Role of BMP Pathway in MDS Progression

NCT ID: NCT06175923

Last Updated: 2024-01-23

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

NOT_YET_RECRUITING

Total Enrollment

60 participants

Study Classification

OBSERVATIONAL

Study Start Date

2024-01-27

Study Completion Date

2034-01-27

Brief Summary

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Myelodysplastic syndromes (MDS) are hematological cancers that can progress to acute myelogenous leukemia (AML). The involvement of the microenvironment in the maintenance, resistance and evolution of MDS is increasingly described.

The Bone Morphogenetic Protein (BMP) pathway is involved in numerous functions, including self-renewal of the hematopoietic stem cell compartment and the regulation of hematopoiesis, via interaction with bone marrow stromal cells. Investigators have demonstrated its involvement in chronic myeloid leukemia (CML) and AML, in particular via the activation of TWIST1, ΔNp73, NANOG; it is responsible for an increased state of quiescence of certain cancer stem cells and their resistance.

Preliminary results based on the analysis of large databases suggest that the BMP pathway is also altered early in MDS. This study explores the alteration of this pathway in MDS and its involvement in the transformation into AML.

If appropriate, the BMP pathway could constitute a very promising therapeutic target to combat transformation into AML.

Detailed Description

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Conditions

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Myelodysplastic Syndromes Acute Myelogenous Leukemia

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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MDS patients

Adult patients with myelodysplastic syndrome or suspected myelodysplastic syndrome according to the criteria defined by the World Health Organization:

* one or more cytopenias,
* and/or dysplasia of one or more lines,
* and/or bone marrow blastosis
* and/or sideroblasts in medullary crowns
* and/or genetic/cytogenetic abnormalities characteristic of MDS.
* Whatever the R-IPSS stage (Revised International Prognostic Scoring System)
* No history of cytotoxic treatment (hydroxycarbamide, azacytidine)

Collection of EDTA (disodium salt of ethylenediaminetetraacetic acid) tubes of marrow during routine care

Intervention Type BIOLOGICAL

When bone marrow is collected as part of a patient's care (diagnosis, follow-up, suspected AML/MDS hemopathy), one or two additional EDTA tubes of marrow are collected. Certain hematological data (NFP, genetic and molecular characteristics) will be collected in anonymized form and correlated with the BMP pathway alterations measured.

AML patients

Adult patients with suspected de novo acute myeloid leukemia at initial management

Collection of EDTA (disodium salt of ethylenediaminetetraacetic acid) tubes of marrow during routine care

Intervention Type BIOLOGICAL

When bone marrow is collected as part of a patient's care (diagnosis, follow-up, suspected AML/MDS hemopathy), one or two additional EDTA tubes of marrow are collected. Certain hematological data (NFP, genetic and molecular characteristics) will be collected in anonymized form and correlated with the BMP pathway alterations measured.

Interventions

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Collection of EDTA (disodium salt of ethylenediaminetetraacetic acid) tubes of marrow during routine care

When bone marrow is collected as part of a patient's care (diagnosis, follow-up, suspected AML/MDS hemopathy), one or two additional EDTA tubes of marrow are collected. Certain hematological data (NFP, genetic and molecular characteristics) will be collected in anonymized form and correlated with the BMP pathway alterations measured.

Intervention Type BIOLOGICAL

Eligibility Criteria

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Inclusion Criteria

* Adult patients with myelodysplastic syndrome or suspected myelodysplastic syndrome according to the criteria defined by the World Health Organization Or
* Adult patient with suspicion of de novo acute myeloid leukemia at initial treatment

Exclusion Criteria

* Frontier MDS/myeloproliferative syndromes including chronic myelomonocytic leukemia
* MDS and AML having already benefited from cytotoxic treatment including hydroxycarbamide, azacytidine, intensive chemotherapy
* Patients objecting to their inclusion in the study
* Pregnant or breastfeeding women
* Patients under legal protection measure
Minimum Eligible Age

20 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Hospices Civils de Lyon

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Hospices Civils de Lyon

Lyon, , France

Site Status

Countries

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France

Central Contacts

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Maël MD Heiblig

Role: CONTACT

0478864340 ext. +33

Facility Contacts

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Maël MD HEIBLIG

Role: primary

Other Identifiers

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69HCL22_0491

Identifier Type: -

Identifier Source: org_study_id

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