Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry
The Registry is a prospective, multi-center, multi-national, non-interventional study that has been designed to capture safety and effectiveness data for Soliris, as well as to compile data on the natural history and management of patients with PNH. The Registry will enroll PNH patients treated or not with Soliris. It is anticipated that patients will be followed at least for 5 years. Data collected in the Registry will be reported to the FDA and EMA.
This registry has been designed and developed by the Registry Sponsor with assistance from ICON Clinical Research (The Registry Program Manager and amp;quot;) and Phase Forward, including its agents, and affililates.
Patients with a diagnosis of PNH or a detected PNH clone, whether treated or not with Soliris will be eligible for enrollment subsequent to providing informed consent. PNH diagnosis is left to Physician discretion (i.e. all diagnostic methods accepted). A detected PNH clone is defined by the identification of GPI-deficient granulocytes, GPI deficient erythrocytes or both. GPI-deficiency can be classified as partial (Type II) or complete (Type III). Patients with any combination of Type II or Type III clones/cells are eligible for enrollment into a registry. A minimum number of PNH cells at a level of .01% is required.