MoleculAr Profiling for Pediatric and Young Adult Cancer Treatment Stratification 2

NCT05691608 | Recruiting

• 2 other identifiers: 2021-A02990-412021-3321
• Study Type: interventional
• Target: 1,800
• Locations: 1 country
• Sites: 29

Brief Summary

FMG2025 continues the previous efforts to propose treatment for patients based on the molecular characteristics of their tumor at treatment failure in cancer precision medicine trials within standard of care in France. However, whereas FMG2025 is a descriptive effort providing the basis for clinical decisions, MAPPYACTS 2 will translate these findings to clinical actions. The symbiosis is critical to advance patient care. Since 2012, the molecular profiling trials "MOlecular Screening for CAncer Treatment Optimization" (MOSCATO-01) and "MoleculAr Profiling for Pediatric and Young Adult Cancer Treatment Stratification" (MAPPYACTS) have included pediatric and adolescent patients with recurrent or refractory malignancy that underwent on-purpose biopsy or surgical intervention. Whole Exome Sequencing of tumor and normal tissue and RNA Sequencing of tumor tissue have been applied to detect genomic alterations that could lead to an adapted targeted treatment. Furthermore, ancillary studies were associated exploring circulating tumor DNA, the immune contexture of tumors and developing Patient-Derived Xenografts (PDX). The FMG2025 project transfers the molecular profiling of advanced pediatric cancers into a global approach that is now considered standard of care in France. Subsequent clinical recommendations and decisions will be made based on discussions with biologists, scientist and physicians in the molecular and clinical molecular tumor boards. Associated ancillary research studies and links to clinical interventional studies remain essential elements of the program to provide clinical, translational and basic research in order to improve scientific knowledge. The program is articulated in two main parts that are closely interacting: FMG2025 - Cancers et leucémies pédiatriques en échec de traitement or equivalent international projects that cover the sequencing of tumor and blood samples and provide molecular reports. The clinical study MAPPYACTS 2 that provides clinical and therapeutic discussions of the sequencing results and therapy recommendations via the clinical molecular tumor board reports. It collects molecular and comprehensive clinical data of the patients registered in FMG2025 or equivalent international projects and thereby constitutes the critical link to clinical interventional studies and its sponsors ensuring facilitated access to these trials. It also covers and coordinates ancillary research studies.

Conditions

Solid Tumor; Leukemia

Outcome Measures

Primary Outcomes (3)

• Overall survival (OS)

  Defined as the time from study entry to death whatever the cause of death, after 3 years, for the whole cohort and according to cancer type.

  3 years

• The number of additional technologies and new treatment strategies that are introduced in the care for advanced pediatric cancers through this research program.

  The number of patients receiving matched targeted treatments The number of new treatment strategies that are introduced in the care for advanced pediatric cancers through this research program The number of additional technologies that are introduced in the care for advanced pediatric cancers through this research program

  3 years

• The types of additional technologies and new treatment strategies that are introduced in the care for advanced pediatric cancers through this research program.

  The type of patients receiving matched targeted treatments The type of new treatment strategies that are introduced in the care for advanced pediatric cancers through this research program The type of additional technologies that are introduced in the care for advanced pediatric cancers through this research program

  3 years

Secondary Outcomes (3)

• Objective response rate on treatment attributed by the molecular tumor board in the clinical trial, overall and according to the treatment decision

  3 years

• Progression-free survival (PFS)

  3 years

• 5-year overall survival

  5 years

Study Arms (1)

Patient with solid tumor or leukemia (no treatment)

EXPERIMENTAL

Patient with solid tumor or leukemia (no treatment)

Biological: Solid tumor and CT DNA

Interventions

Solid tumor and CT DNA BIOLOGICAL

Biopsy and blood sample

Patient with solid tumor or leukemia (no treatment)

Eligibility Criteria

• Age: 6 Years - 25 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Patient referred for sequencing of the tumor within the FMG2025 or equivalent program and written informed consent for FMG2025 "Cancers et leucémies pédiatriques en échec de traitement" or equivalent, according to local regulations
• Written informed consent of MAPPYACTS 2 to collect molecular and comprehensive clinical data on cancer diagnosis, therapies, therapy outcomes, to provide clinical therapeutic recommendations, to collect follow-up data on treatment and patients' outcome; optional written consents to perform to ancillary research studies, according to local regulations. The written consent will include access to reimbursement data from the French national health insurance through linkage with the Système National des Données de Santé (SNDS) or equivalent.
• Patient with histologically/cytologically confirmed solid tumor or leukemia which is relapsed or refractory to standard treatment and who is potentially eligible for an experimental treatment or an early phase clinical trial
• Planned tumor biopsy, surgical resection, bone marrow or blood sample or recently (preferably within the last 3 months) archived frozen tumor material available of the current recurrent or refractory disease
• Patients aged ≤ 25 years at the time of initial diagnosis
• Performance status and life expectancy \> 3 months expected to allow enrolment into an clinical trial
• Patients affiliated with a Social Security Regimen or beneficiary of the same, as per local regulatory requirements

You may not qualify if:

• Any concurrent illness or laboratory abnormality that, in the opinion of the investigator, is likely to interfere with the interpretation of study results
• Pregnant women

Sponsors & Collaborators

• Gustave Roussy, Cancer Campus, Grand Paris: lead

Study Sites (29)

Gustave Roussy

Paris, Villejuif, 94800, France

RECRUITING

CHU Amiens-Picardie

Amiens, 80054, France

RECRUITING

CHU Angers

Angers, 49933 Cedex 9, France

RECRUITING

CHU Besançon

Besançon, 25030, France

RECRUITING

CHU Pellegrin

Bordeaux, 33076, France

RECRUITING

CHRU Morvan

Brest, 29200, France

RECRUITING

CHU de Caen

Caen, 14000, France

RECRUITING

CHU Estaing

Clermont-Ferrand, 63000, France

RECRUITING

CHU Dijon François Mitterand

Dijon, 21079, France

RECRUITING

CHU Grenoble Alpes

Grenoble, 38043, France

RECRUITING

Centre Oscar Lambret

Lille, 59020, France

RECRUITING

Chu Limoges

Limoges, 87000, France

RECRUITING

IHOP

Lyon, 69373, France

RECRUITING

CHU La Timone

Marseille, 13005, France

RECRUITING

CHU Arnaud de Villeneuve

Montpellier, 34295, France

RECRUITING

CHU Nantes

Nantes, 44093, France

RECRUITING

CHU de Nice

Nice, 06202, France

RECRUITING

Institut Curie

Paris, 75005, France

RECRUITING

Hopital Armand Trousseau

Paris, 75012, France

RECRUITING

CHU Poitiers

Poitiers, 86022 Cedex, France

RECRUITING

CHU Reims- Hôpital Américain

Reims, 51100, France

RECRUITING

CHU Rennes Hôpital Sud

Rennes, 35203, France

RECRUITING

CHU de Rouen

Rouen, 76000, France

RECRUITING

CHU de La Réunion site nord

Saint-Denis, 97400, France

RECRUITING

CHU Saint Etienne

Saint-Etienne, 42270, France

RECRUITING

Hôpital de Hautepierre

Strasbourg, 67200, France

RECRUITING

Hôpital des Enfants - CHU de Toulouse

Toulouse, 31059, France

RECRUITING

Hôpital Clocheville

Tours, 37000, France

RECRUITING

CHRU Nancy Hôpital Enfants

Vandœuvre-lès-Nancy, 54500, France

RECRUITING

MeSH Terms

Conditions

Leukemia

Condition Hierarchy (Ancestors)

Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Birgit GEOERGER, MD

  Gustave Roussy, Cancer Campus, Grand Paris

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Birgit GEOERGER, MD

CONTACT

+33 (0)1 42 11 46 61; Birgit.GEOERGER@gustaveroussy.fr

Jean-Luc JOANNIC, PhD

CONTACT

+33 (0)1 42 11 47 94; jeanluc.joannic@gustaveroussy.fr

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: DIAGNOSTIC
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 12, 2022
• First Posted: January 20, 2023
• Study Start: September 9, 2022
• Primary Completion (Estimated): September 9, 2030
• Study Completion (Estimated): September 9, 2030
• Last Updated: August 22, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, ICF
• Time Frame: Data will become available in January 2023 The duration of the availability is up to 2030.

Locations

FR (29)