Study of Safety and Efficacy of MY008211A in Patients With Residual Anemia Despite Anti-C5 Antibody Treatment

Korábbi kezelés

• Previous bone marrow or hematopoietic stem cell transplantation.
• Previous splenectomy.

Más betegség

• History of recurrent invasive infections caused by encapsulated organisms,e.g. meningococcus or pneumococcus.
• A history of malignancy within 5 years before screening, except cured local basal cell carcinoma of the skin and carcinoma in situ of the cervix.

Laborértékek

• Patients with reticulocytes <100x10\^9/L; platelets <30x10\^9/L; neutrophils <0.5x10\^9/L.

Egyéb

• Known or suspected hereditary complement deficiency.

• 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.