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The purpose of this study is to evaluate the efficacy of HSK39297 tablets in paroxysmal nocturnal hemoglobinuria (PNH) patients with anemia after stable treatment of anti-C5 antibody.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| HSK39297 | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| HSK39297 tablets | Drug | 200mg QD |
|
| Measure | Description | Time Frame |
|---|---|---|
| Proportion of participants achieving hemoglobin levels ≥ 120 g/L at least on three out of four measurements in the absence of red blood cell transfusions | 18 to 24 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Proportion of participants with increase in hemoglobin levels from baseline of ≥20 g/L at least on three out of four measurements in the absence of red blood cell transfusions | 18 to 24 weeks | |
| Percentage of patients who did not receive a blood transfusion. |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| The First Affiliated Hospital of Nanjing Medical University | Nanjing | Jiangsu | China | |||
| The First Affiliated Hospital, College of Medicine, Zhejiang University |
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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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| 18 to 24 weeks |
| Change from baseline in hemoglobin | Baseline, week 18 to 24 |
| Change From Baseline in Reticulocyte Count | Baseline, week 18 to 24 |
| Change from baseline in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue score | The FACIT-F is a 13-item questionnaire with a 0-52 range. A higher total score indicates less fatigue and better function. | Baseline, week 18 to 24 |
| Percent change from baseline in lactate dehydrogenase (LDH) | Baseline, week 18 to 24 |
| Rate of breakthrough hemolysis (BTH) | 24 weeks |
| Proportion of participants with Major Adverse Vascular Events (MAVEs) | 24 weeks |
| Hangzhou |
| Zhejiang |
| China |
| D009190 |
| Myelodysplastic Syndromes |
| D001855 | Bone Marrow Diseases |