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Studies how leukemia cells resist azacitidine treatment in high-risk blood disease

Studies why azacitidine stops working in adults with high-risk myelodysplastic syndromes.

Plain-language title generated from the registry record
Registry title: Characterization of the Mechanisms of Resistance to Azacitidine

Key facts

Where
Monaco, MonacoSee the site

AI summary: who the study is looking for

From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.

The trial is looking for adults with high-risk myelodysplastic syndrome or acute myeloid leukemia with multilineage dysplasia who have been treated with Azacitidine.

Age
18 years or older
Sex
All sexes can join.
Disease stage
High-risk or Intermediate 2 MDS (IPSS), or AML with multilineage dysplasia (WHO classification).
Earlier treatment
At least three to six cycles of Azacitidine.

Main reasons for not taking part

  • Treatment with other chemotherapies, alone or combined

Read by machine on 12 November 2026.

Where in Monaco

  • CH Princesse GraceMonaco, MonacoContact: Vanina OLIVERI, Project ManagerTaking people

Questions to ask the study team

  1. How often do I need to come in, and for how long each time?
  2. Are travel costs paid?
  3. Can I keep my own doctor?
  4. What happens if I want to stop?

Contact the study team: see the registry record → Contacts and Locations

Print a page for my doctor

Trial details

Who runs it
Centre Hospitalier Universitaire de Nice
Trial code
NCT01210274
Record updated
19 March 2025
Original description
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.Read full original descriptionShow less

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).

Inclusion: 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.

Exclusion: Treatment with others chemotherapies alone or in association.

ClinicalTrials.gov NCT01210274