An open label study to evaluate the efficacy and safety of FXS6837 capsules compared to eculizumab in naive PNH patients. About 90 PNH patients who are naive to complement inhibitor therapies will be enrolled to take FXS6837 capsules or eculizumab for 24 weeks according to protocol.
Male or female subjects aged ≥ 18 years at screening;
Body weight ≥ 40 kg and body mass index (BMI) ≥ 18 kg/m2 at screening;
Diagnosis of PNH by the investigator according to the PNH diagnostic criteria, with red blood cell and white blood cell (monocytes or neutrophil) clone levels > 10% detected by high sensitivity flow cytometry within 6 months before screening or during the screening period;
PNH patients with no prior treatment involving any complement inhibitors;
With laboratory evidence of bone marrow failure during screening (reticulocyte count < 100 × 109/L, or platelet count < 30 × 109/L, or neutrophil count < 0.5 × 109/L); Received acute treatment (such as platelet transfusion, granulocyte colony-stimulating factor) for thrombocytopenia or neutropenia within 30 days before screening;
Erythropoietin for at least 8 weeks
Immunosuppressants for at least 8 weeks, systemic corticosteroids (≤ 15 mg/day) for at least 4 weeks
Iron, vitamin B12, or folic acid supplementation for at least 4 weeks
Parallel
FXS6837 Capsule
DrugFXS6837 capsule orally taken for 24 weeks
Eculizumab infusion
Biological/VaccineEculizumab infusion treated for 24 weeks
Proportion of participants with hemoglobin (Hb) level ≥ 120 g/L in at least 3 of 4 measurements between W18 and W24 in the absence of RBC transfusion (defined as no RBC transfusion after W2 through W24)
Proportion of participants with an increase from baseline in Hb level of ≥ 20 g/L in at least 3 of 4 measurements between W18 and W24 in the absence of RBC transfusion (defined as no RBC transfusion after Week 2 through Week 24)
Proportion of subjects without RBC transfusion during the treatment period from Week 2 to Week 24
Change from baseline in Hb during the treatment period from Week 18 to Week 24
Change from baseline in reticulocyte count during the treatment period from Week 18 to Week 24
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