Actively Recruiting
Global PNH Patient Registry
Led by Aplastic Anemia and MDS International Foundation · Updated on 2025-02-20
500
Participants Needed
1
Research Sites
521 weeks
Total Duration
On this page
Sponsors
A
Aplastic Anemia and MDS International Foundation
Lead Sponsor
N
National Organization for Rare Disorders
Collaborating Sponsor
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Global PNH Patient Registry
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Individuals of any age with a confirmed diagnosis of PNH or diagnosis consistent with PNH
- Willingness to provide informed consent (by participant or legally authorized representative)
- Ability to access the internet periodically and comply with web-based study procedures
You will not qualify if you...
- Inability to read and understand English
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Aplastic Anemia and MDS International Foundation
Bethesda, Maryland, United States, 20814
Actively Recruiting
Research Team
A
Alice Houk, MS
CONTACT
E
Elizabeth Kottke
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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