Clinical trial NCT03496402: Biological Characterisation of High Risk CHildhood Cancer in Children, Adolescents and Young Adults (MICCHADO)
Official title: Molecular and Immunological Characterisation of High Risk CHildhood Cancer At DiagnOsis, Treatment and Follow-up - Biological Evaluation in Children, Adolescents and Young Adults -
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: NA
Conditions: Neuroblastoma
Rhabdomyosarcoma
Ewing Sarcoma Family of Tumors
Osteosarcoma
Leukemia
Central Nervous System Tumor
Lead sponsor: Institut Curie
Sponsor class: OTHER
First posted: 2018-04-12
Enrollment: 600
Sex: ALL
Minimum age (years): 1
Maximum age (years): 25
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: OTHER

Brief summary: Methodology:

Prospective, multicentric, open, non-randomised, non-therapeutic, interventional study

Detailed description: To identify and characterise:

* meaningful molecular genetic alterations,
* meaningful immunological features of high risk childhood, adolescents and young adult cancers, at diagnosis, during patient treatment and follow-up (time dimension).

Eligibility criteria: Inclusion Criteria:

1. Inclusion within 3 months after diagnosis
2. Availability of a cryopreserved tumour sample (primary and/or metastatic and/or lymph nodes) or peripheral blood or bone marrow samples (if invasion more than 30% of lymphoblasts) for leukaemias, obtained at the time of diagnosis during a routine procedure
3. Availability of a formalin-fixed paraffin-embedded (FFPE) tumour sample (primary and/or metastasis and/or lymph nodes), obtained at the time of diagnosis during a routine procedure (except for leukaemia patients)
4. Age: ≤ 25 years at diagnosis
5. Written patient informed consent, or parents or legal representative written informed consent and assent of the child and the adolescent
6. Compulsory affiliation to a social security scheme

   Additional inclusion criteria for the study:

   To avoid multiple sampling for children, adolescents and young adults with cancer, patients already included or to be included in a study with similar analyses and/or objectives might also be included in MICCHADO study and in this case, samples or data might be exchanged on a collaborative basis.

   Cohort 1:
   * High risk neuroblastoma:

     - Any type of neuroblastoma with MYCN amplification, except INSS stage 1

     - Stage 4 neuroblastoma in children older than one year at diagnosis
   * High risk rhabdomyosarcoma:

     * Foxo1 rearrangement any stage;
     * and / or N1 ;
     * and / or metastatic rhabdomyosarcoma
   * High risk Ewing sarcoma:

     * Metastatic Ewing sarcoma family of tumours (ESFT)
     * Localised inoperable Ewing sarcoma with primary tumours ≥ 200 ml
   * High risk osteosarcoma:

     - Metastatic osteosarcoma

     - Localised inoperable osteosarcoma
   * High risk leukaemia:

     * Secondary acute myeloid leukaemia
     * Biphenotypic acute leukaemia

   Cohort 2:

   • Extra cerebral or cerebral high risk tumours including:
   * other metastatic sarcomas,
   * other rare high risk cancers,
   * high risk renal tumours with surgery after an initial chemotherapy
   * rhabdoid brain tumours (AT/RT) and extra cerebral rhabdoid tumours
   * high risk or metastatic cancers of unclear histological diagnosis • Lymphoblastic leukaemia with high MRD at Day 78 (time point 2) • Very high risk T-cells acute lymphoblastic leukaemia:
   * MRD ≥ 10-2 at the end of the induction ;
   * or MRD ≥ 10-3 at Day 78

   Cohort 3:

   Children, adolescents and young adults, with low/intermediate risk cancers belonging to the following types:

   • Neuroblastoma:

   - Localised, without MYCN amplification
   * Localised, INSS stage 1, with MYCN amplification
   * Stage 4s, in infants (younger than one year at diagnosis), without MYCN amplification

     • Rhabdomyosarcoma:
   * Localised, without Foxo1 rearrangement

     • ESFT:
   * All non-high risk localised ESFT • Osteosarcoma:
   * All non-high risk localised osteosarcoma

   Exclusion Criteria:

   Main non-inclusion Criteria common to all study cohorts:

1) Age: patients \> 25 years old at diagnosis 2) Absence of patient or parents or legal representative written informed consent 3) Patient for whom follow-up by the investigating centre does not appear feasible

Primary outcomes:
measure: Number of patients with meaningful molecular genetic alterations
timeFrame: At the end of study (6 years)
measure: Number of patients with meaningful immunological features
timeFrame: At the end of study (6 years)
measure: Number of patients with identification of new tumor-specific genetic characteristics during follow-up (clonal evolution)
timeFrame: up to 6 years
Secondary outcomes:
measure: Correlation between disease recurrence and molecular and/or immunological biomarkers
timeFrame: up to 6 years
measure: Correlation between genetic variations and immune parameters
timeFrame: up to 6 years
measure: Correlation between disease staging and immunological features
timeFrame: up to 6 years
Other outcomes:
Locations:
city: Amiens
country: France
facility: Chu D'Amiens Picardie
city: Angers
country: France
facility: CHU Angers
city: Besançon
country: France
facility: CHRU de Besançon - Hôpital Jean-Minjoz
city: Bordeaux
country: France
facility: CHU de Bordeaux - Hôpital des enfants - Groupe Hospitalier Pellegrin
city: Brest
country: France
facility: CHRU de Brest
city: Caen
country: France
facility: CHU CAEN
city: Clermont-Ferrand
country: France
facility: Centre Régional de Cancérologie et Thrapie Cellulaire Pdiatrique (CRCTCP)
city: Dijon
country: France
facility: CHU Hôpital d'Enfants
city: Grenoble
country: France
facility: CHU GRENOBLE Alpes - Hôpital Couple-Enfant
city: Lille
country: France
facility: Centre Oscar Lambret
city: Limoges
country: France
facility: CHU de Limoges - Hôpital Mère-Enfant
city: Lyon
country: France
facility: Centre Léon Bérard
city: Lyon
country: France
facility: Hospices Civils de Lyon
city: Marseille
country: France
facility: Hôpital d'Enfants de la Timone (AP-HM)
city: Montpellier
country: France
facility: CHU Arnaud de Villeneuve
city: Nantes
country: France
facility: CHU Nantes - Hôpital Mère Enfant
city: Nice
country: France
facility: Hôpital l'Archet 2
city: Paris
country: France
facility: Hôpital d'Enfants Armand-Trousseau
city: Paris
country: France
facility: Hôpital universitaire Robert-Debré (AP-HP)
city: Paris
country: France
facility: Institut Curie
city: Poitiers
country: France
facility: CHU de Poitiers
city: Reims
country: France
facility: CHU de Reims - Hôpital Américain
city: Rennes
country: France
facility: Chu Hopital Sud Rennes
city: Rouen
country: France
facility: CHU de Rouen - Hôp. Charles NICOLLE
city: Saint-Étienne
country: France
facility: CHU Saint-Etienne - Hôpital Nord
city: Strasbourg
country: France
facility: Hôpitaux Universitaires de Strasbourg - Hôpital de Hautepierre
city: Toulouse
country: France
facility: CHU Hôpital des Enfants
city: Tours
country: France
facility: CHU TOURS - Hôpital Clocheville
city: Vandoeuvre les Nancy
country: France
facility: CHU Nancy - Hôpital d'Enfants
city: Villejuif
country: France
facility: Gustave Roussy
Study officials:
affiliation: Institut Curie
name: Gudrun SCHLEIERMACHER, MD
role: PRINCIPAL_INVESTIGATOR
References: