The main purpose of this study is to evaluate the efficacy of MY008211A in adult patients with PNH, showing signs of active hemolysis.
Male and female participants ≥ 18 years of age and BMI ≥ 18.0 kg/m2 with a diagnosis of PNH confirmed by high-sensitivity flow cytometry with clone size ≥ 10%.
Mean hemoglobin level <100 g/L.
LDH > 1.5 x Upper Limit of Normal (ULN).
Vaccination against Neisseria meningitidis infection is required prior to the start of study treatment. If not received previously, vaccination against Streptococcus pneumoniae and Haemophilus influenzae infections should be given.
Patients with reticulocytes <100x10^9/L; platelets <30x10^9/L; neutrophils <0.5x10^9/L.
Were using a complement inhibitor before the first administration of MY008211A tablets or had discontinued a previous complement inhibitor for less than five half-lives or 120 days, whichever was the longest.
History of recurrent invasive infections caused by encapsulated organisms, e.g. meningococcus or pneumococcus.
Known or suspected hereditary complement deficiency.
Parallel
MY008211A tablets
Drugdose 1 (400 mg BID) and dose 2 (600 mg BID) in a 1:1 ratio by central randomization
The proportion of subjects with an increase in hemoglobin concentration ≥ 20 g/L from baseline among subjects who do not receive RBC transfusion after 4 weeks of dosing
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
The proportion of patients with an increase in hemoglobin ≥ 20 g/L from baseline among those without RBC transfusion
The proportion of patients with an increase in hemoglobin ≥ 20 g/L from baseline among those without RBC transfusion
The proportion of patients with hemoglobin ≥ 120 g/L among those without RBC transfusion
The proportion of patients with hemoglobin ≥ 120 g/L among those without RBC transfusion
Change in hemoglobin concentration from baseline in patients without RBC transfusion
Change in hemoglobin concentration from baseline in patients without RBC transfusion
Change in LDH level from baseline
Change in LDH level from baseline
Click on the lead sponsor to view all of their trials.
This trial is not recruiting at the moment. You can still explore other options:
