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The main purpose of this study is to evaluate the efficacy of MY008211A compared to eculizumab in PNH patients.
This is a multi-center, randomized, open-label, active comparator-controlled, parallel group study. The purpose of this study is to determine whether MY008211A compared to eculizumab is efficacious and safe for the treatment of PNH patients who were naive to complement inhibitor therapy.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| MY008211A tablets | Experimental | MY008211A tablets 400mg BID |
|
| Eculizumab | Active Comparator | Eculizumab Injection |
|
| 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 for 24 weeks |
|
| Measure | Description | Time Frame |
|---|---|---|
| The proportion of Participants With Sustained Hemoglobin Levels of ≥ 120 g/L in the Absence of Red Blood Cell Transfusions | The proportion of patients with sustained hemoglobin levels ≥ 120 g/L among those without RBC transfusion (defined as no red blood cell infusion after D14 to D168). | between Day 126 and Day 168 |
| Measure | Description | Time Frame |
|---|---|---|
| The proportion of subjects with an increase in hemoglobin concentration ≥ 20 g/L from baseline among subjects who do not receive RBC transfusion | Proportion of participants achieving a sustained increase from baseline in hemoglobin levels of ≥ 20 g/L assessed among those without RBC transfusion (defined as no red blood cell infusion after D14 to D168). | between Day 126 and Day 168 |
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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 | +86 | lcyxzx@createrna.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Peking Union Medical College Hospital, Chinese Academy of Medical Sciences | Not yet recruiting | Beijing | Beijing Municipality | 100032 | China |
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| Eculizumab Injection | Drug | Eculizumab Injection for 24 weeks |
|
| The proportion of patients with LDH < 1.5 ULN among those without RBC transfusion. | The proportion of patients with hemolysis controlled (defined as LDH < 1.5 ULN) among those without RBC transfusion | between Day 126 and Day 168 |
| Change (Expressed as Percentages) in LDH level from baseline | Change (Expressed as Percentages) in LDH level from baseline | between Day 126 and Day 168 |
| Change in reticulocyte count from baseline | Change in reticulocyte count from baseline | between Day 126 and Day 168 |
| The proportion of patients without RBC transfusion | The proportion of patients without RBC transfusion | between Day 14 and Day 168 |
| Change in FACIT-F score from baseline | Change from baseline in FACIT-Fatigue scores. The FACIT-Fatigue is a 13-item questionnaire with support for its validity and reliability in PNH that assesses patient self-reported fatigue and its impact on daily activities and function. All FACIT scales are scored so that a high score is better. As each of the 13 items of the FACIT-F Scale ranges from 0-4, the range of possible scores is 0-52, with 0 being the worst possible score and 52 the best. | between Day 126 and Day 168 |
| The Clinical BTH Rate | The Clinical BTH Rate | between Day 1 and Day 168 |
| The Major Adverse Vascular Events Rate | The Major Adverse Vascular Events Rate | between Day 1 and Day 168 |
| Chinese Academy of Medical Sciences Hematology Hospital | Recruiting | Tianjin | Tianjin Municipality | 300020 | China |
|
| ID | Term |
|---|---|
| D006457 | Hemoglobinuria, Paroxysmal |
| ID | Term |
|---|---|
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D009190 | Myelodysplastic Syndromes |
| D001855 | Bone Marrow Diseases |
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| ID | Term |
|---|---|
| C481642 | eculizumab |
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