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This trial is a multi-center, single-arm, open-label phase III clinical trial. A total of approximately 35 patients with paroxysmal nocturnal hemoglobinuria who remained anemic despite stable use of C5 complement inhibitor (eculizumab/Kevacumab) for the first 6 months before randomization were included in the study. Approximately 40% of the subjects had received at least one red blood cell (RBC) transfusion within the first 6 months before receiving the experimental intervention. Subjects who met the criteria were all treated with HRS-5965 capsules. This trial includes an 8-week screening period, a 24-week treatment period, a 2-week dose reduction period, and a 4-week safety follow-up period.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| HRS-5965 capsule | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| HRS-5965 capsule | Drug | HRS-5965 capsule treatment |
|
| Measure | Description | Time Frame |
|---|---|---|
| In the absence of red blood cell infusion (defined as no red blood cell infusion after week 2 to week 24), the proportion of subjects with at least 3 times of ≥12 g/dL of hemoglobin level measured 4 times between weeks 18 and 24. | From the 18th to the 24th week. |
| Measure | Description | Time Frame |
|---|---|---|
| The proportion of subjects who did not receive red blood cell infusion from week 2 to week 24 | From the 18th to the 24th week | |
| The average change of hemoglobin compared with baseline during weeks 18 to 24 | From the 18th to the 24th week |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Hematology Hospital of Chinese Academy of Medical Sciences | Tianjin | Tianjin Municipality | 30000 | 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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| The average percentage change of lactate dehydrogenase (LDH) level compared with the baseline during weeks 18 to 24 | From the 18th to the 24th week |
| The average change of reticular red blood cell count compared with the baseline during weeks 18 to 24 | From the 18th to the 24th week |
| Chronic Disease Treatment Function Assessment (FACIT) from weeks 18 to 24 - the average change of the score compared to the baseline | From the 18th to the 24th week |
| The incidence of breakthrough hemolysis from week 1 to week 24 | From the 18th to the 24th week |
| The incidence of MAVEs (including thrombosis) from week 1 to week 24 | From the 1st to the 24th week |
| The incidence and severity of adverse events from week 1 to week 24 | From the 1st to the 24th week |
| The concentration of HRS-5965 in plasma | From the 1st to the 24th week |
| The percentage of the change of complement bypass pathway (AP) activity at each point in time compared with the baseline | From the 1st to the 24th week |
| Tianjin Medical University General Hospital |
| Tianjin |
| Tianjin Municipality |
| 30000 |
| China |
| D009190 |
| Myelodysplastic Syndromes |
| D001855 | Bone Marrow Diseases |