Efficacy and Safety of Hetrombopag in Non-severe Aplastic Anemia
Study Details
Study Description
Brief Summary
This is a prospective one arm study to explore the efficacy and safety of Hetrombopag in non-severe aplastic anemia. Patients meeting the inclusion and exclusion criteria would be recruited. Treatment of Hetrombopag would be started with 5mg/day. The dosage would be increased by 2.5mg/day every 2 weeks if the platelet count remains less than 20×10e9/L and reduced if the platelet count reaches over than 150×10e9/L. The maximum dosage is 15mg/day. All patients would receive treatment for at least 6 months except that the platelet <20×10e9/L at the dosage of 15mg/day for 4 weeks or the platelet ≥200×10e9/L at the dosage of 5mg/week for 2 weeks. The hematological response rate and safety will be recorded and compared at D15, 1month, 1.5month, 2month, 3month, 4month, 5month, 6month, 8month, 10month and 1year.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
| Phase 2/Phase 3 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: treatment group Hetrombopag would be started with 5mg/day. The dosage would be increased by 2.5mg/day every 2 weeks if the platelet count remains less than 20×10e9/L and reduced if the platelet count reaches over than 150×10e9/L. The maximum dosage is 15mg/day. |
Drug: Hetrombopag
Hetrombopag starting at 5mg/day and increased by 2.5mg/day every 2 weeks if the platelet count remains less than 20×10e9/L and reduced if the platelet count reaches over than 150×10e9/L.
|
Outcome Measures
Primary Outcome Measures
- ORR at 6 Months [6 month]
Overall Response Rate (ORR) Defined as the Number of Participants Who Met the Criteria of Either Complete Response (CR) or Partial Response (PR) at 6 months
Secondary Outcome Measures
- ORR at 3 Months [3 month]
ORR will be calculated after 3 months of treatment by measuring platelet, reticulocyte, neutrophil and transfusion independence.
- Volume of Platelet Transfusions [12 months]
Volume of Platelet Transfusions every month
- percentage of side effects at 12 months [12 months]
percentage of side effects would be recorded during the study and be calculated according to CTCAE 5.0 at 12 months
- Percentage of patients with clonal evolution at 12 months [12 months]
Percentage of patients with clonal evolution would be evaluated by bone marrow biopsy at 12 months follow up.
Eligibility Criteria
Criteria
Inclusion Criteria:
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Patients diagnosed to be non-severe aplastic anemia
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Patients with platelet count < 30×109/L and have at least one of the followings: ①absolute neutrophil count < 1.5×109/L, ②platelet count < 50×109/L, ③ hemoglobin level < 100g/L
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Patients have no response or relapsed following at least one treatment course in a period time of > 6 months of immunosuppression containing CsA or CsA+anti-thymocyte globulin (ATG);
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Eastern Cooperative Oncology Group (ECOG) performance status 0-2;
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Patients able to understand and comply with protocol requirements and instructions and have signed and dated informed consent.
Exclusion Criteria:
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Congenital aplastic anemia;
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Presence of chromosomal aberration;
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Evidence of a clonal hematologic bone marrow disorder (MDS, AML) on cytogenetics;
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Presence with PNH clone ≥50%;
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Patients received HSCT before;
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Uncontrolled infection or bleeding with standard treatment;
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Allergic to Hetrombopag or accessories;
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HIV, HCV or HBV active infection or liver cirrhosis or portal hypertension;
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Patient with QTcF (Fridericia's QT correction formula) at screening <450 msec, or<480 msec with bundle branch block, as determined via the mean of a triplicate ECG and assessed at site, unstable angina pectoris, uncontrolled hypertension(>180/100mmHg)，pulmonary artery hypertension;
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Have any concomitant malignancies within 5 years expect for local basal cell carcinoma of the skin;
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Past history of thromboembolic event, heart attack or stroke (including anti-phospholipid antibody syndrome) and current use of anticoagulants;
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Pregnant or nursing (lactating) woman;
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Have attended other clinical trials within 3 months;
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Peking Union Medical College Hospital | Beijing | China | 100730 |
Sponsors and Collaborators
- Peking Union Medical College Hospital
Investigators
- Study Director: Bing Han, Master, Peking Union Medical College Hospital
Study Documents (Full-Text)
None provided.More Information
Publications
- HBP-NSAA-001