirmed diagnosis of PNH or a diagnosis consistent with PNH.
✕Probably not a fit
Patients who cannot read and understand English may not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
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.
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 gone: Other patient registries have shown success in enhancing understanding of rare diseases, making this approach promising.
Who can join: the full rules
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 it's running
-
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
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
-
Oral HS-10542 for paroxysmal nocturnal hemoglobinuria
This study will test whether the oral drug HS-10542 can reduce hemolysis and raise hemoglobin in adults with PNH, including those not fully helped by C5 inhibitor therapy.
-
Danicopan added to ravulizumab or eculizumab for PNH in Korea
This will see if adding Danicopan to ravulizumab or eculizumab is safe and helps people with PNH who are treated in routine care in Korea.
-
Iptacopan use in Korean patients with PNH or C3G
This post-marketing surveillance study tests how well iptacopan works and how safe it is for Korean adults with PNH or C3G in real-world care.
-
Pozelimab + cemdisiran for adults with PNH whose current treatment isn't working
This trial tests whether combining pozelimab and cemdisiran can better reduce hemolysis in adults with PNH whose C5 inhibitor treatment isn’t controlling their disease.
-
Evaluating MY008211A for treating anemia in PNH patients on anti-C5 therapy
This study is testing a new treatment called MY008211A to see if it can help people with PNH who still have anemia even while on anti-C5 therapy.
-
Iptacopan treatment for adults with PNH in China
This study will try iptacopan (LNP023) in adults with PNH in China to see if it improves clinical and laboratory outcomes and changes healthcare use.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.