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.
調査の概要
状態
状態
条件
条件
介入・治療
介入・治療
詳細な説明
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.
研究の種類
研究の種類
入学 (推定)
入学
連絡先と場所
研究連絡先
研究連絡先
- 名前:Carole CAGNOT
- 電話番号:+33 1 47 11 18 91
- メール:carole.cagnot@curie.fr
研究場所
-
-
-
Amiens、フランス、80054
- Chu Amiens
-
主任研究者:
- Antoine GOURMEL, MD
-
Angers、フランス、49033
- Chu Angers
-
主任研究者:
- Stéphanie PROUST, MD
-
Besançon、フランス、25030
- Chu Besancon
-
主任研究者:
- Victor PEREIRA, MD
-
Bordeaux、フランス、33000
- Chu Pellegrin
-
主任研究者:
- Jordane CHAIX, MD
-
Brest、フランス、29609
- Chru Brest
-
主任研究者:
- Liana CARAUSU, MD
-
Caen、フランス、14033
- CHU CAEN
-
主任研究者:
- Marianna DEPARIS, MD
-
Clermont-Ferrand、フランス、63000
- Chu Clermont Ferrand
-
主任研究者:
- Pauline GIRARD-MONIN, MD
-
Dijon、フランス、21000
- Chu Dijon
-
主任研究者:
- Florent NEUMANN, MD
-
Grenoble、フランス、38043
- Chu Grenoble
-
主任研究者:
- Séverine BOBILLIER-CHAUMONT, MD
-
Lille、フランス、59020
- Centre Oscar Lambret
-
主任研究者:
- Anne-Sophie DEFACHELLES-THOMASSIN, MD
-
Limoges、フランス、87042
- Chu Limoges
-
主任研究者:
- Thomas LAUVRAY, MD
-
Lyon、フランス、69008
- IHOPe - CENTRE LEON BERARD
-
主任研究者:
- Nadège CORRADINI, MD
-
Marseille、フランス、13005
- Chu La Timone - Hopital Enfants
-
コンタクト:
- MD
-
主任研究者:
- Angélique ROME, MD
-
Montpellier、フランス、34090
- Chu Montpellier
-
主任研究者:
- Stéphanie HAOUY, MD
-
コンタクト:
-
Nantes、フランス、44093
- CHU Nantes - Hôpital Mère Enfant
-
コンタクト:
-
主任研究者:
- Morgane CLEIREC, MD
-
Nice、フランス、06200
- CHU NICE
-
主任研究者:
- Morgane PODROM, MD
-
Paris、フランス、75005
- Institut Curie
-
主任研究者:
- Daniel ORBACH, MD
-
Paris、フランス、75012
- Ap-Hp - Hopital Trousseau
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主任研究者:
- Hélène BOUTROUX, MD
-
Poitiers、フランス、86021
- CHU Poitiers
-
主任研究者:
- Chrystelle DUPRAZ, MD
-
Rennes、フランス、35203
- Chu de Rennes - Hôpital Sud
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主任研究者:
- Jamie PROBERT, MD
-
Rouen、フランス、76031
- Chu Rouen
-
主任研究者:
- Aude MARIE-CARDINE, MD
-
Saint-Etienne、フランス、42055
- Chu Saint Etienne
-
主任研究者:
- Marina GOUTTENOIRE, MD
-
Strasbourg、フランス、67000
- Chu Strasbourg - Hôpital Hautepierre
-
主任研究者:
- Sarah JANNIER, MD
-
Toulouse、フランス、31059
- Chu Toulouse
-
主任研究者:
- Marie-Pierre CASTEX, MD
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Tours、フランス、37044
- Chu Tours - Hôpital Clocheville
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主任研究者:
- Jill SERRE, MD
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Vandœuvre-lès-Nancy、フランス、54500
- CHU NANCY- Hôpital d'Enfants
-
主任研究者:
- Ludovic MANSUY, MD
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Villejuif、フランス、94800
- Gustave Roussy
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コンタクト:
- 電話番号:+33 1 42 11 60 5
-
主任研究者:
- Lucy METAYER, MD
-
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La Réunion
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Saint-Denis、La Réunion、フランス、97400
- Chr Felix Guyon
-
主任研究者:
- Yves REGUERRE, MD
-
-
参加基準
適格基準
適格基準
就学可能な年齢
健康ボランティアの受け入れ
受講資格のある性別
サンプリング方法
調査対象母集団
Children and young adults (aged from 0 to 25 years) with a new diagnosis of NRSTS.
説明
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.
研究計画
研究はどのように設計されていますか?
デザインの詳細
- 観測モデル:CASE_ONLY
- 時間の展望:OTHER
グループ/コホートの数
コホートと介入
グループ/コホートグループ/コホート |
介入・治療介入・治療 |
|---|---|
|
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 |
この研究は何を測定していますか?
主要な結果の測定
主要な結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Histopathological profiling of NRSTS tumors
時間枠:Baseline
|
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
|
|
Molecular profiling of NRSTS tumors
時間枠:Baseline
|
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
|
二次結果の測定
二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Percentage of patients with associated cfDNA and tumor tissue DNA methylation profiles in NRSTS
時間枠:Through study completion, an average of 4 years
|
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
時間枠:Baseline
|
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
|
|
Percentage of patients with detectable cfDNA molecular changes associated with disease status or relapse.
時間枠:Through study completion, an average of 4 years
|
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
|
|
Identification of prognostic and predictive biomarkers a in NRSTS
時間枠: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
|
|
Percentage of synovial sarcoma organoids showing similar histological and molecular profiles to their parental tumors
時間枠: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
|
|
Impact of systematic national review on tumor diagnosis
時間枠:Baseline
|
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
|
協力者と研究者
協力者
協力者
捜査官
捜査官
- スタディチェア:Daniel ORBACH, MD、Institut Curie
研究記録日
主要日程の研究
研究開始 (推定)
研究開始
一次修了 (推定)
一次修了
研究の完了 (推定)
研究の完了
試験登録日
最初に提出
最初に提出
QC基準を満たした最初の提出物
QC基準を満たした最初の提出物
最初の投稿 (実際)
最初の投稿
学習記録の更新
投稿された最後の更新 (実際)
投稿された最後の更新
QC基準を満たした最後の更新が送信されました
QC基準を満たした最後の更新が送信されました
最終確認日
最終確認日
詳しくは
本研究に関する用語
キーワード
その他の研究ID番号
その他の研究ID番号
- IC 2025-02
- ID RCB : 2026-A00153-48 (その他の識別子:ANSM)
個々の参加者データ (IPD) の計画
個々の参加者データ (IPD) を共有する予定はありますか?
IPD プランの説明
Sponsor will share de-identified data sets. Documents generated under the project will be disseminated in accordance with Institut Curie policies.
IPD 共有時間枠
Data requests can be submitted starting 9 months after last article publication and will be made accessible for up to 12 months.
IPD 共有アクセス基準
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 共有サポート情報タイプ
- STUDY_PROTOCOL
- SAP
医薬品およびデバイス情報、研究文書
米国FDA規制医薬品の研究
米国FDA規制機器製品の研究
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