Clinical Phase II Trial to Evaluate the Safety and Efficacy of Treosulfan Based Conditioning Prior to Allogeneic Haematopoietic Stem Cell Transplantation in Patients With Acute Myeloid Leukaemia
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- medac GmbH
- 入组人数
- 75
- 试验地点
- 1
- 主要终点
- Efficacy - Evaluation of engraftment. Safety - Evaluation of the incidence of the following CTC grade 3 and 4 adverse events between day -6 and day +28 - hyperbilirubinemia and mucositis / stomatitis - veno-occlusive disease - seizures
研究概览
简要总结
This is a multicenter, multinational, non-randomized, non-controlled open-label phase II trial to evaluate the safety and efficacy of treosulfan in a combination regimen with fludarabine as conditioning therapy prior to allogeneic stem cell transplantation (SCT) in patients with AML.
The aim is to demonstrate a clinical benefit compared with historical data on intravenous busulfan (BusulfexTM, BusilvexTM), the only drug so far registered in the indication conditioning before allogeneic stem cell transplantation.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Supportive Care
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 60 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with acute myeloid leukaemia (AML) according to WHO classification (> 20% myeloblasts in peripheral blood or bone marrow at initial diagnosis) with < 5% myeloblast in the bone marrow, indicated for allogeneic transplantation
- •Availability of an HLA-identical sibling donor (MRD) or HLA-identical unrelated donor (MUD) HLA-identity defined by the following markers: A, B, DRB1, DQB
- •Target graft size (unmanipulated)
- •bone marrow: 2 - 10 x 106 CD34+ cells/kg BW recipient or > 2 x 108 nucleated cells/kg BW recipient or
- •peripheral blood: 4 - 10 x 106 CD34+ cells/kg BW recipient
- •Age > 18 and < 60 years
- •Karnofsky Index > 80 %
- •Adequate contraception in female patients of child-bearing potential
- •Written informed consent
排除标准
- •Therapy related secondary AML
- •AML with t(8;21)(q22;q22) in CR1
- •Acute promyelocytic leukaemia with t(15;17)(q22;q12) in CR1
- •Secondary malignancies
- •Previous allogeneic transplantation
- •Severe concomitant illnesses / medical conditions (e.g. impaired respiratory and/or cardiac function)
- •Known and manifested malignant involvement of the CNS
- •Active infectious disease
- •HIV- positivity or active hepatitis infection
- •Impaired liver function (Bilirubin > upper normal limit; Transaminases > 3.0 x upper normal limit)
- •Impaired renal function (Creatinine-clearance < 60 ml/min; Serum Creatinine > 1.5 x upper normal limit).
- •Pleural effusion or ascites > 1.0 L
- •Pregnancy or lactation
- •Known hypersensitivity to treosulfan and/or fludarabine
- •Participation in another experimental drug trial within 4 weeks before day -6
- •Non-co-operative behaviour or non-compliance
- •Psychiatric diseases or conditions that might impair the ability to give informed consent
研究组 & 干预措施
Treosulfan
Patients with acute myeloid leukaemia (AML) according to WHO classification (> 20% myeloblasts in peripheral blood or bone marrow at initial diagnosis) with < 5% myeloblasts in the bone marrow, indicated for allogeneic transplantation
干预措施: Treosulfan (Drug)
结局指标
主要结局
Efficacy - Evaluation of engraftment. Safety - Evaluation of the incidence of the following CTC grade 3 and 4 adverse events between day -6 and day +28 - hyperbilirubinemia and mucositis / stomatitis - veno-occlusive disease - seizures
时间窗: 3.5 years
次要结局
- Efficacy - Evaluation of disease free survival (DFS) - Evaluation of overall survival (OS) - Evaluation of relapse incidence (RI) - Donor chimerism on day +28, +56 and +100. Safety - Evaluation of NRM on days +28 and +100(3.5 years)