The International PNH Interest Group PNH Registry

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.

Study Overview

• Status: Recruiting
• Conditions: Paroxysmal Nocturnal Hemoglobinuria

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 Type: Observational
• Enrollment (Estimated): 2000

Contacts and Locations

• Study Contact: Name: IPIG Registry Coordinator; Phone Number: Please email; Email: registry@pnhinterestgroup.org

Study Locations

• United States
  • Florida
    • Altamonte Springs, Florida, United States, 32714
      • Recruiting
      • International PNH Interest Group
      • Contact: IPIG Registry Coordinator; Email: registry@pnhinterestgroup.org

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

PNH patients with a confirmed diagnosis of PNH by flow cytometry

Description

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 Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort
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 the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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	5-10 years

Collaborators and Investigators

• Sponsor: International PNH Interest Group
• Investigators: Principal Investigator: Richard Kelly, MBChB PhD, International PNH Interest Group; Study Chair: Jeff Szer, MB BS FRACP, International PNH Interest Group

Publications and helpful links

Helpful Links

• IPIG Website

Study record dates

Study Major Dates

• Study Start (Actual): May 10, 2024
• Primary Completion (Estimated): May 10, 2029
• Study Completion (Estimated): May 10, 2029

Study Registration Dates

• First Submitted: July 23, 2024
• First Submitted That Met QC Criteria: July 23, 2024
• First Posted (Actual): July 29, 2024

Study Record Updates

• Last Update Posted (Actual): July 29, 2024
• Last Update Submitted That Met QC Criteria: July 23, 2024
• Last Verified: July 1, 2024

More Information

Terms related to this study

• Keywords: Anemia; Registry; Hematologic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases; PNH; Paroxysmal Nocturnal Hemoglobinuria; Hemoglobinuria; Patient Registry; Anemia, Hemolytic; Hemoglobinuria, Paroxysmal
• Additional Relevant MeSH Terms: Urologic Diseases; Urological Manifestations; Bone Marrow Diseases; Hematologic Diseases; Urination Disorders; Anemia; Proteinuria; Anemia, Hemolytic; Myelodysplastic Syndromes; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Hemoglobinuria; Hemoglobinuria, Paroxysmal
• Other Study ID Numbers: 5674-0001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: Aggregate information about the registry data will be made available to funding pharmaceutical partners and participating sites in the form of an annual report.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No