A Phase II Study of Pentostatin For the Treatment of High Risk or Refractory Chronic GVHD in Children
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 60
- 试验地点
- 49
- 主要终点
- To estimate the response rate (CR and PR) of pentostatin when used to treat refractory-chronic GVHD in children.
研究概览
简要总结
This is a multicenter trial through the Pediatric Blood and Marrow Transplant Consortium.
The Primary hypothesis of this study is that because of its effect as a potent immunosuppressive agent targeting lymphocytes, pentostatin will show a sustained response in pediatric subjects with severe chronic GVHD. Secondary hypotheses include that the infection and toxicity rate of pentostatin in this setting will be acceptable given its lack of severe myelosuppression, and subjects with refractory chronic GVHD will have significant QOL impairment and symptomatology. These may change as subjects are being treated for their chronic GVHD with pentostatin.
详细描述
To participate in this study, subjects must have diagnosed chronic Graft versus Host Disease that is refractory to therapy or that is considered high risk (i.e. low platelet count, progressive onset and greater than 50% of body surface area affected). Subjects must have not failed more than 2 immunosuppressive regimens in order to be considered for this trial. Eligible subjects will receive intravenous pentostatin every 2 weeks for 24 weeks. If the subject has had a complete response, the therapy will end at 24 weeks. If the subject has had a partial or mixed response or stable disease, they will continue on study receiving pentostatin for 52 weeks.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 1 Month 至 21 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- •More than two previous regimens to treat chronic GVHD. A regimen for the purpose of this study is defined as an adequate trial of an immunosuppressive or immunosuppressive combination (patient was given drug or drugs for 3 months unless there was clear progression during regimen).
- •Calculated Cr. Clearance<30 ml/min/1.73m2
- •Active infection not responding to oral or IV antibiotics.
- •Subjects and families unwilling to firmly commit to return for follow-up doses and appointments.
- •Use of any investigational agent in the past 30 days (this does not include the use of licensed agents for indications not listed in the package insert).
- •Pregnancy or nursing mother; Females of child-bearing potential and sexually active males should use effective contraception while on study.
- •Active veno-occlusive disease of the liver.
- •Bronchiolitis obliterans (defined for the purpose of this study by respiratory symptoms such as shortness of breath or cough and FEV1<50%).
- •Karnofsky performance score less than 40 (or Lansky performance less than 40 in subjects under 16 years of age).
结局指标
主要结局
To estimate the response rate (CR and PR) of pentostatin when used to treat refractory-chronic GVHD in children.
时间窗: To end of study
次要结局
- To gather initial efficacy and safety data in high-risk untreated chronic GVHD in children.(To end of study)
- To evaluate toxicities of pentostatin when used to treat chronic GVHD in children.(To end of study)
- To evaluate quality of life (QOL) and symptoms at diagnosis and after therapy with pentostatin in pediatric patients with refractory chronic GVHD.(To end of study)