Cyclophosphamide Added to Standard Immunosuppressive Therapy With Eltrombopag as Front-line Therapy in Patients With Severe Aplastic Anemia
Study Details
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 2 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Experiemental ALG/CsA(Cyclosporine) + eltrombopag + moderate-dose cyclophosphamide |
Drug: Cyclophosphamide added to standard immunosuppressive therapy with eltrombopag
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.
|
Outcome Measures
Primary Outcome Measures
- Overall response rate [Within 3 months]
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 [Within 3 months]
Percentage of patients with robust response, including CR, near CR, VGPR and mPR.
- All-cause mortality [Within 3 months]
- Time to achieve robust hematological response [Within 6 months]
- Incidence of the adverse event [Within 6 months]
Use Common Terminology Criteria for Adverse Events (CTCAE) Version 5 to assess the adverse event
Eligibility Criteria
Criteria
Inclusion Criteria:
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Male or female age ≥ 12 years
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Subject has a diagnosis of naïve severe or very severe aplastic anemia
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ECOG performance status ≤2
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Unwilling or unable to receive allogeneic hematopoietic stem cell transplantation.
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Willing and able to comply with the requirements for this study and written informed consent.
Exclusion Criteria:
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Previously received immunosuppressive therapy > 4 weeks
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Previously treated with TPO-RA > 4 weeks
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Have an allergy or intolerance to either eltrombopag or cyclophosphamide.
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Have an allergy to ALG
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Uncontrolled systemic fungal, bacterial, or viral infection
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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)
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Abnormal liver or kidney function: ALT or AST >3 ULN, or serum creatinine (sCr)≥ 2.5 ULN.
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History of radiotherapy and chemotherapy for malignant solid tumors
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Combined with other serious disorders
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Pregnant or breast-feeding patients
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Patients considered to be ineligible for the study by the investigator for reasons other than the above.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Regenerative Medicine Center | Tianjin | Tianjin | China | 300131 |
Sponsors and Collaborators
- Institute of Hematology & Blood Diseases Hospital
Investigators
- Principal Investigator: Liwei Fang, MD, Institute of Hematology & Blood Diseases Hospital
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- ALG-HPAG-CTX2023