This is a multicenter, randomized, open-label phase 2 study. Adult Patients with paroxysmal nocturnal hemoglobinuria naïve to complement inhibitor therapy were included. Subjects were treated with HSK39297 for 24 weeks.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
47
HSK39297 tablets for 24 weeks
The First Affiliated Hospital of Nanjing Medical University
Nanjing, Jiangsu, China
Proportion of participants with increase in hemoglobin levels from baseline of ≥20 g/L in the absence of red blood cell transfusions
Time frame: Baseline, 24 weeks
Proportion of participants with at least 60% reduction in LDH compared to baseline or LDH below the upper limit of normal
Time frame: Baseline, 24 weeks
Change from baseline in hemoglobin
Time frame: Baseline, 24 weeks
Change from baseline in reticulocyte count
Time frame: Baseline, 24 weeks
Change from baseline in LDH
Time frame: Baseline, 24 weeks
Change from baseline in Indirect bilirubin
Time frame: Baseline, 24 weeks
Change from baseline in free hemoglobin
Time frame: Baseline, 24 weeks
Proportion of participants without requiring red blood cells (RBC) transfusions
Time frame: From week 4 to week 24
Change in the average number of RBC transfused per week
Time frame: From week 4 to week 24
Change from baseline in PNH RBC clone size
Time frame: Baseline, 24 weeks
Change from baseline in C3 fragment deposition on PNH RBC
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Time frame: Baseline, 24 weeks
Change from baseline in FACIT-Fatigue score
Time frame: Baseline, 24 weeks
Incidence and severity of adverse events
Time frame: 28 weeks