Myelodysplastic SyndromesFerMDSNCT05924074
Ferroptosis Study in SF3B1-mutant Myelodysplastic Syndromes (FerMDS)
A study testing Biological sampling in people with Myelodysplastic Syndromes and Ferroptosis, run by University Hospital, Bordeaux at 3 sites in France.
Trial summary
Myelodysplastic syndromes (MDS) are clonal diseases of hematopoietic stem cells (HSC) characterized by dysplastic and inefficient hematopoiesis related to excessive progenitor cell death. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndromes, Ferroptosis
- What is being tested?
- Biological sampling
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 80 participants
- Where
- 3 sitesFrance
- Study dates
- 21 Feb 2025to Feb 2027
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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)
Show all 9Show fewer
- 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).
Who cannot join
- 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
- Subject who is in an exclusion period after another study or who has participated in another interventional drug study within 30 days prior to entry into the protocol
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Charles DUSSIAU contact
France3 sites, 3 recruiting
- Not yet recruiting
CHU de Bordeaux, Laboratoire d'Hématologie
Pessac
Victor-Emmanuel BRETT, victor.brett@chu-bordeaux.fr
- Not yet recruiting
CHU de Bordeaux, Service de Médecine Interne
Pessac
Estibaliz LAZARO, estibaliz.lazaro@chu-bordeaux.fr
- Recruiting
CHU de Bordeaux, Service Hématologie Clinique et Thérapie Cellulaire
Pessac
Sophie DIMICOLI-SALAZAR, sophie.dimicoli-salazar@chu-bordeaux.fr
About this study
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.
Read the detailed description
Myelodysplastic syndromes (MDS) are hematological malignancies characterized by a defect in blood cells production. Their pathophysiology remains poorly understood, but an excessive death of progenitor cells is considered as a key mechanism contributing to the appearance of cytopenia. Furthermore, it is known that there are abnormalities of iron metabolism in MDS, especially in patients with ring sideroblasts and SF3B1 mutation. The classical therapeutic strategy in MDS relies on symptomatic management of cytopenias (transfusions, growth factors) associated with demethylating agents in high-risk forms. Unfortunately, these treatments only stabilize the disease and only allogeneic bone marrow transplantation (reserved to limited number of patients) can cure the patients. Therefore, there is a urgent need to identify new therapeutic targets in these diseases.
An excessive apoptosis activation has been shown in MDS for a long time. However, other cell death pathways could also contribute to their pathophysiology. Among them, ferroptosis, a cell death process triggered by the accumulation of free iron in the cell, seems to be a promising candidate.
The project is proposed to study ferroptosis in SF3B1-mutant MDS patients. An additionnal bone marrow sample will be aspirate at diagnosis. Ferroptosis will be analyzed using flow cytometry (labeling of peroxidized lipids with C11-BODIPY). The percentage of cells in ferroptosis will be compared between SF3B1-mutant MDS patients and control patients (patients evaluated for Monoclonal Gammapathy of Unknown Significance-MGUS). The presence of an excess of ferroptosis in SF3B1-mutant MDS patients will be correlated to clinico-biological parameters. No follow up will be be performed.
- Conditions
- Myelodysplastic Syndromes, Ferroptosis
- Being tested
- Biological sampling
- Medical subject headings
- Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Biological samplingBiological
The procedure will consist of an additional bone marrow sample and blood sample
| Study group | Role | What they receive |
|---|---|---|
| SF3B1 mutant Myelodysplastic syndromes patients (MDS) | Experimental | Patients diagnosed with MDS carrying the SF3B1 somatic mutation associated myelodysplastic neoplasm with ring sideroblasts Receives: Biological: Biological sampling |
| Monoclonal Gammapathy of Unknown Significance patients (MGUS) | Active comparator | MGUS patients, referred to as normal bone marrow controls Receives: Biological: Biological sampling |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
Measured: 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
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)
Measured: At Baseline
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
Measured: At baseline
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
Measured: Through study completion, up to 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jun 2023
- Study start21 Feb 2025
- Primary completionFeb 2027 (estimated)
- Study completionFeb 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelodysplastic SyndromesStudies of the same condition, open and under way.
- More trials run by University Hospital, BordeauxThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Diagnosis
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 80 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University Hospital, Bordeaux
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05924074
- Sponsor's protocol ID
- CHUBX 2022/43
- Acronym
- FerMDS
- Status last verified
- Mar 2025
- First submitted
- 7 Jun 2023
- Last updated
- 19 Mar 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Ferroptosis Study in SF3B1-mutant Myelodysplastic Syndromes (FerMDS)
- ClinicalTrials.gov ID
- NCT05924074
- Acronym / sponsor ID
- FerMDS / CHUBX 2022/43
- Conditions
- Myelodysplastic Syndromes, Ferroptosis
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 80 participants (estimated)
- Lead sponsor
- University Hospital, BordeauxUniversity, hospital or other
- Study start
- 21 Feb 2025 (actual)
- Primary completion
- Feb 2027 (estimated)
- Study completion
- Feb 2027 (estimated)
- First posted
- 29 Jun 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Myelodysplastic syndromes (MDS) are hematological malignancies characterized by a defect in blood cells production. Their pathophysiology remains poorly understood, but an excessive death of progenitor cells is considered as a key mechanism contributing to the appearance of cytopenia. Furthermore, it is known that there are abnormalities of iron metabolism in MDS, especially in patients with ring sideroblasts and SF3B1 mutation. The classical therapeutic strategy in MDS relies on symptomatic management of cytopenias (transfusions, growth factors) associated with demethylating agents in high-risk forms. Unfortunately, these treatments only stabilize the disease and only allogeneic bone marrow transplantation (reserved to limited number of patients) can cure the patients. Therefore, there is a urgent need to identify new therapeutic targets in these diseases.
An excessive apoptosis activation has been shown in MDS for a long time. However, other cell death pathways could also contribute to their pathophysiology. Among them, ferroptosis, a cell death process triggered by the accumulation of free iron in the cell, seems to be a promising candidate.
The project is proposed to study ferroptosis in SF3B1-mutant MDS patients. An additionnal bone marrow sample will be aspirate at diagnosis. Ferroptosis will be analyzed using flow cytometry (labeling of peroxidized lipids with C11-BODIPY). The percentage of cells in ferroptosis will be compared between SF3B1-mutant MDS patients and control patients (patients evaluated for Monoclonal Gammapathy of Unknown Significance-MGUS). The presence of an excess of ferroptosis in SF3B1-mutant MDS patients will be correlated to clinico-biological parameters. No follow up will be be performed.
Study design
- Primary purpose
- Diagnosis
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| SF3B1 mutant Myelodysplastic syndromes patients (MDS) | Experimental | Biological: Biological samplingPatients diagnosed with MDS carrying the SF3B1 somatic mutation associated myelodysplastic neoplasm with ring sideroblasts |
| Monoclonal Gammapathy of Unknown Significance patients (MGUS) | Active comparator | Biological: Biological samplingMGUS patients, referred to as normal bone marrow controls |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- 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)
- Presence of ring sideroblasts on bone marrow smear
Key exclusion criteria (8 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Biological samplingThe procedure will consist of an additional bone marrow sample and blood sample | Biological | Monoclonal Gammapathy of Unknown Significance patients (MGUS), SF3B1 mutant Myelodysplastic syndromes patients (MDS) |
Primary outcome
Percentage of cells undergoing ferroptosis in SF3B1-mutant MDS patients compared to MGUS patients
Time frame: At Baseline
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)
3 secondary outcomes
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
Time frame: At Baseline
Biological characteristics of SF3B1-mutant MDS patients with an excess of cells undergoing ferroptosis in the bone marrow compared to MGUS controls
Time frame: At baseline
Clinical characteristics of SF3B1-mutant MDS patients with an excess of cells undergoing ferroptosis in the bone marrow compared to MGUS controls
Time frame: Through study completion, up to 2 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Mar 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Charles DUSSIAU contact
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Biological sampling in people with Myelodysplastic Syndromes and Ferroptosis, run by University Hospital, Bordeaux at 3 sites in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 19 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Victor-Emmanuel BRETT (0556774357, victor.brett@chu-bordeaux.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of cells undergoing ferroptosis in SF3B1-mutant MDS patients compared to MGUS patients (time frame: At Baseline). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Mar 2025.