Personalized Targeted Therapy in Refractory or Relapsed Cancer in Childhood(TRICEPS Study)
试验速览
- 阶段
- 不适用
- 发起方
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- Feasibility of performing genomic data-based targeted therapy clinical trials in childhood cancers with poor prognosis, including relapsed or refractory cancers.
研究概览
简要总结
A new research paradigm that involves sequencing tumor DNA/RNA to identify driver mutations, select among the Health Canada approved drugs (for adult cancers) known to block certain oncogenic pathways, and recommend these drugs to the treating physician, without taking into account the tumor histology.
In this paradigm, the treatment is targeted to the actionable mutation(s) i.e. those driving oncogenesis. It is also personalized to the molecular signature of the patient's tumor, irrespective of its histopathological subtype. The experience of the investigators team in genomics, including next generation sequencing and bioinformatic analysis combined with the clinical expertise, bring at last this approach within our technical capacities. In parallel, the number of Health Canada-approved drugs (which have been tested in a pediatric setting) designed to interfere with oncogenesis pathways is increasing exponentially.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Prospective
入排标准
- 年龄范围
- 1 Month 至 21 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •At the time of enrollment:
- •21 year-old or less
- •Poor prognosis biopsy-proven cancer of any type :
- •Cancer (at initial diagnosis) known to be refractory to treatment
- •Or cancer refractory to treatment
- •Or relapsed cancer
- •Written informed consent by patient, parents, or the legal guardians
排除标准
- •Estimated life expectancy of less than 3 months.
结局指标
主要结局
Feasibility of performing genomic data-based targeted therapy clinical trials in childhood cancers with poor prognosis, including relapsed or refractory cancers.
时间窗: 24 months
The study team will evaluate the timeline between decision of biopsy, the actual biopsy, availability of results of the whole-genome analysis, interpretation of results and divulgation of results to patient and family.
次要结局
- Number and type of driver mutation(s) found in our population of recurrent or refractory cancers.(24 months)
- Feasibility of performing whole genome sequencing and data analysis, identifying a drug based on the genomic data and offering this information to the medical team, the patient and the family within 10-week time frame from diagnosis(24 months)
- Number of cancer patients who harbour actionable driver mutation(s) that can be targeted with a Health Canada approved targeted drug.(24 months)
- Number of children with cancer who are suitable candidates for targeted therapy at our institution each year.(24 months)
研究者
Dr Monia Marzouki
Hematologist/Oncologist
St. Justine's Hospital