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.
The purpose of this open-label, single arm, multicenter study is to evaluate the long-term safety, tolerability and efficacy of MY008211A tablets in patients with PNH and to provide access to patients who have completed (without tapering down) Phase 2 and Phase 3 trials and derived benefit from MY008211A treatment.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
120
Participants will receive MY008211A at a dose of 400 mg orally b.i.d
Chinese Academy of Medical Sciences Hematology Hospital (Institute of Hematology, Chinese Academy of Medical Sciences)
Tianjin, Tianjin Municipality, China
RECRUITINGProportion 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.
Time frame: About 100 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.
Time frame: About 100 weeks
Change From Baseline in Hemoglobin
Change in hemoglobin concentration from baseline in patients without RBC transfusion every 12 weeks.
Time frame: About 100 weeks
The proportion of patients without RBC transfusion.
The proportion of patients without RBC transfusion.
Time frame: About 100 weeks
The Clinical BTH Rate
The Clinical BTH Rate
Time frame: About 100 weeks
The Major Adverse Vascular Events Rate
The Major Adverse Vascular Events Rate
Time frame: About 100 weeks
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