Prognostic and Predictive Factors in Childhood Soft Tissue Sarcomas (MAKI-France)
The goal of this study is to better understand biological characteristics of non-rhabdomyosarcoma soft tissue sarcomas (NRSTS), a rare group of cancers that affect children, adolescents, and young adults. Researchers also aim to identify factors that could help predict how the disease will behave and respond to treatment.
The main questions it aims to answer are:
- Can advanced molecular analyses improve the classification and diagnosis of NRSTS?
- Can blood- and tumor-based biomarkers help predict outcomes and detect relapse earlier?
- Can researchers identify biological features that may guide future personalized treatments?
Participants aged 0 to 25 years with a recent diagnosis of NRSTS will take part in the prospective study. Researchers will collect archived tumor samples and blood samples during routine follow-up (approximately every 3 to 4 months) and will analyze these samples using advanced laboratory techniques. Clinical information will also be collected over a follow-up period of up to 4 years.
150 participants will be enrolled in the prospective part from 28 investigating centers across France.
Tumor samples already collected as part of your care, together with blood samples taken during follow-up visits, will be stored for future researches.
研究概览
详细说明
MAKI-France is an ambispective study. In addition to the 150 participants to include in the prospective part, 300 tumors collected as part of routine care will be analyzed.
研究类型
注册 (估计的)
联系人和位置
学习联系方式
- 姓名:Carole CAGNOT
- 电话号码:+33 1 47 11 18 91
- 邮箱:carole.cagnot@curie.fr
学习地点
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Amiens、法国、80054
- Chu Amiens
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首席研究员:
- Antoine GOURMEL, MD
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Angers、法国、49033
- Chu Angers
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首席研究员:
- Stéphanie PROUST, MD
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Besançon、法国、25030
- Chu Besancon
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首席研究员:
- Victor PEREIRA, MD
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Bordeaux、法国、33000
- Chu Pellegrin
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首席研究员:
- Jordane CHAIX, MD
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Brest、法国、29609
- Chru Brest
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首席研究员:
- Liana CARAUSU, MD
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Caen、法国、14033
- CHU CAEN
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首席研究员:
- Marianna DEPARIS, MD
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Clermont-Ferrand、法国、63000
- Chu Clermont Ferrand
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首席研究员:
- Pauline GIRARD-MONIN, MD
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Dijon、法国、21000
- Chu Dijon
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首席研究员:
- Florent NEUMANN, MD
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Grenoble、法国、38043
- Chu Grenoble
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首席研究员:
- Séverine BOBILLIER-CHAUMONT, MD
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Lille、法国、59020
- Centre Oscar Lambret
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首席研究员:
- Anne-Sophie DEFACHELLES-THOMASSIN, MD
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Limoges、法国、87042
- Chu Limoges
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首席研究员:
- Thomas LAUVRAY, MD
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Lyon、法国、69008
- IHOPe - CENTRE LEON BERARD
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首席研究员:
- Nadège CORRADINI, MD
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Marseille、法国、13005
- Chu La Timone - Hopital Enfants
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接触:
- MD
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首席研究员:
- Angélique ROME, MD
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Montpellier、法国、34090
- Chu Montpellier
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首席研究员:
- Stéphanie HAOUY, MD
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接触:
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Nantes、法国、44093
- CHU Nantes - Hôpital Mère Enfant
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接触:
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首席研究员:
- Morgane CLEIREC, MD
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Nice、法国、06200
- CHU NICE
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首席研究员:
- Morgane PODROM, MD
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Paris、法国、75005
- Institut Curie
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首席研究员:
- Daniel ORBACH, MD
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Paris、法国、75012
- Ap-Hp - Hopital Trousseau
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首席研究员:
- Hélène BOUTROUX, MD
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Poitiers、法国、86021
- CHU Poitiers
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首席研究员:
- Chrystelle DUPRAZ, MD
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Rennes、法国、35203
- Chu de Rennes - Hôpital Sud
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首席研究员:
- Jamie PROBERT, MD
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Rouen、法国、76031
- Chu Rouen
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首席研究员:
- Aude MARIE-CARDINE, MD
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Saint-Etienne、法国、42055
- Chu Saint Etienne
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首席研究员:
- Marina GOUTTENOIRE, MD
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Strasbourg、法国、67000
- Chu Strasbourg - Hôpital Hautepierre
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首席研究员:
- Sarah JANNIER, MD
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Toulouse、法国、31059
- Chu Toulouse
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首席研究员:
- Marie-Pierre CASTEX, MD
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Tours、法国、37044
- Chu Tours - Hôpital Clocheville
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首席研究员:
- Jill SERRE, MD
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Vandœuvre-lès-Nancy、法国、54500
- CHU NANCY- Hôpital d'Enfants
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首席研究员:
- Ludovic MANSUY, MD
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Villejuif、法国、94800
- Gustave Roussy
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接触:
- 电话号码:+33 1 42 11 60 5
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首席研究员:
- Lucy METAYER, MD
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La Réunion
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Saint-Denis、La Réunion、法国、97400
- Chr Felix Guyon
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首席研究员:
- Yves REGUERRE, MD
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参与标准
资格标准
适合学习的年龄
接受健康志愿者
有资格学习的性别
取样方法
研究人群
Children and young adults (aged from 0 to 25 years) with a new diagnosis of NRSTS.
描述
Inclusion Criteria:
- Patients with local diagnosis of intermediate or malignant NRSTS, at the time of initial diagnosis only.
- Localized or metastatic disease.
- Age: 0-25 years.
- Whatever strategy is decided (observation, surgery, chemotherapy, radiotherapy, other systemic therapies).
- Within the 3 months after initial diagnosis.
- Written informed consent from adult patients, or the 2 parents/legal representatives. Assent from minor patients should be verified.
- Availability of archived tissue samples. Adult participants or parents must be willing to provide archival tissue. A minimum of 1 FFPE block / 1 HE slide with IHC slides available is required.
- Patient affiliated to a health insurance system.
Exclusion Criteria:
- NRSTS at relapse.
- Any significant medical, psychological, social or geographic conditions that, in the opinion of the Investigator, would impair study participation or cooperation.
- Patients deprived of their liberty or under guardianship.
学习计划
研究是如何设计的?
设计细节
- 观测模型:CASE_ONLY
- 时间观点:OTHER
队列和干预
团体/队列 |
干预/治疗 |
|---|---|
|
NRSTS group
Single arm for all patients. Tumor samples will be taken for histological and molecular profiling, at baseline (FFPE for routine care or tissue sample from surgery. Blood samples will be taken at baseline and every 3- months up to end of study post surgery. |
Tumor samples will be taken for histological and molecular characterization at baseline (routine care biopsy) or surgery. Blood samples (liquid biopsies) will be taken at baseline and every 3-4 months up to end of study for early relapse detection |
研究衡量的是什么?
主要结果指标
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Histopathological profiling of NRSTS tumors
大体时间:Baseline
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Histopathological description of NRSTS tumors based on analysis of tumor tissue in paraffin. Results will be reported as the number and percentage of tumors classified into each histological subtype. |
Baseline
|
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Molecular profiling of NRSTS tumors
大体时间:Baseline
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Molecular profiling of NRSTS tumors combining DNA and RNA profiling, DNA methylation analysis, and clustering. Results will be reported as the number and percentage of tumors presenting identified molecular alterations. |
Baseline
|
次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Percentage of patients with associated cfDNA and tumor tissue DNA methylation profiles in NRSTS
大体时间:Through study completion, an average of 4 years
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Assessment of the association between DNA methylation profiles obtained from cell-free DNA (cfDNA) and matched tumor tissue DNA samples |
Through study completion, an average of 4 years
|
|
Classification Accuracy of DNA Methylation-Based Classifiers for NRSTS Subtypes
大体时间:Baseline
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Classification results for NRSTS subtypes obtained using the Institut Curie classifier based on DNA methylation and RNA profiling compared with those obtained using the Heidelberg classifier. Agreement in subtype classification and the distribution of classification results between the two classifiers will be reported. |
Baseline
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Percentage of patients with detectable cfDNA molecular changes associated with disease status or relapse.
大体时间:Through study completion, an average of 4 years
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Detection and characterization of prognostic and epigenetic biomarkers using cell-free DNA (cfDNA) analysis at initial diagnosis and during follow-up assessments performed every 3-4 months. Changes in cfDNA methylation profiles over time and their association with disease status and relapse will be reported, including the detection of molecular changes potentially indicative of relapse. |
Through study completion, an average of 4 years
|
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Identification of prognostic and predictive biomarkers a in NRSTS
大体时间:Through study completion, an average of 4 years
|
Sensitivity and specificity of molecular biomarkers, including epigenetic alterations at diagnosis and relapse, such as fragmentomic profiles and nucleosome positioning, genomic index, CINCARC, and other prognostic molecular signatures, compared with conventional clinical assessment and imaging. |
Through study completion, an average of 4 years
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Percentage of synovial sarcoma organoids showing similar histological and molecular profiles to their parental tumors
大体时间:At the time of surgical resection, up to 4 years
|
Assessment of the similarity between synovial sarcoma organoids and their corresponding parental tumor tissues based on histological and molecular characterization. |
At the time of surgical resection, up to 4 years
|
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Impact of systematic national review on tumor diagnosis
大体时间:Baseline
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Number and proportion of tumors with concordant diagnoses or diagnostic modifications following systematic pathological and molecular review at the National Reference Center in Lyon. Diagnostic modifications will be classified as minor, without significant clinical impact, or major, with clinical impact. |
Baseline
|
合作者和调查者
研究记录日期
研究主要日期
学习开始 (估计的)
初级完成 (估计的)
研究完成 (估计的)
研究注册日期
首次提交
首先提交符合 QC 标准的
首次发布 (实际的)
研究记录更新
最后更新发布 (实际的)
上次提交的符合 QC 标准的更新
最后验证
更多信息
与本研究相关的术语
其他研究编号
- IC 2025-02
- ID RCB : 2026-A00153-48 (其他标识符:ANSM)
计划个人参与者数据 (IPD)
计划共享个人参与者数据 (IPD)?
IPD 计划说明
Sponsor will share de-identified data sets. Documents generated under the project will be disseminated in accordance with Institut Curie policies.
IPD 共享时间框架
Data requests can be submitted starting 9 months after last article publication and will be made accessible for up to 12 months.
IPD 共享访问标准
Access to trial individual participant data can be requested by qualified researchers engaging in independent scientific research, and will be provided following review and approval of a research proposal and Statistical Analysis Plan (SAP) and execution of a data sharing agreement (DSA).
IPD 共享支持信息类型
- 研究方案
- 树液
药物和器械信息、研究文件
研究美国 FDA 监管的药品
研究美国 FDA 监管的设备产品
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