Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry

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

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

Official Title
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Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry

Conditions
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Paroxysmal Nocturnal Hemoglobinuria

Participation Criteria
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Eligibility Criteria
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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with a diagnosis of PNH or a detected PNH clone, including patients previously treated with Soliris or Ultomiris and withdrawn from treatment.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.
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Receiving Soliris or Ultomiris<br>PNH patients of any age, including minors, that are receiving Soliris or Ultomiris | |
| Not receiving Soliris or Ultomiris<br>PNH patients of any age, including minors, that are not receiving Soliris or Ultomiris | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | Ongoing (up to 13 years) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | Ongoing (up to 13 years) | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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PNH, Paroxysmal Nocturnal hemoglobinuria, Soliris, Eculizumab, Ravulizumab, Ultomiris