A Multicenter, Randomized, Single-blind Phase II Study Evaluating the Efficacy and Safety of XH-S003 Capsules in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
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;
• LDH \> 1.5×ULN detected two times during the screening period (interval of 2 to 8 weeks);
• Hb meets one of the following conditions: (1) Hb \<100 g/L at the first screening visit, and subjects receive RBC transfusion because of PNH-related anemia during the screening period; (2) The average Hb of two tests during the screening period \<100 g/L (interval of 2\
⁃ 8 weeks);
• Vaccination against Neisseria meningitidis and Streptococcus pneumoniae before the first administration. If the subject has not been vaccinated previously or requires booster vaccination (according to local vaccination policies), vaccination must be administered at least 2 weeks before the first administration. If the first administration must begin less than 2 weeks after vaccination, preventive antibiotic treatment must begin at least 2 weeks after vaccination;