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Global PNH Patient Registry

NCT05755867 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 500

Last updated 2025-02-20

No results posted yet for this study

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.

Conditions

Sponsors & Collaborators

  • National Organization for Rare Disorders

    collaborator OTHER

  • Aplastic Anemia and MDS International Foundation

    lead OTHER

Principal Investigators

  • Alice Houk, MS · Aplastic Anemia and MDS International Foundation

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-05-06
Primary Completion
2026-05-06
Completion
2031-05-06

Countries

  • United States

Study Locations

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Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05755867 on ClinicalTrials.gov