Prospective, Interventional Diagnostic, Multicenter, Non-treatment Clinical Study Identifying Specific Molecular Changes by Using Genomic Sequencing Technologies in Refractory/Recurrent or Very High-risk Pediatric CNS Tumors.
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 80
- 试验地点
- 5
- 主要终点
- The proportion of patients in which F1Heme molecular testing identified at least 1 clinically relevant alteration at the time of MTB decision.
研究概览
简要总结
PPROSPERITIT is a prospective clinical study assessing the use of comprehensive molecular profiling to define the best matching targeted and immune treatment for relapsed, refractory or very high risk pediatric CNS tumors.
详细描述
PPROSPERITIT will identify specific molecular changes by using genomic sequencing technologies in refractory/recurrent or very high-risk pediatric CNS tumors. The study will employ an analytically validated comparison of a selection of targeted agents/immune therapies on the basis of commercially available comprehensive genomic profiling FoundationOneHeme panel (F1Heme, comprising DNA and RNA analysis) vs selection of agents based on more complex DNA/RNA/Protein based analyses. This will be coupled to a computer algorithm that uses preexisting definitions and prioritization of target-agent pairs to assign patients by actionable mutation results to a targeted treatment. The selection of targeted agents will be performed by a multidisciplinary molecular tumor board, but the recommended treatment will not be a part of the PPROSPERTIT study.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Diagnostic
- 盲法
- None
入排标准
- 年龄范围
- 1 Year 至 19 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patient Informed consent form must be appropriately obtained under the applicable local and regulatory requirements. Each patient must sign a consent form prior the enrollment to document their willingness to participate.
- •The subject is male or female, aged 1 - 19 years
- •The subject must have a histologically proven recurrent/ refractory or very high-risk CNS tumors
- •Patients must be in good overall physical condition, which allows tumor biopsy
- •Patients must have a life expectancy of at least 3 months.
- •Patients must have a tumor amenable to image-guided or direct vision biopsy and be willing and able to undergo a tumor biopsy and/or blood taking for molecular profiling.
- •Patients must be accessible for follow-up.
排除标准
- •Patients with known psychiatric or substance abuse disorders that would interfere with cooperation with the requirements of the trial.
- •Pregnant and/or breastfeeding women, if applicable
- •No intention to treat the patient.
研究组 & 干预措施
Diagnostic group
Tumor and blood samples will be collected from each patient and broad molecular profiling will be performed. The results of the evaluation of the tumor specimens will determine if the patient's tumor has an actionable mutation for which treatment is available.
干预措施: FoundationOneHeme (Device)
结局指标
主要结局
The proportion of patients in which F1Heme molecular testing identified at least 1 clinically relevant alteration at the time of MTB decision.
时间窗: Diagnostic assessment is done within 28 days from enrolment patient in the study.
Evaluation of the feasibility of FoundationOneHeme
次要结局
未报告次要终点