Securing Access to Innovative Molecules in Oncology and Hematology for Children, Adolescents and Young Adults in Therapeutic Failure or Relapse and Not Eligible for a Clinical Trial: a Project of the SFCE
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 1,000
- 试验地点
- 31
- 主要终点
- Collect access data
研究概览
简要总结
It involves collecting safety and efficacy data, under the actual conditions of use of medicines in children and adolescents, using a validated tool (Ennov EDC) and relying on the network of Interregional pediatric oncology appeal organizations (RIOs) identified by INCa since 2010 and responsible for the organization of Pluridisciplinary Pediatric Interregional (RCPPI) and National Consultative Meetings which discuss each case of relapse in order to define the best therapeutic options.
详细描述
The objective is to aim for completeness throughout the national territory. This is why this project will be carried out in close collaboration with the RIOs and RCPPI and that of the 31 centers of the SFCE which bring together more than 400 doctors pediatric oncologists and hematologists on the French territory
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 25 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age ≤ 25 years old at the time of inclusion in the study
- •Patient with a pediatric tumor or leukemia in therapeutic failure or relapse without standard therapeutic options. Exceptionally, patients on the first line of treatment without standard therapeutic options may also be included (eg g: inoperable plexiform neurofibroma and MEK inhibitors, childhood fibrosarcoma and NTRK inhibitors).
- •Patient not eligible for an early phase clinical trial open to inclusion on French territory or refusal of inclusion
- •Patient treated with a new drug discussed at a RCPPI as part of a compassionate use issued by the ANSM or an off-label prescription of a drug of interest already authorized in adults.
- •Patients treated in one of the SFCE centers authorized to prescribe chemotherapy
- •Patient who did not object to participate after being informed of the study. The patient must be able and willing to cooperate in the study.
排除标准
- •Patient included in an early phase clinical trial open to inclusions on French territory.
- •Oral refusal to participate by the patient or their legal representatives, in reading the information note specific to the study
- •Patient under guardianship or curatorship, deprived of liberty or in the impossibility of expressing opposition to participating in the study
结局指标
主要结局
Collect access data
时间窗: up to 1 year
Collect access data of children, adolescents and young adults in therapeutic failures and not eligible for a clinical trial with innovative molecules, whether they are targeted therapies, immunotherapies or chemotherapies
次要结局
未报告次要终点