About this trial
Pediatric acute myeloid leukemias are disease with poor prognosis (overall survival of 60-75%) and high relapse rate of 35-45% require further understanding of the underlying biological mechanisms.
The main objective of this study is to establish a biological collection to evaluate the genomic profiling of leukemic cells from primary blasts at diagnosis and/or relapse to improve identification of the main genetic hits involved in resistance and could predict a high risk of relapse. Other objectives include the study of bone marrow mesenchymal stem cells and ex vivo drug testing.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
0-25 years old
Newly diagnosed de novo or secondary Acute Myeloid Leukemia (AML) or
Relapsed or refractory AML or
Patients with genetic predisposition to develop AML or
Disqualifiers
Refuse to participate
Chronic myeloid leukemia (CML)
Lack of health insurance (French social security)
Under protection (tutelle, curatelle or sauvegarde de justice)
Trial population
Children or young adult with Newly diagnosed de novo or secondary Acute Myeloid Leukemia (AML) or Relapsed or refractory AML or patients with genetic predisposition to develop AML or patients without haematological malignancy nor AML genetic predisposition syndrome who undergo bone marrow aspirate as part of standard of care
Trial design
Cohort
Prospective
Treatments tested in this trial
Collection of blood sample of bone marrow (cohort 1)
Other intervention* 3 additional tubes of blood sample (cohort 1), at diagnosis and upon relapse if relapse occurs * Bone marrow aspirate : 3 additional tubes (cohort 1), at diagnosis and upon relapse if relapse occurs
Collection of blood sample of bone marrow (cohort 2 and 3)
Other intervention* 1 additional tube of blood sample (cohort 2 and 3 at inclusion) * Bone marrow aspirate: 1 additional tube (cohort 2 and 3 at inclusion)
Treatment groups
Trial outcomes
Primary outcomes
Number of somatic mutations in leukemic cells between diagnosis and relapse identified by Next-Generation Sequencing (NGS)
Secondary outcomes
Cumulative incidence of relapse (CIR) from remission status.
Relapse is defined as: Bone marrow blasts ≥ 5% and/or evidence of extramedullary disease
Event Free Survival (EFS)
Event Free Survival (EFS) is defined as the time from start of chemotherapy to failure, relapse, or death which ever occurs first
Disease Free Survival (DFS)
Disease Free Survival (DFS) is defined as the time from remission status to relapse or death.
Number of mutations identified by WGS
Number of mutations identified by Whole-Genome-Sequencing (WGS) as compared to Next-Generation Sequencing (NGS) in leukemic cells
Sponsors and contacts
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