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Global PNH Patient Registry

Introductory image: People in discussion at a conference

We are pleased to announce the launch of the Global PNH Patient Registry, a collaborative effort between the Aplastic Anemia and MDS International Foundation (AAMDSIF) and the National Organization for  Rare Disorders (NORD®) to study PNH.

Overview of Research Study

The Global PNH Patient Registry is more than a versatile online system that securely collects and stores data for medical research; it is an interactive dynamic participant-driven resource that can empower and unite the PNH community through shared knowledge. Registry participants not only can complete surveys about their own disease experiences, but also can learn about other participants’ experiences by viewing aggregated survey data. As the registry sponsor,* AAMDSIF will ensure that data privacy and confidentiality are strictly maintained. Participation in the Global PNH Patient Registry is free and voluntary, and participants may withdraw at any time.
The Global PNH Patient Registry will include data collection on demographics, quality of life, treatment, diagnosis, and PNH symptoms. It can be completed in stages throughout the course of the study.

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.

Community Involvement

The Global PNH Patient Registry is a powerful opportunity for individuals with PNH and their family members to contribute directly to research that will enhance our understanding of PNH, thus facilitating the development of new diagnostic and treatment options. Participation is especially vital given the rarity of PNH. Every patient experience is a unique and invaluable part of the natural history of PNH.

For more information, visit pnh.iamrare.org

Some Frequently Asked Questions

WHO CAN JOIN THE STUDY?

This study is open to anyone who has been diagnosed with PNH.

WHAT TYPES OF DATA WILL BE COLLECTED IN THE GLOBAL PNH PATIENT REGISTRY?

The data collected is uniform and includes but is not limited to:

  • Socio-demographics
  • Medical and diagnostics
  • Treatment and disease progression 
  • Management of care
  • Quality of life

HOW IS THE DATA COLLECTED?

Data is collected through a secure web-based system developed by the National Organization for Rare Disorders, Inc. (NORD®), an independent non-profit committed to the identification, treatment, and cure of all 7,000 rare diseases. Study participants respond to questions grouped within a series of surveys developed per study standards and in collaboration with disease specific experts.

WHO IS A STUDY PARTICIPANT?

A study participant is the individual who takes part in a research study and whose information is collected for that research. Study participants may consent to enter and share their own personal data.

WHO IS A REPORTER/RESPONDENT?

A reporter/respondent is an individual who completes the surveys on behalf of the patient/study participant, when that individual is unable to do so on their own behalf.

WHAT IS A LEGALLY AUTHORIZED REPRESENTATIVE (LAR)?

An individual who is authorized under applicable law to consent, on behalf of a prospective subject, to the subject’s participation in a clinical trial. The LAR may be a parent, grandparent, caregiver or guardian who has the legal authority to grant consent on behalf of another who is eligible to participate in research. When a LAR acts on behalf of a study participant, they are considered to be the reporter/respondent in the research. 

WHAT IS AN INFORMED CONSENT?

The Office for Human Research Protections (OHRP) states that, “… the informed consent process is the critical communication link between the prospective human subject and an investigator beginning with the initial approach of an investigator to the potential subject (e.g. through a flyer, brochure, or any advertisement regarding the research study) and continuing until the completion of the research study. […] The informed consent process involves three key features: (1) disclosing to potential research subjects’ information needed to make an informed decision; (2) facilitating the understanding of what has been disclosed; and (3) promoting the voluntariness of the decision about whether or not to participate in the research.”

IS THERE A COST TO PARTICIPATE?

There is no cost for patients to join this study. AAMDSIF absorbs the cost of the registry for its community. 

*=The Global PNH Patient Registry is a collaborative effort between AAMDSIF and NORD, along with the support of industry partners, Apellis Pharmaceuticals, Inc., Genentech, Inc., and BioCryst Pharmaceuticals, Inc.

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