Phase I Study of Eltrombopag for Promoting Thrombopoiesis in Patients Undergoing Stem Cell Transplantation After Total Body Irradiation
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 19
- 主要终点
- Maximum Tolerated Dose (MTD) of Eltrombopag
研究概览
简要总结
Patients who undergo total body irradiation (TBI) for stem cell transplantation have prolonged periods of low counts of specific blood cells called platelets. These low platelets counts can cause bleeding and infection. Thus far, no drug is available for use to speed the recovery of platelets, and therefore transfusions are often necessary.
The purpose of this study is to test the safety of a drug called eltrombopag in patients who have received TBI. The investigators want to find out what effects, good or bad, it has on people with low platelet counts due to treatment with TBI. The investigators will also be testing how well eltrombopag may work at different doses and determine if this drug speeds up the recovery of the platelets.
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Able to give written informed consent for a clinical trial
- •Scheduled to undergo an autologous or an allogeneic stem cell transplantation from a sibling, related donor, or unrelated donor using a conditioning regimen containing at least 400 cGy TBI
- •Transplantation is being performed for one of the following medical conditions:
- •Acute myelogenous leukemia
- •Acute lymphoblastic leukemia
- •Chronic myelogenous leukemia in chronic, accelerated, or blastic phase
- •Myelodysplastic syndrome
- •Myeloproliferative diseases such as chronic myelomonocytic leukemia, agnogeneic myeloid metaplasia with myelofibrosis, polycythemia vera, or essential thrombocythemia
- •Hodgkin's lymphoma
- •Non-Hodgkin's lymphoma
- •Multiple myeloma
- •Chronic lymphocytic leukemia
- •Other malignancies or marrow disease such as aplastic anemia where transplant would be appropriate with approval of principal investigator
- •Patients with therapy-related AML or MDS may be included if their prior malignancy has been in remission for at least 12 months. If the remission is less than 12 months, approval of the principal investigator is required. Entry could be allowed if the malignancy is controlled and not expected to relapse e.g. localized prostate cancer treated with XRT.
- •Patients must meet all other pre-transplantation criteria of the transplant center including acceptable tests of heart, liver, kidney, and lung function (Standard screening for HSCT per PI, and co-investigators).
- •Karnofsky performance status must be ≥70%.
排除标准
- •TBI dose less than 400 cGY
- •Cord blood transplantation
- •HIV infection
- •Pregnancy or breastfeeding
- •Creatinine or bilirubin or ALT or AST greater than two times the upper limit of normal, unless the abnormal bilirubin is due to Gilbert's syndrome
- •Active infection requiring systemic antibiotic therapy with antibacterial, antifungal, or antiviral agents
- •Concomitant enrollment in another therapeutic clinical study except with PI approval
- •Must not have previously received eltrombopag
- •Patients with moderate or severe liver disease (ALT, AST, or bilirubin ≥ 2X the upper limit of normal, unless the abnormal bilirubin is due to Gilbert's syndrome) will be excluded
- •Patients with high risk of thromboembolism based on genetic syndromes , or past thromboembolic disease in the past 6 months will be excluded from the study, with the exception of those with catheter related clots
研究组 & 干预措施
Eltrombopag
干预措施: Eltrombopag (Drug)
结局指标
主要结局
Maximum Tolerated Dose (MTD) of Eltrombopag
时间窗: 1.5 years
The MTD was defined as the highest dose if no dose limiting toxicity was observed, or the highest dose at which less than one-third of the patients experienced toxicities not expected in the standard stem cell transplantation setting.
次要结局
- Median Number of Platelet Transfusions up to the Day of Engraftment(baseline to day of engraftment)
- Median Time to Platelet Engraftment(1.5 years)
研究者
Jane Liesveld
Professor M&D-Hematology/Oncology
University of Rochester