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.
Studienübersicht
Status
Intervention / Behandlung
Detaillierte Beschreibung
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.
Studientyp
Einschreibung (Geschätzt)
Kontakte und Standorte
Studienkontakt
- Name: Carole CAGNOT
- Telefonnummer: +33 1 47 11 18 91
- E-Mail: carole.cagnot@curie.fr
Studienorte
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-
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Amiens, Frankreich, 80054
- Chu Amiens
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Hauptermittler:
- Antoine GOURMEL, MD
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Angers, Frankreich, 49033
- Chu Angers
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Hauptermittler:
- Stéphanie PROUST, MD
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Besançon, Frankreich, 25030
- Chu Besancon
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Hauptermittler:
- Victor PEREIRA, MD
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Bordeaux, Frankreich, 33000
- Chu Pellegrin
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Hauptermittler:
- Jordane CHAIX, MD
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Brest, Frankreich, 29609
- Chru Brest
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Hauptermittler:
- Liana CARAUSU, MD
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Caen, Frankreich, 14033
- CHU CAEN
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Hauptermittler:
- Marianna DEPARIS, MD
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Clermont-Ferrand, Frankreich, 63000
- Chu Clermont Ferrand
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Hauptermittler:
- Pauline GIRARD-MONIN, MD
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Dijon, Frankreich, 21000
- Chu Dijon
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Hauptermittler:
- Florent NEUMANN, MD
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Grenoble, Frankreich, 38043
- Chu Grenoble
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Hauptermittler:
- Séverine BOBILLIER-CHAUMONT, MD
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Lille, Frankreich, 59020
- Centre Oscar Lambret
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Hauptermittler:
- Anne-Sophie DEFACHELLES-THOMASSIN, MD
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Limoges, Frankreich, 87042
- Chu Limoges
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Hauptermittler:
- Thomas LAUVRAY, MD
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Lyon, Frankreich, 69008
- IHOPe - CENTRE LEON BERARD
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Hauptermittler:
- Nadège CORRADINI, MD
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Marseille, Frankreich, 13005
- Chu La Timone - Hopital Enfants
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Kontakt:
- MD
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Hauptermittler:
- Angélique ROME, MD
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Montpellier, Frankreich, 34090
- Chu Montpellier
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Hauptermittler:
- Stéphanie HAOUY, MD
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Kontakt:
- E-Mail: Nadege.corradini@ihope.fr
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Nantes, Frankreich, 44093
- CHU Nantes - Hôpital Mère Enfant
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Kontakt:
- E-Mail: Nadege.corradini@ihope.fr
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Hauptermittler:
- Morgane CLEIREC, MD
-
Nice, Frankreich, 06200
- CHU NICE
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Hauptermittler:
- Morgane PODROM, MD
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Paris, Frankreich, 75005
- Institut Curie
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Hauptermittler:
- Daniel ORBACH, MD
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Paris, Frankreich, 75012
- Ap-Hp - Hopital Trousseau
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Hauptermittler:
- Hélène BOUTROUX, MD
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Poitiers, Frankreich, 86021
- CHU Poitiers
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Hauptermittler:
- Chrystelle DUPRAZ, MD
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Rennes, Frankreich, 35203
- Chu de Rennes - Hôpital Sud
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Hauptermittler:
- Jamie PROBERT, MD
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Rouen, Frankreich, 76031
- Chu Rouen
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Hauptermittler:
- Aude MARIE-CARDINE, MD
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Saint-Etienne, Frankreich, 42055
- Chu Saint Etienne
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Hauptermittler:
- Marina GOUTTENOIRE, MD
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Strasbourg, Frankreich, 67000
- Chu Strasbourg - Hôpital Hautepierre
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Hauptermittler:
- Sarah JANNIER, MD
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Toulouse, Frankreich, 31059
- Chu Toulouse
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Hauptermittler:
- Marie-Pierre CASTEX, MD
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Tours, Frankreich, 37044
- Chu Tours - Hôpital Clocheville
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Hauptermittler:
- Jill SERRE, MD
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Vandœuvre-lès-Nancy, Frankreich, 54500
- CHU NANCY- Hôpital d'Enfants
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Hauptermittler:
- Ludovic MANSUY, MD
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Villejuif, Frankreich, 94800
- Gustave Roussy
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Kontakt:
- Telefonnummer: +33 1 42 11 60 5
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Hauptermittler:
- Lucy METAYER, MD
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La Réunion
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Saint-Denis, La Réunion, Frankreich, 97400
- Chr Felix Guyon
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Hauptermittler:
- Yves REGUERRE, MD
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Teilnahmekriterien
Zulassungskriterien
Studienberechtigtes Alter
Akzeptiert gesunde Freiwillige
Studienberechtigte Geschlechter
Probenahmeverfahren
Studienpopulation
Children and young adults (aged from 0 to 25 years) with a new diagnosis of NRSTS.
Beschreibung
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.
Studienplan
Wie ist die Studie aufgebaut?
Designdetails
- Beobachtungsmodelle: CASE_ONLY
- Zeitperspektiven: OTHER
Kohorten und Interventionen
Gruppe / Kohorte |
Intervention / Behandlung |
|---|---|
|
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 |
Was misst die Studie?
Primäre Ergebnismessungen
Ergebnis Maßnahme |
Maßnahmenbeschreibung |
Zeitfenster |
|---|---|---|
|
Histopathological profiling of NRSTS tumors
Zeitfenster: 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
Zeitfenster: 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
|
Sekundäre Ergebnismessungen
Ergebnis Maßnahme |
Maßnahmenbeschreibung |
Zeitfenster |
|---|---|---|
|
Percentage of patients with associated cfDNA and tumor tissue DNA methylation profiles in NRSTS
Zeitfenster: 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
Zeitfenster: 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.
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
|
Mitarbeiter und Ermittler
Sponsor
Mitarbeiter
Ermittler
- Studienstuhl: Daniel ORBACH, MD, Institut Curie
Studienaufzeichnungsdaten
Haupttermine studieren
Studienbeginn (Geschätzt)
Primärer Abschluss (Geschätzt)
Studienabschluss (Geschätzt)
Studienanmeldedaten
Zuerst eingereicht
Zuerst eingereicht, das die QC-Kriterien erfüllt hat
Zuerst gepostet (Tatsächlich)
Studienaufzeichnungsaktualisierungen
Letztes Update gepostet (Tatsächlich)
Letztes eingereichtes Update, das die QC-Kriterien erfüllt
Zuletzt verifiziert
Mehr Informationen
Begriffe im Zusammenhang mit dieser Studie
Schlüsselwörter
Zusätzliche relevante MeSH-Bedingungen
Andere Studien-ID-Nummern
- IC 2025-02
- ID RCB : 2026-A00153-48 (Andere Kennung: ANSM)
Plan für individuelle Teilnehmerdaten (IPD)
Planen Sie, individuelle Teilnehmerdaten (IPD) zu teilen?
Beschreibung des IPD-Plans
Sponsor will share de-identified data sets. Documents generated under the project will be disseminated in accordance with Institut Curie policies.
IPD-Sharing-Zeitrahmen
Data requests can be submitted starting 9 months after last article publication and will be made accessible for up to 12 months.
IPD-Sharing-Zugriffskriterien
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).
Art der unterstützenden IPD-Freigabeinformationen
- STUDIENPROTOKOLL
- SAFT
Arzneimittel- und Geräteinformationen, Studienunterlagen
Studiert ein von der US-amerikanischen FDA reguliertes Arzneimittelprodukt
Studiert ein von der US-amerikanischen FDA reguliertes Geräteprodukt
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