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Avatrombopag Usage in NSAA

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ClinicalTrials.gov Identifier: NCT04728789
Recruitment Status : Not yet recruiting
First Posted : January 28, 2021
Last Update Posted : January 28, 2021
Sponsor:
Information provided by (Responsible Party):
Bing Han, Peking Union Medical College Hospital

Study Description
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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.

Condition or disease Intervention/treatment Phase
Aplastic Anemia Drug Effect Drug: Avatrombopag 20 MG Phase 2 Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 40 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Efficacy and Safety of Avatrombopag in Non-severe Aplastic Anemia - a Multicenter Prospective Single Arm Study
Estimated Study Start Date : February 1, 2021
Estimated Primary Completion Date : December 31, 2022
Estimated Study Completion Date : June 30, 2023

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Arms and Interventions
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Arm Intervention/treatment
Experimental: Avatrombopag treatment group
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.
 Drug: Avatrombopag 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.


Outcome Measures
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Primary Outcome Measures :
  1. ORR at 6 Months [ Time Frame: 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 Outcome Measures :
  1. ORR at 3 Months [ Time Frame: 3 months ]
    ORR will be calculated after 3 months of treatment by measuring platelet, reticulocyte, neutrophil and transfusion independence.

  2. Percentage of patients with clonal evolution at 6 months [ Time Frame: 6 months ]
    Percentage of patients with clonal evolution would be evaluated by bone marrow biopsy at 6 months follow up.

  3. percentage of side effects at 6 months [ Time Frame: 6 months ]
    percentage of side effects would be recorded during the study and be calculated according to CTCAE 5.0 at 6 months

  4. Total volume of Platelet Transfusions [ Time Frame: 1 month ]
    Total volume of Platelet Transfusions

  5. Total volume of Red Blood Cells Transfusions [ Time Frame: 1 month ]
    Total volume of Red Blood Cells Transfusions


Eligibility Criteria
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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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;
Contacts and Locations
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To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04728789


Locations
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China
Peking Union Medical College Hospital
Beijing, China, 100730
Sponsors and Collaborators
Peking Union Medical College Hospital
More Information
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Responsible Party: Bing Han, Professor, Peking Union Medical College Hospital
ClinicalTrials.gov Identifier: NCT04728789    
Other Study ID Numbers: Avatrombopag-1
First Posted: January 28, 2021    Key Record Dates
Last Update Posted: January 28, 2021
Last Verified: January 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: individual participant data would be accepted upon request
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Informed Consent Form (ICF)
Clinical Study Report (CSR)
Time Frame: 10 years
Access Criteria: email request

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Anemia
Anemia, Aplastic
Hematologic Diseases
Bone Marrow Failure Disorders
Bone Marrow Diseases