Brief Title
Avatrombopag Usage in NSAA
Official Title
Efficacy and Safety of Avatrombopag in Non-severe Aplastic Anemia - a Multicenter Prospective Single Arm Study
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.
Study Phase
Phase 2/Phase 3
Study Type
Interventional
Primary Outcome
ORR at 6 Months
Secondary Outcome
ORR at 3 Months
Condition
Aplastic Anemia
Intervention
Avatrombopag 20 MG
Study Arms / Comparison Groups
Avatrombopag treatment group
Description: 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.
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
40
Start Date
February 1, 2021
Completion Date
June 30, 2023
Primary Completion Date
December 31, 2022
Eligibility Criteria
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;
Gender
All
Ages
18 Years - 75 Years
Accepts Healthy Volunteers
No
Contacts
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Location Countries
China
Location Countries
China
Administrative Informations
NCT ID
NCT04728789
Organization ID
Avatrombopag-1
Responsible Party
Principal Investigator
Study Sponsor
Peking Union Medical College Hospital
Study Sponsor
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Verification Date
January 2021