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

November 2, 2026 updated by: 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.

Study Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

Detailed Description

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.

Study Type

Observational

Enrollment (Estimated)

150

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

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

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 25 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

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

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: CASE_ONLY
  • Time Perspectives: OTHER

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
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.
Other: 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Histopathological profiling of NRSTS tumors
Time Frame: 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
Time Frame: 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

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Percentage of patients with associated cfDNA and tumor tissue DNA methylation profiles in NRSTS
Time Frame: 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
Time Frame: 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.
Time Frame: 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
Time Frame: 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
Time Frame: 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
Time Frame: 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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Institut Curie

Collaborators

Cancer Research Center of Lyon

Investigators

  • Study Chair: Daniel ORBACH, MD, Institut Curie

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

December 1, 2026

Primary Completion (Estimated)

December 1, 2034

Study Completion (Estimated)

December 1, 2034

Study Registration Dates

First Submitted

October 14, 2026

First Submitted That Met QC Criteria

November 2, 2026

First Posted (Actual)

November 9, 2026

Study Record Updates

Last Update Posted (Actual)

November 9, 2026

Last Update Submitted That Met QC Criteria

November 2, 2026

Last Verified

November 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • IC 2025-02
  • ID RCB : 2026-A00153-48 (Other Identifier: ANSM)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

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

IPD Sharing Time Frame

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

IPD Sharing Access Criteria

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 Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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