This is a multicenter, single-arm, open-label study to characterize long-term safety and tolerability of MY008211A tablets and to provide access to MY008211A tablets to patients with PNH who have completed Phase 2 or 3 studies with MY008211A tablets.
Patients who have previously received and completed MY008211A study treatment, and are judged by the investigator to have treatment benefit and may benefit from continued treatment of MY008211A.
Prior vaccinations against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae infections.
History of recurrent invasive infections caused by encapsulated organisms, e.g. meningococcus or pneumococcus.
Known or suspected hereditary complement deficiency.
Any comorbidity or medical condition (including but not limited to any active systemic bacterial, viral or fungal infection or malignancy) that, in the opinion of the investigator, could put the subject at increased risk or potentially confound study data.
Single group
MY008211A tablets
DrugParticipants will receive MY008211A at a dose of 400 mg orally b.i.d
Proportion of participants with adverse events, safety laboratory parameters, vital signs, ECG.
Safety evaluations including but not limited to adverse events, laboratory parameters, vital signs, ECG through End of Study visit every 12 weeks.
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 the absence of red blood cell transfusions evaluated every 12 weeks.
Change From Baseline in Hemoglobin
Change in hemoglobin concentration from baseline in patients without RBC transfusion every 12 weeks.
The proportion of patients without RBC transfusion.
The proportion of patients without RBC transfusion.
The Clinical BTH Rate
The Clinical BTH Rate
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