The primary objective is to evaluate the safety of eculizumab in patients with transfusion-dependent hemolytic PNH
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
85
Acceptable safety (adverse events [AEs], labs, electrocardiograms [ECGs], vital signs)
Primary surrogate of efficacy endpoint is hemolysis measured by LDH area under the curve.
Hemolysis measured by the change of LDH from baseline;
Quality of Life
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...and 38 more locations