Acute Myeloid Leukemia At Initial Diagnosis and/or Relapse in Children, Teenagers and Young Adults: Molecular Profiling, Multidrug Testing and MSC Interaction Studies

Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeUp to 25
SponsorAssistance Publique - Hôpitaux de Paris

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 volunteers

Qualifiers

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

Design model

Cohort

Time perspective

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

500 Participants
are divided into 3 treatment groups
Group A: Cohort 1 : Patients with acute myeloid leukemia1 intervention
Group B: Cohort 2 : Patients with genetic predisposition to develop acute myeloid leukemia1 intervention
Group C: Cohort 3 : Patients who undergo bone marrow aspirate1 intervention

Trial outcomes

Primary outcomes

1

Number of somatic mutations in leukemic cells between diagnosis and relapse identified by Next-Generation Sequencing (NGS)

Time frame
Up to 5 years

Secondary outcomes

1

Cumulative incidence of relapse (CIR) from remission status.

Relapse is defined as: Bone marrow blasts ≥ 5% and/or evidence of extramedullary disease

Time frame
Up to 5 years
2

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

Time frame
Up to 5 years
3

Disease Free Survival (DFS)

Disease Free Survival (DFS) is defined as the time from remission status to relapse or death.

Time frame
Up to 5 years
4

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

Time frame
Up to 5 years

Other outcomes

Sponsors and contacts

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