Paroxysmal Nocturnal Hemoglobinuria (PNH)
Conditions
Brief summary
Safety evaluations including but not limited to adverse events/serious adverse events, safety laboratory parameters, vital signs, etc., through End of Study visit
Detailed description
Response defined as maintaining sustained hemoglobin levels ≥ 12 g/dL in the absence of transfusions evaluated over yearly follow up intervals, Absence of administration of packed-red blood cell transfusions evaluated over yearly follow up intervals, Occurrences of breakthrough hemolysis and of Major Adverse Vascular Events, MAVE occurring evaluated over yearly follow up intervals
Interventions
Sponsors
Eligibility
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety evaluations including but not limited to adverse events/serious adverse events, safety laboratory parameters, vital signs, etc., through End of Study visit | — |
Secondary
| Measure | Time frame |
|---|---|
| Response defined as maintaining sustained hemoglobin levels ≥ 12 g/dL in the absence of transfusions evaluated over yearly follow up intervals, Absence of administration of packed-red blood cell transfusions evaluated over yearly follow up intervals, Occurrences of breakthrough hemolysis and of Major Adverse Vascular Events, MAVE occurring evaluated over yearly follow up intervals | — |
Countries
Czechia, France, Germany, Italy, Lithuania, Netherlands, Spain