This trial is a multi-center, single-arm, open-label phase III clinical trial. A total of approximately 35 patients with paroxysmal nocturnal hemoglobinuria who remained anemic despite stable use of C5 complement inhibitor (eculizumab/Kevacumab) for the first 6 months before randomization were included in the study. Approximately 40% of the subjects had received at least one red blood cell (RBC) transfusion within the first 6 months before receiving the experimental intervention. Subjects who met the criteria were all treated with HRS-5965 capsules. This trial includes an 8-week screening period, a 24-week treatment period, a 2-week dose reduction period, and a 4-week safety follow-up period.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
39
HRS-5965 capsule treatment
Hematology Hospital of Chinese Academy of Medical Sciences
Tianjin, Tianjin Municipality, China
Tianjin Medical University General Hospital
Tianjin, Tianjin Municipality, China
In the absence of red blood cell infusion (defined as no red blood cell infusion after week 2 to week 24), the proportion of subjects with at least 3 times of ≥12 g/dL of hemoglobin level measured 4 times between weeks 18 and 24.
Time frame: From the 18th to the 24th week.
The proportion of subjects who did not receive red blood cell infusion from week 2 to week 24
Time frame: From the 18th to the 24th week
The average change of hemoglobin compared with baseline during weeks 18 to 24
Time frame: From the 18th to the 24th week
The average percentage change of lactate dehydrogenase (LDH) level compared with the baseline during weeks 18 to 24
Time frame: From the 18th to the 24th week
The average change of reticular red blood cell count compared with the baseline during weeks 18 to 24
Time frame: From the 18th to the 24th week
Chronic Disease Treatment Function Assessment (FACIT) from weeks 18 to 24 - the average change of the score compared to the baseline
Time frame: From the 18th to the 24th week
The incidence of breakthrough hemolysis from week 1 to week 24
Time frame: From the 18th to the 24th week
The incidence of MAVEs (including thrombosis) from week 1 to week 24
Time frame: From the 1st to the 24th week
The incidence and severity of adverse events from week 1 to week 24
Time frame: From the 1st to the 24th week
The concentration of HRS-5965 in plasma
Time frame: From the 1st to the 24th week
The percentage of the change of complement bypass pathway (AP) activity at each point in time compared with the baseline
Time frame: From the 1st to the 24th week
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.