Denne side blev automatisk oversat, og nøjagtigheden af ​​oversættelsen er ikke garanteret. Der henvises til engelsk version for en kildetekst.

Prognostic and Predictive Factors in Childhood Soft Tissue Sarcomas (MAKI-France)

2. november 2026 opdateret af: Institut Curie

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.

Studieoversigt

Status

Ikke rekrutterer endnu

Betingelser

Intervention / Behandling

Detaljeret beskrivelse

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.

Undersøgelsestype

Observationel

Tilmelding (Anslået)

150

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiekontakt

Studiesteder

  • Frankrig
      • Amiens, Frankrig, 80054
        • Chu Amiens
        • Ledende efterforsker:
          • Antoine GOURMEL, MD
      • Angers, Frankrig, 49033
        • Chu Angers
        • Ledende efterforsker:
          • Stéphanie PROUST, MD
      • Besançon, Frankrig, 25030
        • Chu Besancon
        • Ledende efterforsker:
          • Victor PEREIRA, MD
      • Bordeaux, Frankrig, 33000
        • Chu Pellegrin
        • Ledende efterforsker:
          • Jordane CHAIX, MD
      • Brest, Frankrig, 29609
        • Chru Brest
        • Ledende efterforsker:
          • Liana CARAUSU, MD
      • Caen, Frankrig, 14033
        • CHU CAEN
        • Ledende efterforsker:
          • Marianna DEPARIS, MD
      • Clermont-Ferrand, Frankrig, 63000
        • Chu Clermont Ferrand
        • Ledende efterforsker:
          • Pauline GIRARD-MONIN, MD
      • Dijon, Frankrig, 21000
        • Chu Dijon
        • Ledende efterforsker:
          • Florent NEUMANN, MD
      • Grenoble, Frankrig, 38043
        • Chu Grenoble
        • Ledende efterforsker:
          • Séverine BOBILLIER-CHAUMONT, MD
      • Lille, Frankrig, 59020
        • Centre Oscar Lambret
        • Ledende efterforsker:
          • Anne-Sophie DEFACHELLES-THOMASSIN, MD
      • Limoges, Frankrig, 87042
        • Chu Limoges
        • Ledende efterforsker:
          • Thomas LAUVRAY, MD
      • Lyon, Frankrig, 69008
        • IHOPe - CENTRE LEON BERARD
        • Ledende efterforsker:
          • Nadège CORRADINI, MD
      • Marseille, Frankrig, 13005
        • Chu La Timone - Hopital Enfants
        • Kontakt:
          • MD
        • Ledende efterforsker:
          • Angélique ROME, MD
      • Montpellier, Frankrig, 34090
      • Nantes, Frankrig, 44093
      • Nice, Frankrig, 06200
        • CHU NICE
        • Ledende efterforsker:
          • Morgane PODROM, MD
      • Paris, Frankrig, 75005
        • Institut Curie
        • Ledende efterforsker:
          • Daniel ORBACH, MD
      • Paris, Frankrig, 75012
        • Ap-Hp - Hopital Trousseau
        • Ledende efterforsker:
          • Hélène BOUTROUX, MD
      • Poitiers, Frankrig, 86021
        • CHU Poitiers
        • Ledende efterforsker:
          • Chrystelle DUPRAZ, MD
      • Rennes, Frankrig, 35203
        • Chu de Rennes - Hôpital Sud
        • Ledende efterforsker:
          • Jamie PROBERT, MD
      • Rouen, Frankrig, 76031
        • Chu Rouen
        • Ledende efterforsker:
          • Aude MARIE-CARDINE, MD
      • Saint-Etienne, Frankrig, 42055
        • Chu Saint Etienne
        • Ledende efterforsker:
          • Marina GOUTTENOIRE, MD
      • Strasbourg, Frankrig, 67000
        • Chu Strasbourg - Hôpital Hautepierre
        • Ledende efterforsker:
          • Sarah JANNIER, MD
      • Toulouse, Frankrig, 31059
        • Chu Toulouse
        • Ledende efterforsker:
          • Marie-Pierre CASTEX, MD
      • Tours, Frankrig, 37044
        • Chu Tours - Hôpital Clocheville
        • Ledende efterforsker:
          • Jill SERRE, MD
      • Vandœuvre-lès-Nancy, Frankrig, 54500
        • CHU NANCY- Hôpital d'Enfants
        • Ledende efterforsker:
          • Ludovic MANSUY, MD
      • Villejuif, Frankrig, 94800
        • Gustave Roussy
        • Kontakt:
          • Telefonnummer: +33 1 42 11 60 5
        • Ledende efterforsker:
          • Lucy METAYER, MD
    • La Réunion
      • Saint-Denis, La Réunion, Frankrig, 97400
        • Chr Felix Guyon
        • Ledende efterforsker:
          • Yves REGUERRE, MD

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

Ikke ældre end 25 år (Barn, Voksen)

Tager imod sunde frivillige

Ingen

Køn, der er berettiget til at studere

Alle

Prøveudtagningsmetode

Ikke-sandsynlighedsprøve

Studiebefolkning

Children and young adults (aged from 0 to 25 years) with a new diagnosis of NRSTS.

Beskrivelse

Inclusion Criteria:

  1. Patients with local diagnosis of intermediate or malignant NRSTS, at the time of initial diagnosis only.
  2. Localized or metastatic disease.
  3. Age: 0-25 years.
  4. Whatever strategy is decided (observation, surgery, chemotherapy, radiotherapy, other systemic therapies).
  5. Within the 3 months after initial diagnosis.
  6. Written informed consent from adult patients, or the 2 parents/legal representatives. Assent from minor patients should be verified.
  7. 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.
  8. Patient affiliated to a health insurance system.

Exclusion Criteria:

  1. NRSTS at relapse.
  2. Any significant medical, psychological, social or geographic conditions that, in the opinion of the Investigator, would impair study participation or cooperation.
  3. Patients deprived of their liberty or under guardianship.

Studieplan

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

  • Observationsmodeller: CASE_ONLY
  • Tidsperspektiver: OTHER

Kohorter og interventioner

Gruppe / kohorte
Intervention / Behandling
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.
Andet: Intervention

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

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Histopathological profiling of NRSTS tumors
Tidsramme: 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
Tidsramme: 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

Sekundære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Percentage of patients with associated cfDNA and tumor tissue DNA methylation profiles in NRSTS
Tidsramme: 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
Tidsramme: 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.
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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

Samarbejdspartnere og efterforskere

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Sponsor

Institut Curie

Samarbejdspartnere

Cancer Research Center of Lyon

Efterforskere

  • Studiestol: Daniel ORBACH, MD, Institut Curie

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart (Anslået)

1. december 2026

Primær færdiggørelse (Anslået)

1. december 2034

Studieafslutning (Anslået)

1. december 2034

Datoer for studieregistrering

Først indsendt

14. oktober 2026

Først indsendt, der opfyldte QC-kriterier

2. november 2026

Først opslået (Faktiske)

9. november 2026

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

9. november 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

2. november 2026

Sidst verificeret

1. november 2026

Mere information

Begreber relateret til denne undersøgelse

Nøgleord

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

  • IC 2025-02
  • ID RCB : 2026-A00153-48 (Anden identifikator: ANSM)

Plan for individuelle deltagerdata (IPD)

Planlægger du at dele individuelle deltagerdata (IPD)?

JA

IPD-planbeskrivelse

Sponsor will share de-identified data sets. Documents generated under the project will be disseminated in accordance with Institut Curie policies.

IPD-delingstidsramme

Data requests can be submitted starting 9 months after last article publication and will be made accessible for up to 12 months.

IPD-delingsadgangskriterier

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-deling Understøttende informationstype

  • STUDY_PROTOCOL
  • SAP

Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter

Studerer et amerikansk FDA-reguleret lægemiddelprodukt

Ingen

Studerer et amerikansk FDA-reguleret enhedsprodukt

Ingen

Disse oplysninger blev hentet direkte fra webstedet clinicaltrials.gov uden ændringer. Hvis du har nogen anmodninger om at ændre, fjerne eller opdatere dine undersøgelsesoplysninger, bedes du kontakte register@clinicaltrials.gov. Så snart en ændring er implementeret på clinicaltrials.gov, vil denne også blive opdateret automatisk på vores hjemmeside .

Abonner