Global PNH Patient Registry
Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Patient Registry
1 other identifier
observational
500
1 country
1
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for all trials
Started May 2021
Longer than P75 for all trials
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
May 6, 2021
CompletedFirst Submitted
Initial submission to the registry
February 23, 2023
CompletedFirst Posted
Study publicly available on registry
March 6, 2023
CompletedPrimary Completion
Last participant's last visit for primary outcome
May 6, 2026
CompletedStudy Completion
Last participant's last visit for all outcomes
May 6, 2031
ExpectedFebruary 20, 2025
February 1, 2025
5 years
February 23, 2023
February 18, 2025
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Characterize and describe the Global PNH population
Understand disease prevalence and phenotype as well as the rate of progression of disease characteristics. Gather patient experiences including demographics, diagnostics, treatment, symptoms, and quality of life.
5-10 years
Secondary Outcomes (1)
Develop a communications registry
5-10 years
Study Arms (1)
PNH Patients
Individuals of any age with a confirmed diagnosis of PNH or diagnosis consistent with PNH are eligible for inclusion.
Eligibility Criteria
Individuals of any age with a confirmed diagnosis of PNH or diagnosis consistent with PNH.
You may qualify if:
- 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
You may not qualify if:
- Individuals not able to read and understand English.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
Aplastic Anemia and MDS International Foundation
Bethesda, Maryland, 20814, United States
Related Links
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Alice Houk, MS
Aplastic Anemia and MDS International Foundation
Central Study Contacts
Study Design
- Study Type
- observational
- Observational Model
- OTHER
- Time Perspective
- PROSPECTIVE
- Target Duration
- 10 Years
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
February 23, 2023
First Posted
March 6, 2023
Study Start
May 6, 2021
Primary Completion
May 6, 2026
Study Completion (Estimated)
May 6, 2031
Last Updated
February 20, 2025
Record last verified: 2025-02
Data Sharing
- IPD Sharing
- Will share
- Shared Documents
- CSR
- Time Frame
- Data will be available 2 years after study start and will be analyzed through study ending.
- Access Criteria
- Sharing is subject to review by the Medical Advisory Committee
It is anticipated that the Global PNH Patient Registry will be a valuable resource for current and future research. A Global PNH Patient Registry Advisory Board will ensure proper evaluation of protocols that request use of registry data and/or contact registry participants. To promote use of the repository, aggregate information about database contents will be updated quarterly or semi-annually and made available to the public. Such information may include number of registrants, prevalence of common diagnoses of registrants, demographic information, and percent willing to be contacted for future research. Investigators wanting to use the registry or contact participants will need to apply to the Advisory Board. The application will require information concerning: Principal Investigator, aims and hypotheses of the proposed research, where the research will be performed, and how the research will be funded.