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Azacitidine Combined to Epoetin Beta in International Prognostic Scoring System (IPSS) Low-risk and Intermediate-1 Myelodysplastic Syndrome (MDS) Patients, Resistant to Erythropoetin-stimulating Agents (ESA)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01015352
Recruitment Status : Completed
First Posted : November 18, 2009
Last Update Posted : March 19, 2014
Celgene Corporation
Roche Pharma AG
Information provided by (Responsible Party):
Groupe Francophone des Myelodysplasies

Brief Summary:
The study is aimed to treat low-risk MDS patients,who are dependent on red-blood cell transfusion due to disease-related anemia, and who have a proven resistance towards treatment with erythropoetin-stimulating agents (ESA). The study randomizes patients to receive a treatment with the demethylating agent 5-azacytidine alone or in combination with an ESA. The study thus evaluates, if efficacy of 5-azacytidine, notably on the red-blood cell transfusion-dependence is comparable/inferior to a combination treatment with azacitidine and an ESA (that is if 5-azacytidine can overcome the resistance towards ESA). Being a phase II study, the study assesses, duration of erythroid response, overall survival and time to progression as well as toxicity.

Condition or disease Intervention/treatment Phase
Myelodysplastic Syndromes Drug: Azacitidine Drug: Epoetin beta Phase 2

  Show Detailed Description

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 98 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase II Study of Azacitidine (Vidaza®) Combined to Epoetin Beta (NeoRecormon®) in IPSS Low-risk and Intermediate-1 MDS Patients, Resistant to ESA
Study Start Date : February 2009
Actual Primary Completion Date : March 2014
Actual Study Completion Date : March 2014

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Active Comparator: Arm A
Azacitidine 75mg/sqm SQ per day for 5 days every 28 days for 6 courses and 12 additional maintenance courses in responders.
Drug: Azacitidine
Azacitidine 75mg/sqm SQ per day for 5 days every 28 days
Other Name: Vidaza®

Active Comparator: Arm B

Azacitidine: 75mg/sqm SQ per day for 5 days every 28 days for 6 courses AND

Epoetin beta : 60000U weekly SQ injections (to be adapted according to Hb as described above)

12 additional maintenance courses are planned in responders

Drug: Azacitidine
Azacitidine 75mg/sqm SQ per day for 5 days every 28 days
Other Name: Vidaza®

Drug: Epoetin beta

Epoetin beta : 60000U weekly SQ injections


Other Name: Epoetin beta : 60000U weekly SQ injections

Primary Outcome Measures :
  1. To determine the major erythroid response rate after 6 courses, assessed according to IWG 2000 criteria [ Time Frame: after 6 courses of treatment in the respective treatment arm ]

Secondary Outcome Measures :
  1. Degree and duration of erythroid response (including red blood cell transfusion independence),overall survival and time to progression and toxicity [ Time Frame: after 4 and 6 months of treatment until the end of study ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

MDS defined as

  • RCMD, RA with or without ring sideroblasts
  • RAEB 1, or CMML 1, if WBC < 13 G /l according to the WHO classification
  • with a low or int-1 IPSS score AND
  • primary or secondary resistance to epoetin alpha/ beta (> 60000 U/w) or darbepoetin (> 250ug/w), administered for at least 12 weeks
  • requirement of RBC transfusions > 4 U in the previous 8 weeks
  • Aged 18 years or more
  • Adequate contraception, if relevant
  • Negative pregnancy test if relevant
  • Written Informed consent
  • Ability to participate to a clinical trial and adhere to study procedures
  • Health insurance

Exclusion Criteria:

  • Therapy-related MDS (after chemo- or radiotherapy for a previous neoplasm or immune disorder)
  • Patients with a planned allogeneic bone marrow transplantation
  • Creatininemia >1.5 upper normal value or estimated Ccr less than 30ml/mn
  • ALAT and ASAT >2.5 upper normal value
  • Bilirubin >2N, except unconjugated hyperbilirubinemia due to MDS-related dyserythropoiesis
  • Heart failure NYHA > II
  • Known allergy to mannitol
  • Other tumor, unstable for the last three years, except in situ uterine carcinoma or basal skin tumor
  • ECOG > 2
  • Life expectancy less than 3 months

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01015352

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CHU d'Amiens
Amiens, France, 80054
Hôpital Angers
Angers, France, 49033
Hôpital Avignon
Avignon, France, 84000
Hôpital de la Côte Basque
Bayonne, France, 64100
Hopital Avicenne
Bobigny, France, 93009
Hôpital Boulogne Sur Mer
Boulogne Sur Mer, France, 62321
Hopital Clémenceau
Caen, France, 14033
Hôpital le Bocage
Dijon, France, 21034
Hôpital kremlin Bicêtre
Kremlin Bicêtre, France, 94275
Hôpital Versailles
Le Chesnay, France, 78157
Hôpital Saint Vincent
Lille, France, 59020
Hôpital Huriez
Lille, France, 59037
Hôpital Limoges
Limoges, France, 87046
Hôpital Edouard Herriot
Lyon, France, 69437
Hôpital Paoli-Calmettes
Marseille, France, 13273
Hôpital Brabois
Nancy, France, 54511
Hôpital Hôtel Dieu
Nantes, France, 44035
Hôpital Archet1
Nice, France, 06202
Hôpital La Source
Orléans, France, 45067
Hôpital Lariboisière
Paris, France, 75475
Hôpital Saint Louis
Paris, France, 75475
Hôpital Saint Antoine
Paris, France, 75571
Hôpital Cochin
Paris, France, 75679
Hôpital Maréchal Joffre
Perpignan, France, 66046
Hôpital Jean-Bernard
Poitiers, France, 86021
Hôpital Reims
Reims, France, 51092
Hôpital Henri Becquerel
Rouen, France, 76038
Hôpital Hautepierre
Strasbourg, France, 67098
Hôpital Purpan
Toulouse, France, 31059
Sponsors and Collaborators
Groupe Francophone des Myelodysplasies
Celgene Corporation
Roche Pharma AG
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Principal Investigator: Simone Boehrer, MD Groupe Francophone des Myélodysplasies
Principal Investigator: Claude Gardin, MD Groupe Francophone des Myélodysplasies

Additional Information:
Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: Groupe Francophone des Myelodysplasies Identifier: NCT01015352     History of Changes
Other Study ID Numbers: GFM-Aza-Epo-2008-01
First Posted: November 18, 2009    Key Record Dates
Last Update Posted: March 19, 2014
Last Verified: November 2009

Keywords provided by Groupe Francophone des Myelodysplasies:
Myelodysplastic Syndromes

Additional relevant MeSH terms:
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Myelodysplastic Syndromes
Pathologic Processes
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Epoetin Alfa
Antimetabolites, Antineoplastic
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Enzyme Inhibitors