A RANDOMIZED CONTROLLED STUDY IN NEWLY DIAGNOSED SEVERE APLASTIC ANEMIA PATIENTS RECEIVING ANTITHYMOCYTE GLOBULIN (ATG), CYCLOSPORIN A, WITH OR WITHOUT G-CSF
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 入组人数
- 205
- 试验地点
- 76
- 主要终点
- Failure free survival
研究概览
简要总结
The purpose of this study is to examine the effect of G-CSF on disease free survival and overall survival in aplastic anaemia patients who also receive ATG and Cyclosporin A.
详细描述
Open label, randomized, controlled study of G-CSF, ATG and Cyclosporin A versus ATG and Cyclosporin A. Subjects will be evaluated for hematologic response through day 240. Subjects who do not demonstrate a partial or complete remission by day 120 will be randomized to receive either a second course of ATG or continue their current regimen. Subjects who do demonstrate a partial or complete remission will continue their current regimen through day 240 or maintenance of a complete remission for 30 days. The last day of study treatment will be day 240.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 90 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Severe or very severe aplastic anemia
- •Less than 6 months from diagnosis of severe aplastic anemia by bone marrow biopsy
- •Ethical - Before randomization is done the subject or legally acceptable representative must give written informed consent for participation in the study
排除标准
- •Eligibility for an HLA-matched sibling donor transplant
- •Prior therapy with ATG
- •Cyclosporin A <4 weeks before enrollment
- •Treatment with G-CSF <2 weeks before enrollment
- •Other growth factors <4 weeks before enrollment
- •Diagnosis of Fanconi anemia, dyskeratosis congenita or congenital bone marrow failure syndrome
- •Evidence of myelodysplastic disease
- •Diagnosis or previous history of carcinoma (except local cervical, basal cell, squamous cells, or melanoma)
- •Subjects who have infection, hepatic, renal cardiac, metabolic or other concurrent diseases of such severity that death is imminent
- •Subject is pregnant (e.g. positive HCG test) or is breast feeding
研究组 & 干预措施
Yes G-CSF, No 2nd ATG
Patients randomised to receive G-CSF (alongside ATG and CSA) and to not receive early retreatment in case of no response.
干预措施: G-CSF (Drug)
Yes G-CSF, Yes 2nd ATG
Patients randomised to receive G-CSF (alongside ATG and CSA) and to receive early retreatment in case of no response.
干预措施: G-CSF (Drug)
No G-CSF, yes 2nd ATG
Patients randomised not to receive G-CSF (alongside ATG and CSA) but they do receive early retreatment in case of no response.
干预措施: Early retreatment with ATG (Drug)
Yes G-CSF, No 2nd ATG
Patients randomised to receive G-CSF (alongside ATG and CSA) and to not receive early retreatment in case of no response.
干预措施: Early retreatment with ATG (Drug)
Yes G-CSF, Yes 2nd ATG
Patients randomised to receive G-CSF (alongside ATG and CSA) and to receive early retreatment in case of no response.
干预措施: Early retreatment with ATG (Drug)
No G-CSF, No 2nd ATG
Patients randomised not to receive G-CSF (alongside ATG and CSA) and to not receive early retreatment in case of no response.
干预措施: Early retreatment with ATG (Drug)
No G-CSF, No 2nd ATG
Patients randomised not to receive G-CSF (alongside ATG and CSA) and to not receive early retreatment in case of no response.
干预措施: G-CSF (Drug)
No G-CSF, yes 2nd ATG
Patients randomised not to receive G-CSF (alongside ATG and CSA) but they do receive early retreatment in case of no response.
干预措施: G-CSF (Drug)
结局指标
主要结局
Failure free survival
时间窗: day 240
To evaluate the effect of G-CSF on failure free survival and mortality in study subjects also receiving ATG and Cyclosporin A \& time to hematologic response (failure defined as death, non-response or requirement of further treatment).
次要结局
- Retreatment with ATG(day 240)
- Safety(6year)
- Relapse rate(2year)
- Blood count(day 240)
- Severity of the disease(day 365)
- Complete remission(day 120)
- Severe Infections(day 240)
- Haematological response(day 240)
- Benefit of addition of G-CSF(day 240)