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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| MY008211A tablets | Experimental | MY008211A tablets 400mg BID |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| MY008211A tablets | Drug | Participants will receive MY008211A at a dose of 400 mg orally b.i.d |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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. | About 100 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | About 100 weeks |
| Change From Baseline in Hemoglobin |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Wuhan Createrna Science and Technology Co.,Ltd | Contact | 027-68788900 | lcyxzx@createrna.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Chinese Academy of Medical Sciences Hematology Hospital (Institute of Hematology, Chinese Academy of Medical Sciences) | Recruiting | Tianjin | Tianjin Municipality | 300020 | China |
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| ID | Term |
|---|---|
| D006457 | Hemoglobinuria, Paroxysmal |
| ID | Term |
|---|---|
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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Change in hemoglobin concentration from baseline in patients without RBC transfusion every 12 weeks. |
| About 100 weeks |
| The proportion of patients without RBC transfusion. | The proportion of patients without RBC transfusion. | About 100 weeks |
| The Clinical BTH Rate | The Clinical BTH Rate | About 100 weeks |
| The Major Adverse Vascular Events Rate | The Major Adverse Vascular Events Rate | About 100 weeks |
|
| D009190 |
| Myelodysplastic Syndromes |
| D001855 | Bone Marrow Diseases |