A Study of MY008211A in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Korábbi kezelés

• 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.
• 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, BMI≥18 kg/m2，with a diagnosis of PNH confirmed by laboratory tests, according to the PNH diagnostic criteria in the Chinese Guidelines for the Diagnosis and Treatment of Rare Diseases (2019 edition) , and 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.