Paroxysmal Nocturnal Hemoglobinuria
Conditions
Keywords
PNH, Paroxysmal Nocturnal hemoglobinuria, Soliris, Eculizumab, Ravulizumab, Ultomiris
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).
Interventions
None listed
Sponsors
Alexion Pharmaceuticals, Inc.
Study design
Observational model
OTHER
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
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with a diagnosis of PNH or a detected PNH clone, including patients previously treated with Soliris or Ultomiris and withdrawn from treatment.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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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. |
Countries
United States
Contacts
STUDY_DIRECTORPhillipe Gustovic
Alexion Pharmaceuticals, Inc.
Outcome results
None listed