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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
54
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.
Tangshan Central Hospital
Tangshan, Hebei, China
Zhoukou Central Hospital
Zhoukou, Henan, China
The Second Affilated Hospital of Shandong First Medical University
Tai’an, Shandong, China
Regenerative Medicine Center
Tianjin, Tianjin Municipality, China
Overall response rate
Percentage of patients with hematological response. Hematological response is evaluated by hemoglobin, platelet and neutrophil count in the routine blood test.
Time frame: 24 weeks
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.
Time frame: 24 weeks
Proportion of patients with abnormal karyotype changes
The abnormal karyotype was examined by karyotype test
Time frame: Baseline and 24 weeks
Time duration for patients achieving hematological response
Duration time was calculated from response to relapse.
Time frame: 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
Time frame: Baseline and 24 weeks
Incidence of the adverse event
Use Common Terminology Criteria for Adverse Events (CTCAE) Version 5 to assess the adverse event.
Time frame: 24 weeks
Severity of the adverse event
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Use Common Terminology Criteria for Adverse Events (CTCAE) Version 5 to assess the severity.
Time frame: 24 weeks