High Dose Ruxolitinib and Allogeneic Stem Cell Transplantation in Myelofibrosis Patients With Splenomegaly
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- Safety and adverse events (AEs)
研究概览
简要总结
To learn if giving ruxolitinib and busulfan before a stem cell transplant can help to reduce spleen size and help the transplant to succeed.
详细描述
Primary Objective
1) Compare the proportion of patients alive, disease free, engrafted, and without poor graft function at 100 days post-transplant with the historical rate of 45%.
Secondary Objectives:
- Overall survival
- Progression-free survival
- Graft vs host disease relapse free survival
- Relapse rate
- Non-relapse Mortality
- Time to Neutrophil and platelet engraftment
- Time to red cell transfusion independence
- Graft failure
- Acute and chronic GVHD
- Grade 3 -5 Toxicity
- Incidence of poor graft function5
- Need for growth factors (myeloid or thrombopoietic) at 100 days
- Spleen response around day -7, -1, 30, and 100 days
- Need for transfusions at 100 days
- Time to discontinuation of immunosuppressives
Exploratory Objectives:
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
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants 18 years to less than or equal to 75 years.
- •Able to provide written consent.
- •Primary or secondary Myelofibrosis (may have received Jak inhibitors including ruxolitinib)
- •Enlarged spleen by palpation or imaging. For the purpose of this study, splenomegaly is defined as any clinically palpable spleen or spleen larger than 12 cms on imaging.
- •Has a fully matched (8/8:HLA A, B, C, DRB1) related or matched unrelated donor.
- •Adequate renal function, including:
- •a. Serum creatinine /= 40 ml/min/1.73 m
- •Adequate liver function, including:
排除标准
- •Positive beta HCG in females of child-bearing potential defined as not postmenopausal for 24 months or no previous surgical sterilization or lactating females.
- •Ejection fraction <40%
- •Corrected DLCO < 50%
- •Evidence of other clinically significant uncontrolled condition(s) including, but not limited to:
- •Uncontrolled and/or active systemic infection (viral, bacterial or fungal)
- •Active hepatitis B virus (HBV), hepatitis C (HCV), HIV or TB infection or requiring treatment for the same.
- •Thrombosis including MI, Stroke, PE, DVT in the past 6 months
- •Note: subjects with serologic evidence of prior vaccination to HBV (i.e. hepatitis B surface (HBs) antigen negative-, anti-HBs antibody positive and anti-hepatitis B core (HBc) antibody negative) or positive anti-HBc antibody from intravenous immunoglobulins (IVIG) may participate.
研究组 & 干预措施
Ruxolitinib and Allogeneic Stem Cell Transplantation
Participants will be asked to receive study drugs and a stem cell transplant and attend study visits, at which various tests and procedures will be performed. Participants are expected to receive treatment for about 100 days, followed by a year of follow-up.
干预措施: Allogeneic Stem Cell Transplantation (Procedure)
Ruxolitinib and Allogeneic Stem Cell Transplantation
Participants will be asked to receive study drugs and a stem cell transplant and attend study visits, at which various tests and procedures will be performed. Participants are expected to receive treatment for about 100 days, followed by a year of follow-up.
干预措施: Ruxolitinib (Drug)
Ruxolitinib and Allogeneic Stem Cell Transplantation
Participants will be asked to receive study drugs and a stem cell transplant and attend study visits, at which various tests and procedures will be performed. Participants are expected to receive treatment for about 100 days, followed by a year of follow-up.
干预措施: Mesna (Drug)
Ruxolitinib and Allogeneic Stem Cell Transplantation
Participants will be asked to receive study drugs and a stem cell transplant and attend study visits, at which various tests and procedures will be performed. Participants are expected to receive treatment for about 100 days, followed by a year of follow-up.
干预措施: Levetiracetam (Drug)
Ruxolitinib and Allogeneic Stem Cell Transplantation
Participants will be asked to receive study drugs and a stem cell transplant and attend study visits, at which various tests and procedures will be performed. Participants are expected to receive treatment for about 100 days, followed by a year of follow-up.
干预措施: Eltrombopag (Drug)
Ruxolitinib and Allogeneic Stem Cell Transplantation
Participants will be asked to receive study drugs and a stem cell transplant and attend study visits, at which various tests and procedures will be performed. Participants are expected to receive treatment for about 100 days, followed by a year of follow-up.
干预措施: Busulfan (Drug)
Ruxolitinib and Allogeneic Stem Cell Transplantation
Participants will be asked to receive study drugs and a stem cell transplant and attend study visits, at which various tests and procedures will be performed. Participants are expected to receive treatment for about 100 days, followed by a year of follow-up.
干预措施: Romiplostim (Drug)
Ruxolitinib and Allogeneic Stem Cell Transplantation
Participants will be asked to receive study drugs and a stem cell transplant and attend study visits, at which various tests and procedures will be performed. Participants are expected to receive treatment for about 100 days, followed by a year of follow-up.
干预措施: Cyclophosphamide (Drug)
Ruxolitinib and Allogeneic Stem Cell Transplantation
Participants will be asked to receive study drugs and a stem cell transplant and attend study visits, at which various tests and procedures will be performed. Participants are expected to receive treatment for about 100 days, followed by a year of follow-up.
干预措施: Fludarabine phosphate (Drug)
Ruxolitinib and Allogeneic Stem Cell Transplantation
Participants will be asked to receive study drugs and a stem cell transplant and attend study visits, at which various tests and procedures will be performed. Participants are expected to receive treatment for about 100 days, followed by a year of follow-up.
干预措施: Tacrolimus (Drug)
结局指标
主要结局
Safety and adverse events (AEs)
时间窗: Through study completion; an average of 1 year.
Incidence of Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 5.0
次要结局
未报告次要终点