RECRUITING

Global PNH Patient Registry

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Patient Registry

Quick Facts

Study Start:2021-05-06
Study Completion:2031-05-06
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT05755867

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:Not specified
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * legal adult participants who are able to provide their own consent;
  2. * 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.
  3. * Individuals must have at least periodic access to the internet and be able to comply with web-based study procedures and data collections
  1. * Individuals not able to read and understand English.

Contacts and Locations

Study Contact

Alice Houk, MS
CONTACT
3012797202
houk@aamds.org
Elizabeth Kottke
CONTACT
475.289.6855
ekottke@rarediseases.org

Principal Investigator

Alice Houk, MS
PRINCIPAL_INVESTIGATOR
Aplastic Anemia and MDS International Foundation

Study Locations (Sites)

Aplastic Anemia and MDS International Foundation
Bethesda, Maryland, 20814
United States

Collaborators and Investigators

Sponsor: Aplastic Anemia and MDS International Foundation

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

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2021-05-06
Study Completion Date2031-05-06

Study Record Updates

Study Start Date2021-05-06
Study Completion Date2031-05-06

Terms related to this study

Keywords Provided by Researchers

  • PNH
  • Registry
  • Patient Registry
  • Paroxysmal Nocturnal Hemoglobinuria

Additional Relevant MeSH Terms

  • Paroxysmal Nocturnal Hemoglobinuria