Paroxysmal Nocturnal HemoglobinuriaNCT05755867
Global PNH Patient Registry
An observational study of Paroxysmal Nocturnal Hemoglobinuria, run by Aplastic Anemia and MDS International Foundation at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria
- How many people
- 500 participants
- Where
- 1 siteUnited States
- Study dates
- 6 May 2021to 6 May 2031
- How long will I take part?
- 10 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Individuals of any age with a confirmed diagnosis of PNH or diagnosis consistent with PNH.
Who may be able to join
- 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
Who cannot join
- Individuals not able to read and understand English.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Aplastic Anemia and MDS International Foundation
Bethesda, Maryland, 20814
Nyaradzo Sirewu, MPH, 301-279-7202 ext. 126, sirewu@aamds.org
Alice Houk, MS
About this study
Official title: 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.
Read the 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.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Keywords
- PNH, Registry, Patient Registry, Paroxysmal Nocturnal Hemoglobinuria
- Medical subject headings
- Hemoglobinuria, Paroxysmal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| PNH Patients | Individuals of any age with a confirmed diagnosis of PNH or diagnosis consistent with PNH are eligible for inclusion. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Characterize and describe the Global PNH population
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.
Measured: 5-10 years
Secondary outcomes (1)
Develop a communications registry
- 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.
- Notify patients of research studies and clinical trials as appropriate.
Measured: 5-10 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Mar 2023
- Study start6 May 2021
- Primary completion6 May 2026 (estimated)
- Study completion6 May 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Paroxysmal Nocturnal HemoglobinuriaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Prospective
- Enrolment
- 500 participants (estimated)
- Target duration
- 10 Years
Sponsor and investigators
- Lead sponsor
- Aplastic Anemia and MDS International Foundation
- Sponsor type
- University, hospital or other
- Collaborators
- National Organization for Rare Disorders
- Responsible party
- Sponsor
- Investigators
- Alice Houk, MS, Principal investigator, Aplastic Anemia and MDS International Foundation
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05755867
- Sponsor's protocol ID
- AAMDSIF-PNH1
- Status last verified
- Feb 2025
- First submitted
- 23 Feb 2023
- Last updated
- 20 Feb 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: It is anticipated that the Global PNH Patient Registry will be a valuable resource for current and future research. A Global PNH Patient Registry Advisory Board will ensure proper evaluation of protocols that request use of registry data and/or contact registry participants. To promote use of the repository, aggregate information about database contents will be updated quarterly or semi-annually and made available to the public. Such information may include number of registrants, prevalence of common diagnoses of registrants, demographic information, and percent willing to be contacted for future research. Investigators wanting to use the registry or contact participants will need to apply to the Advisory Board. The application will require information concerning: Principal Investigator, aims and hypotheses of the proposed research, where the research will be performed, and how the research will be funded.
Study at a glance
From the registry record, last updated by the sponsor on 20 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Patient Registry
- ClinicalTrials.gov ID
- NCT05755867
- Acronym / sponsor ID
- AAMDSIF-PNH1
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Keywords
- PNH, Registry, Patient Registry, Paroxysmal Nocturnal Hemoglobinuria
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 500 participants (estimated)
- Lead sponsor
- Aplastic Anemia and MDS International FoundationUniversity, hospital or other
- Collaborators
- National Organization for Rare Disorders
- Study start
- 6 May 2021 (actual)
- Primary completion
- 6 May 2026 (estimated)
- Study completion
- 6 May 2031 (estimated)
- First posted
- 6 Mar 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Observational model
- Other
- Time perspective
- Prospective
- Target follow-up
- 10 Years
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| PNH Patients | – | –Individuals of any age with a confirmed diagnosis of PNH or diagnosis consistent with PNH are eligible for inclusion. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Individuals of any age with a confirmed diagnosis of PNH or diagnosis consistent with PNH.
Key inclusion criteria (5 listed)
- 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.
- 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
Key exclusion criteria (1 listed)
- Individuals not able to read and understand English.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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.
1 secondary outcome
Develop a communications registry
Time frame: 5-10 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Feb 2025.
- Description of the patient registry on the sponsor websiteLink provided by the sponsor.
- Link to registry platform where participants can enroll into the studyLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. It is anticipated that the Global PNH Patient Registry will be a valuable resource for current and future research. A Global PNH Patient Registry Advisory Board will ensure proper evaluation of protocols that request use of registry data and/or contact registry participants. To promote use of the repository, aggregate information about database contents will be updated quarterly or semi-annually and made available to the public. Such information may include number of registrants, prevalence of common diagnoses of registrants, demographic information, and percent willing to be contacted for future research. Investigators wanting to use the registry or contact participants will need to apply to the Advisory Board. The application will require information concerning: Principal Investigator, aims and hypotheses of the proposed research, where the research will be performed, and how the research will be funded.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Alice Houk, MS principal investigator
Aplastic Anemia and MDS International Foundation
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Paroxysmal Nocturnal Hemoglobinuria, run by Aplastic Anemia and MDS International Foundation at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 20 Feb 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Alice Houk, MS (3012797202 ext. 101, houk@aamds.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Characterize and describe the Global PNH population (time frame: 5-10 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Feb 2025.