Myelodysplastic Syndromes or Acute Myeloid Leukemia With Multilineage DysplasiaNCT01210274
Characterization of the Mechanisms of Resistance to Azacitidine
An observational study of Myelodysplastic Syndromes or Acute Myeloid Leukemia With Multilineage Dysplasia, run by Centre Hospitalier Universitaire de Nice at 4 sites in 2 countries.
Trial summary
Myelodysplastic syndromes (MDS) are frequent diseases in elderly patients (median age: 71 years). IPSS classification defines low risk (Low and Intermediate 1), and high risk (Intermediate 2 and High) MDS. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndromes or Acute Myeloid Leukemia With Multilineage Dysplasia
- How many people
- 250 participants
- Where
- 4 sitesin 2 countries
- Study dates
- Sep 2010to Sep 2025
- Phase
- Observational
- 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
Study population: Patients with myelodysplastic syndromes or acute myeloid leukemia with multilineage dysplasia treated with Azacitidine
Who may be able to join
- Age ≥ 18 years
- High Risk or Intermediate 2 MDS (IPSS)
- AML-MD (WHO classification)
- Treatment with minimum three to six cycles of Azacitidine
- Informed consent form signed
Who cannot join
- Treatment with others chemotherapies alone or in association
Trial locations and contacts
4 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
4 sites in 2 countries
France3 sites, 3 recruiting
- Recruiting
CH d'Antibes
Antibes
Vanina OLIVERI, Project Manaer, oliveri.v@chu-nice.fr
Daniel RE, PH
- Recruiting
CHU de Nice - Hôpital de l'Archet
Nice, 06200
Vanina OLIVERI, Project Manager, oliveri.v@chu-nice.fr
Thomas CLUZEAU, PH
- Recruiting
Centre Antoine Lacassagne
Nice
Vanina OLIVERI, Project Manager, oliveri.v@chhu-nice.fr
Antoine THYSS, PU-PH
Monaco1 site, 1 recruiting
- Recruiting
CH Princesse Grace
Monaco
Vanina OLIVERI, Project Manager, oliveri.v@chu-nice.fr
Georges GARNIER, PH
About this study
Official title: Characterization of the Mechanisms of Action of Resistance to Azacitidine in High-risk Myelodysplastic Syndromes and Acute Myeloid Leukemia With Multilineage Dysplasia
Myelodysplastic syndromes (MDS) are frequent diseases in elderly patients (median age: 71 years). IPSS classification defines low risk (Low and Intermediate 1), and high risk (Intermediate 2 and High) MDS. High-risk MDS (MDS-HR) have a high risk of transformation into acute leukemia with multilineage dysplasia (AML-DML). The success of Azacitidine has been mainly achieved through a rigorous empirical and clinical research, but the molecular mechanisms by which this molecule exerts its effects remain poorly characterized. The primary mode of action of Azacytidine is through DNA demethylation, and integration in to mRNA that favor traduction inhibition. The impact of this molecule on various cell death programs involved in the elimination of leukemic cells : apoptosis and autophagy is currently poorly known.
The research program and clinical studies we proposed focus on two major aspects:
- Main objective: Molecular mechanism of action and resistance to Azacitidine: Role of apoptosis versus autophagy.
- Secondary Objective: Reversion of Azacytidine resistance using different drugs targeting apoptosis and/or autophagy. Our laboratory has identified new molecules to selectively induce different types of cell death (apoptosis or autophagy).
- Medical subject headings
- Myelodysplastic Syndromes
What is being measured
The primary outcome is the main question the study is designed to answer.
hematological response
Hematological response evaluated by the International Working Group (IWG) response of Cheson
Measured: at 3 months
hematological response
Hematological response evaluated by the International Working Group (IWG) response of Cheson
Measured: at 6 months
Secondary outcomes (2)
Overall survival
Overall survival (OS) defined as the time from start of treatment
Measured: Day 1 of treatment
Overall survival
Overall survival (OS) defined as the time from start of treatment
Measured: at the death
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Sep 2010 (estimated)
- Study startSep 2010
- Primary completionSep 2014
- Study completionSep 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Myelodysplastic Syndromes or Acute Myeloid Leukemia With Multilineage DysplasiaStudies of the same condition, open and under way.
- More trials run by Centre Hospitalier Universitaire de NiceThe 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
- Case only
- Time perspective
- Prospective
- Enrolment
- 250 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Centre Hospitalier Universitaire de Nice
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01210274
- Sponsor's protocol ID
- 10-PP-10
- Status last verified
- Mar 2025
- First submitted
- 27 Sep 2010
- Last updated
- 19 Mar 2025
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Publications
Cluzeau T, Robert G, Mounier N, Karsenti JM, Dufies M, Puissant A, Jacquel A, Renneville A, Preudhomme C, Cassuto JP, Raynaud S, Luciano F, Auberger P. BCL2L10 is a predictive factor for resistance to azacitidine in MDS and AML patients. Oncotarget. 2012 Apr;3(4):490-501. doi: 10.18632/oncotarget.481.
PubMed 22577154
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
- Characterization of the Mechanisms of Action of Resistance to Azacitidine in High-risk Myelodysplastic Syndromes and Acute Myeloid Leukemia With Multilineage Dysplasia
- ClinicalTrials.gov ID
- NCT01210274
- Acronym / sponsor ID
- 10-PP-10
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 250 participants (estimated)
- Lead sponsor
- Centre Hospitalier Universitaire de NiceUniversity, hospital or other
- Study start
- Sep 2010
- Primary completion
- Sep 2014 (actual)
- Study completion
- Sep 2025 (estimated)
- First posted
- 28 Sep 2010 (estimated)
- 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 frequent diseases in elderly patients (median age: 71 years). IPSS classification defines low risk (Low and Intermediate 1), and high risk (Intermediate 2 and High) MDS. High-risk MDS (MDS-HR) have a high risk of transformation into acute leukemia with multilineage dysplasia (AML-DML). The success of Azacitidine has been mainly achieved through a rigorous empirical and clinical research, but the molecular mechanisms by which this molecule exerts its effects remain poorly characterized. The primary mode of action of Azacytidine is through DNA demethylation, and integration in to mRNA that favor traduction inhibition. The impact of this molecule on various cell death programs involved in the elimination of leukemic cells : apoptosis and autophagy is currently poorly known.
The research program and clinical studies we proposed focus on two major aspects:
- Main objective: Molecular mechanism of action and resistance to Azacitidine: Role of apoptosis versus autophagy.
- Secondary Objective: Reversion of Azacytidine resistance using different drugs targeting apoptosis and/or autophagy. Our laboratory has identified new molecules to selectively induce different types of cell death (apoptosis or autophagy).
Study design
- Observational model
- Case only
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients with myelodysplastic syndromes or acute myeloid leukemia with multilineage dysplasia treated with Azacitidine
Key inclusion criteria (5 listed)
- Age ≥ 18 years
- High Risk or Intermediate 2 MDS (IPSS)
- AML-MD (WHO classification)
- Treatment with minimum three to six cycles of Azacitidine
- Informed consent form signed
Key exclusion criteria (1 listed)
- Treatment with others chemotherapies alone or in association
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
hematological response
Time frame: at 3 months
Hematological response evaluated by the International Working Group (IWG) response of Cheson
hematological response
Time frame: at 6 months
Hematological response evaluated by the International Working Group (IWG) response of Cheson
2 secondary outcomes
Overall survival
Time frame: Day 1 of treatment
Overall survival
Time frame: at the death
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.
- Derived from this study: PubMed 22577154Cluzeau T, Robert G, Mounier N, Karsenti JM, Dufies M, Puissant A, Jacquel A, Renneville A, Preudhomme C, Cassuto JP, Raynaud S, Luciano F, Auberger P. BCL2L10 is a predictive factor for resistance to azacitidine in MDS and AML patients. Oncotarget. 2012 Apr;3(4):490-501. doi: 10.18632/oncotarget.481.
- Individual participant dataSharing planned: No
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.
4 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Myelodysplastic Syndromes or Acute Myeloid Leukemia With Multilineage Dysplasia, run by Centre Hospitalier Universitaire de Nice at 4 sites in 2 countries.
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 1 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 4 sites in France, Monaco. 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?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
hematological response (time frame: at 3 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01210274, which also lists 1 linked publication.
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.