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)
A Phase II Study of Azacitidine (Vidaza®) Combined to Epoetin Beta (NeoRecormon®) in IPSS Low-risk and Intermediate-1 MDS Patients, Resistant to ESA
1 other identifier
interventional
98
1 country
29
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_2
Started Feb 2009
Longer than P75 for phase_2
29 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
February 1, 2009
CompletedFirst Submitted
Initial submission to the registry
November 17, 2009
CompletedFirst Posted
Study publicly available on registry
November 18, 2009
CompletedPrimary Completion
Last participant's last visit for primary outcome
March 1, 2014
CompletedStudy Completion
Last participant's last visit for all outcomes
March 1, 2014
CompletedMarch 19, 2014
November 1, 2009
5.1 years
November 17, 2009
March 18, 2014
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
To determine the major erythroid response rate after 6 courses, assessed according to IWG 2000 criteria
after 6 courses of treatment in the respective treatment arm
Secondary Outcomes (1)
Degree and duration of erythroid response (including red blood cell transfusion independence),overall survival and time to progression and toxicity
after 4 and 6 months of treatment until the end of study
Study Arms (2)
Arm A
ACTIVE COMPARATORAzacitidine 75mg/sqm SQ per day for 5 days every 28 days for 6 courses and 12 additional maintenance courses in responders.
Arm B
ACTIVE COMPARATORAzacitidine: 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
Interventions
Azacitidine 75mg/sqm SQ per day for 5 days every 28 days
Epoetin beta : 60000U weekly SQ injections NeoRecormon®
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Groupe Francophone des Myelodysplasieslead
- Celgene Corporationcollaborator
- Roche Pharma AGcollaborator
Study Sites (29)
CHU d'Amiens
Amiens, 80054, France
Hôpital Angers
Angers, 49033, France
Hôpital Avignon
Avignon, 84000, France
Hôpital de la Côte Basque
Bayonne, 64100, France
Hopital Avicenne
Bobigny, 93009, France
Hôpital Boulogne Sur Mer
Boulogne-sur-Mer, 62321, France
Hopital Clémenceau
Caen, 14033, France
Hôpital le Bocage
Dijon, 21034, France
Hôpital Versailles
Le Chesnay, 78157, France
Hôpital kremlin Bicêtre
Le Kremlin-Bicêtre, 94275, France
Hôpital Saint Vincent
Lille, 59020, France
Hôpital Huriez
Lille, 59037, France
Hôpital Limoges
Limoges, 87046, France
Hôpital Edouard Herriot
Lyon, 69437, France
Hôpital Paoli-Calmettes
Marseille, 13273, France
Hôpital Brabois
Nancy, 54511, France
Hôpital Hôtel Dieu
Nantes, 44035, France
Hôpital Archet1
Nice, 06202, France
Hôpital La Source
Orléans, 45067, France
Hôpital Lariboisière
Paris, 75475, France
Hôpital Saint Louis
Paris, 75475, France
Hôpital Saint Antoine
Paris, 75571, France
Hôpital Cochin
Paris, 75679, France
Hôpital Maréchal Joffre
Perpignan, 66046, France
Hôpital Jean-Bernard
Poitiers, 86021, France
Hôpital Reims
Reims, 51092, France
Hôpital Henri Becquerel
Rouen, 76038, France
Hôpital Hautepierre
Strasbourg, 67098, France
Hôpital Purpan
Toulouse, 31059, France
Related Publications (1)
Thepot S, Ben Abdelali R, Chevret S, Renneville A, Beyne-Rauzy O, Prebet T, Park S, Stamatoullas A, Guerci-Bresler A, Cheze S, Tertian G, Choufi B, Legros L, Bastie JN, Delaunay J, Chaury MP, Sanhes L, Wattel E, Dreyfus F, Vey N, Chermat F, Preudhomme C, Fenaux P, Gardin C; Groupe Francophone des Myelodysplasies (GFM). A randomized phase II trial of azacitidine +/- epoetin-beta in lower-risk myelodysplastic syndromes resistant to erythropoietic stimulating agents. Haematologica. 2016 Aug;101(8):918-25. doi: 10.3324/haematol.2015.140988. Epub 2016 May 26.
PMID: 27229713DERIVED
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Simone Boehrer, MD
Groupe Francophone des Myélodysplasies
- PRINCIPAL INVESTIGATOR
Claude Gardin, MD
Groupe Francophone des Myélodysplasies
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
November 17, 2009
First Posted
November 18, 2009
Study Start
February 1, 2009
Primary Completion
March 1, 2014
Study Completion
March 1, 2014
Last Updated
March 19, 2014
Record last verified: 2009-11