This is a multicenter, randomized, single-blind Phase II trial to evaluate the efficacy and safety of XH-S003 capsules in PNH patients. About 24 PNH patients will be enrolled and randomized to three dose levels and take XH-S003 capsules orally
Male or female with aged ≥18 years old;
Weight ≥40 kg and BMI≥18 kg/m2 ;
Diagnosed with PNH: with red blood cell or granulocyte clone levels >10% detected by flow cytopy within 6 months prior to screening or during screening;
Patients who have not previously received any complement inhibitor therayp;
Subjects with laboratory evidence of bone marrow failure during the screening period (reticulocyte count <100×109/L, platelet count <30×109/L, or neutrophil count <0.5×109/L);
A history of bone marrow/hematopoietic stem cell or solid organ transplantation;
Alanine aminotransferase (ALT), γ-glutamyl transpeptidase (GGT), or alkaline phosphatase (ALP) >3×ULN at screening; - Positive HIV antibody, active syphilis infection, positive HBsAg, active HCV infection, or active tuberculosis infection at screening;
Known or suspected immunodeficiency diseases or hereditary complement deficiency at screening;
Parallel
XH-S003 Capsule
Drug25mg \& 100mg
Changes of hemoglobin compared with baseline
Changes of hemoglobin compared with baseline
Proportion of subjects with an increase in Hb ≥20g/L compared with baseline (without RBC transfusion);
Proportion of subjects with an increase in Hb ≥20g/L compared with baseline (without RBC transfusion);
Changes in Indirect Bilirubin compared to baseline;
Changes in Indirect Bilirubin compared to baseline;
Changes in reticulocyte counts compared to baseline;
Changes in reticulocyte counts compared to baseline;
Proportion of subjects without RBC transfusion
Proportion of subjects without RBC transfusion
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