A Phase II Study Using Fostamatinib to Treat Post-Hematopoietic Stem Cell Transplant Immune-Mediated Cytopenias
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 1
- 试验地点
- 1
- 主要终点
- Number of Participants Who Were Able to Maintain Hematologic Recovery
研究概览
简要总结
Background:
People who have a blood stem cell transplant can sometimes develop cytopenia. This means that their levels of one or more types of blood cell, such as the red cells or platelets, are lower than they should be. This can occur because a person s immune system might attack these cells after a stem cell transplant. Cytopenia can lead to anemia, severe bleeding, infections, and other problems. Treatments are needed to help keep blood cell levels stable after blood stem cell transplant.
Objective:
To test a study drug (fostamatinib) in people who have cytopenia after a blood stem cell transplant.
Eligibility:
People aged 18 to 75 years who have cytopenia after a blood stem cell transplant.
Design:
Participants will be screened. They will have a physical exam. They will have blood, urine, and stool tests.
Fostamatinib is an oral tablet taken by mouth. Participants will take the pills 2 times a day for 12 weeks.
Participants will have a medical assessment every 2 weeks; their vital signs will be checked, and they will have blood and stool tests. Participants must come to the NIH clinic for these visits in weeks 4 and 12. Other visits may be done by telephone or telehealth; the blood and stool tests can be sent to the researchers from a local lab.
After 4 weeks, some participants may begin taking a higher dose of the drug.
Participants will return for a final medical assessment 2 weeks after they finish taking the drug.
Participants who complete this study and show evidence that fostamatinib has increased their blood cell counts may enroll in an extension study to continue taking fostamatinib.
详细描述
Study Description:
This open label phase II trial is designed to evaluate the efficacy of fostamatinib in the treatment of post-transplant cytopenias as assessed by hematologic improvement in anemia and/or thrombocytopenia following a 12-week treatment course. Patients who respond to the 12-week treatment course on this single arm study are eligible and have the option to enroll on the extended access trial.
Objectives:
The primary objective is to assess efficacy of fostamatinib for stable hematologic recovery during post-hematopoietic stem cell transplant immune mediated anemia and/or thrombocytopenia.
The secondary objective is to assess efficacy of fostamatinib for clinically-relevant outcomes in post-hematopoietic stem cell transplant patients.
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
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Fostamatinib in Participants Post-Hematopoietic Stem Cell Transplant with Immune-Mediated Cytopenias
Participants Post-Hematopoietic Stem Cell Transplant with Immune-Mediated Cytopenias will receive fostamatinib 100 mg BID by mouth for 4 weeks. On the 4-week evaluation, a) if cytopenia improves (hemoglobin =10 g/dL, platelets = 50 x 109/L), patients will continue the same dose for a total of 12 weeks, b) if refractory cytopenias persist (hemoglobin < 10 g/dL, platelets < 50 x 109/L), the dose will be increased to 150 mg BID. Subjects with persistent (=2 readings, 2 weeks apart) loss of hematologic response after week 5 can increase their dose to 150 mg BID until at end of the study on week 12.
干预措施: fostamatinib (Drug)
结局指标
主要结局
Number of Participants Who Were Able to Maintain Hematologic Recovery
时间窗: Baseline, Week 2, Week 4, Week 6, Week 8, Week 10
Stable hematologic recovery (improvement documented in 2 consecutive available readings) without recent blood product transfusion support (in the past 48-72 hours). Hematologic recovery is defined as: * Hemoglobin =10 g/dL (or at least =2 g/dL above baseline) in participants enrolled with posttransplant anemia. In participants with symptomatic anemia, a hemoglobin increase of at least =2 g/dL above baseline is required OR * Platelets = 50 x 10\^9/L (or at least =20 x 10\^9/L above baseline) in participants enrolled with posttransplant thrombocytopenia OR * Both of the above criteria in participants with posttransplant Evans syndrome
次要结局
- Number of Participants Who Achieve Objective Hematologic Recovery(Up to 10 weeks)
- Median Change in Requirement of Transfused Blood Component(Baseline, Week 2, Week 4, Week 6, Week 8, Week 10)
- Median Change in Requirement of Growth Factor Injections(Baseline, Week 2, Week 4, Week 6, Week 8, Week 10)
- Median Change in Requirement Corticosteroid Dose (Prednisone)(Baseline, Week 2, Week 4, Week , Week 8, Week 10)
- Median Change in Other Immunosuppressant Dose(Baseline, Week 2, Week 4, Week 6, Week 8, Week 10, Week 12)
- Number of Participants Who Developed Mild, Moderate or Severe Chronic Graft vs Host Disease (GVHD) According to 2014 NIH Consensus Criteria(Up to 10 weeks)
- Number of Patients Who Achieved 50% Steroid Dose Reduction(Up to Week 10)