Ruxolitinib Versus Allogeneic Stem Cell Transplantation for Patients with Myelofibrosis According to Donor Availability: a Prospective Phase II Trial (MMM 02 Study)
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 87
- 试验地点
- 14
- 主要终点
- Event free survival
研究概览
简要总结
The present study will be a multicenter, prospective phase II-study comparing efficacy of allogeneic SCT for patients with myelofibrosis who have a suitable stem cell donor after a 3 months Ruxolitinib induction therapy with patients who lack a suitable stem cell donor and will continue to receive Ruxolitinib.
详细描述
This study is a multicenter, prospective phase II-study compares efficacy of allogeneic SCT for patients with myelofibrosis who have a suitable stem cell donor after a 3 months Ruxolitinib induction therapy with patients who lack a suitable stem cell donor and will continue to receive Ruxolitinib.
In this study will further assess and compare the safety and efficacy of study treatments/ induction therapy in both study arms on spleen reduction, improvement of constitutional symptoms, QOL, toxicity, fibrosis regression, development of GvHD as well as chimerism, engraftment, relapse incidence, disease related mortality, outcome and overall survival.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Symptomatic primary myelofibrosis or myelofibrosis post polycythaemia vera or essential thrombocythemia stage intermediate 2- or high-risk according to IPSS or DIPSS [46] or intermediate 1-risk with high risk cytogenetics, other than normal karyotype, sole del 20q, del 13q, or sole+9, or transfusion-dependency
- •Patients age: 18 - 70 years at time of inclusion (female and male)
- •Patients understand and voluntarily sign an informed consent form
- •Platelet count ≥ 50 x 109/L
- •No prior Ruxolitinib treatment
排除标准
- •Severe renal, hepatic, pulmonary or cardiac disease, such as:
- •Total bilirubin, SGPT or SGOT > 3 times upper the normal level
- •Left ventricular ejection fraction < 30 %
- •Creatinine clearance < 30 ml/min
- •DLCO < 35 % and/or receiving supplementary continuous oxygen
- •Positive serology for HIV
- •Pregnant or lactating women (positive serum pregnancy test)
- •Age < 18 and ≥ 71 years.
- •Uncontrolled invasive fungal infection at time of screening (baseline)
- •Serious psychiatric or psychological disorders
- •Participation in another study with ongoing use of unlicensed investigational product from 28 days before study enrollment
- •Transformation to AML
研究组 & 干预措施
Arm A
Treatment with Allogeneic Stem cell Transplantation after 3 months of Ruxolitinib induction therapy
干预措施: Allogeneic stem cell transplantation (Procedure)
Arm B
Treatment with Ruxolitinib continuous therapy
干预措施: Ruxolitinib continuous therapy (Drug)
结局指标
主要结局
Event free survival
时间窗: 3 years
Compare to event free survival of patients at 3 years after allogeneic SCT and in Ruxolitinib continuous therapy in patients without a suitable donor
次要结局
- Acute graft-versus-host disease(Day +100 after allogeneic SCT)
- Non-relapsed mortality(1 and 3 years)
- Evaluation of QOL (FACT-BMT)(baseline, confinement to Ruxolitinib continous therapy, +180d, +1 year, +2 years and +3 years)
- Evaluation of QOL (MPN-SAF-TSS)(baseline, confinement to Ruxolitinib continous therapy, +180d, +1 year, +2 years and +3 years)
- Chimerism on relapse(30d, 100d, 180 d, 1 year, 2 years and 3 years)
- Bone marrow fibrosis regression(30d, 100d, 1 year and 3 years)
- Overall Survival(3 years)
- Improvement of bone marrow fibrosis(3 months)
- Toxicity of Ruxolitinib(till 3 years)
- Toxicity of conditioning therapy(till 3 years)
- Relapse(3 years)
- Evaluation of Sorror Risk Score(at baseline)
- Spleen reduction(3 months)
- Improvement of constitutional symptoms(3 months)
- Chronic graft-versus-host disease(1, 2 and 3 years after allogeneic SCT)
- Disease-related mortality(3 years)
- Discontinuation rate(3 years)