Reduced Dose of Cyclophosphamide Combined With Standard Immunosuppressive Therapy as Front-line Therapy in Patients With Severe Aplastic Anemia
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 75
- 试验地点
- 1
- 主要终点
- Overall response rate
研究概览
简要总结
This is a prospective, single-center, single-arm, phase 2 study. This study aims to evaluate the efficacy and safety of anti-lymphocyte globulin plus herombopag in combination with the reduced dose of cyclophosphamide (hypo-CASH) for severe aplastic anemia.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subject has a diagnosis of naïve severe or very severe aplastic anemia
- •Male or female age ≥ 12 years
- •Unwilling or unable to receive allogeneic hematopoietic stem cell transplantation.
- •ECOG performance status ≤2
- •Willing and able to comply with the requirements for this study and written informed consent.
排除标准
- •Previously received immunosuppressive therapy > 4 weeks
- •Previously treated with TPO-RA > 4 weeks
- •Have an allergy or intolerance to anti-lymphocyte globulin, cyclosporine, herombopag or cyclophosphamide.
- •Uncontrolled fungal, bacterial, or viral infection. Hepatitis-associated aplastic anemia is allowed; hepatitis B or C infection is permitted unless it causes severe liver failure.
- •Tested positive for HIV or syphilis
- •Presence of severe liver, kidney, or heart failure, or other life-threatening comorbidities.
- •History of radiotherapy and chemotherapy for malignant solid tumors in recent 5 years
- •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.
研究组 & 干预措施
hypo-CASH
ALG/CsA and herombopag and reduced dose of cyclophosphamide
干预措施: Reduced dose of cyclophosphamide combined with standard immunosuppressive therapy (Drug)
结局指标
主要结局
Overall response rate
时间窗: Within 3 months
Percentage of patients with hematological response. Hematological response includes complete rate, near complete rate(CR), very good partial response(VGPR), good partial response(GPR) and partial response(PR).
次要结局
- Superior response rate(With in 6 months)
- Overall response rate(With in 6 months)
- Incidence of the adverse event(Within 6 months)
- Time to achieve robust superior response(Within 6 months)
- First time to response(Within 6 months)
研究者
Jun Shi
Director of the Red Blood Cell Disorders Center & Director of the Regenerative Medicine Clinic
Institute of Hematology & Blood Diseases Hospital, China