A Survival Study in Patients With High Risk Myelodysplastic Syndromes Comparing Azacitidine Versus Conventional Care

A Multicenter, Randomized, Open-label, Parallel-group, Phase 3 Trial of Subcutaneous Azacitidine Plus Best Supportive Care Versus Conventional Care Regimens Plus Best Supportive Care for the Treatment of Myelodysplastic Syndromes (MDS)

The purpose of this study is to determine whether patients with high-risk myelodysplastic syndromes (MDS) treated with azacitidine have improved survival compared to conventional care treatments. The study will also assess the effect of treatments on response, duration of response, and transformation to acute myeloid leukemia (AML). The study will continue for 12 months following last patient enrolled.

See study AZA PH GL 2003 CL 001 E for information about the extension to this study.

Study Overview

• Status: Completed
• Conditions: Myelodysplastic Syndromes
• Intervention / Treatment: Drug: Azacitidine; Other: Physician Choice

Detailed Description

Comparison/Control Interventions offered the physician three options:

• Best supportive care (BSC) alone,
• Low-dose cytarabine subcutaneously for 14 days every 28 to 42 days, or
• Standard chemotherapy administered for induction as a continuous intravenous infusion of cytarabine over 7 days plus an anthracycline (daunorubicin, idarubicin, or mitoxantrone) on Days 1, 2, and 3; and, for those eligible, 1 or 2 consolidation cycles administered as continuous intravenous infusions of cytarabine for 3 to 7 days with the same anthracycline that was used at induction on Days 1 and 2 (each cycle between 28 to 70 days from the start of the previous cycle).

All three options included best supportive care. Neither the experimental group (azacitidine) nor any of the comparison/control options allowed use of erythropoietin.

Duration of Intervention: Patients will be treated until death, withdrawal, unacceptable toxicity or conclusion of the study.

• Study Type: Interventional
• Enrollment (Actual): 358
• Phase: Phase 3

Contacts and Locations

Study Locations

• Australia
  • New South Wales
    • Liverpool, New South Wales, Australia, 2170
      • Liverpool Hospital
    • St. Leonards, New South Wales, Australia, 2065
      • Royal North Shore Hospital
    • Warratah, New South Wales, Australia, 2298
      • The Newcastle Mater Miseriecordiae Hospital
  • Queensland
    • Hersten, Queensland, Australia, 4029
      • Royal Brisbane Hospital
    • Woolloongabba, Queensland, Australia, 4102
      • Princess Alexandra Hospital
  • South Australia
    • Adelaide, South Australia, Australia, 5000
      • Royal Adelaide Hospital
  • Victoria
    • East Melbourne, Victoria, Australia, 3002
      • Peter Maccallum Cancer Institute
    • Melbourne, Victoria, Australia, 3050
      • Royal Melbourne Hospital
    • Melbourne, Victoria, Australia, 3181
      • The Alfred Hospital
  • Western Australia
    • Perth, Western Australia, Australia, 6847
      • The Royal Perth Hospital
• Bulgaria
  • Pleven, Bulgaria, 5800
    • First Clinical Base - Clinic of Hematology, MHAT - Pleven
  • Plovdiv, Bulgaria, 4004
    • III-rd Internal Department, District Dispensary for Oncology diseases with stationary(DDOncDIU)
  • Plovdiv, Bulgaria, 4002
    • MHAT "St George" Clinic of Hematology, Plovdiv
  • Sofia, Bulgaria, 1756
    • National Centre of Hematology and Transfusiology, Sofia
  • Varna, Bulgaria, 9010
    • University Multiprofile Hospital for Active Treatment "Sveta Marina"
  • Varna, Bulgaria, 3010
    • Multiprofile Hospital for Active Treatment (MHAT), "St. Marina" Clinic of Hematology
• Czechia
  • Olomouc, Czechia, 775 20
    • Fakultní Nemocnice Olomouc
  • Praha, Czechia, 2 128 08
    • Vseobecna Fakultni Nemocnice
  • Praha, Czechia, 2 128 20
    • Uslav Hematologie a Krevni Transfuze
  • Brno
    • Jihlavska, Brno, Czechia, 639 00
      • Fakultní Nemocnice Brno
  • Hradec Kralove
    • Sokolska, Hradec Kralove, Czechia, 500 05
      • Fakultni nemocnice Hradec Kralove
• France
  • Angers, France, 49033
    • CHU d'Angers
  • Clichy, France, 92110
    • Hopital Beaujon
  • Lille, France, 59037
    • Che De Lille
  • Lyon, France, 69437
    • Hospital Edouard Herriot
  • Marseille, France, 13009
    • Institute Paoli Calmettes
  • Nantes, France, 44093
    • CHU de Nantes
  • Paris, France, 75679
    • Hôpital Cochin
  • Paris, France, 75010
    • Hospital Saint Louis
  • Rouen, France, 76038
    • Centre Henri Becquerel
  • Toulouse, France, 31059
    • CHU Purpan
• Germany
  • Bonn, Germany, 53105
    • Universitätsklinikum Bonn
  • Chemnitz, Germany, 9113
    • Klinikum Chemnitz gGmbH
  • Dresden, Germany, 1307
    • Universitätsklinikum Carl Gustav Carus
  • Duisburg, Germany, 47166
    • St Johannes Hospital
  • Dusseldorf, Germany, 40225
    • Heinrich-Heine University Düsseldorf
  • Essen, Germany, 45147
    • University Essen
  • Gottingen, Germany, 37075
    • Gerorg-August-Universitat Gottingen
  • Hamburg, Germany, D-20099
    • Allgemeines Krankenhaus St. Georg
  • Hamburg, Germany, D-20246
    • Universitatsklinikum Hambur-Eppendorf
  • Kiel, Germany, D-24116
    • Universitatsklinikum Kiel II
  • Ulm, Germany, 89070
    • Universitätsklinikum Ulm
  • Berlin
    • Hindenburgdamm, Berlin, Germany, D-12203
      • Universitatsklinikum Benjamin Franklin
• Greece
  • Athens, Greece, 11527
    • District General Hospital of Athens
  • Athens, Greece, 11527
    • General Hospital of Chest Disease
  • Ioannina, Greece, 45500
    • University General Hospital of Ioannina
  • Patra, Greece, 26500
    • University General Hospital of Patra Rio
  • Athens
    • Haidari, Athens, Greece, 12462
      • University Hospital-Attikon
  • Crete
    • Heraklio, Crete, Greece, 71110
      • University General Hospital of Heraklio Voutes
• Hungary
  • Budapest, Hungary, 1135
    • Orszagos Gyogyintezeti Kozpont
  • Pecs, Hungary, 7624
    • University of Pecs, 1st Dept of Internal Medicine
  • Szeged, Hungary, 6701
    • University of Szeged, 2nd Department of Internal Medicine
• Italy
  • Bologna, Italy, 40138
    • Policlinico S. Orsola-Malpighi
  • Firenze, Italy, 50139
    • Universita Di Firenze
  • Genova, Italy, I-16132
    • Ospedale San Martino
  • Milano, Italy, 20133
    • Instituto Nazionale Dei Tumori
  • Modena, Italy, 41100
    • Centro Oncologico Modenese
  • Roma, Italy, 00168
    • Policlinico Gemelli
  • Roma, Italy, 00144
    • Ospedale San Eugenio
  • Roma, Italy, 144
    • Instituto Nazionale Tumori "Regina Elena"
  • San Giovanni Rotondo, Italy, 71013
    • Ospedale Casa Sollievo Della Sofferenza - Irrc
  • Sassari, Italy, 7100
    • Università degli Studi di Sassari
• Netherlands
  • Amsterdam, Netherlands, 1081 HV
    • VU University Medical Center Amsterdam
  • Nijmejen, Netherlands
    • Univ Hospital St. Radboud
• Poland
  • Gdansk, Poland, 80-952
    • Samodzielny Publiczny Szpital Kliniczny Nr 1
  • Lodz, Poland, 93-510
    • Wojewodzki Szpital Specjalistyczny
  • Lublin, Poland, 20081
    • Samodzielny Publiczny Szpital Kliniczny
  • Warszawa, Poland, 00-909
    • Wojskowy Instytut Medyczny
  • Warszawa, Poland, 02-097
    • Samodzelny Publiczny Centralny Szpital Kliniczny
  • Wroclaw, Poland, 50-367
    • Samodzielny Publiczny Szpital Kliniczny Nr 1
• Russian Federation
  • Moscow, Russian Federation, 105299
    • Burdenko Central Military Clinical Hospital
  • Moscow, Russian Federation, 115487
    • Blokhin Cancer Research Center
  • Moscow, Russian Federation, 125167
    • Scientific Haematology Center, Moscow
  • St. Petersburg, Russian Federation, 197089
    • Pavlov State Medical University
  • St. Petersburg, Russian Federation, 197110
    • City Hospital #31
  • St. Petersburg, Russian Federation, 193024
    • Institute of Haematology & Blood Transfusion
  • St. Petersburg, Russian Federation, 197022
    • Pavlov State Medical University
• Spain
  • Barcelona, Spain, 08036
    • Hospital Clinic
  • Barcelona, Spain, 08025
    • Hospital Santa Creu i Sant Pau
  • Barcelona, Spain
    • Hospital Universitario Germans Trias i Pujol
  • Leon, Spain, 24071
    • Hospital de León
  • Madrid, Spain, 28006
    • Hospital Universitario de La Princesa
  • Madrid, Spain, 28034
    • Hospital Ramón y Cajal
  • Madrid, Spain, 28046
    • Hospital La Paz, Madrid
  • Madrid, Spain, 28048
    • Hospital Clinico San Carlos
  • Palma de Mallorca, Spain, 07198
    • Hospital Son Llatzer
  • Salamanca, Spain, 37007
    • Hospital Universitario Del Salamanca
  • Valencia, Spain, 46009
    • Hospital Universitario La Fe
• Sweden
  • Goteborg, Sweden, S-413 45
    • Sahlgrenska University Hospital
  • Lund, Sweden, 22185
    • Lund Universtiy Hospital
  • Malmo, Sweden, S-205 02
    • University Hospital MAS
  • Stockholm, Sweden, 14186
    • Huddinge University Hospital
  • Uppsala, Sweden, S-751 85
    • Uppsala University Hospital
• United Kingdom
  • Bournemouth, United Kingdom, BH7 7DW
    • Royal Bournemouth General Hospital
  • London, United Kingdom, EC1A 7BE
    • St. Bartholomew's Hospital
  • London, United Kingdom
    • Kings College Hospital NHS Trust
  • Manchester, United Kingdom, M20 4BX
    • Christie Hospital
  • Norwich, United Kingdom, NR4 7UY
    • Norfolk and Norwich University Hospital
  • Oxford, United Kingdom, OX3 9DU
    • John Radcliffe Hospital
  • Truro, United Kingdom, TR1 3LJ
    • Royal Cornwall Hospital
• United States
  • Alabama
    • Birmingham, Alabama, United States, 35294
      • University of Alabama School of Medicine
  • Indiana
    • Indianapolis, Indiana, United States, 46202
      • Indiana University Cancer Center
  • Missouri
    • Saint Louis, Missouri, United States, 63110
      • Washington University School of Medicine
  • New York
    • New York, New York, United States, 10029-6574
      • Mount Sinai Medical Center
  • Ohio
    • Cleveland, Ohio, United States, 44106
      • Case Western Reserve University
  • Oregon
    • Portland, Oregon, United States, 97201
      • Oregon Cancer Center
  • Pennsylvania
    • Pittsburgh, Pennsylvania, United States, 15224
      • Western Pennsylvania Cancer Institute
  • Wisconsin
    • Milwaukee, Wisconsin, United States, 53226
      • Froedtert Memorial Lutheran Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Have a diagnosis of refractory anemia with excess blasts or refractory anemia with excess blasts in transformation according to the French-American-British classification system for myelodysplastic syndromes (MDS) and a relatively high risk of acute myeloid leukemia (AML) transformation, with an International Prognostic Scoring System score of INT-2 or High.
• Be 18 years of age or older
• Have a life expectancy of at least 3 months
• Be unlikely to proceed to bone marrow or stem cell transplantation therapy following remission
• Have serum bilirubin levels less than or equal to 1.5 times the upper limit of normal range for the laboratory
• Have serum glutamic-oxaloacetic transaminase (aspartate aminotransferase) or serum glutamic-pyruvic transaminase (alanine aminotransferase) levels less than or equal to 2 times the upper limit of normal (unless these are considered to be related to transfusion-induced secondary hemosiderosis)
• Have serum creatinine levels less than or equal to 1.5 times the upper limit of normal

Exclusion Criteria:

• Secondary myelodysplastic syndromes (MDS)
• Prior treatment with azacitidine;
• Prior history of acute myeloid leukemia (AML);
• Malignant disease diagnosed within prior 12 months;
• Metastatic disease;
• Hepatic tumors;
• Radiation, chemotherapy, cytotoxic therapy for non-MDS conditions within prior 12 months;
• Prior transplantation or cytotoxic therapy to treat MDS;
• Serious medical illness likely to limit survival to 12 months or less;
• Treatment with erythropoietin or myeloid growth factors during prior 21 days or androgenic hormones during prior 13 days;
• Active HIV, viral hepatitis type B or C;
• Treatment with investigational drugs during prior 30 days;
• Within the 28-day screening period, documented red cell folate deficiency, as evidenced by red blood cell folate (not serum folate) or vitamin B12 deficiency

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Other
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Azacitidine; Study Drug plus best supportive care. Treatment with erythropoietin was not permitted	Drug: Azacitidine; Azacitidine was injected subcutaneously (SC) at an initial dose of 75mg/m^2/day for 7 days. The 7-day dosing was repeated every 28 days with dose adjustment based on predefined hematology and renal laboratory results. Number of cycles: Azacitidine treatment was to be continued until the end of the study unless treatment was discontinued due to unacceptable toxicity, relapse after complete or partial response, transformation to AML or disease progression.; Other Names: AZA
Active Comparator: Conventional Care; Physician choice of low dose cytarabine (plus best supportive care), standard chemotherapy (plus best supportive care) or best supportive care (only). Treatment with erythropoietin was not permitted	Other: Physician Choice; Physician Choice was one of three options: Best supportive care (BSC) alone,; Low-dose cytarabine subcutaneously for 14 days every 28 to 42 days, or; Standard chemotherapy administered for induction as a continuous intravenous infusion of cytarabine over 7 days plus an anthracycline (daunorubicin, idarubicin, or mitoxantrone) on Days 1, 2, and 3; and, for those eligible, 1 or 2 consolidation cycles administered as continuous intravenous infusions of cytarabine for 3 to 7 days with the same anthracycline that was used at induction on Days 1 and 2 (each cycle between 28 to 70 days from the start of the previous cycle). All three options included best supportive care; Other Names: cytarabine; anthracycline

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Kaplan-Meier Estimates for Median Time to Death From Any Cause	Kaplan-Meier estimates for the median months until death from any cause within the intent-to-treat population. Patients surviving at the end of the follow-up period were censored at the date of last contact. If a patient withdrew consent to follow-up or was lost to follow-up, the patient was censored as of the last date of contact.	Day 1 (randomization) to 42 months
Summary of Subgroup Analyses for Kaplan-Meier Estimates for Time to Death From Any Cause	Kaplan-Meier estimates for the median months until death from any cause within the intent-to-treat population. Patients surviving at the end of the follow-up period were censored at the date of last contact. If a patient withdrew consent to follow-up or was lost to follow-up, the patient was censored as of the last date of contact. Subgroups that were analyzed are age, gender, French-American-British (FAB) classification, World Health Organization (WHO) classification and International Prognostic Scoring System (IPSS) classification.	Day 1 (randomization) to 42 months
Number of Participants Who Died	Count of participants who died during the study	42 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Kaplan-Meier Estimate for Median Time to Transformation to Acute Myeloid Leukemia (AML) or Death From Any Cause, Whichever Occurred First	The time to transformation to AML or death from any cause (whichever occurred first) was defined as the number of days from the date of randomization until the date of documented AML transformation or death from any cause. Patients who did not transform to AML or die were censored at the date of last follow-up.	Day 1 (randomization) to 42 months
Kaplan-Meier Estimates for Median Time to Transformation to Acute Myeloid Leukemia (AML)	The time to transformation to AML was defined as the number of days from the date of randomization until the date of documented AML transformation, defined as a bone marrow blast count ≥ 30% independent of baseline bone marrow count. Patients who did not transform to AML were censored at the date of last follow-up or date of death.	Day 1 (randomization) to 42 months
Summary of Participants' Red Blood Cell (RBC) Transfusion Status for Participants Who Were Transfusion Dependent at Baseline	Summary of dependence and independence from red blood cell (RBC) transfusion at baseline and during treatment, for patients who were dependent at baseline. A patient was considered transfusion independent at baseline if the patient had no transfusions during the 56 days prior to randomization. During study, a patient was considered transfusion independent during the on-treatment period if the patient had no transfusions during any 56 consecutive days or more. Otherwise, the patient was considered transfusion dependent.	Day 1 (randomization) to 42 months
Summary of Participants' Red Blood Cell (RBC) Transfusion Status for Participants Who Were Transfusion Independent at Baseline	Summary of dependence and independence from red blood cell (RBC) transfusion at baseline and during treatment, for patients who were independent at baseline. A patient was considered transfusion independent at baseline if the patient had no transfusions during the 56 days prior to randomization. During study, a patient was considered transfusion independent during the on-treatment period if the patient had no transfusions during any 56 consecutive days or more. Otherwise, the patient was considered transfusion dependent.	Day 1 (randomization) to 42 months
Summary of Participants' Platelet Transfusion Status for Participants Who Were Transfusion Dependent at Baseline	Summary of dependence and independence from platelet transfusion at baseline and during treatment for patients who were dependent at baseline. A patient was considered transfusion independent at baseline if the patient had no transfusions during the 56 days prior to randomization. During study, a patient was considered transfusion independent during the on-treatment period if the patient had no transfusions during any 56 consecutive days or more. Otherwise, the patient was considered transfusion dependent.	Day 1 (randomization) to 42 months
Summary of Participants' Platelet Transfusion Status for Participants Who Were Transfusion Independent at Baseline	Summary of dependence and independence from platelet transfusion at baseline and during treatment for patients who were independent at baseline. A patient was considered transfusion independent at baseline if the patient had no transfusions during the 56 days prior to randomization. During study, a patient was considered transfusion independent during the on-treatment period if the patient had no transfusions during any 56 consecutive days or more. Otherwise, the patient was considered transfusion dependent.	Day 1 (randomization) to 42 months
Number of Participants Considered Hematologic Responders by Investigator Determinations Using International Working Group (IWG 2000) Criteria for Myelodysplastic Syndrome (MDS)	Investigator determined responses followed IWG criteria for; complete remission(CR): repeat bone marrow show <5% myeloblasts, and peripheral blood evaluations lasting >=2 months of hemoglobin(>110 g/L), neutrophils(>=1.5x10^9/L), platelets(>=100x10^9/L), blasts (0%) and no dysplasia; partial remission(PR) is the same as CR for peripheral blood: bone marrow shows blasts decrease by >=50% or a less advanced FAB classification from pretreatment; stable disease(SD) is a failure to achieve at least a partial remission, but with no evidence of progression for at least 2 months.	Day 1 to 42 months
Number of Participants Showing Hematologic Improvement Using International Working Group (IWG 2000) Criteria for Myelodysplastic Syndrome (MDS) Assessed by Independent Review Committee	IWG 2000 Criteria: Pretreatment=hemoglobin <100g/L or RBC transfusion-dependent, platelet count <100x10^9/L or platelet transfusion dependent, absolute neutrophil count <1.5x10^9/L. Erythroid response: Major->20g/L increase or transfusion independent. Minor- 10-20g/L increase or >=50% decrease in transfusion requirements. Platelet response: Major-absolute increase of >=30x10^9/L or platelet transfusion independence. Minor->=50% increase. Neutrophil response: Major->=100% increase or an absolute increase of >0.5x10^9/L. Minor->=100% increase and absolute increase of <0.5x10^9/L.	Day 1 to 42 months
Time to Disease Progression, Relapse After Complete or Partial Remission, or Death From Any Cause	The time to disease progression, relapse after complete or partial remission (CR, PR), or death from any cause was defined as the time from the date of randomization until the first date of documented disease progression, relapse after CR or PR, or death from any cause.	Day 1 (randomization) to 42 months
Duration of Any Hematologic Improvement	The duration of improvement was defined as the time from the date of hematologic improvement until the date of first documented progression or relapse after hematologic improvement or death from any cause.	Day 1 (randomization) to 42 months
Number of Infections Per Treatment Year Requiring Intravenous Antibiotics, Antifungals or Antivirals	The on-treatment adverse event rate of infection requiring IV antibiotics, antifungals, or antivirals per patient-years. The on-treatment period was considered the period from the date of randomization to the last treatment study visit.	Day 1 (randomization) to 42 months
Number of Participants in Different Categories of Adverse Experiences During Core Study Period	Patient counts for a variety of subsets of adverse experiences for the core study period (day 1 to 42 months). The individual options for Conventional Care Regimens (Best Supportive Care Only, Low-Dose Cytarabine, and Standard Chemotherapy) are presented as separate treatments.	Day 1 (randomization) to 42 months

Collaborators and Investigators

• Sponsor: Celgene
• Investigators: Study Director: CL Beach, Celgene Corporation

Publications and helpful links

General Publications

• Fenaux P, Mufti GJ, Hellstrom-Lindberg E, Santini V, Finelli C, Giagounidis A, Schoch R, Gattermann N, Sanz G, List A, Gore SD, Seymour JF, Bennett JM, Byrd J, Backstrom J, Zimmerman L, McKenzie D, Beach C, Silverman LR; International Vidaza High-Risk MDS Survival Study Group. Efficacy of azacitidine compared with that of conventional care regimens in the treatment of higher-risk myelodysplastic syndromes: a randomised, open-label, phase III study. Lancet Oncol. 2009 Mar;10(3):223-32. doi: 10.1016/S1470-2045(09)70003-8. Epub 2009 Feb 21.
• Fenaux P, Mufti GJ, Hellstrom-Lindberg E, Santini V, Gattermann N, Germing U, Sanz G, List AF, Gore S, Seymour JF, Dombret H, Backstrom J, Zimmerman L, McKenzie D, Beach CL, Silverman LR. Azacitidine prolongs overall survival compared with conventional care regimens in elderly patients with low bone marrow blast count acute myeloid leukemia. J Clin Oncol. 2010 Feb 1;28(4):562-9. doi: 10.1200/JCO.2009.23.8329. Epub 2009 Dec 21.
• Fenaux P, Gattermann N, Seymour JF, Hellstrom-Lindberg E, Mufti GJ, Duehrsen U, Gore SD, Ramos F, Beyne-Rauzy O, List A, McKenzie D, Backstrom J, Beach CL. Prolonged survival with improved tolerability in higher-risk myelodysplastic syndromes: azacitidine compared with low dose ara-C. Br J Haematol. 2010 Apr;149(2):244-9. doi: 10.1111/j.1365-2141.2010.08082.x. Epub 2010 Feb 5. Erratum In: Br J Haematol. 2010 Jun;149(6):919.
• Santini V, Fenaux P, Mufti GJ, Hellstrom-Lindberg E, Silverman LR, List A, Gore SD, Seymour JF, Backstrom J, Beach CL. Management and supportive care measures for adverse events in patients with myelodysplastic syndromes treated with azacitidine*. Eur J Haematol. 2010 Aug;85(2):130-8. doi: 10.1111/j.1600-0609.2010.01456.x. Epub 2010 Apr 12.
• Seymour JF, Fenaux P, Silverman LR, Mufti GJ, Hellstrom-Lindberg E, Santini V, List AF, Gore SD, Backstrom J, McKenzie D, Beach CL. Effects of azacitidine compared with conventional care regimens in elderly (>/= 75 years) patients with higher-risk myelodysplastic syndromes. Crit Rev Oncol Hematol. 2010 Dec;76(3):218-27. doi: 10.1016/j.critrevonc.2010.04.005. Epub 2010 May 6.
• Gore SD, Fenaux P, Santini V, Bennett JM, Silverman LR, Seymour JF, Hellstrom-Lindberg E, Swern AS, Beach CL, List AF. A multivariate analysis of the relationship between response and survival among patients with higher-risk myelodysplastic syndromes treated within azacitidine or conventional care regimens in the randomized AZA-001 trial. Haematologica. 2013 Jul;98(7):1067-72. doi: 10.3324/haematol.2012.074831. Epub 2013 Apr 12.

Helpful Links

• Study record for the extension study of AZA PH GL 2003 CL 001

Study record dates

Study Major Dates

• Study Start (Actual): November 1, 2003
• Primary Completion (Actual): July 1, 2007
• Study Completion (Actual): July 1, 2007

Study Registration Dates

• First Submitted: October 31, 2003
• First Submitted That Met QC Criteria: November 4, 2003
• First Posted (Estimate): November 5, 2003

Study Record Updates

• Last Update Posted (Actual): October 29, 2019
• Last Update Submitted That Met QC Criteria: October 16, 2019
• Last Verified: October 1, 2019

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Neoplasms; Disease; Bone Marrow Diseases; Hematologic Diseases; Precancerous Conditions; Syndrome; Myelodysplastic Syndromes; Preleukemia; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Anti-Infective Agents; Antiviral Agents; Enzyme Inhibitors; Antimetabolites, Antineoplastic; Antimetabolites; Antineoplastic Agents; Immunosuppressive Agents; Immunologic Factors; Azacitidine; Cytarabine
• Other Study ID Numbers: AZA PH GL 2003 CL001