Back to resultsAvatrombopag Usage in NSAA
Primary Purpose
Aplastic Anemia, Drug Effect
Status
Unknown status
Phase
Phase 2
Locations
China
Study Type
Interventional
Intervention
Avatrombopag 20 MG
Sponsored by
About this trial
This is an interventional treatment trial for Aplastic Anemia
Eligibility Criteria
Inclusion Criteria:
- Patients diagnosed to be non-severe aplastic anemia
- 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
- 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);
- Eastern Cooperative Oncology Group (ECOG) performance status 0-2;
- Patients able to understand and comply with protocol requirements and instructions and have signed and dated informed consent.
Exclusion Criteria:
- Congenital aplastic anemia;
- Presence of chromosomal aberration;
- Evidence of a clonal hematologic bone marrow disorder (MDS, AML) on cytogenetics;
- Presence with PNH clone ≥50%;
- Patients received HSCT before;
- Uncontrolled infection or bleeding with standard treatment;
- Allergic to Avatrombopag or accessories;
- HIV, HCV or HBV active infection or liver cirrhosis or portal hypertension;
- 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;
- Have any concomitant malignancies within 5 years expect for local basal cell carcinoma of the skin;
- Past history of thromboembolic event, heart attack or stroke (including anti-phospholipid antibody syndrome) and current use of anticoagulants;
- Pregnant or nursing (lactating) woman;
- Have attended other clinical trials within 3 months;
Sites / Locations
- Peking Union Medical College Hospital
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Avatrombopag treatment group
Arm 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.
Outcomes
Primary Outcome Measures
ORR at 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
ORR at 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
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
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
Total volume of Platelet Transfusions
Total volume of Red Blood Cells Transfusions
Total volume of Red Blood Cells Transfusions
Full Information
NCT ID
NCT04728789
First Posted
December 1, 2020
Last Updated
January 27, 2021
Sponsor
Peking Union Medical College Hospital
1. Study Identification
Unique Protocol Identification Number
NCT04728789
Brief Title
Avatrombopag Usage in NSAA
Official Title
Efficacy and Safety of Avatrombopag in Non-severe Aplastic Anemia - a Multicenter Prospective Single Arm Study
Study Type
Interventional
2. Study Status
Record Verification Date
January 2021
Overall Recruitment Status
Unknown status
Study Start Date
February 1, 2021 (Anticipated)
Primary Completion Date
December 31, 2022 (Anticipated)
Study Completion Date
June 30, 2023 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Peking Union Medical College Hospital
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Aplastic Anemia, Drug Effect
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
40 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Avatrombopag treatment group
Arm Type
Experimental
Arm 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.
Intervention Type
Drug
Intervention Name(s)
Avatrombopag 20 MG
Intervention Description
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.
Primary Outcome Measure Information:
Title
ORR at 6 Months
Description
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
Time Frame
6 months
Secondary Outcome Measure Information:
Title
ORR at 3 Months
Description
ORR will be calculated after 3 months of treatment by measuring platelet, reticulocyte, neutrophil and transfusion independence.
Time Frame
3 months
Title
Percentage of patients with clonal evolution at 6 months
Description
Percentage of patients with clonal evolution would be evaluated by bone marrow biopsy at 6 months follow up.
Time Frame
6 months
Title
percentage of side effects at 6 months
Description
percentage of side effects would be recorded during the study and be calculated according to CTCAE 5.0 at 6 months
Time Frame
6 months
Title
Total volume of Platelet Transfusions
Description
Total volume of Platelet Transfusions
Time Frame
1 month
Title
Total volume of Red Blood Cells Transfusions
Description
Total volume of Red Blood Cells Transfusions
Time Frame
1 month
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
75 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Patients diagnosed to be non-severe aplastic anemia
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
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);
Eastern Cooperative Oncology Group (ECOG) performance status 0-2;
Patients able to understand and comply with protocol requirements and instructions and have signed and dated informed consent.
Exclusion Criteria:
Congenital aplastic anemia;
Presence of chromosomal aberration;
Evidence of a clonal hematologic bone marrow disorder (MDS, AML) on cytogenetics;
Presence with PNH clone ≥50%;
Patients received HSCT before;
Uncontrolled infection or bleeding with standard treatment;
Allergic to Avatrombopag or accessories;
HIV, HCV or HBV active infection or liver cirrhosis or portal hypertension;
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;
Have any concomitant malignancies within 5 years expect for local basal cell carcinoma of the skin;
Past history of thromboembolic event, heart attack or stroke (including anti-phospholipid antibody syndrome) and current use of anticoagulants;
Pregnant or nursing (lactating) woman;
Have attended other clinical trials within 3 months;
Facility Information:
Facility Name
Peking Union Medical College Hospital
City
Beijing
ZIP/Postal Code
100730
Country
China
12. IPD Sharing Statement
Plan to Share IPD
Yes
IPD Sharing Plan Description
individual participant data would be accepted upon request
IPD Sharing Time Frame
10 years
IPD Sharing Access Criteria
email request
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Avatrombopag Usage in NSAA
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