EPIgenetics and in Vivo Resistance of Chronic Myeloid Leukemia Stem Cells to Tyrosine Kinase Inhibitors

NCT ID: NCT03481868

Last Updated: 2024-04-09

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

NA

Total Enrollment

160 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-02-01

Study Completion Date

2026-02-01

Brief Summary

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Primary objective : To identify epigenetic dysregulations of in vivo TKI-resisting CML cells Hypothesis : An epigenetic dysregulation is involved in the in vivo survival of a CML cell subclone despite the use of TKIs

Detailed Description

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Chronic myeloid leukemia (CML) is a model of leukemogenesis because the malignant transformation of a hematopoietic stem cell is considered as the consequence of a unique major event: the translocation t(9;22) which is able to produce the BCR-ABL chimeric protein. The treatment of CML has made considerable progress with the development of tyrosine kinase inhibitors (TKI) targeting the BCR-ABL protein activity. Despite the efficacy of these drugs, several studies showed the existence of intra-clonal heterogeneity and the in vivo survival of a leukemic stem cell (LSC) subset by:

* A detectable residual disease in the majority of cases, and after treatment for several years.
* The relapse of about half of patients after stopping TKI; these relapses are the proof of the in vivo persistence of LSC, even when CML clone is particularly sensitive to TKI.
* Cases of unexplained TKI resistance (no BCR-ABL mutation etc…)

The mechanisms involved in in vivo survival of LSC remain largely unknown. Mechanisms independent of BCR-ABL TK activity could be responsible of LSC survival. However, the fact that CML is the consequence of t(9; 22) if it appears in a HSC, suggests that a stem cell specific biological status should play a role in the emergence of the disease and probably the special feature of this cell subset. Several studies showed the essential role of epigenetic factors in stem cell behavior (quiescence, self-renewal, or differentiation process). Epigenetic dysregulation of some gene expression was observed in CML cells and changes in DNA methylation are involved in CML progression towards accelerated or blast phase, more resistant to TKIs. These observations led to clinical trial combining TKI with epigenetic drugs which results confirmed the in vivo involvement of epigenetic mechanisms during CML progression. However, the role of epigenetics in the early resistance of a chronic phase CML cell subset remains unknown.

The hypothesis is that epigenetic features could participate in TKI resistance of CML LSC and their survival in bone marrow.

In order to identify new mechanisms and/or new targets involved in LSC resistance, investigator choose a global approach of DNA methylation profile with an HM450K microarray. Investigator analyzed the sorted CML CD34+ CD15- cell subset (n=6) in comparison with: 1) CD34+ CD15- cells from healthy donors (hematopoietic grafts), in order to eliminate specificities of normal hematopoietic hierarchy, 2) the CD34-CD15+ sub-clone from the CML clone before any treatment in order to eliminate characteristic of CML mature compartment. The CD34+CD15- cells showed a specific DNA methylation profile, from diagnosis and from this level of cell hierarchy ((Bourgne et al., oral communication, SFH meeting 2017, manuscript submitted). In order to identify biomarkers specific to CML cells, investigator removed abnormally methylated regions that are differently methylated during hematopoietic differentiation. After this step, 825, 2210 and 1461 probes identified regions specifically dysmethylated in CD34-CD15+, CD34+ CD15- CML cells and both respectively. Investigator also observed changes in expression of epigenetic actors. These results validate our hypothesis. With the recent data published in literature, they strongly argue in favor of the involvement of epigenetic dysregulation in native intra-clonal heterogeneity, and justify this original translational research project.

Conditions

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Chronic Myeloid Leukemia (CML) Chronic Phase

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

OTHER

Blinding Strategy

NONE

Study Groups

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Chronic Myeloid Leukemia

Patients newly diagnosed for Chronic Myeloid Leukemia, according to inclusion and exclusion criteria

Group Type EXPERIMENTAL

Collection of blood and bone marrow

Intervention Type BIOLOGICAL

Collection of blood and bone marrow in order to identify epigenetic abnormalities and their consequences in surviving CML cells after 3 months of TKI treatment

Interventions

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Collection of blood and bone marrow

Collection of blood and bone marrow in order to identify epigenetic abnormalities and their consequences in surviving CML cells after 3 months of TKI treatment

Intervention Type BIOLOGICAL

Eligibility Criteria

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Inclusion Criteria

* Patient newly diagnosed for Chronic Myeloid Leukemia in chronic phase
* Patient older than 18 years old
* Patient who received no treatment for CML at the time of sampling on D0
* Intention of prescription with first-line treatment with TKI only
* Choice of first-line CML treatment by TKI only
* Patient having signed an informed consent
* Patient with a social security system

Exclusion Criteria

* Contra-indication to the use of TKI
* Probability of poor compliance during treatment
* Patients already treated for CML
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Institut Paoli-Calmettes

OTHER

Sponsor Role collaborator

CHRU Lille, hematology department

UNKNOWN

Sponsor Role collaborator

CH Annecy Genevois

OTHER

Sponsor Role collaborator

Institut Bergonié Bordeaux, hematology department

UNKNOWN

Sponsor Role collaborator

Central Hospital, Nancy, France

OTHER

Sponsor Role collaborator

CHU Saint-Etienne, hematology department

UNKNOWN

Sponsor Role collaborator

Institut Universitaire du Cancer de Toulouse

OTHER

Sponsor Role collaborator

CHU Caen, hematology department

UNKNOWN

Sponsor Role collaborator

CHU Nice, hematology department

UNKNOWN

Sponsor Role collaborator

Hospices Civils de Lyon

OTHER

Sponsor Role collaborator

AP-HP, hematology department

UNKNOWN

Sponsor Role collaborator

AP-HP Hôpital Henri-Mondor, hematology department

UNKNOWN

Sponsor Role collaborator

Versailles Hospital

OTHER

Sponsor Role collaborator

Centre Leon Berard

OTHER

Sponsor Role collaborator

University Hospital, Grenoble

OTHER

Sponsor Role collaborator

University Hospital, Clermont-Ferrand

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Marc BERGER

Role: PRINCIPAL_INVESTIGATOR

University Hospital, Clermont-Ferrand

Locations

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CHU Annecy-Genevois

Annecy, , France

Site Status RECRUITING

Institut Bergonié

Bordeaux, , France

Site Status RECRUITING

CHU Caen

Caen, , France

Site Status RECRUITING

CHU Clermont-Ferrand

Clermont-Ferrand, , France

Site Status RECRUITING

CHU Grenoble Alpes

Grenoble, , France

Site Status RECRUITING

CHU Lille

Lille, , France

Site Status RECRUITING

Centre Léon Bérard

Lyon, , France

Site Status RECRUITING

Hospices Civils de Lyon

Lyon, , France

Site Status RECRUITING

CHU Nancy

Nancy, , France

Site Status RECRUITING

CHU Nice

Nice, , France

Site Status RECRUITING

Hôpital Bicêtre

Paris, , France

Site Status RECRUITING

Hôpital Henri Mondor

Paris, , France

Site Status RECRUITING

Hôpital Paul Brousse

Paris, , France

Site Status RECRUITING

Hôpital Saint Louis

Paris, , France

Site Status RECRUITING

CHU Saint-Etienne

Saint-Etienne, , France

Site Status RECRUITING

CHU Versailles

Versailles, , France

Site Status RECRUITING

Countries

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France

Central Contacts

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Patrick LACARIN

Role: CONTACT

04 73 75 11 95

Facility Contacts

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Patrick LACARIN

Role: primary

04 73 75 11 95

Other Identifiers

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2017-A01332-51

Identifier Type: OTHER

Identifier Source: secondary_id

CHU-376

Identifier Type: -

Identifier Source: org_study_id

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