This was a phase 1b/2,open label, multi-center study to assess efficacy and safety of HS-10542 in adulte patients with paroxysmal nocturnal hemoglobinuria (PNH) with signs of active hemolysis.
Men or women aged more than or equal to (≥) 18 years, and less than (≤) 75 years.
It was confirmed to be PNH during screening, and the clone size of red blood cells or/and granulocytes or/and monocytes was detected by flow cytopy ≥10%
Stable use of C5 complement inhibitor ikuzumab/covalimab for the first 6 months of random treatment
Have at least one blood transfusion record within the last 4 months, or sustain a hemoglobin level below 100g/L the last 4 months prior to screening.
Known or suspected hereditary or acquired complement deficiency
Currently active primary or secondary immunodeficiency
History of infection with pod bacteria (such as Neisseris meningitis, Streptococcus pneumoniae, etc.)
Patients with laboratory evidence of bone marrow failure (reticulocytes <100x109/L; platelets <30x109/L; neutrophils <0.5x109/L);
Parallel
HS-10542
DrugHS-10542 low dose,QD
HS-10542
DrugHS-10542 high dose, QD
HS-10542
DrugOrally QD ，The recommended dose from Ph1b
Phase 1b: Incidence and severity of adverse events
Phase 2: In the absence of red blood cell infusion the proportion of subjects with at least 3 times of ≥120 g/L of hemoglobin level measured 4 times between weeks 18 and 24
Phase 1b: In the absence of red blood cell infusion the proportion of subjects of ≥120 g/L of hemoglobin level between weeks 3 and 12
Phase 1b: the Proportion of Participants With Sustained Increase in Hemoglobin Levels From Baseline of ≥ 20g/L in the Absence of Red Blood Cell Transfusions
Phase 1b: The proportion of subjects who did not receive red blood cell infusion from week 3 to week 12
Phase 1b: Change in LDH level from baseline
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