The main purpose of this study is to evaluate the efficacy of MY008211A in adult patients with PNH , showing signs of active hemolysis, in China.
The purpose of this study is to determine whether MY008211A is efficacious and safe for the treatment of PNH patients who are naive to complement inhibitor therapy, including anti-C5 antibody.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
34
The first 10 participants will be received low-dose MY008211A tablets, and the next 30 participants will be randomized to low-dose or high-dose treatment arms in a 1:2 ratio.
Institute of Hematology & Blood Disease Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College
Tianjin, Tianjin Municipality, China
Proportion of participants achieving a sustained increase in hemoglobin levels of ≥ 20 g/L in the absence of red blood cell transfusion.
Proportion of participants achieving a sustained increase from baseline in hemoglobin levels of ≥ 20 g/L assessed , in the absence of red blood cell transfusions
Time frame: up to 84 days
Proportion of participants achieving sustained hemoglobin levels ≥ 120 g/L in the absence of red blood cell transfusions.
Proportion of participants achieving sustained hemoglobin levels ≥ 120 g/L in absence of red blood cell transfusion
Time frame: up to 84 days
Change from baseline in hemoglobin concentration.
Change from baseline in hemoglobin concentration (g/L) in absence of red blood cell transfusion
Time frame: up to 84 days
Change from baseline in serum LDH levels.
Change from baseline in serum LDH levels (U/L)
Time frame: up to 84 days
Change from baseline in Reticulocyte count.
Change from baseline in Reticulocyte count (×10\^9/L)
Time frame: up to 84 days
Changes from baseline in transfusion volume.
The average number of red blood cells transfused per week
Time frame: up to 84 days
Change in the level of PNH red cell clones.
Change from baseline in the level of PNH red cell clones.
Time frame: up to 84 days
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Occurrences of AEs occurring between Day 1 and Day 84.
Adverse Events (AEs)
Time frame: up to 84 days