The International PNH Interest Group PNH Registry

NCT06524726 | Recruiting DMC

• 1 other identifier: 5674-0001
• Study Type: observational
• Target: 2,000
• Locations: 1 country
• Sites: 1

Brief 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.

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

Outcome Measures

Primary Outcomes (1)

• 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

Study Arms (2)

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

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

PNH patients with a confirmed diagnosis of PNH by flow cytometry

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• International PNH Interest Group: lead

Study Sites (1)

International PNH Interest Group

Altamonte Springs, Florida, 32714, United States

RECRUITING

Related Links

• IPIG Website

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal; Anemia, Hemolytic; Anemia; Hematologic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases; Hemoglobinuria

Condition Hierarchy (Ancestors)

Hemic and Lymphatic Diseases; Proteinuria; Urination Disorders; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Urological Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Officials

• Richard Kelly, MBChB PhD

  International PNH Interest Group

  PRINCIPAL INVESTIGATOR

• Jeff Szer, MB BS FRACP

  International PNH Interest Group

  STUDY CHAIR

Central Study Contacts

IPIG Registry Coordinator

CONTACT

Please email; registry@pnhinterestgroup.org

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 23, 2024
• First Posted: July 29, 2024
• Study Start: May 10, 2024
• Primary Completion (Estimated): May 10, 2029
• Study Completion (Estimated): May 10, 2029
• Last Updated: July 29, 2024

Record last verified: 2024-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)