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Translational Research - Observational Study for Identification of New Possible Prognostic Factors and Future Therapeutic Targets in Children With Acute Lymphoblastic Leukemia (ALL)

1 Year
17 Years
Open (Enrolling)
Leukemia, Lymphoma

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Trial Information

Translational Research - Observational Study for Identification of New Possible Prognostic Factors and Future Therapeutic Targets in Children With Acute Lymphoblastic Leukemia (ALL)


- Collect clinical data in parallel with biological data and samples from children with
newly diagnosed acute lymphoblastic leukemia for biobanking, and use part of the
biobanked material to perform specific translational projects to achieve objectives

- To identify new prognostic factors (e.g., minimal-residual disease [MRD] significance
in small subgroups, miRNAs expression profile, PAX5 mutation, genetic abnormalities in
T-cell acute lymphoblastic leukemia [T-ALL], and RAS pathway activation) and future
therapeutic targets in children with newly diagnosed acute lymphoblastic leukemia.

- To identify leukemia cell genetic alterations (e.g., mutations in T-ALL and miRNA
expression in B-cell acute lymphoblastic leukemia [B-ALL]) and related molecular
pathways (e.g., RAS pathway) underlying leukemogenesis.

- To identify patient pharmacogenetic polymorphisms impacting individual response to
corticosteroids as part of standard therapy and investigate their prognostic

OUTLINE: This is a prospective observational biobanking study.

Patients undergo clinical evaluation, laboratory tests, and imaging periodically. Data are
collected before, during, and after first-line standard therapy. Clinical data are collected
from all patients in parallel with the biological data and samples. Biological samples are
partly used to perform specific translational research (TR) projects. Remaining biological
materials are stored for future research.

The following TR projects are performed on the biological samples for this study. Biological
samples are analyzed for allele-specific amplification of Ig/TCR clonal rearrangements to
quantify minimal-residual disease (MRD) via real-time PCR (TR1 Project); miRNA expression
via qPCR (TR 2 Project); the detection of main point mutations via high-resolution melting
PCR (TR 3 Project); genetic polymorphisms via real-time TaqMan allelic-discrimination method
(TR 4 Project); clinical significance of genetic abnormalities via quantitative real-time
RT-PCR, direct sequencing, and fluorescence in situ hybridization (TR 5 Project); and RAS
pathway activation via single-nucleotide polymorphism (SNP) analysis and gene-expression
analysis (TR 6 Project).

Inclusion Criteria


- Newly diagnosed acute lymphoblastic leukemia (ALL), meeting the following criteria:

- FAB L1 or L2 morphology (any immunophenotype) and acute leukemias of ambiguous
lineage (including biphenotypic or bilineal acute lymphoblastic leukemia)

- Patients with mature B-cell acute lymphoblastic leukemia (B-ALL) (FAB L3
morphology and immunophenotypical mature B phenotype or B-ALL with
documented presence of karyotype t(8;14), t(2;8) t(8;22) or breakpoints as
in mature B-ALL) are excluded from this study

- Patients must also meet the following criteria for participating in individual
translational research (TR) project:

- TR 1 project (MRD prognostic significance in small ALL subgroups):

- All patients, categorized according to response to pre-phase (< or > 1,000
peripheral blasts/mm³ at day 8) and minimal-residual disease (MRD) level at
end of the induction therapy

- iAmp(21q) detected at presentation

- Hypodiploidy detected at presentation by karyotype and/or fluorescence in
situ hybridization (FISH) and/or DNA index

- TR 2 project (miRNAs expression in pediatric ALL):

- Initially, average-risk 1 (AR1) patients

- In a second stage, the analysis might be extended to low-risk patients that
still show treatment failure and high-risk ALL patients

- TR 3 project (Prognostic value of newly described mutations in childhood B-ALL)

- Initially, only B-cell precursor ALL patients

- TR 4 project (Pharmacogenetics of the response to prephase and induction

- All ALL patients

- TR 5 project (Clinical significance of genetic abnormalities in childhood T-ALL)

- All patients with T-cell ALL, as defined by expression of T-cell surface

- TR 6 project (RAS pathway activation in childhood B-ALL):

- All patients with B-lineage ALL

- Patients with Philadelphia-chromosome positive ALL (documented presence of
t(9;22)(q34;q11) and/or of the BCR/ABL fusion transcript) are eligible

- Scheduled to receive therapy as per institutional standard practice and have not
started therapy (except for a maximum of 7 days of systemic corticosteroids prior to

- May only be registered to this study once


- No psychological, familial, sociological, or geographical condition potentially
hampering participation in the study protocol and follow-up schedule

- Patients with Down syndrome are eligible


- See Disease Characteristics

Type of Study:


Study Design:


Outcome Measure:

Event-free survival

Safety Issue:


Principal Investigator

Helene Cave

Investigator Affiliation:

CHU - Hopital Robert Debre



Study ID:




Start Date:

May 2011

Completion Date:

Related Keywords:

  • Leukemia
  • Lymphoma
  • untreated childhood acute lymphoblastic leukemia
  • L1 childhood acute lymphoblastic leukemia
  • L2 childhood acute lymphoblastic leukemia
  • B-cell childhood acute lymphoblastic leukemia
  • T-cell childhood acute lymphoblastic leukemia
  • stage I childhood lymphoblastic lymphoma
  • stage II childhood lymphoblastic lymphoma
  • stage III childhood lymphoblastic lymphoma
  • stage IV childhood lymphoblastic lymphoma
  • Philadelphia chromosome positive childhood precursor acute lymphoblastic leukemia
  • Leukemia
  • Leukemia, Lymphoid
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma
  • Lymphoma