Avatrombopag Usage in NSAA

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 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

, , 

Location Countries

China

Location Countries

China

NCT ID

NCT04728789

Organization ID

Avatrombopag-1

Responsible Party

Principal Investigator

Study Sponsor

Peking Union Medical College Hospital

Study Sponsor

, , 

Verification Date

January 2021