The Aplastic Anemia and MDS International Foundation and the National Organization for Rare Disorders (NORD) have set up the Global PNH Patient Registry to improve knowledge about paroxysmal nocturnal hemoglobinuria (PNH) and its course and pace over time.
“The Global PNH Patient Registry provides a convenient online platform for participants; facilitates communication about PNH; gives researchers the ability to characterize the PNH population as a whole; assists the PNH community with the development of recommendations and standards of care and; and serves as a case-finding resource to be used for researchers to study the pathophysiology of PNH,” according to the study’s page on ClinicalTrials.gov.
This prospective, longitudinal, web-based, observational natural history study will enroll 500 participants of any age with a confirmed diagnosis of PNH or a diagnosis consistent with PNH. Participants will be followed throughout their lives, with a target follow-up duration of 10 years.
They will share data on various topics—demographics, quality of life, medical history, disease phenotypes, disease-related events, personal experience with PNH, general health status, medications, and diagnoses. Data will be collected at the start of the study and then at varying intervals (at least once per year).
Read about PNH etiology
The study’s primary outcome measure is to characterize and describe the worldwide population of patients with PNH. The secondary outcome measure is to develop a communications registry to be a case-finding resource for researchers who aim to study the disease, retrospectively collect intervention outcomes, and design prospective trials of novel treatments, and to notify patients of research studies and clinical trials as appropriate.
The study participants will also be enrolled in NORD’s Natural History Study Program. Their de-identified information may be used for additional research by NORD or other databases, including the Rare Disease Cures Accelerator-Data and Analytics Platform.
The Global PNH Patient Registry is currently recruiting.
Global PNH patient registry. ClinicalTrials.gov. March 6, 2023. Accessed March 7, 2023.