Aplastic Anemia, Drug Effect
Conditions
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.
Interventions
DRUGHetrombopag
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.
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 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 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 Hetrombopag 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 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
| Measure | Time frame | Description |
|---|---|---|
| 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. |
Countries
China
Contacts
STUDY_DIRECTORBing Han, Master
Peking Union Medical College Hospital
Outcome results
None listed