A Study to Evaluate the Safety and Efficacy of Hetrombopag Olamine in Severe Aplastic Anemia （SAA） Patient

Study Overview
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Brief Summary
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This is a Multicentre, Open-label, single-arm, Phase II Study to Evaluate the Safety and Efficacy of Hetrombopag Olamine in Patients With Severe Aplastic Anemia. 55 adult patients with SAA will be enrolled in the study. Treatment with Hetrombopag will be started at 7.5 mg/day and uptitrated according to the platelet count. The primary objective of the study is to assess the safety and efficacy of Hetrombopag in patients with SAA.

Official Title
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A Multicentre, Open-label, Single-arm, Phase II Study to Evaluate the Safety and Efficacy of Hetrombopag Olamine in Patients With Severe Aplastic Anemia

Conditions
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Severe Aplastic Anemia

Intervention / Treatment
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* Drug: Hetrombopag Olamine


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Previous diagnosis of refractory severe aplastic anemia, not meeting partial response criteria after at least one treatment course of immunosuppressive therapy, not suitable or unwilling to do hematopoietic stem cell transplantation. Platelet count ≤ 30×109/L. Signed informed consent. Exclusion Criteria: Bleeding and/or Infection not adequately responding to appropriate therapy. Patients with a PNH clone size in neutrophils of ≥50%. Treatment with immunosuppressive therapy within 6 months prior to study entry. Any laboratory or clinical evidence for HIV infection. Any clinical history for hepatitis C infection; chronic hepatitis B infection; or any evidence for active hepatitis at the time of subjects screening. ALT> 2.5 x upper limit of normal (ULN), AST> 2.5 x upper limit of normal (ULN) DBLI> 1.5 x upper limit of normal (ULN), Scr> upper limit of normal (ULN). Subjects diagnosed with cirrhosis or portal hypertension. Subjects diagnosed with tumor. Patients with any prior history of congestive heart failure, arrhythmia, and peripheral arteriovenous thrombosis within 1 year. With a history of myocardial infarction or cerebral infarction within 3 months. ECOG Performance Status of 3 or greater. Female subjects who are nursing or pregnant within 6 months. Subjects cannot take effective contraception. Subjects have participated in other clinical trial within the 3 months prior to study entry.

Ages Eligible for Study
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Minimum Age: 18 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
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Hetrombopag Olamine<br>Hetrombopag will be started at 7.5 mg/day and uptitrated according to the platelet count. | Drug: Hetrombopag Olamine<br>* once daily<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Proportion of Subjects With Hematological Response | Hematological Response is defined by changes in the platelet count or platelet transfusion requirements, hemoglobin levels or number of red blood cell transfusions, neutrophil counts or G-CSF usage requirements. | up to 18 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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SAA