Use of Specific Genetic Alteration s of Tumoral Cells Identified by the Next Generation Sequencing Techniques (NGS) to Follow Peripheral Samples of Children With Metastatic and/or High Risk Solid Tumor - NGSKids (NGSKids)

Use of Specific Genetic Alteration s of Tumoral Cells Identified by NGS to Follow Peripheral Samples of Children With Metastatic and/or High Risk Solid Tumor -

The search for genetic alterations in primary tumor by NGS techniques followed by the detection of these alterations in circulating tumor DNA and/or CTC/DTC present in peripheral samples (blood, cerebrospinal fluid, bone marrow, possibly urine) collected during several steps and after the treatment could be a tool to monitor the response during and after the treatment.

Study Overview

• Status: Completed
• Conditions: Metastatic and/or High Risk Solid Tumor of Children
• Intervention / Treatment: Biological: Tumoral specific genetic alterations; Biological: Tumoral specific genetic alterations

• Study Type: Interventional
• Enrollment (Actual): 30
• Phase: Not Applicable

Contacts and Locations

Study Locations

• France
  • Paris, France, 75005
    • Institut Curie

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 18 years (Child, Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Children with metastatic and/or high risk solid tumor, of the following pathologies :

Neuroblastoma, sarcoma, malignant brain tumor (medulloblastoma, high-grade glioma), bone tumors, rhabdoid tumors, others rare tumors

• Availability of a frozen tumoral sample (primary tumor or metastasis whatever the localization) at diagnosis allowing analysis of genetic alterations by a NGS technique
• Age < 18 years
• Signed informed consent by parents or legal representatives
• Patient having health care insurance

Exclusion Criteria :

• Age ≥ 18 years
• No signed informed consent by parents or legal representatives

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Diagnostic
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Other: Tumoral specific genetic alterations; NGS techniques (next generation sequencing) will be used to identify specific genetic alterations of tumoral cells of a patient. If specific genetic alterations is detected, they will be used to detect circulating tumor DNA and/or circulating/disseminated tumoral cells (CTC/DTC) in peripheral samples (blood, bone marrow, cerebral spinal fluid) collected before, during and after treatment.

Biological: Tumoral specific genetic alterations; A buccal swab and a blood sample will be used at the diagnostic to identify the specific genetic alterations of tumoral cells.; Biological: Tumoral specific genetic alterations; Collection of blood (maximum 9 samples of 3 to 5 ml), bone marrow (maximum 3 samples of 3 to 5 ml) and cerebral spinal fluid (maximum 3 samples 500µl to 1ml).

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Detection by Polymerase Chain Reaction (PCR) of specific genetic alterations	Genetic alterations which have been previously detected by NGS technique in the tumor, in circulating tumoral DNA and/or CTC/DTC present in a blood sample at the inclusion.	at the inclusion
Detection of specific genetic alterations of tumoral cells in peripheral samples	Detection of specific genetic alterations of tumoral cells in peripheral samples for which presence of tumoral cells has been confirmed by conventional clinic techniques (cytology, anatomopathology, immunohistochemistry	Up to 6 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Detection of genetic alterations in solid tumor pediatric samples	Use of identified genetic alterations in solid tumor pediatric samples to help to confirm diagnosis and prognosis and to search for new therapeutic targets	At the inclusion
Change of CTC/DTC/circulating tumoral DNA levels detected by PCR targeting specific genetic alterations of tumoral cells in peripheral samples will be confronted to clinical features including patient outcome		Up to 6 years

Collaborators and Investigators

• Sponsor: Institut Curie

Study record dates

Study Major Dates

• Study Start (Actual): September 1, 2014
• Primary Completion (Actual): January 1, 2021
• Study Completion (Actual): January 1, 2021

Study Registration Dates

• First Submitted: April 23, 2015
• First Submitted That Met QC Criteria: September 8, 2015
• First Posted (Estimated): September 10, 2015

Study Record Updates

• Last Update Posted (Actual): January 31, 2024
• Last Update Submitted That Met QC Criteria: January 29, 2024
• Last Verified: January 1, 2024

More Information

Terms related to this study

• Keywords: high risk solid tumor pediatric, genetic alteration, Metastatic solid tumor pediatric
• Additional Relevant MeSH Terms: Neoplasms
• Other Study ID Numbers: IC 2014-01

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: SAP