Extension Study (Extended Access) of Syk-inhibition Using Fostamatinib to Treat Post-Transplant Immune-mediated Cytopenias
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Proportion of subjects who are 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. Up to 20% of people who have blood stem cell transplants develop cytopenias, which 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 evaluate the long-term effects of a study drug (fostamatinib) in people with cytopenia after a blood stem cell transplant.
Eligibility:
People who responded well to fostamatinib in an earlier study.
Design:
Participants will be screened. They will have a physical exam and blood tests.
Fostamatinib is an oral tablet taken by mouth. Participants will take the pills at the same dose and frequency as they did during the previous study. They will take the pills for up to 21 months. The dosage of the drug may be reduced over time if their blood cell levels are stable.
Participants will have a medical assessment every month. This can be with their local doctor or at the NIH clinic.
Participants will have blood tests every 3 months.
Participants will have a follow-up visit after they stop taking the drug. Their vital signs will be taken, and they will have blood drawn. They will answer questions about their health.
详细描述
Study Description:
This is an extension trial of a phase II study which is designed to evaluate the long-term safety and efficacy of fostamatinib in the treatment of post-transplant cytopenias as assessed by hematologic improvement in anemia and/or thrombocytopenia following an additional 12-84 weeks of treatment on the initial phase II trial. The subjects who are deemed responders to the initial 12-week treatment, will be candidates to enroll onto the extension trial for up to 2 years of treatment.
Objectives:
The primary objective is to evaluate the efficacy for the use of fostamatinib in subjects with post-transplant anemia and/or thrombocytopenia at the end of total 24 weeks of treatment.
The secondary objectives are to evaluate the long-term efficacy of subjects on fostamatinib while weaning off fostamatinib treatment either partially or completely.
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 Arm
The subjects will receive oral fostamatinib daily for up to 2 years.
干预措施: fostamatinib (Drug)
结局指标
主要结局
Proportion of subjects who are able to maintain hematologic recovery
时间窗: 12 weeks
Proportion of subjects who are able to maintain hematologic recovery (as defined below) by the end of 12 weeks on the extended access trial (total of 24 weeks fostamatinib treatment). Hematologic recovery is defined as: Hemoglobin \>=10 g/dL (or at least \>=2 g/dL above baseline) in subjects enrolled with post-transplant anemia. In subjects with symptomatic anemia, a hemoglobin increase of at least \>=2 g/dL above baseline is required OR Platelets \>= 50 x 109/L (or at least =20 x 10\^9/L above baseline) in subjects enrolled with post-transplant thrombocytopenia OR Both of the above criteria in subjects with post-transplant Evan s syndrome
次要结局
- Change in corticosteroid dose(12 weeks)
- Proportion of subjects who are able to taper off fostamatinib by >33% while maintaining hematologic recovery(106 weeks)
- Change in the dose of other immunosuppressive agents(12 weeks)
- Proportion of subjects who are able to completely taper off fostamatinib while maintaining hematologic recovery(106 weeks)