ClinicalTrials.gov record NCT06524726

The International PNH Interest Group PNH Registry

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Jul 27, 2026

The International PNH Interest Group PNH Registry

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.The registry record was last updated on Jul 29, 2024, about 2 years ago, so the status shown may be out of date. The study team or the registry can confirm.
Study typeObservationalA study in which researchers do not assign participants to a treatment or other intervention. They observe or measure what happens, which may include study visits, tests, questionnaires or a review of medical records.
SexAll
LocationUnited States
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This is an international, prospective patient registry for people with Paroxysmal Nocturnal Hemoglobinuria (PNH). It collects clinical outcomes, patient-reported outcomes, health-resource use, and long-term safety data, using a core dataset for all patients and product-specific 'silos' for those on PNH therapies. Data are recorded at enrollment and then every six months.

What it is studying. To build a long-term real-world dataset describing PNH disease course, patterns of treatment use, patient-reported quality of life, health-resource use, and safety of PNH therapies.

What the researchers hope to learn. Describe the natural history of PNH; characterize treatment patterns and long-term safety of PNH therapies; assess patient-reported quality of life and health-resource utilization to support research and regulatory post-approval commitments.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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.

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

  • 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.

Exclusion criteria

  • 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.

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Show the detailed description from the registry

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).

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

After giving consent, participants provide medical and health information at enrollment. Data are collected at enrollment and then every 6 months about clinical outcomes, patient-reported outcomes (quality of life), health-resource use, and long-term safety; patients treated with specific marketed therapies may have extra data collected in product-specific silos. If a participant later joins an interventional PNH trial, data collection for the registry is paused during that trial.

How the study is structured. A prospective observational registry: participants do not receive treatments from the study. The registry records a defined core set of data on all enrolled PNH patients; additional, product-specific silos collect extra data for patients treated with particular PNH therapies. Data are collected at enrollment and approximately every six months thereafter; the registry is managed by the International PNH Interest Group (IPIG).

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • This is an observational study, so the study itself gives no treatment. What would taking part involve for me, and would it change my usual care?
  • The listed sites are in United States. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on paroxysmal nocturnal hemoglobinuria

Clinical trials44 trials listed for paroxysmal nocturnal hemoglobinuriaEvery trial the registry lists for paroxysmal nocturnal hemoglobinuria, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in paroxysmal nocturnal hemoglobinuria researchLatest monthly update: September 2026. Includes: Treatment access. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
May 10, 2024
Primary completion
May 10, 2029 (estimated)
Study completion
May 10, 2029 (estimated)
Target enrollment
2000 participants (estimated)
Last updated on registry
Jul 29, 2024
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
International PNH Interest Group

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (1)

  • International PNH Interest GroupRecruiting

    Altamonte Springs, Florida, 32714 · United States

Terms used on this page · explanations by MedicalUpdates.ai

registry
An organised collection of information about people with a particular condition, gathered over time for research. Joining a registry usually means sharing medical information, not receiving a treatment.
legally authorized representative
A person who is legally allowed to make decisions, including consent to research, on behalf of someone who cannot consent for themselves.
written informed consent
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
assent
A child's or dependent adult's agreement to take part, given alongside the formal consent of a parent or legal representative.
interventional
A study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
extension study
A continuation study in which participants who completed an earlier study can keep receiving the treatment, usually with everyone knowing what they are given.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
enrollment
The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
follow-up
The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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