Global PNH Patient Registry

Part of paid clinical trials in Bethesda, Maryland.

Sponsor
Aplastic Anemia and MDS International Foundation
Study ID
NCT05755867
Status
Recruiting
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Conditions

  • Paroxysmal Nocturnal Hemoglobinuria

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Not accepted

Study Details

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.

Key Dates

Start date
May 6, 2021
Status verified
Feb 2025
Primary completion
May 6, 2026
Completion
May 6, 2031

Study Design

Enrollment
500 participants (estimated)

Arms

  • Arm: PNH Patients
    Individuals of any age with a confirmed diagnosis of PNH or diagnosis consistent with PNH are eligible for inclusion.

Primary Outcome Measure

Characterize and describe the Global PNH population [ Time Frame: 5-10 years ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Aplastic Anemia and MDS International FoundationBethesdaMaryland20814
Nyaradzo Sirewu, MPH
[email protected]
301-279-7202
Alice Houk, MS (PRINCIPAL_INVESTIGATOR)

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