Paroxysmal Nocturnal HemoglobinuriaNCT06524726
The International PNH Interest Group PNH Registry
An observational study of Paroxysmal Nocturnal Hemoglobinuria, run by International PNH Interest Group at 1 site in United States.
Trial summary
The aim of this International PNH Interest Group (IPIG) registry is to develop an international database to prospectively collect data on patients with PNH covering clinical outcomes, patient reported outcomes (PROs), and health-resource utilization (HRU) on all enrolled patients, as well as long term safety data. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria
- How many people
- 2,000 participants
- Where
- 1 siteUnited States
- Study dates
- 10 May 2024to 10 May 2029
- 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: PNH patients with a confirmed diagnosis of PNH by flow cytometry
Who may be able to join
- Patients with PNH confirmed by flow cytometry.
- Patient and/or parent/legally authorized representative provide written informed consent/assent to participate in the registry in a manner approved by the Institutional Review Board/Independent Ethics Committee and local regulations.
Who cannot join
- Participating in an interventional PNH clinical trial. Note: A patient included in the registry, who enrolls in an interventional PNH clinical trial during the course of the registry, will be kept in the registry but data collection will be paused in the registry during their involvement in the clinical trial/extension study. Data collection in the registry will continue after patient involvement in the clinical trial/extension study has ended or trial protocol mandated data collection ceases.
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
International PNH Interest Group
Altamonte Springs, Florida, 32714
IPIG Registry Coordinator, registry@pnhinterestgroup.org
About this study
The aim of this International PNH Interest Group (IPIG) registry is to develop an international database to prospectively collect data on patients with PNH covering clinical outcomes, patient reported outcomes (PROs), and health-resource utilization (HRU) on all enrolled patients, as well as long term safety data.
Read the detailed description
The International PNH Interest Group (IPIG) PNH Registry is an international, observational database collecting real-world health information about PNH patients over time. The registry is owned and managed by IPIG, a not-for-profit network of international PNH experts dedicated to improving care and treatment for PNH patients. Several pharmaceutical partners are collaborating with IPIG to fund the registry, which will provide data to support their post-approval regulatory commitments.
The IPIG PNH Registry aims to collect data about all PNH patients including clinical outcomes, patient reported outcomes and health resource usage, as well as long term safety data for patients treated with anti-complement therapies.
The IPIG PNH Registry is comprised of a 'core' PNH disease registry collecting a defined set of core variables on all patients with PNH. In addition, there are several product-specific 'silos' initiated by marketing authorization holders including patients treated with PNH-specific therapies. The silos collect both the core dataset and additional variables to address specific objectives or requests from regulatory authorities (e.g. post-authorization safety data).
Data is collected at enrollment and at 6 monthly intervals during follow-up.
This clinical trial information was submitted voluntarily under the applicable law and, therefore, certain submission deadlines may not apply. (That is, clinical trial information for this applicable clinical trial was submitted under section 402(j)(4)(A) of the Public Health Service Act and 42 CFR 11.60 and is not subject to the deadlines established by sections 402(j)(2) and (3) of the Public Health Service Act or 42 CFR 11.24 and 11.44).
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Keywords
- PNH, Registry, Patient Registry, Paroxysmal Nocturnal Hemoglobinuria, Anemia, Hemolytic, Anemia, Hematologic Diseases, Myelodysplastic Syndromes, Bone Marrow Diseases, Hemoglobinuria, Hemoglobinuria, Paroxysmal
- Medical subject headings
- Hemoglobinuria, Paroxysmal, Anemia, Hemolytic, Anemia, Hematologic Diseases, Myelodysplastic Syndromes, Bone Marrow Diseases, Hemoglobinuria
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 not receiving anti-complement treatment | PNH patients of any age who are not receiving any anti-complement treatment | |
| PNH patients receiving anti-complement treatment | PNH patients of any age who are receiving an approved anti-complement treatment |
What is being measured
The primary outcome is the main question the study is designed to answer.
Increase knowledge of PNH and describe the PNH patient population
To describe PNH disease natural history and PNH treatment course, the patterns of treatment usage, long-term safety of PNH treatments, assess patient reported quality of life and describe health resource usage
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 posted29 Jul 2024
- Study start10 May 2024
- Primary completion10 May 2029 (estimated)
- Study completion10 May 2029 (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
- 2,000 participants (estimated)
Sponsor and investigators
- Lead sponsor
- International PNH Interest Group
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Richard Kelly, MBChB PhD, Principal investigator, International PNH Interest GroupJeff Szer, MB BS FRACP, Study chair, International PNH Interest Group
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06524726
- Sponsor's protocol ID
- 5674-0001
- Status last verified
- Jul 2024
- First submitted
- 23 Jul 2024
- Last updated
- 29 Jul 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Aggregate information about the registry data will be made available to funding pharmaceutical partners and participating sites in the form of an annual report.
Study at a glance
From the registry record, last updated by the sponsor on 29 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The International PNH Interest Group PNH Registry
- ClinicalTrials.gov ID
- NCT06524726
- Acronym / sponsor ID
- 5674-0001
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Keywords
- PNH, Registry, Patient Registry, Paroxysmal Nocturnal Hemoglobinuria, Anemia, Hemolytic, Anemia, Hematologic Diseases, Myelodysplastic Syndromes
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 2,000 participants (estimated)
- Lead sponsor
- International PNH Interest GroupUniversity, hospital or other
- Study start
- 10 May 2024 (actual)
- Primary completion
- 10 May 2029 (estimated)
- Study completion
- 10 May 2029 (estimated)
- First posted
- 29 Jul 2024 (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 aim of this International PNH Interest Group (IPIG) registry is to develop an international database to prospectively collect data on patients with PNH covering clinical outcomes, patient reported outcomes (PROs), and health-resource utilization (HRU) on all enrolled patients, as well as long term safety data.
Detailed description
The International PNH Interest Group (IPIG) PNH Registry is an international, observational database collecting real-world health information about PNH patients over time. The registry is owned and managed by IPIG, a not-for-profit network of international PNH experts dedicated to improving care and treatment for PNH patients. Several pharmaceutical partners are collaborating with IPIG to fund the registry, which will provide data to support their post-approval regulatory commitments.
The IPIG PNH Registry aims to collect data about all PNH patients including clinical outcomes, patient reported outcomes and health resource usage, as well as long term safety data for patients treated with anti-complement therapies.
The IPIG PNH Registry is comprised of a 'core' PNH disease registry collecting a defined set of core variables on all patients with PNH. In addition, there are several product-specific 'silos' initiated by marketing authorization holders including patients treated with PNH-specific therapies. The silos collect both the core dataset and additional variables to address specific objectives or requests from regulatory authorities (e.g. post-authorization safety data).
Data is collected at enrollment and at 6 monthly intervals during follow-up.
This clinical trial information was submitted voluntarily under the applicable law and, therefore, certain submission deadlines may not apply. (That is, clinical trial information for this applicable clinical trial was submitted under section 402(j)(4)(A) of the Public Health Service Act and 42 CFR 11.60 and is not subject to the deadlines established by sections 402(j)(2) and (3) of the Public Health Service Act or 42 CFR 11.24 and 11.44).
Study design
- Observational model
- Other
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| PNH patients not receiving anti-complement treatment | – | –PNH patients of any age who are not receiving any anti-complement treatment |
| PNH patients receiving anti-complement treatment | – | –PNH patients of any age who are receiving an approved anti-complement treatment |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- PNH patients with a confirmed diagnosis of PNH by flow cytometry
Key inclusion criteria (2 listed)
- Patients with PNH confirmed by flow cytometry.
- Patient and/or parent/legally authorized representative provide written informed consent/assent to participate in the registry in a manner approved by the Institutional Review Board/Independent Ethics Committee and local regulations.
Key exclusion criteria (1 listed)
- Participating in an interventional PNH clinical trial. Note: A patient included in the registry, who enrolls in an interventional PNH clinical trial during the course of the registry, will be kept in the registry but data collection will be paused in the registry during their involvement in the clinical trial/extension study. Data collection in the registry will continue after patient involvement in the clinical trial/extension study has ended or trial protocol mandated data collection ceases.
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
Increase knowledge of PNH and describe the PNH patient population
Time frame: 5-10 years
To describe PNH disease natural history and PNH treatment course, the patterns of treatment usage, long-term safety of PNH treatments, assess patient reported quality of life and describe health resource usage
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 29 Jul 2024.
- IPIG WebsiteLink provided by the sponsor.
- Individual participant dataSharing planned: No. Aggregate information about the registry data will be made available to funding pharmaceutical partners and participating sites in the form of an annual report.
Related trials for Paroxysmal Nocturnal Hemoglobinuria
Enrolling by invitationObservational study
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Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells
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A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works
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Not yet recruitingPhase 3
A Study of Efficacy and Safety of FXS6837 in Anti-C5 Treated PNH Patients.
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Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis
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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.
Richard Kelly, MBChB PhD principal investigator
International PNH Interest Group
Jeff Szer, MB BS FRACP study chair
International PNH Interest Group
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 International PNH Interest Group 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 2 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 29 Jul 2024. 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 IPIG Registry Coordinator (Please email, registry@pnhinterestgroup.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Increase knowledge of PNH and describe the PNH patient 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 29 Jul 2024.