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A Single-center, Single-arm, Prospective Clinical Study on the Efficacy and Safety of CsA+AVA in the Treatment of NSAA in the Elderly

Primary Purpose

Aplastic Anemia

Status
Not yet recruiting
Phase
Phase 4
Locations
China
Study Type
Interventional
Intervention
Avatrombopag+CsA
Sponsored by
Peking Union Medical College Hospital
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Aplastic Anemia

Eligibility Criteria

65 Years - 90 Years (Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: Elderly patients with well-defined NSAA anemia who meet the diagnostic criteria for aplastic anemia (AA) but do not meet the diagnostic criteria for severe aplastic anemia (SAA). AA is diagnosed if at least two of the following conditions are met: hemoglobin <100 g/L, platelet count <50×109/L, and neutrophil count <1.5×10^9/L. SAA diagnostic criteria: Meet the following diagnostic criteria, at least two of the following three criteria meet PLT < 20×10^9/L, ANC < 0.5×10^9/L, ARC < 60×10^9/L or red blood cell corrected volume <1%. Age 65 years or older, male or female. Subjects must complete all screening assessments listed in the trial protocol. Able to swallow or administer orally. Cannot tolerate or refuse ATG treatment. No previous treatment with cyclosporine, tacrolimus or hormones or treatment for less than 1 month. No TPO receptor agonists (including thrombopoietin, aitripopar, tritripopar, etc.) were used or TPO receptor agonists were used to treat the total dose of thrombopoietin, Eltrombopag, Herombopag, Avatrombopag and other TPO receptor agonists ≤1 week. Informed consent must be signed before the start of all specific research procedures. Considering the patient's condition, if the patient's signature is not conducive to the treatment of the disease, the informed consent shall be signed by the patient's immediate family. Exclusion Criteria: Known congenital hematopoietic exhaustion diseases (such as Fanconi anemia) and other causes of pancytopenia and bone marrow hypoproliferative diseases (such as hemolytic PNH, hypoproliferative MDS/AML, autoantibody-mediated pancytopenia, etc.); Patients with uncontrolled bleeding and/or infection after standard treatment. Patients with a history of hematopoietic stem cell transplantation. History of high blood pressure. History of thrombosis. Patients with underlying cancer who also have malignant tumors or are receiving immunosuppressive therapy. Participants considered unsuitable for inclusion by the researchers. Renal function indicators: serum creatinine >1.2 times the upper limit of normal (ULN) or albumin <0.9 times the lower limit of normal (LLN); Patients with severe renal insufficiency with creatinine clearance <30ml/min; Liver function indicators: alanine aminotransferase (ALT) is 2.5 times higher than the upper limit of normal or AST is 2.5 times higher than the upper limit of normal or total bilirubin is 2.5 times higher than the upper limit of normal or serum creatinine is 1.5 times higher than the upper limit of normal; Serious heart, liver and kidney disease.

Sites / Locations

  • Peking union medical college hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

treatment group

Arm Description

Cyclosporine: 3mg/kg/d orally divided into two times, the valley concentration of cyclosporine maintained at 100~150ng/ml, to achieve the maximum effect and began to reduce after 3 months, the reduction of 25mg every 3 months; Avatrombopag: The dosage was 40~60mg orally, once a day, and the dosage was adjusted according to the subject's platelet count. The drug was administered for 24 weeks (6 months).

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 Week 26

Secondary Outcome Measures

Changes in Haemoglobin in the Absence of Red Blood Cells Transfusion
The change in hematology values ( haemoglobin) were evaluated
Changes in Platelet in the Absence of Platelet Transfusion
The change in hematology values (platelet) were evaluated
Percentage of patients with clonal evolution to myelodysplasia, PNH, acute leukemia
Clonal evolution to myelodysplasia is defined as a new marrow cytogenic abnormality with or without characteristic dysplastic marrow findings. Evolution to leukemia is defined as greater than 20% peripheral blood and/or marrow blasts. Evolution to paroxysmal nocturnal hemoglobinuria (PNH) is defined as a clone at baseline < 10% that rose to greater than 50% on study.

Full Information

First Posted
August 16, 2023
Last Updated
August 16, 2023
Sponsor
Peking Union Medical College Hospital
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1. Study Identification

Unique Protocol Identification Number
NCT06004752
Brief Title
A Single-center, Single-arm, Prospective Clinical Study on the Efficacy and Safety of CsA+AVA in the Treatment of NSAA in the Elderly
Official Title
A Single-center, Single-arm, Prospective Clinical Study on the Efficacy and Safety of Cyclosporine Combined With Avatrombopag in the Treatment of Non-severe Aplastic Anemia in the Elderly
Study Type
Interventional

2. Study Status

Record Verification Date
August 2023
Overall Recruitment Status
Not yet recruiting
Study Start Date
August 2023 (Anticipated)
Primary Completion Date
August 2024 (Anticipated)
Study Completion Date
August 2025 (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
For elderly patients who cannot tolerate anti-thymocyte globulin (ATG) treatment, the addition of avatrombopag (AVA), which has a slight adverse reaction, can theoretically improve the hematological response rate in elderly patients with non-severe aplastic anemia (NSAA) without significantly increasing adverse reactions. Based on this, this study treated NSAA patients older than 60 with AVA combined with CsA to evaluate the hematological response rate and safety of AVA in the elderly who could not tolerate ATG therapy.
Detailed Description
Aplastic anemia (AA) can be divided into severe AA (SAA) and non-severe AA (NSAA), according to the severity of the disease. Anti-thymocyte globulin (ATG) in combination with CsA is the most typical combined immunosuppressive therapy regimen. For elderly patients who cannot tolerate anti-thymocyte globulin (ATG) treatment, the addition of avatrombopag, which has a slight adverse reaction, can theoretically improve the hematological response rate in elderly patients with NSAA without significantly increasing adverse reactions. Based on this, this study treated NSAA patients older than 60 with AVA combined with CsA to evaluate the hematological response rate and safety of AVA in the elderly who could not tolerate ATG therapy.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Aplastic Anemia

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
30 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
treatment group
Arm Type
Experimental
Arm Description
Cyclosporine: 3mg/kg/d orally divided into two times, the valley concentration of cyclosporine maintained at 100~150ng/ml, to achieve the maximum effect and began to reduce after 3 months, the reduction of 25mg every 3 months; Avatrombopag: The dosage was 40~60mg orally, once a day, and the dosage was adjusted according to the subject's platelet count. The drug was administered for 24 weeks (6 months).
Intervention Type
Drug
Intervention Name(s)
Avatrombopag+CsA
Intervention Description
Cyclosporine was taken orally at 3mg/kg/d twice, and the valley concentration of cyclosporine was maintained at 100-150ng /ml. Avatrombopag:The dosage is 40~60mg, once a day, orally.(If the patient has a hematological reaction and platelets rapidly rise to PLT>200×10^9/L, the drug should be stopped and observed until the PLT<100×109/L and then reduced by one titration dose; If the platelet recovered to 100-200 ×10^9/L for more than 2 months, the titration dose was reduced by one. After the reduction to 20mg, platelets can still be maintained at 100~200×10^9/L, and for more than 2 months, the reduction to 20mg 1/ every other day; After the reduction, platelets can still be maintained at 100-200 ×10^9/L for more than 2 months
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 Week 26
Time Frame
6 months
Secondary Outcome Measure Information:
Title
Changes in Haemoglobin in the Absence of Red Blood Cells Transfusion
Description
The change in hematology values ( haemoglobin) were evaluated
Time Frame
6 months
Title
Changes in Platelet in the Absence of Platelet Transfusion
Description
The change in hematology values (platelet) were evaluated
Time Frame
6 months
Title
Percentage of patients with clonal evolution to myelodysplasia, PNH, acute leukemia
Description
Clonal evolution to myelodysplasia is defined as a new marrow cytogenic abnormality with or without characteristic dysplastic marrow findings. Evolution to leukemia is defined as greater than 20% peripheral blood and/or marrow blasts. Evolution to paroxysmal nocturnal hemoglobinuria (PNH) is defined as a clone at baseline < 10% that rose to greater than 50% on study.
Time Frame
12 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
65 Years
Maximum Age & Unit of Time
90 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Elderly patients with well-defined NSAA anemia who meet the diagnostic criteria for aplastic anemia (AA) but do not meet the diagnostic criteria for severe aplastic anemia (SAA). AA is diagnosed if at least two of the following conditions are met: hemoglobin <100 g/L, platelet count <50×109/L, and neutrophil count <1.5×10^9/L. SAA diagnostic criteria: Meet the following diagnostic criteria, at least two of the following three criteria meet PLT < 20×10^9/L, ANC < 0.5×10^9/L, ARC < 60×10^9/L or red blood cell corrected volume <1%. Age 65 years or older, male or female. Subjects must complete all screening assessments listed in the trial protocol. Able to swallow or administer orally. Cannot tolerate or refuse ATG treatment. No previous treatment with cyclosporine, tacrolimus or hormones or treatment for less than 1 month. No TPO receptor agonists (including thrombopoietin, aitripopar, tritripopar, etc.) were used or TPO receptor agonists were used to treat the total dose of thrombopoietin, Eltrombopag, Herombopag, Avatrombopag and other TPO receptor agonists ≤1 week. Informed consent must be signed before the start of all specific research procedures. Considering the patient's condition, if the patient's signature is not conducive to the treatment of the disease, the informed consent shall be signed by the patient's immediate family. Exclusion Criteria: Known congenital hematopoietic exhaustion diseases (such as Fanconi anemia) and other causes of pancytopenia and bone marrow hypoproliferative diseases (such as hemolytic PNH, hypoproliferative MDS/AML, autoantibody-mediated pancytopenia, etc.); Patients with uncontrolled bleeding and/or infection after standard treatment. Patients with a history of hematopoietic stem cell transplantation. History of high blood pressure. History of thrombosis. Patients with underlying cancer who also have malignant tumors or are receiving immunosuppressive therapy. Participants considered unsuitable for inclusion by the researchers. Renal function indicators: serum creatinine >1.2 times the upper limit of normal (ULN) or albumin <0.9 times the lower limit of normal (LLN); Patients with severe renal insufficiency with creatinine clearance <30ml/min; Liver function indicators: alanine aminotransferase (ALT) is 2.5 times higher than the upper limit of normal or AST is 2.5 times higher than the upper limit of normal or total bilirubin is 2.5 times higher than the upper limit of normal or serum creatinine is 1.5 times higher than the upper limit of normal; Serious heart, liver and kidney disease.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Bing Han
Phone
+861069151235
Email
hanbing_li@sina.com
Facility Information:
Facility Name
Peking union medical college hospital
City
Beijing
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
always
IPD Sharing Access Criteria
email request

Learn more about this trial

A Single-center, Single-arm, Prospective Clinical Study on the Efficacy and Safety of CsA+AVA in the Treatment of NSAA in the Elderly

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