Molecular Analysis of Samples From Patients With Diffuse Intrinsic Pontine Glioma and Brainstem Glioma
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 100
- 试验地点
- 1
- 主要终点
- Protein expression patterns as assessed by immunohistochemistry and western blot compared to normal brainstem tissue
研究概览
简要总结
The purpose of this study is to prospectively collect specimens from pediatric patients with diffuse intrinsic pontine glioma or brainstem glioma, either during therapy or at autopsy, in order to characterize the molecular abnormalities of this tumor.
详细描述
High grade diffuse intrinsic pontine glioma (DIPG) accounts for approximately 80% of pediatric brainstem tumors and 10% of pediatric brain tumors, and is the most lethal form of brainstem gliomas in children. There is currently no effective therapy to treat these tumors. We hypothesize that this tumor exhibits unique molecular abnormalities leading to altered RNA and protein expression. The aim of this trial is to collect specimens from pediatric patients with diffuse intrinsic pontine glioma including serum, cerebrospinal fluid, urine, brain tumor and other constitutional tissue, during therapy and/or at autopsy. Our goal is to study this tissue to characterize the genetic abnormalities that lead to tumor formation in order to identify key molecules as biomarkers which we can target to design and test new and more effective treatments.
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
- — 至 21 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients of any age with clinical and radiologic diagnosis of diffuse intrinsic pontine glioma
- •Patients with other high-grade gliomas originating in the brainstem
- •Patients with focal gliomas (WHO grade I/II) of the brainstem
排除标准
- •Patients with any type of infiltrative low grade (WHO grade I and II) or high grade glioma (WHO grade III and IV) originating outside the brainstem
- •Patients harboring primary brainstem tumors with other histologic diagnoses (e.g., PNET)
结局指标
主要结局
Protein expression patterns as assessed by immunohistochemistry and western blot compared to normal brainstem tissue
时间窗: 5 years
Collected tumor and normal samples will have the immunochemistry and western blot compared to assess protein expression variation.
Validation of results of the genome-wide analysis
时间窗: 5 years
The molecular analysis done on collected samples will be validated through whole genome sequencing.
In vitro and in vivo molecular analysis of collected samples
时间窗: 5 years
Collected samples will potentially be used for in vitro analysis and generation of animal models of this tumor.
Genome-wide expression patterns of RNA in tumor samples, normal brainstem tissue and cerebrospinal fluid using Affymetrix gene expression profiling
时间窗: 5 years
Collected tumor and normal samples will potentially be used for RNA genome-wide expression pattern profiling.
Genome-wide analysis of tumor samples and normal brainstem tissue
时间窗: 5 years
To obtain full characterization of collected samples, whole genome sequencing will be done on tumor and normal samples collected.
Proteomic profiling of tumor, normal brainstem tissue and cerebrospinal fluid
时间窗: 5 years
To obtain full characterization of collected samples, proteomic profiling will be done on tumor and normal samples collected.
次要结局
- Assess aspects associated with specimen acquisition, including potential benefits and drawbacks(5 years)
研究者
Javad Nazarian
Associate Professor
Children's National Research Institute