Campath-1h as Immunoablative Therapy for Children and Adolescents With Treatment Refractory Systemic Sclerosis
试验速览
- 阶段
- 不适用
- 状态
- 撤回
- 主要终点
- Campath
研究概览
简要总结
This phase I/II pilot trial seeks to demonstrate that prolonged administration of Campath-1H without prior marrow or stem cell harvesting can result in immunoablation similar to that achieved by hematopoietic stem cell transplantation (HSCT) from either bone marrow or peripheral blood stem cell sources in children and adolescents with severe treatment refractory systemic sclerosis (SSc).
详细描述
Patients, 8 to18 years of age, will be included if they have a proven diagnosis of diffuse cutaneous or systemic SSc as defined by the ACR criteria with evidence of active inflammatory disease Plus at least 1 of the following:SSc-related pulmonary disease with forced vital capacity (FVC) or hemoglobin-adjusted DLCO < 70% and evidence of alveolitis by high-resolution CT scan or bronchoalveolar lavage.
OR:History of SSc-related renal crisis or disease, not active at the time of screening
OR:Moderate to severe upper and/or lower gastrointestinal involvement
AND:Unacceptable toxicity or steroid dependence > 0.3 mg/kg/d,
OR:Failure to respond to, or unacceptable toxicity of MTX > 1mg/kg in combination with cyclosporine or azathioprine or cyclophosphamide 2 kg/d or Rituximab 375 mg/m2 x 4 doses or Imatinib 800 mg/
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 8 Years 至 18 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
- 未提供
结局指标
主要结局
Campath
时间窗: The site will follow patients for 6 months post adverse event.
Number of Participants with Adverse Events as a Measure of Safety and Tolerability
Primary outcome
时间窗: 2 years
To determine why the extended administration of Campath-1H results in immune ablation in some patients and immunosuppression in others, Number of Participants with Adverse Events as a Measure of Safety and Tolerability Campath-1H antibody levels during and after the completion of the Campath administration. (47) Thus, both the peak Campath-1H levels as well as the duration of circulating Campath will be determined.
次要结局
未报告次要终点
研究者
Diane Brown
Co-Principal Investigator
Children's Hospital Los Angeles