A Multicenter, Single-arm, Open-label Phase III Study to Evaluate Efficacy and Safety of MY008211A Tablets in Patients With PNH and Residual Anemia, Despite Treatment With Anti-C5 Antibody.
The main purpose of this study is to evaluate the efficacy of MY008211A in PNH patients with residual anemia despite treatment with anti-C5 antibody.
• 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%.
• Stable regimen of anti-C5 antibody treatment for at least 6 months before treatment, and Hb was still \< 100 g/L.
• The average hemoglobin level of at least two tests in 4 months before screening \< 100 g/L.
• The average hemoglobin level of two tests in the central laboratory during screening \< 100 g/L.
• 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.