High Risk Myelodysplasia Treated by Azacytidine : Genetic and Epigenetic (MYRAGE) (MYRAGE)

August 5, 2019 updated by: Central Hospital, Nancy, France

Myelodysplastic syndromes (MDS) are the most frequent myeloid neoplasms in Western Countries.They mainly affect patients aged 65 years or older. This is a very heterogenous group of diseases, which prognosis is evaluated with International Prognosis Scoring System. High risk MDS present with high frequency of transformation into acute myeloid leukemia. Treatment of high risk MDS often is based on hypomethylating agents, such as 5'-azacytidine (Azacytidine), with a complete response in approximativel 20% of cases..

This treatment is based on 4-week cycles, with daily injection during the first week and rest during the 3 next weeks of the cycle.

Azacytidine efficacy is commonly evaluated with clinical and biological parameters determined by the International Working Group 2006. These parameters are usually evaluated after at least 6 cycles of treatments.

There is a response with Azacytidine treatment in 60% of cases, including 40% of partial responses and 20% of complete responses. In 40% of patients, there is no response, which means that the disases is stable or in progression under therapy.

In this regard, early evaluation of treatment response is an issue. We want to improve our knowledge about early response criteria in Azacytidine-treated high-risk MDS, focusing on SMD with excess blasts, which represent 30 to 40% of total MDS.

Then, the investigator team want to compare DNA methylation profile at diagnosis and after 3 cycles of Azacytidine treatment.

Main objective :

Identify DNA methylation profiles related to response to Azacytidine therapy, after only 3 cycles of treatment, in high risk MDS with excess blasts.

Secondary objective :

Identify at diagnosis DNA methylation profiles that are predicitive of response to Azacytidin, in high risk MDS with excess blasts.

Study Overview

Status

Unknown

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Anticipated)

32

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • France
      • Nancy, France, 54035
        • Not yet recruiting
        • CHRU de NANCY
        • Contact:
          • Julien BROSEUS, MCU-PH
      • Vandoeuvre les Nancy, France
        • Recruiting
        • BROSEUS
        • Contact:
          • Julien BROSEUS

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

14 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Pre-inclusion Criteria:

  • patient benefiting from social welfcare
  • patient followed at the University Hospital of Nancy
  • patient aged 18 years or older
  • patient informed on research organization and having signed an informed pre-inclusion consent
  • No personal history of myelodysplastic syndrome
  • clinical exam adapted to research
  • one or more blood cytopenia

Inclusion Criteria:

  • patient benefiting from social welfcare
  • patient followed at the University Hospital of Nancy
  • patient aged 18 years or older
  • patient informed on research organization and having signed an informed inclusion consent
  • definitive diagnosis of high risk myelodysplastic syndrome with excess blasts
  • eligibility to an Azacytidine therapy as first-line treatment

Exclusion Criteria:

  • personal history or current other cancer
  • immediate acute myeloid leukemia
  • personal history of demethylation treatment
  • pregnant or breast feeding women
  • life-theatening condition
  • guardianship
  • imprisoned patients

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Screening
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Patients with High-risk MDS With Excess Blasts
Diagnostic test: Myelogram
Bone marrow aspiration after 3 cycles of Azacytidine treatment

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Methylation level of the Differentially Methylated Regions (DMR)
Time Frame: 3 months (after 3 cycles of treatment)
3 months (after 3 cycles of treatment)
Overall response by IWG 2006 response criteria (complete remission / partial remission / non response)
Time Frame: At the treatment response assessment (After 6-12 cycles of treatment up to 52 weeks)
At the treatment response assessment (After 6-12 cycles of treatment up to 52 weeks)

Secondary Outcome Measures

Outcome Measure
Time Frame
Methylation level of the Differentially Methylated Regions (DMR)
Time Frame: At diagnosis
At diagnosis
Cytogenetic response by IWG 2006 response criteria (major / minor / no response)
Time Frame: At the treatment response assessment (After 6-12 cycles of treatment up to 52 weeks)
At the treatment response assessment (After 6-12 cycles of treatment up to 52 weeks)
Hematologic improvement by IWG 2006 response criteria (major / minor / no response)
Time Frame: At the treatment response assessment (After 6-12 cycles of treatment up to 52 weeks)
At the treatment response assessment (After 6-12 cycles of treatment up to 52 weeks)
Transfusion independence (yes/no)
Time Frame: At the treatment response assessment (After 6-12 cycles of treatment up to 52 weeks)
At the treatment response assessment (After 6-12 cycles of treatment up to 52 weeks)
General condition improvement (yes/no)
Time Frame: At the treatment response assessment (After 6-12 cycles of treatment up to 52 weeks)
At the treatment response assessment (After 6-12 cycles of treatment up to 52 weeks)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Central Hospital, Nancy, France

Collaborators

Inserm U954 - N-GERE (Nutrition, Genetics and Exposition to Environmental Risk)

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

October 12, 2017

Primary Completion (Anticipated)

January 1, 2022

Study Completion (Anticipated)

January 1, 2022

Study Registration Dates

First Submitted

July 3, 2017

First Submitted That Met QC Criteria

July 12, 2017

First Posted (Actual)

July 14, 2017

Study Record Updates

Last Update Posted (Actual)

August 6, 2019

Last Update Submitted That Met QC Criteria

August 5, 2019

Last Verified

August 1, 2019

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • MYRAGE

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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