ALXN1210-PNH-303
A Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label, Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With Eculizumab
Study treatment
- Arm 1: Ravulizumab subcutaneous
- Arm 2: Ravulizumab intravenous
Inclusion criteria
- 18 years or older
- Treated with eculizumab according to the labeled dosing recommendation for PNH (900 mg every 14 days ± 2 days) for at least 6 months prior to study entry with no missed doses within 2 months prior to study entry and no more than 2 doses outside of the visit window
- Lactate dehydrogenase levels ≤ 1.5 × ULN
- Documented diagnosis of PNH confirmed by high-sensitivity flow cytometry evaluation
- Vaccinated against meningococcal infections within 3 years prior to, or at the time of, initiating study drug to reduce the risk of meningococcal infection (N meningitidis)
Exclusion criteria
- More than 1 LDH value > 2 × ULN within the 6 months prior to study entry
- Major adverse vascular event in the 6 months prior to study entry
- Platelet count < 30,000/mm3 (30 × 109/L) at Screening
- Absolute neutrophil count < 500/μL (0.5 × 109/L) at Screening
- History of bone marrow transplantation
- History of N meningitidis infection
Participating sites
- CHU UCL St-Luc