The purpose of this study is to evaluate the efficacy of HSK39297 tablets in paroxysmal nocturnal hemoglobinuria (PNH) patients with anemia after stable treatment of anti-C5 antibody.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
36
200mg QD
The First Affiliated Hospital of Nanjing Medical University
Nanjing, Jiangsu, China
The First Affiliated Hospital, College of Medicine, Zhejiang University
Hangzhou, Zhejiang, China
Proportion of participants achieving hemoglobin levels ≥ 120 g/L at least on three out of four measurements in the absence of red blood cell transfusions
Time frame: 18 to 24 weeks
Proportion of participants with increase in hemoglobin levels from baseline of ≥20 g/L at least on three out of four measurements in the absence of red blood cell transfusions
Time frame: 18 to 24 weeks
Percentage of patients who did not receive a blood transfusion.
Time frame: 18 to 24 weeks
Change from baseline in hemoglobin
Time frame: Baseline, week 18 to 24
Change From Baseline in Reticulocyte Count
Time frame: Baseline, week 18 to 24
Change from baseline in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue score
The FACIT-F is a 13-item questionnaire with a 0-52 range. A higher total score indicates less fatigue and better function.
Time frame: Baseline, week 18 to 24
Percent change from baseline in lactate dehydrogenase (LDH)
Time frame: Baseline, week 18 to 24
Rate of breakthrough hemolysis (BTH)
Time frame: 24 weeks
Proportion of participants with Major Adverse Vascular Events (MAVEs)
Time frame: 24 weeks
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