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| Name | Class |
|---|---|
| Qilu Pharmaceutical Co., Ltd. | INDUSTRY |
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This prospective, single center, single-arm study aims to evaluate the efficacy and safety of anti-human T lymphocyte porcine immunoglobulin (p-ALG) in combination with cyclosporine and Romiplostim in treating severe aplastic anemia (SAA). It will include 48 patients with newly diagnosed SAA.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| p-ALG+Romiplostim N01+CsA | Experimental | The maximum screening period was 4 weeks, and after entering the treatment period, the patients were treated with p-ALG combined with cyclosporine and Romiplostim N01. Symptomatic leukocyte elevation, blood transfusion support, and anti-infective therapy are allowed during this period, according to the patient's blood routine. If a patient has a serious adverse event during treatment, it will be treated according to the criteria for suspension and recovery. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| standard IST combined with Romiplostim N01 | Drug | Romiplostim N01 is a class of TPO receptor agonists. Its TPO peptide can bind to TPO receptors on megakaryocyte precursors in the bone marrow and activate them, triggering a series of intracellular signalling pathways, including JAK2/STAT5, PI3K/Akt, MEK/ERK, and p38, which ultimately lead to gene transcription and increased megakaryocyte proliferation and differentiation. |
| Measure | Description | Time Frame |
|---|---|---|
| Overall response rate | Percentage of patients with hematological response, including complete response (CR) or partial response (PR). Hematological response is evaluated by hemoglobin (Hb), platelet count (PLT) and absolute neutrophil count (ANC). | Within 27 weeks post treatment |
| Measure | Description | Time Frame |
|---|---|---|
| Overall response rate | Percentage of patients with hematological response, including complete response (CR) or partial response (PR). Hematological response is evaluated by hemoglobin (Hb), platelet count (PLT) and absolute neutrophil count (ANC). | Within 3 months post treatment |
| Time to hematological response |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Lele Zhang, PhD | Contact | 15811139278 | zhanglele@ihcams.ac.cn | |
| Meili Ge, PhD | Contact |
| Name | Affiliation | Role |
|---|---|---|
| Jun Shi, PhD | Institute of Hematology & Blood Diseases Hospital, China | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Regenerative Medicine Center and Red Blood Cell Disorders Center | Recruiting | Tianjin | Tianjin Municipality | China |
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Duration time was calculated from enrollment to achieve hematological response. |
| Within 27 weeks post treatment |
| Incidence of adverse events | Use Common Terminology Criteria for Adverse Events (CTCAE) Version 5 to assess the adverse event | Within 27 weeks post treatment |
| 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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