- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT05755867
Global PNH Patient Registry
Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Patient Registry
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.
Study Overview
Status
Conditions
Detailed Description
The Global PNH Patient Registry is a prospective, longitudinal, web-based, observational natural history study. Participants with PNH will be followed throughout the course of their lives with either the participant or authorized respondents contributing data at varying intervals throughout the course of the study. Data will be collected at the start of the study (baseline), at least once per year or can be updated by the participant as needed. Data will be collected on demographics, quality of life, medical history, disease phenotypes, disease-related events, personal experience with PNH, general health status, medications, and diagnoses. 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.
A Registry Advisory Board, that may include scientists, doctors, and patient advocates, will be assembled to oversee the conduct of the study. The Advisory Board will review aggregate registry data and the use of this registry, ensure proper evaluation of protocols requesting to use registry data and/or contact registry participants, and review any protocol or confidentiality deviations on a case- by by-case basis and ensure that any such deviations are reported to the IRB.
The registry will use a web-based interface to maximize accessibility to participants and clinicians world-wide. Following informed consent, participants will be invited to enter their data and information which will be stored indefinitely or until a participant revokes their consent to participate in the study. No experimental intervention is involved in participation in the Global PNH Patient registry study.
Annual maintenance will be funded by the Aplastic Anemia and MDS International Foundation (AAMDSIF) with support from industry partners. Registry participants will be automatically enrolled in NORD's Natural History Study Program (NHS), and their de-identified information aggregated with information from other rare diseases may be used for the purposes of cross disease analysis and cross-disease research to facilitate advocacy and further NORD's mission. De-identified information may be shared with other databases such as the Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP). This will allow more researchers to use the information to do research.
Study Type
Enrollment (Anticipated)
Contacts and Locations
Study Contact
- Name: Alice Houk, MS
- Phone Number: 101 3012797202
- Email: houk@aamds.org
Study Contact Backup
- Name: Elizabeth Kottke
- Phone Number: 275 475.289.6855
- Email: ekottke@rarediseases.org
Study Locations
-
-
Maryland
-
Bethesda, Maryland, United States, 20814
- Recruiting
- Aplastic Anemia and MDS International Foundation
-
Contact:
- Nyaradzo Sirewu, MPH
- Phone Number: 126 301-279-7202
- Email: sirewu@aamds.org
-
Principal Investigator:
- Alice Houk, MS
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
- Child
- Adult
- Older Adult
Accepts Healthy Volunteers
Genders Eligible for Study
Sampling Method
Study Population
Description
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 Plan
How is the study designed?
Design Details
Cohorts and Interventions
Group / Cohort |
---|
PNH Patients
Individuals of any age with a confirmed diagnosis of PNH or diagnosis consistent with PNH are eligible for inclusion.
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Characterize and describe the Global PNH population
Time Frame: 5-10 years
|
Understand disease prevalence and phenotype as well as the rate of progression of disease characteristics.
Gather patient experiences including demographics, diagnostics, treatment, symptoms, and quality of life.
|
5-10 years
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Develop a communications registry
Time Frame: 5-10 years
|
|
5-10 years
|
Collaborators and Investigators
Collaborators
Investigators
- Principal Investigator: Alice Houk, MS, Aplastic Anemia and MDS International Foundation
Publications and helpful links
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Anticipated)
Study Completion (Anticipated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Estimate)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- AAMDSIF-PNH1
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
IPD Plan Description
IPD Sharing Time Frame
IPD Sharing Access Criteria
IPD Sharing Supporting Information Type
- CSR
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Paroxysmal Nocturnal Hemoglobinuria
-
Apellis Pharmaceuticals, Inc.RecruitingParoxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal HemoglobinuriaMalaysia, United States, Czechia, France, Netherlands, Serbia, Spain, Thailand, United Kingdom
-
Novartis PharmaceuticalsCompletedParoxysmal Nocturnal Hemoglobinuria PNHLithuania, Japan, Czechia
-
Ra PharmaceuticalsCompletedParoxysmal Nocturnal Hemoglobinuria (PNH)United States
-
Alexion PharmaceuticalsAchillion, a wholly owned subsidiary of AlexionCompletedParoxysmal Nocturnal Hemoglobinuria (PNH)United Kingdom, New Zealand, Korea, Republic of, Italy
-
AKARI TherapeuticsCompletedParoxysmal Nocturnal Hemoglobinuria (PNH)Kazakhstan, Lithuania, Sri Lanka
-
Alexion PharmaceuticalsTerminatedParoxysmal Nocturnal Hemoglobinuria (PNH)United States, Czech Republic, Italy, Poland, United Kingdom
-
AlexionCompletedParoxysmal Nocturnal Hemoglobinuria (PNH)United States, Korea, Republic of, Canada, France, Germany, Spain, United Kingdom, Japan, Australia, Italy, Netherlands
-
Ra PharmaceuticalsCompletedParoxysmal Nocturnal Hemoglobinuria (PNH)United Kingdom, Australia, Canada, Denmark, Finland, Germany, Hungary, New Zealand
-
Alnylam PharmaceuticalsCompletedParoxysmal Nocturnal Hemoglobinuria (PNH)Spain, United Kingdom
-
Alexion PharmaceuticalsCompletedParoxysmal Hemoglobinuria, NocturnalUnited States, Australia, Belgium, Canada, Germany, Italy, Spain, Sweden, United Kingdom, France, Ireland, Netherlands