A Phase II Study of Intraventricular Methotrexate With Systemic Topotecan and Cyclophosphamide in Children With Recurrent or Progressive Malignant Brain Tumors
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 发起方
- 入组人数
- 3
- 试验地点
- 1
- 主要终点
- Tumor response
研究概览
简要总结
The purpose of this research study is to test an experimental treatment method for recurrent or progressive brain tumors in children aged from 0-22 years. The use of methotrexate and chemotherapy (topotecan and cyclophosphamide) is experimental in this study. This means that their use by themselves or together has not been approved by the U.S. Food and Drug Administration for this usage.
详细描述
The purpose of this research study is to test an experimental treatment method for recurrent or progressive brain tumors in children aged from 0-22 years. The use of methotrexate and chemotherapy (topotecan and cyclophosphamide) is experimental in this study. This means that their use by themselves or together has not been approved by the U.S. Food and Drug Administration for this usage. This study will attempt to determine via serial MRI scans if methotrexate administration into the lateral or fourth ventricle in combination with systemic intravenous topotecan and cyclophosphamide is effective in reducing tumor burden in the brain and spine for patients with recurrent or progressive brain tumors, including medulloblastoma, ependymoma, PNET (Primitive Neuroectodermal Tumor), atypical teratoid/rhabdoid tumor (AT/RT), and other malignant embryonal tumors. In addition, the study will evaluate the toxicity of the above mentioned experimental treatment during a two-year progression-free survival and survival of children with recurrent malignant brain tumors.
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
- — 至 22 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Recurrent or progressive supratentorial or posterior fossa tumors with measurable disease on imaging studies of the brain and spine or CSF cytology.
- •Tumor histology, including: Medulloblastoma , PNET, or other embryonal tumor, Atypical teratoid/rhaboid tumor (AT/RT), and Ependymoma.
- •Leptomeningeal dissemination of a previously diagnosed CNS tumor.
- •Diagnosis of tumor pathology will be based upon pathology diagnosis from previous surgeries for patients with recurrent tumors.
- •Patients must have received prior radiation before current recurrence, unless the patient is less than 36 months old at diagnosis and has progressed after at least one upfront chemotherapy regimen in which case no prior radiation is required.
- •Patients must have a life expectancy of at least 12 weeks as indicated by the patient's oncologist and/or neurosurgeon.
- •Lansky or Karnofsky Performance status of at least
- •Negative pregnancy test.
- •Specific organ function requirements for: Central Nervous System, Bone Marrow, renal and liver.
排除标准
- •Patients that do not meet the inclusion criteria above.
- •Pregnant or lactating female patients.
- •Patients currently enrolled in another experimental treatment protocol.
- •Patients with documented allergies to any of the chemotherapy agents used in this study.
- •Patient/Parent refuses study participation.
- •Patient is severely somnolent or comatose.
- •Unable or unwilling to commit to return or to follow-up visits.
研究组 & 干预措施
Intra thecal methotrexate
IT methotrexate via Ommaya reservoir with concomitant systemic topotecan and cyclophosphamide
干预措施: Intra thecal methotrexate (Drug)
Intra thecal methotrexate
IT methotrexate via Ommaya reservoir with concomitant systemic topotecan and cyclophosphamide
干预措施: topotecan (Drug)
Intra thecal methotrexate
IT methotrexate via Ommaya reservoir with concomitant systemic topotecan and cyclophosphamide
干预措施: cyclophosphamide (Drug)
结局指标
主要结局
Tumor response
时间窗: 6 months
MRI response
次要结局
- Toxicity of chemotherapy regimen (topotecan and cyclophosphamide)(6 months)
研究者
Ziad Khatib
Director of Neuro-Oncology
Nicklaus Children's Hospital f/k/a Miami Children's Hospital