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This is a prospective, single-center, single-arm, phase 2 study. This study aims to evaluate the efficacy and safety of Anti-lymphocyte globulin plus herombopag in combination with moderate-dose cyclophosphamide for severe aplastic anemia.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Experiemental | Experimental | ALG/CsA(Cyclosporine) and herombopag and moderate-dose cyclophosphamide |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Cyclophosphamide added to standard immunosuppressive therapy with herombopag | Drug | Severe aplastic anemia patients will receive a daily dose of Anti-lymphocyte globulin (25mg/kg) for the initial five days at the beginning of the treatment. Cyclosporine will be administered daily at a dosage of 3-5mg/kg. Herombopag will be administered daily at a dosage of 15mg starting from the first day of treatment and continuing for a duration of six months. The moderate-dose cyclophosphamide (20mg/kg) will be administered on days 29-30 and days 43-44. |
| Measure | Description | Time Frame |
|---|---|---|
| Overall response rate | Percentage of patients with hematological response. Hematological response includes complete rate, near complete rate(CR), very good partial response(VGPR), good partial response(GPR) and partial response(PR). | Within 3 months |
| Measure | Description | Time Frame |
|---|---|---|
| Robust response | Percentage of patients with robust response, including CR, near CR, VGPR and GPR. | Within 3 months |
| All-cause mortality | Within 3 months |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Liwei Fang, MD | Institute of Hematology & Blood Diseases Hospital, China | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Regenerative Medicine Center | Tianjin | Tianjin Municipality | 300131 | China |
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| Time to achieve robust hematological response | Within 6 months |
| Incidence of the adverse event | Use Common Terminology Criteria for Adverse Events (CTCAE) Version 5 to assess the adverse event | Within 6 months |
| ID | Term |
|---|---|
| D000741 | Anemia, Aplastic |
| ID | Term |
|---|---|
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D000080983 | Bone Marrow Failure Disorders |
| D001855 | Bone Marrow Diseases |
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