Aplastic Anemia, Drug Effect
Conditions
Brief summary
This is a multicenter, open-label, prospective one arm study to explore the efficacy and safety of Avatrombopag in non-severe aplastic anemia. Patients meeting the inclusion and exclusion criteria would be recruited. Treatment of Avatrombopag would be started with 20mg/day. The dosage would be increased by 20 mg/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 dosage could range from 20mg/week to 60mg/day. All patients would receive treatment for at least 6 months except that the platelet \<20×10e9/L at the dosage of 60mg/day for 4 weeks or the platelet ≥200×10e9/L at the dosage of 20mg/week for 2 weeks. The hematological response rate and safety will be recorded and compared at every month after starting the study treatment. The patients would be followed up for at least 6 months.
Interventions
DRUGAvatrombopag 20 MG
Avatrombopag 20mg/week to 60mg/day, starting at 20mg/day and increased by 20 mg/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.
Sponsors
Peking Union Medical College Hospital
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
1. Patients diagnosed to be non-severe aplastic anemia 2. Patients have at least one of the followings: ①absolute neutrophil count \< 1.5×109/L, ②platelet count \< 30×109/L, ③ hemoglobin level \< 100g/L 3. 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); 4. Eastern Cooperative Oncology Group (ECOG) performance status 0-2; 5. Patients able to understand and comply with protocol requirements and instructions and have signed and dated informed consent.
Exclusion criteria
1. Congenital aplastic anemia; 2. Presence of chromosomal aberration; 3. Evidence of a clonal hematologic bone marrow disorder (MDS, AML) on cytogenetics; 4. Presence with PNH clone ≥50%; 5. Patients received HSCT before; 6. Uncontrolled infection or bleeding with standard treatment; 7. Allergic to Avatrombopag or accessories; 8. HIV, HCV or HBV active infection or liver cirrhosis or portal hypertension; 9. 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; 10. Have any concomitant malignancies within 5 years expect for local basal cell carcinoma of the skin; 11. Past history of thromboembolic event, heart attack or stroke (including anti-phospholipid antibody syndrome) and current use of anticoagulants; 12. Pregnant or nursing (lactating) woman; 13. Have attended other clinical trials within 3 months;
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| ORR at 6 Months | 6 months | 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
| Measure | Time frame | Description |
|---|---|---|
| ORR at 3 Months | 3 months | ORR will be calculated after 3 months of treatment by measuring platelet, reticulocyte, neutrophil and transfusion independence. |
| Percentage of patients with clonal evolution at 6 months | 6 months | Percentage of patients with clonal evolution would be evaluated by bone marrow biopsy at 6 months follow up. |
| percentage of side effects at 6 months | 6 months | percentage of side effects would be recorded during the study and be calculated according to CTCAE 5.0 at 6 months |
| Total volume of Platelet Transfusions | 1 month | Total volume of Platelet Transfusions |
| Total volume of Red Blood Cells Transfusions | 1 month | Total volume of Red Blood Cells Transfusions |
Countries
China
Outcome results
None listed