NCT05755867Recruiting
Global PNH Patient Registry
Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Patient Registry
Source: ClinicalTrials.gov · Last updated 20 Feb 2025 · Not updated for over 12 months - ask the site whether it is still open
Brief summary (registry, in English)
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.
- Status (registry)
- Recruiting
- Study type
- Observational
- Enrollment
- 500 (estimated)
- First submitted
- 23 Feb 2023
- Start date
- 6 May 2021
- Primary completion date
- 6 May 2026
- Lead sponsor
- Aplastic Anemia and MDS International Foundation
- NCT number
- NCT05755867
What you commit to
- Study type: Observational
- Planned participants: 500
- From 6 May 2021 to the primary completion date, 6 May 2026
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other
- Individuals not able to read and understand English.
Who the study is looking for
- \- 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
Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Aplastic Anemia and MDS International Foundation Coordinator of this site? Confirm its status | Bethesda | United States | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.