Recruiting

Observational Study

Sponsor:

Aplastic Anemia and MDS International Foundation

Code:

NCT05755867

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

Study Details

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.

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Study ID

NCT05755867

Start date

May 6, 2021

Status verified date

Feb, 2025

Completion date

May 6, 2031

Anticipated

Primary completion date

May 6, 2026

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

Inclusion Criteria:

- Individuals of any age with a confirmed diagnosis of PNH or diagnosis consistent with PNH are eligible for inclusion. PNH is defined as a genetic mutation in the PIG-A gene.

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

Exclusion Criteria:

  • Individuals not able to read and understand English.

Study Design

Enrollment

500 participants

Anticipated

Interventions and Outcome Measures

Arms

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 and Locations

Central contacts

Alice Houk, MS

3012797202houk@aamds.org

Elizabeth Kottke

475.289.6855ekottke@rarediseases.org

Locations

Aplastic Anemia and MDS International Foundation

Recruiting

Bethesda, Maryland, United States, 20814

Contacts

Nyaradzo Sirewu, MPH

301-279-7202sirewu@aamds.org

Principal Investigator:

Alice Houk, MS

More Information

Sponsor

Aplastic Anemia and MDS International Foundation

Last update posted

Feb 20, 2025

Last verified

Feb, 2025

Keywords

  • PNH
  • Registry
  • Patient Registry
  • Paroxysmal Nocturnal Hemoglobinuria

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Aplastic Anemia and MDS International Foundation on 2025-02-20.