The main purpose of this study is to evaluate the efficacy of MY008211A compared to eculizumab in PNH patients.
Male and female participants ≥ 18 years of age and BMI ≥ 18.0 kg/m2 with a diagnosis of PNH confirmed by high-sensitivity flow cytometry with clone size ≥ 10%.
Mean hemoglobin level <100 g/L at screening.
LDH > 1.5 x Upper Limit of Normal (ULN) at screening.
Vaccination against Neisseria meningitidis infection is required prior to the start of study treatment. If not received previously, vaccination against Streptococcus pneumoniae and Haemophilus influenzae infections should be given.
Patients with reticulocytes <100x10^9/L; platelets <30x10^9/L; neutrophils <0.5x10^9/L.
History of recurrent invasive infections caused by encapsulated organisms,e.g. meningococcus or pneumococcus.
Known or suspected hereditary complement deficiency.
Previous bone marrow or hematopoietic stem cell transplantation.
Parallel
MY008211A tablets
DrugParticipants will receive MY008211A at a dose of 400 mg orally b.i.d for 24 weeks
Eculizumab Injection
DrugEculizumab Injection for 24 weeks
The proportion of Participants With Sustained Hemoglobin Levels of ≥ 120 g/L in the Absence of Red Blood Cell Transfusions
The proportion of patients with sustained hemoglobin levels ≥ 120 g/L among those without RBC transfusion (defined as no red blood cell infusion after D14 to D168).
The proportion of subjects with an increase in hemoglobin concentration ≥ 20 g/L from baseline among subjects who do not receive RBC transfusion
Proportion of participants achieving a sustained increase from baseline in hemoglobin levels of ≥ 20 g/L assessed among those without RBC transfusion (defined as no red blood cell infusion after D14 to D168).
The proportion of patients with LDH < 1.5 ULN among those without RBC transfusion.
The proportion of patients with hemolysis controlled (defined as LDH \< 1.5 ULN) among those without RBC transfusion
Change (Expressed as Percentages) in LDH level from baseline
Change (Expressed as Percentages) in LDH level from baseline
Change in reticulocyte count from baseline
Change in reticulocyte count from baseline
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