Ferroptosis Study in SF3B1-mutant Myelodysplastic Syndromes (FerMDS)
Trial statusRecruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
Age18+
SponsorUniversity Hospital, Bordeaux
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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
Sampling at diagnosis for MDS patients (WHO 2016 criteria)
Disqualifiers
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)
Trial design
Design model
Parallel
Treatments tested in this trial
Biological sampling
Biological/VaccineThe procedure will consist of an additional bone marrow sample and blood sample
Treatment groups
80 Participants
are divided into 2 treatment groupsGroup A: SF3B1 mutant Myelodysplastic syndromes patients (MDS)Experimental treatment 1 intervention
Group B: Monoclonal Gammapathy of Unknown Significance patients (MGUS)Active comparator 1 intervention
Trial outcomes
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)
Time frame
At BaselineSecondary outcomes
1
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
Mean values for the proportion of ferroptose cells among the different bone marrow subpopulations will be compared between arms, using comparative statistical methods (Student's t test, Student's test for unequal variances or Wilcoxon test)
Time frame
At Baseline2
Biological characteristics of SF3B1-mutant MDS patients with an excess of cells undergoing ferroptosis in the bone marrow compared to MGUS controls
Biological differences in number (1 to 3) and type of cytopenias (anemia, thrombocytopenia, neutropenia) in SF3B1-mutated MDS patients will be studied and statistically compared with the MGUS control group, based on the number of ferroptosis cells observed by cytometry
Time frame
At baseline3
Clinical characteristics of SF3B1-mutant MDS patients with an excess of cells undergoing ferroptosis in the bone marrow compared to MGUS controls
Overall survival after diagnosis, progression-free survival (evolution of MDS towards a higher-grade hemopathy or death) in SF3B1-mutated MDS patients will be studied and statistically compared with the MGUS control group, based on the number of ferroptosis cells observed by cytometry
Time frame
Through study completion, up to 2 yearsSponsors and contacts
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Source ClinicalTrials.gov