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| Name | Class |
|---|---|
| Jiangsu Hengrui Pharmaceutical Co., Ltd. | INDUSTRY |
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This is a prospective, multicenter, single-arm, phase 2 trial. The aim of this study is to evaluate the efficacy and safety of herombopag combined with cyclosporine for patients with non severe aplastic anemia (NSAA).
This study aims to improve the 24 weeks response rate. The sample size is calculated based on Simon's two-stage design. The first stage of the study enrolled a cohort of 15 patients. If after 24 weeks at least 9 patients achieved a response, then enrollment was expanded to a total of 43 patients. The null hypothesis was unaccepted if more than 26 of 43 patients achieved the response. Accounting for a 20% dropout rate, the estimated final sample size was 54 patients.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| CsA + Herombopag | Experimental | Herombopag combined with cyclosporine |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Herombopag | Drug | Hetrombopag is a TPO receptor agonist approved in China in 2021 for idiopathic thrombocytopenic purpura (ITP) and second-line severe aplastic anemia (SAA). Indications of chemotherapy-induced thrombocytopenia (CIT), pediatric/juvenile ITP and naive severe aplastic anemia are under development. Hetrombopag was granted Orphan Drug Designation by FDA for the treatment of CIT. Cyclosporine A is a calcineurin inhibitor, which has an effect on reducing T-cell proliferation and activation, can reverse pancytopenia and alleviate transfusion requirements in NSAA. |
| Measure | Description | Time Frame |
|---|---|---|
| Overall response rate | Percentage of patients with hematological response. Hematological response is evaluated by hemoglobin, platelet and neutrophil count in the routine blood test. | 24 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Robust response rate | Percentage of patients with robust response, including complete response, near complete response, very good partial response(VGPR) and Meaningful partial response(mPR). These are evaluated by hemoglobin, platelet and neutrophil count in the routine blood test. | 24 weeks |
| Proportion of patients with abnormal karyotype changes |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Tangshan Central Hospital | Tangshan | Hebei | China | |||
| Zhoukou Central Hospital |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 41489450 | Derived | Zhang L, Li R, Liang Q, Li W, Pan H, Gao Z, Fang L, Zhao J, Yu X, Kuang Z, Nie N, Li J, Huang J, Zhao X, Ge M, Zheng Y, Li J, Zhang H, Shi J. Hetrombopag Added to Cyclosporine as the First-Line Treatment for Patients With Non-Severe Aplastic Anemia: A Phase 2 Multicenter Trial. Am J Hematol. 2026 Mar;101(3):467-476. doi: 10.1002/ajh.70183. Epub 2026 Jan 5. |
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| ID | Term |
|---|---|
| D016572 | Cyclosporine |
| ID | Term |
|---|---|
| D003524 | Cyclosporins |
| D010456 | Peptides, Cyclic |
| D047028 | Macrocyclic Compounds |
| D011083 | Polycyclic Compounds |
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The abnormal karyotype was examined by karyotype test |
| Baseline and 24 weeks |
| Time duration for patients achieving hematological response | Duration time was calculated from response to relapse. | A minimum of 2 years of planned follow-up |
| Change of the health-related quality of life | Medical Outcomes Study Questionnaire Short Form 36 Health Survey (SF-36) is used to assess the health-related quality of life of patients. The SF-36 has eight scaled scores; the scores are weighted sums of the questions in each section. Scores range from 0 - 100. Lower scores = more disability, higher scores = less disability | Baseline and 24 weeks |
| Incidence of the adverse event | Use Common Terminology Criteria for Adverse Events (CTCAE) Version 5 to assess the adverse event. | 24 weeks |
| Severity of the adverse event | Use Common Terminology Criteria for Adverse Events (CTCAE) Version 5 to assess the severity. | 24 weeks |
| Zhoukou |
| Henan |
| China |
| The Second Affilated Hospital of Shandong First Medical University | Tai’an | Shandong | China |
| Regenerative Medicine Center | Tianjin | Tianjin Municipality | China |
| D010455 |
| Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |