Actively Recruiting
Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Patient Registry
Led by Aplastic Anemia and MDS International Foundation · Updated on 2025-02-20
500
Participants Needed
1
Research Sites
260 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
Researchers are conducting the Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Patient Registry to better understand the natural history, progression, and characteristics of PNH over time. This observational study aims to gather comprehensive data on the disease to support recommendations, standards of care, and future research, including clinical trials of new treatments. The registry also provides a platform for participants or caregivers to self-report PNH cases and facilitates communication within the PNH community. Participants with PNH will be followed prospectively through a web-based platform that allows them or authorized respondents to contribute information at varying intervals, at least once per year or as needed. Data collected includes demographics, quality of life, medical history, disease phenotypes, disease-related events, medications, and general health status. The study is overseen by a Registry Advisory Board to ensure proper conduct and data use. During the study, participants will provide data online, which will be stored indefinitely unless consent is withdrawn. Researchers will use this information to characterize the global PNH population and support further research and advocacy efforts. There is no experimental treatment involved, and participant data may be shared in de-identified form with related rare disease databases for cross-disease research. The study will continue for several years, with ongoing data collection and communication development.
CONDITIONS
Brief 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
- Willing to provide informed consent or have a legal guardian provide consent
- Ability to access the internet periodically
- Ability to comply with web-based study procedures and data collection
You will not qualify if you...
- Unable to read and understand English
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person or remote)
Duration - Baseline at enrollment
Participants provide baseline data including demographics, medical history, and disease characteristics through a web-based interface.
1 baseline data entry session (online)
Duration - Up to 10 years or until withdrawal
Participants are followed over time with periodic updates of health status, quality of life, and medical events via the online platform.
At least annual data updates or as needed (online)
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
E
Elizabeth Kottke
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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