Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry

Completed

• Conditions: Paroxysmal Nocturnal Hemoglobinuria

• Registration Number: NCT01374360
• Lead Sponsor: Alexion Pharmaceuticals, Inc.

Brief Summary

This study is a collection of data to evaluate safety and characterize progression of Paroxysmal Nocturnal Hemoglobinuria (PNH).

Detailed Description

Collection of data to evaluate safety and characterize progression of Paroxysmal Nocturnal Hemoglobinuria (PNH).

Study Timeline

• Study Start: 2004-10-29
• Study First Submitted: 2011-04-15
• Study First Submitted QC: 2011-06-15
• Study First Posted: 2011-06-16
• Primary Completion: 2024-08-06
• Study Completion: 2024-08-06
• Status Verified: 2025-02
• Last Update Submitted: 2025-02-21
• Last Update Posted: 2025-02-24

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 6061

Inclusion Criteria

• Patients of any age, including minors, with a diagnosis of PNH or a detected PNH clone, including patients previously treated with Soliris or Ultomiris and withdrawn from treatment. Patients who are minors must have parent/legal guardian consent and must be willing and able to give assent, if applicable as determined by the Ethics Committees/Institutional Review Boards. Upon attaining adulthood, these patients must be re-consented.
• Ability to comprehend and sign consent to have data entered in the PNH Registry.

Exclusion Criteria

• Inability or unwillingness to sign informed consent.
• Patients currently enrolled in an interventional clinical trial for treatment of PNH cannot be enrolled in the Registry at the same time.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Evaluate safety data specific to the use of Soliris and Ultomiris	Ongoing (up to 13 years)	Primary analyses will assess safety endpoints, including occurrence and time to first event for the following: meningococcal infections, infections with serious outcomes, formation of human anti-drug antibodies (ADA) to Soliris and Ultomiris, malignancy, thrombotic events, pulmonary hypertension, impaired renal function, impaired hepatic function, hemolysis, pregnancies, lactation, infusion reactions, bone marrow transplant, serious adverse events, and mortality.

Secondary Outcome Measures

Name	Time	Method
Collect data to characterize the progression of PNH as well as clinical outcomes, mortality and morbidity in Soliris or Ultomiris and non-Soliris or non- Ultomiris treated patients	Ongoing (up to 13 years)	Secondary analyses will include descriptions of patient populations, PNH specific treatments, concomitant medications, progression of disease, PNH clone sites, clinical symptoms, and clinical outcomes.

Trial Locations

Locations (1)

Contact the PNH Registry at Alexion Pharmaceuticals, Inc. for worldwide locations.; 🇺🇸 Boston, Massachusetts, United States