Myelodysplastic SyndromesCOMYRENCT05367583
Cohort Study Assessing the Treatment Strategy for High-Risk Myelodysplastic Syndromes in Patients Under 70
An observational study of Myelodysplastic Syndromes, run by University Hospital, Bordeaux at 10 sites in France.
Trial summary
Myelodysplastic syndromes (MDS) are bone marrow malignant diseases resulting in ineffective haematopoiesis and subsequently, blood cell count decrease. Patients have anaemia responsible of fatigue and high heart frequency, thrombocytopenia responsible of increased risk of bleeding and neutropenia responsible of… Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Myelodysplastic Syndromes
- What is being tested?
- Allogeneic Stem Cell Transplantation
- Type of treatment
- Surgery or procedure
- How many people
- 107 participants
- Where
- 10 sitesFrance
- Study dates
- 9 Oct 2013to Oct 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
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 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Patients with MDS-HR defined by an intermediate or high IPSS score (≥ 1.5), or IPSS revised ≥ to 4.5
Who may be able to join
- Age> 18 years old and <70 years old
- MDS-HR defined by an intermediate or high IPSS score (≥ 1.5), or IPSS revised ≥ to 4.5.
- Diagnosis affirmed by 2 myelograms at least 1 month apart or 1 single characteristic myelogram if prior haematological abnormalities on complete blood count present for more than 6 months
- Free informed consent signed by the patient and the investigator (at the latest on the day of inclusion and before any examination required by the study)
- Affiliate or beneficiary of a social security system
Who cannot join
- Age ≥ 70 years
- MDS-LR defined by a low IPSS score (<1.5), or revised IPSS <4.5
- Women of childbearing potential not using contraception, pregnant or breastfeeding
- Not affiliated to the Social Security system
Trial locations and contacts
10 sites in 1 country.
10 sites in 1 country
France10 sites
CH AGEN
Agen
Ch Bayonne
Bayonne
Chu de Bordeaux
Bordeaux
Institut Bergonie
Bordeaux
CH DAX
Dax
Ch Libourne
Libourne
Ch Marmande
Marmande
Ch Mont de Marsan
Mont-de-Marsan
CH PAU
Pau
Ch Villeneuve/Lot
Villeneuve-sur-Lot
About this study
Official title: Cohort Study Assessing the Treatment Strategy for High-Risk Myelodysplastic
Myelodysplastic syndromes (MDS) are bone marrow malignant diseases resulting in ineffective haematopoiesis and subsequently, blood cell count decrease. Patients have anaemia responsible of fatigue and high heart frequency, thrombocytopenia responsible of increased risk of bleeding and neutropenia responsible of increased risk of infection. The patients suffering from MDS also are at increased risk of developing acute myeloblastic leukemia (AML). Allogeneic stem cell transplantation (alloSCT) remains the only curative option for patients with aggressive MDS. However, these patients are frequently ineligible for this kind of treatment, because of, for instance, age and co-morbidities. Thus, other treatment options are needed and Azacytidine (AZA), a hypomethylating agent is then proposed. With this COMYRE observatory study, we wanted to analyse which patients undergo alloSCT, why they are not eligible to alloSCT if it is the case, the overall survival of all the patients and if there are some factors which can influence this survival. It could help us to better identify the best candidate for alloSCT and those for other treatments such as AZA.
Read the detailed description
Myelodysplastic syndromes (MDS) are a heterogeneous group of myeloid disorders due to qualitative defects of the hematopoietic stem cell, resulting in uneffective hematopoiesis, dysplasia of one or more lineages, and subsequently, various cytopenias. The patients suffering from MDS are at increased risk of developing acute myeloblastic leukemia (AML). Allogeneic stem cell transplantation (alloSCT) remains the only curative option for patients with intermediate-2 or high risk MDS according to International Prognostic Scoring System (IPSS). However, since these patients are frequently ineligible for this kind of treatment, because of age and co-morbidities, other treatment options were needed. Thank to AZA-001 trial results, Azacytidine (AZA), a hypomethylating agent is validated in Europe for the treatment of MDS patients who are not candidate for alloSCT. In most of cases, AZA is a well-tolerated drug comparing to more classical chemotherapy or alloSCT. Indeed, the most frequent reported side effects are fatigue, constipation, joints and muscle pains, cytopenias and these side effects rarely have significant clinical consequences. On the contrary, during alloSCT procedure, we could observe more infectious events, graft versus host disease, more toxicity.
In this COMYRE observatory study, all high risk MDS with age below 70 are included. The aim of the study is to analyse which patients undergo alloSCT and why they are not eligible to alloSCT if it is the case. We also want to analyse the overall survival of all included patients and we will try to identify if there are some factors which can influence this survival. Thus, it could help us to better identify the best candidate for alloSCT and those for other treatments such as AZA, regarding the benefit/toxicity.
- Conditions
- Myelodysplastic Syndromes
- Being tested
- Allogeneic Stem Cell Transplantation
- Keywords
- Myelodysplastic syndromes, Allogeneic Stem Cell Transplantation
- Medical subject headings
- Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Allogeneic Stem Cell TransplantationProcedure or surgery
Allogeneic Stem Cell Transplantation
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of patient treated with Allogeneic stem cell transplantation (alloSCT) among enrolled patients
Measured: 3 months from baseline
Secondary outcomes (6)
Evaluate the feasibility of the alloSCT: Proportion of patients with a donor
Measured: 3 months from baseline
Evaluate the effectiveness of iron chelation by measuring plasma ferritin (PF) (PF ≤ 1000 µg/L or PF > 1000 µg/L
Measured: Baseline and before alloSCT or 1 year from baseline for patient not treated with alloSCT
Evaluate the overall survival of patient treated and not treated with alloSCT
Measured: Until 5 year from baseline
Evaluate overall survival prognostic factors of patient treated and not treated with alloSCT
Measured: Baseline
Evaluate response rate to alloSCT or chemotherapy
Measured: Until 5 years from baseline
Evaluate toxicity to alloSCT (rate of systemic infection, rate of acute GVHD and rate of chronic GVHD) or chemotherapy (rate of systemic infection and rate of severe hemorrhage)
Measured: Until 5 years from baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 May 2022
- Study start9 Oct 2013
- Primary completion9 Oct 2021
- Study completionOct 2024 (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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 107 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital, Bordeaux
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05367583
- Sponsor's protocol ID
- CHUBX 2012/22
- Acronym
- COMYRE
- Status last verified
- Jul 2022
- First submitted
- 2 Jul 2019
- Last updated
- 8 Jul 2022
- 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 8 Jul 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Cohort Study Assessing the Treatment Strategy for High-Risk Myelodysplastic
- ClinicalTrials.gov ID
- NCT05367583
- Acronym / sponsor ID
- COMYRE / CHUBX 2012/22
- Conditions
- Myelodysplastic Syndromes
- Keywords
- Myelodysplastic syndromes, Allogeneic Stem Cell Transplantation
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 107 participants (actual)
- Lead sponsor
- University Hospital, BordeauxUniversity, hospital or other
- Study start
- 9 Oct 2013 (actual)
- Primary completion
- 9 Oct 2021 (actual)
- Study completion
- Oct 2024 (estimated)
- First posted
- 10 May 2022 (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 bone marrow malignant diseases resulting in ineffective haematopoiesis and subsequently, blood cell count decrease. Patients have anaemia responsible of fatigue and high heart frequency, thrombocytopenia responsible of increased risk of bleeding and neutropenia responsible of increased risk of infection. The patients suffering from MDS also are at increased risk of developing acute myeloblastic leukemia (AML). Allogeneic stem cell transplantation (alloSCT) remains the only curative option for patients with aggressive MDS. However, these patients are frequently ineligible for this kind of treatment, because of, for instance, age and co-morbidities. Thus, other treatment options are needed and Azacytidine (AZA), a hypomethylating agent is then proposed. With this COMYRE observatory study, we wanted to analyse which patients undergo alloSCT, why they are not eligible to alloSCT if it is the case, the overall survival of all the patients and if there are some factors which can influence this survival. It could help us to better identify the best candidate for alloSCT and those for other treatments such as AZA.
Detailed description
Myelodysplastic syndromes (MDS) are a heterogeneous group of myeloid disorders due to qualitative defects of the hematopoietic stem cell, resulting in uneffective hematopoiesis, dysplasia of one or more lineages, and subsequently, various cytopenias. The patients suffering from MDS are at increased risk of developing acute myeloblastic leukemia (AML). Allogeneic stem cell transplantation (alloSCT) remains the only curative option for patients with intermediate-2 or high risk MDS according to International Prognostic Scoring System (IPSS). However, since these patients are frequently ineligible for this kind of treatment, because of age and co-morbidities, other treatment options were needed. Thank to AZA-001 trial results, Azacytidine (AZA), a hypomethylating agent is validated in Europe for the treatment of MDS patients who are not candidate for alloSCT. In most of cases, AZA is a well-tolerated drug comparing to more classical chemotherapy or alloSCT. Indeed, the most frequent reported side effects are fatigue, constipation, joints and muscle pains, cytopenias and these side effects rarely have significant clinical consequences. On the contrary, during alloSCT procedure, we could observe more infectious events, graft versus host disease, more toxicity.
In this COMYRE observatory study, all high risk MDS with age below 70 are included. The aim of the study is to analyse which patients undergo alloSCT and why they are not eligible to alloSCT if it is the case. We also want to analyse the overall survival of all included patients and we will try to identify if there are some factors which can influence this survival. Thus, it could help us to better identify the best candidate for alloSCT and those for other treatments such as AZA, regarding the benefit/toxicity.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients with MDS-HR defined by an intermediate or high IPSS score (≥ 1.5), or IPSS revised ≥ to 4.5
Key inclusion criteria (5 listed)
- Age> 18 years old and <70 years old
- MDS-HR defined by an intermediate or high IPSS score (≥ 1.5), or IPSS revised ≥ to 4.5.
- Diagnosis affirmed by 2 myelograms at least 1 month apart or 1 single characteristic myelogram if prior haematological abnormalities on complete blood count present for more than 6 months
- Free informed consent signed by the patient and the investigator (at the latest on the day of inclusion and before any examination required by the study)
- Affiliate or beneficiary of a social security system
Key exclusion criteria (4 listed)
- Age ≥ 70 years
- MDS-LR defined by a low IPSS score (<1.5), or revised IPSS <4.5
- Women of childbearing potential not using contraception, pregnant or breastfeeding
- Not affiliated to the Social Security system
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 |
|---|---|---|
| Allogeneic Stem Cell TransplantationAllogeneic Stem Cell Transplantation | Procedure or surgery | – |
Primary outcome
Proportion of patient treated with Allogeneic stem cell transplantation (alloSCT) among enrolled patients
Time frame: 3 months from baseline
6 secondary outcomes
Evaluate the feasibility of the alloSCT: Proportion of patients with a donor
Time frame: 3 months from baseline
Evaluate the effectiveness of iron chelation by measuring plasma ferritin (PF) (PF ≤ 1000 µg/L or PF > 1000 µg/L
Time frame: Baseline and before alloSCT or 1 year from baseline for patient not treated with alloSCT
Evaluate the overall survival of patient treated and not treated with alloSCT
Time frame: Until 5 year from baseline
Evaluate overall survival prognostic factors of patient treated and not treated with alloSCT
Time frame: Baseline
Evaluate response rate to alloSCT or chemotherapy
Time frame: Until 5 years from baseline
Evaluate toxicity to alloSCT (rate of systemic infection, rate of acute GVHD and rate of chronic GVHD) or chemotherapy (rate of systemic infection and rate of severe hemorrhage)
Time frame: Until 5 years from baseline
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Jul 2022.
- 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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
10 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Myelodysplastic Syndromes, run by University Hospital, Bordeaux at 10 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 5 inclusion and 4 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: status not confirmed, as of the sponsor's last update on 8 Jul 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of patient treated with Allogeneic stem cell transplantation (alloSCT) among enrolled patients (time frame: 3 months from 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 8 Jul 2022.