Global PNH Patient Registry
ConditionParoxysmal Nocturnal Hemoglobinuria
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorAplastic Anemia and MDS International Foundation
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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
Disqualifiers
Individuals not able to read and understand English.
Trial population
Individuals of any age with a confirmed diagnosis of PNH or diagnosis consistent with PNH.
Trial design
Design model
Other
Time perspective
Prospective
Treatments tested in this trial
Not listed
Trial groups
500 Participants
are grouped into 1 trial groupGroup A: PNH Patients
Trial outcomes
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.
Time frame
5-10 yearsSecondary outcomes
1
Develop a communications registry
* 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. * Notify patients of research studies and clinical trials as appropriate.
Time frame
5-10 yearsSponsors and contacts
Click on the lead sponsor to view all of their trials.
Aplastic Anemia and MDS International Foundation
Lead sponsor
National Organization for Rare Disorders
Collaborator
Source ClinicalTrials.gov