Ferroptosis Study in SF3B1-mutant Myelodysplastic Syndromes (FerMDS)

NCT05924074 | Recruiting

• 1 other identifier: CHUBX 2022/43
• Study Type: interventional
• Target: 80
• Locations: 1 country
• Sites: 3

Brief Summary

Myelodysplastic syndromes (MDS) are clonal diseases of hematopoietic stem cells (HSC) characterized by dysplastic and inefficient hematopoiesis related to excessive progenitor cell death. Ferroptosis is a recently described cell death mechanism and we think that it could be a major player in the pathophysiology of MDS, involved in the cell death that characterizes these diseases and contributing to cytopenias. The study aims to demonstrate that there is a significant activation of this phenomenon in MDS patients compared to a population of subjects without MDS.

Conditions

Myelodysplastic Syndromes; Ferroptosis

Outcome Measures

Primary Outcomes (1)

• Percentage of cells undergoing ferroptosis in SF3B1-mutant MDS patients compared to MGUS patients

  Mean values for the proportion of ferroptose cells will be compared between arms, using comparative statistical methods (Student's t test, Student's test for unequal variances or Wilcoxon test)

  At Baseline

Secondary Outcomes (3)

• Percentage of cells undergoing ferroptosis in the different bone marrow subpopulations (stem cells, progenitors, and erythroid and myeloid precursors at different stages of differentiation) of SF3B1-mutant MDS patients

  At Baseline

• Biological characteristics of SF3B1-mutant MDS patients with an excess of cells undergoing ferroptosis in the bone marrow compared to MGUS controls

  At baseline

• Clinical characteristics of SF3B1-mutant MDS patients with an excess of cells undergoing ferroptosis in the bone marrow compared to MGUS controls

  Through study completion, up to 2 years

Study Arms (2)

SF3B1 mutant Myelodysplastic syndromes patients (MDS)

EXPERIMENTAL

Patients diagnosed with MDS carrying the SF3B1 somatic mutation associated myelodysplastic neoplasm with ring sideroblasts

Biological: Biological sampling

Monoclonal Gammapathy of Unknown Significance patients (MGUS)

ACTIVE COMPARATOR

MGUS patients, referred to as normal bone marrow controls

Biological: Biological sampling

Interventions

Biological sampling BIOLOGICAL

The procedure will consist of an additional bone marrow sample and blood sample

Monoclonal Gammapathy of Unknown Significance patients (MGUS); SF3B1 mutant Myelodysplastic syndromes patients (MDS)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• For all :
• Patients of legal age (age ≥ 18 years)
• Subjects affiliated to or benefiting from a social security scheme
• Free, written and informed consent signed by the participant and the investigator
• For MDS patients :
• Sampling at diagnosis for MDS patients (WHO 2016 criteria)
• Presence of ring sideroblasts on bone marrow smear
• For MGUS patients :
• \- Sampling as part of the exploration of monoclonal gammopathy of undetermined significance (MGUS) for controls (WHO 2016 criteria).

You may not qualify if:

• For all
• Patient transfused with red blood cells within 120 days prior to collection
• Patients treated with haematopoietic growth factors (EPO, TPO, G-CSF) within 30 days prior to collection
• Patients with conditions that affect systemic iron metabolism: hemochromatosis, Gaucher disease, ferroportin disease, porphyria cutanea tarda
• Person under a legal protection measure (legal protection, guardianship or curatorship)
• Person deprived of liberty by judicial or administrative decision
• Person who is unable to give consent

Sponsors & Collaborators

• University Hospital, Bordeaux: lead

Study Sites (3)

CHU de Bordeaux, Laboratoire d'Hématologie

Pessac, France

NOT YET RECRUITING

CHU de Bordeaux, Service de Médecine Interne

Pessac, France

NOT YET RECRUITING

CHU de Bordeaux, Service Hématologie Clinique et Thérapie Cellulaire

Pessac, France

RECRUITING

MeSH Terms

Conditions

Myelodysplastic Syndromes

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Central Study Contacts

Victor-Emmanuel BRETT

CONTACT

0556774357; victor.brett@chu-bordeaux.fr

Charles DUSSIAU

CONTACT

charles.dussiau@chu-bordeaux.fr

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: DIAGNOSTIC
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 7, 2023
• First Posted: June 29, 2023
• Study Start: February 21, 2025
• Primary Completion (Estimated): February 1, 2027
• Study Completion (Estimated): February 1, 2027
• Last Updated: March 19, 2025

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will not share

Locations

FR (3)