Extension Study of Hetrombopag in Severe Aplastic Anemia

Study Overview
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Brief Summary
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This is a multicenter, placebo-control, phase 3 study of hetrombopag in patients with treatment-naive severe aplastic anemia. All subjects who have completed or withdrawn from the HR-TPO-SAA-III study will voluntarily participate in this extension study. Subjects will receive the same study drug (hetrombopag or placebo) as in study HR-TPO-SAA-III, with the same doses and administration schedule or with modifications based on the actual conditions. The primary objective of this extension study is to give the subjects participating in the HR-TPO-SAA-III study the continued access to the study drug (hetrombopag or placebo) after the completion of the HR-TPO-SAA-III study.

Official Title
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Extension Study of Hetrombopag Olamine in Patients With Treatment-naive Severe Aplastic Anemia

Conditions
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Treatment-naive Severe Aplastic Anemia

Intervention / Treatment
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* Drug: Hetrombopag Olamine
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Subjects who have completed or withdrawn from the HR-TPO-SAA-III study Subjects who have signed the informed consent form Female and male subjects of childbearing age who agree to take adequate contraceptive measures during the extension study period and within 28 days after the last dose Subjects who have completed the end-of-treatment evaluation in the original study Exclusion Criteria: Any unstable situation or situation that will compromise the safety of the subject Evidence of clonal cytogenetic abnormalities at the end-of-treatment examination of the HR-TPO-SAA-III study Subjects with uncontrollable hemorrhage and/or infection after standard treatment Subjects who have experienced deep vein thrombosis, myocardial infarction, stroke, or peripheral arterial embolism within 1 year Any situation that may compromise the subject and the safety or compliance thereof during the study

Ages Eligible for Study
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Minimum Age: 15 Years
Maximum Age: 75 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Hetrombopag Olamine<br> | Drug: Hetrombopag Olamine<br>* Hetrombopag Olamine; once daily<br>|
| Placebo Comparator: Placebo<br> | Drug: Placebo<br>* Placebo; once daily<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The ratio of subjects with clonal evolution at 6 months and 18 months |  | 6 months and 18 months | 
| All SIEs, regardless of whether they are related to the investigational product |  | 3 years | 
| All SAEs, regardless of whether they are related to the investigational product |  | 3 years | 
| All AEs resulting in discontinuation and withdrawal from study |  | 3 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Red blood cell count |  | by 1day visit | 
| Hemoglobin |  | by 1day visit | 
| Platelet count |  | by 1day visit | 
| White blood cell count |  | by 1day visit | 
| Neutrophil count |  | by 1day visit | 
| Reticulocyte count |  | by 1day visit | 
| The 6-month and 18-month survival rates of subjects |  | 6 months and 18 months | 
| The recurrence rates at 6 months and 18 months |  | 6 months and 18 months |