ETMR One: An International Registry and Research Platform for Children With Embryonal Tumor With Multilayer Rosettes
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 100
- 试验地点
- 2
- 主要终点
- ETMR One Registry
研究概览
简要总结
This research is being done to find out more information about a brain tumor called Embryonal Tumor with Multilayer Rosettes (ETMR) by collecting medical information from children who have this disease.
The purpose of this research study is to create and maintain a research database for patients with ETMR. The database will include information about occurrence rates, patient information, tumor tissue information, and response to treatment. This will help advance our understanding of this rare disease.
In addition, this study will include obtaining survival data and evaluating therapeutic response to expert consensus therapy, and procuring patient tumor tissue.
详细描述
All patients with ETMR are eligible for inclusion within the registry, regardless of their disease status or treatment plan. Epidemiologic, clinical, and molecular data from each patient will be obtained following enrollment. Patients will then be followed longitudinally to obtain clinical outcome data. In an effort to best achieve the registry's secondary objectives, it is preferable, but not required, for patients to be registered soon after their diagnosis, prior to the initiation of any tumor-directed therapy.
This registry protocol contains recommendations for a consensus therapy derived from the ETMR medical literature, the investigators' own experience, and clinical data from other infant brain tumor protocols. The recommended therapy can thus not be viewed as investigational, but rather as a consensus recommendation derived from available data. While not mandated, it is strongly encouraged that patients enrolled in the registry be treated according to the recommended consensus therapy, as this will facilitate the prospective evaluation of ETMR patients using a uniform treatment approach.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients of any age
- •Patients must have either a histologically confirmed primary intracranial CNS Embryonal Tumor with Multilayer Rosettes (as agreed upon by central review or local pathologist OR
- •Patients must have tumor tissue that possesses C19MC amplification . Central Review is not required (but is strongly recommended) if the patient's tumor does not demonstrate C19MC amplification.
- •Patients may be enrolled at point following diagnosis
排除标准
- 未提供
结局指标
主要结局
ETMR One Registry
时间窗: 10 years
To create and maintain a comprehensive registry containing the epidemiologic, clinical, and molecular data of patients with embryonal tumor with multilayer rosettes.
Efficacy of consensus therapy on median event-free survival
时间窗: 10 years
To investigate the efficacy of the consensus regimen of chemotherapy, high-dose chemotherapy with autologous stem cell rescue, and radiotherapy (as indicated) following maximal feasible surgical resection for patients with newly-diagnosed ETMR as measured by median event-free and overall survival in comparison to historical controls.
次要结局
未报告次要终点
