Back to resultsCyclophosphamide Added to Standard Immunosuppressive Therapy With Eltrombopag as Front-line Therapy in Patients With Severe Aplastic Anemia
Primary Purpose
Severe Aplastic Anemia
Status
Recruiting
Phase
Phase 2
Locations
China
Study Type
Interventional
Intervention
Cyclophosphamide added to standard immunosuppressive therapy with eltrombopag
Sponsored by
About this trial
This is an interventional treatment trial for Severe Aplastic Anemia
Eligibility Criteria
Inclusion Criteria: Male or female age ≥ 12 years Subject has a diagnosis of naïve severe or very severe aplastic anemia ECOG performance status ≤2 Unwilling or unable to receive allogeneic hematopoietic stem cell transplantation. Willing and able to comply with the requirements for this study and written informed consent. Exclusion Criteria: Previously received immunosuppressive therapy > 4 weeks Previously treated with TPO-RA > 4 weeks Have an allergy or intolerance to either eltrombopag or cyclophosphamide. Have an allergy to ALG Uncontrolled systemic fungal, bacterial, or viral infection Poorly controlled hypertension (≥140/90mmHg) or diabetes (a fasting plasma glucose concentration ≥7.0mmol/L or a random venous plasma glucose concentration ≥11.1mmol/L) Abnormal liver or kidney function: ALT or AST >3 ULN, or serum creatinine (sCr)≥ 2.5 ULN. History of radiotherapy and chemotherapy for malignant solid tumors Combined with other serious disorders Pregnant or breast-feeding patients Patients considered to be ineligible for the study by the investigator for reasons other than the above.
Sites / Locations
- Regenerative Medicine CenterRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Experiemental
Arm Description
ALG/CsA(Cyclosporine) + eltrombopag + moderate-dose cyclophosphamide
Outcomes
Primary Outcome Measures
Overall response rate
Percentage of patients with hematological response. Hematological response includes complete rate, near complete rate(CR), very good partial response(VGPR), Meaningful partial response(mPR) and partial response(PR).
Secondary Outcome Measures
Robust response
Percentage of patients with robust response, including CR, near CR, VGPR and mPR.
All-cause mortality
Time to achieve robust hematological response
Incidence of the adverse event
Use Common Terminology Criteria for Adverse Events (CTCAE) Version 5 to assess the adverse event
Full Information
NCT ID
NCT05975996
First Posted
July 27, 2023
Last Updated
July 27, 2023
Sponsor
Institute of Hematology & Blood Diseases Hospital, China
1. Study Identification
Unique Protocol Identification Number
NCT05975996
Brief Title
Cyclophosphamide Added to Standard Immunosuppressive Therapy With Eltrombopag as Front-line Therapy in Patients With Severe Aplastic Anemia
Official Title
Cyclophosphamide Added to Standard Immunosuppressive Therapy With Eltrombopag as Front-line Therapy in Patients With Severe Aplastic Anemia
Study Type
Interventional
2. Study Status
Record Verification Date
July 2023
Overall Recruitment Status
Recruiting
Study Start Date
July 10, 2023 (Actual)
Primary Completion Date
January 31, 2027 (Anticipated)
Study Completion Date
June 30, 2027 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Institute of Hematology & Blood Diseases Hospital, China
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
This is a prospective, single-center, single-arm, phase 2 study. The aim of this study is to evaluate the efficacy and safety of Anti-lymphocyte globulin plus eltrombopag in combination with moderate-dose cyclophosphamide for severe aplastic anemia.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Severe Aplastic Anemia
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
43 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Experiemental
Arm Type
Experimental
Arm Description
ALG/CsA(Cyclosporine) + eltrombopag + moderate-dose cyclophosphamide
Intervention Type
Drug
Intervention Name(s)
Cyclophosphamide added to standard immunosuppressive therapy with eltrombopag
Intervention Description
Severe aplastic anemia patients will receive a daily dose of Anti-lymphocyte globulin (25mg/kg) for the initial five days at the beginning of the treatment. Cyclosporine will be administered daily at a dosage of 3-5mg/kg. Eltrombopag will be administered daily at a dosage of 15mg starting from the first day of treatment and continuing for a duration of six months. The moderate-dose cyclophosphamide (20mg/kg) will be administered on days 29-30 and days 43-44.
Primary Outcome Measure Information:
Title
Overall response rate
Description
Percentage of patients with hematological response. Hematological response includes complete rate, near complete rate(CR), very good partial response(VGPR), Meaningful partial response(mPR) and partial response(PR).
Time Frame
Within 3 months
Secondary Outcome Measure Information:
Title
Robust response
Description
Percentage of patients with robust response, including CR, near CR, VGPR and mPR.
Time Frame
Within 3 months
Title
All-cause mortality
Time Frame
Within 3 months
Title
Time to achieve robust hematological response
Time Frame
Within 6 months
Title
Incidence of the adverse event
Description
Use Common Terminology Criteria for Adverse Events (CTCAE) Version 5 to assess the adverse event
Time Frame
Within 6 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Male or female age ≥ 12 years
Subject has a diagnosis of naïve severe or very severe aplastic anemia
ECOG performance status ≤2
Unwilling or unable to receive allogeneic hematopoietic stem cell transplantation.
Willing and able to comply with the requirements for this study and written informed consent.
Exclusion Criteria:
Previously received immunosuppressive therapy > 4 weeks
Previously treated with TPO-RA > 4 weeks
Have an allergy or intolerance to either eltrombopag or cyclophosphamide.
Have an allergy to ALG
Uncontrolled systemic fungal, bacterial, or viral infection
Poorly controlled hypertension (≥140/90mmHg) or diabetes (a fasting plasma glucose concentration ≥7.0mmol/L or a random venous plasma glucose concentration ≥11.1mmol/L)
Abnormal liver or kidney function: ALT or AST >3 ULN, or serum creatinine (sCr)≥ 2.5 ULN.
History of radiotherapy and chemotherapy for malignant solid tumors
Combined with other serious disorders
Pregnant or breast-feeding patients
Patients considered to be ineligible for the study by the investigator for reasons other than the above.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Liwei Fang, MD
Phone
13752253515
Email
fangliwei@ihcams.ac.cn
First Name & Middle Initial & Last Name or Official Title & Degree
Hong Pan, MD
Email
panhong@ihcams.ac.cn
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Liwei Fang, MD
Organizational Affiliation
Institute of Hematology & Blood Diseases Hospital, China
Official's Role
Principal Investigator
Facility Information:
Facility Name
Regenerative Medicine Center
City
Tianjin
State/Province
Tianjin
ZIP/Postal Code
300131
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Liwei Fang, MD
Phone
13752253515
Email
fangliwei@ihcams.ac.cn
12. IPD Sharing Statement
Learn more about this trial
Cyclophosphamide Added to Standard Immunosuppressive Therapy With Eltrombopag as Front-line Therapy in Patients With Severe Aplastic Anemia
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