MoleculAr Profiling for Pediatric and Young Adult Cancer Treatment Stratification 2
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 1,800
- 试验地点
- 57
- 主要终点
- The number of additional technologies and new treatment strategies that are introduced in the care for advanced pediatric cancers through this research program.
研究概览
简要总结
FMG2025 continues the previous efforts to propose treatment for patients based on the molecular characteristics of their tumor at treatment failure in cancer precision medicine trials within standard of care in France. However, whereas FMG2025 is a descriptive effort providing the basis for clinical decisions, MAPPYACTS 2 will translate these findings to clinical actions. The symbiosis is critical to advance patient care.
Since 2012, the molecular profiling trials "MOlecular Screening for CAncer Treatment Optimization" (MOSCATO-01) and "MoleculAr Profiling for Pediatric and Young Adult Cancer Treatment Stratification" (MAPPYACTS) have included pediatric and adolescent patients with recurrent or refractory malignancy that underwent on-purpose biopsy or surgical intervention. Whole Exome Sequencing of tumor and normal tissue and RNA Sequencing of tumor tissue have been applied to detect genomic alterations that could lead to an adapted targeted treatment. Furthermore, ancillary studies were associated exploring circulating tumor DNA, the immune contexture of tumors and developing Patient-Derived Xenografts (PDX).
The FMG2025 project transfers the molecular profiling of advanced pediatric cancers into a global approach that is now considered standard of care in France. Subsequent clinical recommendations and decisions will be made based on discussions with biologists, scientist and physicians in the molecular and clinical molecular tumor boards. Associated ancillary research studies and links to clinical interventional studies remain essential elements of the program to provide clinical, translational and basic research in order to improve scientific knowledge.
The program is articulated in two main parts that are closely interacting:
FMG2025 - Cancers et leucémies pédiatriques en échec de traitement or equivalent international projects that cover the sequencing of tumor and blood samples and provide molecular reports.
The clinical study MAPPYACTS 2 that provides clinical and therapeutic discussions of the sequencing results and therapy recommendations via the clinical molecular tumor board (CMTB) reports. It collects molecular and comprehensive clinical data of the patients registered in FMG2025 or equivalent international projects and thereby constitutes the critical link to clinical interventional studies and its sponsors ensuring facilitated access to these trials. It also covers and coordinates ancillary research studies.
Due to the delay in opening of the MAPPYACTS 2 trial, clinical and molecular data for patients whose tumors were sequenced within FMG2025 or equivalent and not included in MAPPYACTS 2 before CMTB or equivalent, will be collected retrospectively after a specific patient/legal representative information and will contribute to the endpoints of the trial as adequate.
详细描述
MAPPYACTS 2 is an ambispective (prospective and retrospective) international multicentric clinical study to provide clinical therapeutic recommendations, to set up the molecular and comprehensive clinical database of patients with relapsed or refractory pediatric malignancies in FMG2025 - Cancers et leucémies pédiatriques en échec de traitement or equivalent international projects, to collect the follow-up data on treatment and patients' outcome, in order to determine the outcome of the program in regard to benefit to the single patient, all the patients and to health care. It will also serve as a central link to interventional study platforms and international precision medicine programs, and cover and coordinate ancillary research studies that lead to improve treatment and outcome for children with advanced malignancies.
Clinical recommendations and decisions following tumor sequencing are made based on discussions with biologists, scientists and physicians in the molecular and clinical molecular tumor boards run by the FMG2025 and MAPPYACTS 2 study teams.
Associated ancillary research studies and links to clinical interventional studies are essential components of the program to perform clinical, translational and basic research in order to generate scientific knowledge and develop new treatment strategies that improve outcome of these patients. Research projects are discussed and agreed on in the MAPPYACTS 2 study committee.
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
- 6 Years 至 25 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Patient referred for sequencing of the tumor within the FMG2025 or equivalent program and written consent for FMG2025 - Cancers et leucémies pédiatriques en échec de traitement or equivalent signed
- •Written informed consent for MAPPYACTS 2 (or non-opposition for retrospective clinical data collection) according to local or national regulations
- •Patient with confirmed solid tumor or leukemia which is relapsed or refractory to standard treatment and who is potentially eligible for an experimental treatment or an early phase clinical trial at the time of tumor sequencing
- •Planned tumor biopsy, surgical resection, bone marrow or blood sample or recently (possibly within the last 3 months) archived frozen tumor material available of the current recurrent or refractory disease
- •Patients aged ≤ 25 years at the time of initial diagnosis
- •Performance status and life expectancy > 3 months at the time of tumor sequencing that allows enrolment into an experimental trial
- •Patients affiliated with a Social Security Regimen or beneficiary of the same as per local regulatory requirements
排除标准
- •Any concurrent illness or laboratory abnormality that, in the opinion of the investigator, is likely to interfere with the interpretation of study results
- •Pregnant women
研究组 & 干预措施
Patient with solid tumor or leukemia (no treatment)
Patient with solid tumor or leukemia (no treatment)
干预措施: Solid tumor and CT DNA (Biological)
结局指标
主要结局
The number of additional technologies and new treatment strategies that are introduced in the care for advanced pediatric cancers through this research program.
时间窗: 3 years
The number of patients receiving matched targeted treatments The number of new treatment strategies that are introduced in the care for advanced pediatric cancers through this research program The number of additional technologies that are introduced in the care for advanced pediatric cancers through this research program
Overall survival (OS)
时间窗: 3 years
Defined as the time from study entry to death whatever the cause of death, after 3 years, for the whole cohort and according to cancer type.
The types of additional technologies and new treatment strategies that are introduced in the care for advanced pediatric cancers through this research program.
时间窗: 3 years
The type of patients receiving matched targeted treatments The type of new treatment strategies that are introduced in the care for advanced pediatric cancers through this research program The type of additional technologies that are introduced in the care for advanced pediatric cancers through this research program
次要结局
- Objective response rate on treatment attributed by the molecular tumor board in the clinical trial, overall and according to the treatment decision(3 years)
- Progression-free survival (PFS)(3 years)
- 5-year overall survival(5 years)
- Objective response rate on treatment attributed by the molecular tumor board in the clinical trial, overall and according to the treatment decision(5 years)
- Progression-free survival (PFS)(5 years)