Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry

Alexion Pharmaceuticals, Inc.6,061 enrolled

Overview

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

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

Study Type

OBSERVATIONAL

Enrollment

6,061

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Eligibility

Sex: ALL

Medical Language ↔ Plain English

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with a diagnosis of PNH or a detected PNH clone, including patients previously treated with Soliris or Ultomiris and withdrawn from treatment.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.

Locations (1)

Contact the PNH Registry at Alexion Pharmaceuticals, Inc. for worldwide locations.

Boston, Massachusetts, United States

Outcomes

Primary Outcomes

Evaluate safety data specific to the use of Soliris and Ultomiris

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.

Time frame: Ongoing (up to 13 years)

Secondary Outcomes

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

Secondary analyses will include descriptions of patient populations, PNH specific treatments, concomitant medications, progression of disease, PNH clone sites, clinical symptoms, and clinical outcomes.

Time frame: Ongoing (up to 13 years)

Data from ClinicalTrials.gov

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