The main purpose of this study is to evaluate the efficacy of MY008211A in PNH patients with residual anemia despite treatment with anti-C5 antibody.
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%.
Stable regimen of anti-C5 antibody treatment for at least 6 months before treatment, and Hb was still < 100 g/L.
The average hemoglobin level of at least two tests in 4 months before screening < 100 g/L.
The average hemoglobin level of two tests in the central laboratory during screening < 100 g/L.
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.
Single group
MY008211A tablets
DrugMY008211A tablets Participants will receive MY008211A at a dose of 400 mg orally b.i.d
The proportion of Participants With Sustained Hemoglobin Levels of ≥ 120 g/L in the Absence of Red Blood Cell Transfusions (defined as no red blood cell infusion after D14 to D168)
The proportion of patients with sustained hemoglobin levels ≥ 120 g/L among those without RBC transfusion.
The proportion of subjects with an increase in hemoglobin concentration ≥ 20 g/L from baseline among subjects who do not receive RBC transfusion (defined as no red blood cell infusion after D14 to D168)
Proportion of participants achieving a sustained increase from baseline in hemoglobin levels of ≥ 20 g/L assessed among those without RBC transfusion.
Change From Baseline in Hemoglobin
Change in hemoglobin concentration from baseline in patients without RBC transfusion.
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
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