The International PNH Interest Group PNH Registry

Last updated: July 23, 2024
Sponsor: International PNH Interest Group
Overall Status: Active - Recruiting

Phase

N/A

Condition

Hemoglobinuria, Paroxysmal

Aplastic Anemia

Red Blood Cell Disorders

Treatment

N/A

Clinical Study ID

NCT06524726
5674-0001
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Patients with PNH confirmed by flow cytometry.

  • Patient and/or parent/legally authorized representative provide written informedconsent/assent to participate in the registry in a manner approved by theInstitutional Review Board/Independent Ethics Committee and local regulations.

Exclusion

Exclusion Criteria:

  • Participating in an interventional PNH clinical trial. Note: A patient included inthe registry, who enrolls in an interventional PNH clinical trial during the courseof the registry, will be kept in the registry but data collection will be paused inthe registry during their involvement in the clinical trial/extension study. Datacollection in the registry will continue after patient involvement in the clinicaltrial/extension study has ended or trial protocol mandated data collection ceases.

Study Design

Total Participants: 2000
Study Start date:
May 10, 2024
Estimated Completion Date:
May 10, 2029

Study 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).

Connect with a study center

  • International PNH Interest Group

    Altamonte Springs, Florida 32714
    United States

    Active - Recruiting

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