The Efficacy and Safety of Oral Azacitidine Plus Best Supportive Care Versus Placebo and Best Supportive Care in Subjects With Red Blood Cell (RBC) Transfusion-Dependent Anemia and Thrombocytopenia Due to International Prognostic Scoring System (IPSS) Low Risk Myelodysplastic Syndrome (MDS)
NCT ID: NCT01566695
Last Updated: 2025-01-07
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
216 participants
INTERVENTIONAL
2013-04-26
2023-12-21
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Oral Azacitidine
Arm 1: Oral azacitidine tablets 300 mg daily (QD) + best supportive care (BSC) on days 1 through 21 of each 28-day treatment cycle.
Oral Azacitidine
300 mg daily, days 1 to 21 of each 28-day treatment cycle
Best Supportiv Care (BSC)
BSC included and was not limited to packed RBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.
Placebo
Arm 2: Identically matching placebo tablets plus best supportive care on days 1 to 21 of each 28-day treatment cycle.
Placebo
Identically matching placebo tablets on day 1 to 21 of each 28-day treatment cycle.
Best Supportiv Care (BSC)
BSC included and was not limited to packed RBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.
Interventions
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Oral Azacitidine
300 mg daily, days 1 to 21 of each 28-day treatment cycle
Placebo
Identically matching placebo tablets on day 1 to 21 of each 28-day treatment cycle.
Best Supportiv Care (BSC)
BSC included and was not limited to packed RBC (packed red blood cell \[pRBC\] and whole blood), platelet transfusions (single donor or pooled donor), antibiotic, antiviral and/or antifungal therapy, nutritional support, and granulocyte colony stimulating factors (G-CSF) for participants who experienced neutropenic fever/infections.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Have a documented diagnosis of MDS
* Anemia that requires red blood cell transfusions
* Thrombocytopenia (sustained for at least 21 days) within 14 days prior to randomization
* Have an Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2
* Must agree to follow pregnancy precautions as required by protocol.
* Understand and voluntarily sign an informed consent document prior to any study related assessments/procedures being conducted
Exclusion Criteria
* Prior treatment with azacitidine, decitabine, other hypomethylating agents and lenalidomide (for lenalidomide : unless the last dose received is \>= 8 weeks prior to inclusion into the study).
* Prior allogeneic or autologous stem cell transplant
* Eligible for allogenic or autologous stem cell transplant
* History of inflammatory bowel disease (eg, Crohn's disease, ulcerative colitis), celiac disease (ie, sprue), prior gastrectomy or upper bowel removal, or any other gastrointestinal disorder or defect
* Thrombocytopenia secondary to other possible causes, including medication(s), congenital disorder(s), immune disorder(s), or microvascular disorder(s)
* Use of cytotoxic, chemotherapeutic, targeted or investigational agents/therapies, thrombopoiesis-stimulating agents (TSAs), erythropoiesis-stimulating agents (ESAs) and other red blood cell hematopoietic growth factors, and within 28 days prior to randomization
* Ongoing medically significant adverse events from previous treatment, regardless of the time period
* Concurrent use of iron-chelating agents, (except for subjects on a stable or decreasing dose for at least 8 weeks (56 days) prior to randomization), corticosteroid (except for subjects on a stable or decreasing dose for ≥ 1 week prior to randomization for medical conditions other than MDS)
* Prior history of cancer, other than MDS, unless the subject has been free of the disease for ≥ 3 years. (Basal or squamous cell carcinoma of the skin, carcinoma in situ of the cervix, carcinoma in situ of the breast, and incidental histologic finding of prostate cancer) (T1a or T1b using the tumor, nodes, metastasis \[TNM\] clinical staging system is allowed)
* Significant active cardiac disease within the previous 6 months
* Uncontrolled systemic fungal, bacterial, or viral infection
* Known Human Immunodeficiency Virus (HIV) or Hepatitis C (HCV) infection, or evidence of active Hepatitis B Virus (HBV) infection
* Known clinically significant anemia due to iron, vitamin B12, or folate deficiencies, or autoimmune or hereditary hemolytic anemia, or gastrointestinal bleeding
* Abnormal coagulation parameters
* Abnormal liver function test results
* Abnormal kidney function test results
* Known or suspected hypersensitivity to azacitidine or mannitol
* Any significant medical condition, laboratory abnormality, or psychiatric illness
18 Years
ALL
No
Sponsors
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Celgene
INDUSTRY
Responsible Party
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Principal Investigators
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Bristol-Myers Squibb
Role: STUDY_DIRECTOR
Bristol-Myers Squibb
Locations
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Alta Bates Comprehensive Cancer Center
Berkeley, California, United States
Tower Hematology/Oncology Medical Group and Tower Cancer Research Found
Beverly Hills, California, United States
City Of Hope
Duarte, California, United States
California Cancer Associates for Research and Excellence cCARE
Escondido, California, United States
Marin Oncology Associates
Greenbrae, California, United States
UCSD-Thornton Hospital
La Jolla, California, United States
University of Southern California Norris Cancer Center
Los Angeles, California, United States
Local Institution - 942
New Haven, Connecticut, United States
University of Florida Health Cancer Center at Orlando Health
Orlando, Florida, United States
Phoebe Cancer Center of Phoebe Putney Memorial Hospital
Albany, Georgia, United States
Robert H Lurie Comprehensive Cancer Center NW Univ
Chicago, Illinois, United States
University Of Illinois At Chicago
Chicago, Illinois, United States
University Of Chicago Medical Center
Chicago, Illinois, United States
Loyola University Chicago
Maywood, Illinois, United States
University Of Kansas Medical Center
Kansas City, Kansas, United States
University of Louisville, J.G. Brown Cancer Center
Louisville, Kentucky, United States
Hematology And Oncology Specialists, Llc
Metairie, Louisiana, United States
Johns Hopkins Medicine
Baltimore, Maryland, United States
UMASS Memorial Hospital
Worcester, Massachusetts, United States
Mayo Clinic
Rochester, Minnesota, United States
Jackson Oncology Associates PLLC
Jackson, Mississippi, United States
Saint Luke's Cancer Institute
Kansas City, Missouri, United States
Kansas City VA Medical Center University of Kansas Medical Center
Kansas City, Missouri, United States
University Of Nebraska Medical Center
Omaha, Nebraska, United States
Weill Cornell Medical College - New York - Presbyterian Hospital
New York, New York, United States
Icahn School of Medicine at Mount Sinai Medical Center
New York, New York, United States
Levine Cancer Institute
Charlotte, North Carolina, United States
Eastern Institute of Medical Sciences
Greenville, North Carolina, United States
University Hospitals of Cleveland Case Medical Center
Cleveland, Ohio, United States
Ohio State University Medical Center
Columbus, Ohio, United States
Local Institution - 917
Portland, Oregon, United States
Penn Medicine: University of Pennsylvania Health System
Philadelphia, Pennsylvania, United States
Western Pennsylvania Cancer Institute
Pittsburgh, Pennsylvania, United States
Brooke Army Medical Center Francis Street Medical Center
Fort Sam Houston, Texas, United States
Local Institution - 900
Houston, Texas, United States
Local Institution - 924
Houston, Texas, United States
Millennium Physicians - Oncology
Houston, Texas, United States
VA Commonwealth University - Massey Cancer Center
Richmond, Virginia, United States
Local Institution - 904
Seattle, Washington, United States
Waukesha Memorial Hospital
Waukesha, Wisconsin, United States
Local Institution - 137
Garran, Australian Capital Territory, Australia
Local Institution - 129
Adelaide, South Australia, Australia
Local Institution - 134
Clayton, Victoria, Australia
Local Institution - 132
Frankston, Victoria, Australia
Local Institution - 131
Camperdown, , Australia
Local Institution - 127
Epping, VIC, , Australia
Local Institution - 133
Fitzroy, , Australia
Local Institution - 135
Kogarah, , Australia
Local Institution - 130
Malvern, , Australia
Local Institution - 126
Milton, Brisbane, , Australia
Local Institution - 138
Waratah, , Australia
Local Institution - 136
Woolloongabba, , Australia
Local Institution - 203
Brasschaat, , Belgium
Local Institution - 200
Bruges, , Belgium
Local Institution - 202
Charleroi, , Belgium
Local Institution - 201
Leuven, , Belgium
Local Institution - 154
Fortaleza, Ceará, Brazil
Local Institution - 155
Curitiba, Paraná, Brazil
Local Institution - 152
Porto Alegre, Rio Grande do Sul, Brazil
Local Institution - 153
Rio de Janeiro, , Brazil
Local Institution - 151
São Paulo, , Brazil
Local Institution - 182
Edmonton, Alberta, Canada
Local Institution - 178
Vancouver, British Columbia, Canada
Local Institution - 183
Barrie, Ontario, Canada
Local Institution - 180
Hamilton, Ontario, Canada
Local Institution - 179
Toronto, Ontario, Canada
Local Institution - 176
Toronto, Ontario, Canada
Local Institution - 181
Montreal, Quebec, Canada
Local Institution - 177
Montreal, Quebec, Canada
Local Institution - 226
Brno, South Moravian, Czechia
Local Institution - 230
Hradec Králové, , Czechia
Local Institution - 229
Olomouc, , Czechia
Local Institution - 228
Prague, , Czechia
Local Institution - 227
Prague, , Czechia
Local Institution - 253
Aarhus, , Denmark
Local Institution - 252
Odense C, , Denmark
Local Institution - 251
Roskilde, , Denmark
Local Institution - 276
Helsinki, , Finland
Local Institution - 277
Turku, , Finland
Local Institution - 305
Lille, , France
Local Institution - 304
Marseille, , France
Local Institution - 301
Nantes, , France
Local Institution - 308
Paris, , France
Local Institution - 307
Pierre-Bénite, , France
Local Institution - 302
Rennes, , France
Local Institution - 303
Rouen, , France
Local Institution - 306
Strasbourg, , France
Local Institution - 300
Toulouse, , France
Local Institution - 309
Tours, , France
Local Institution - 350
Dresden, , Germany
Local Institution - 361
Dresden, , Germany
Local Institution - 355
Düsseldorf, , Germany
Local Institution - 359
Düsseldorf, , Germany
Local Institution - 356
Hamburg, , Germany
Local Institution - 353
Keil, , Germany
Local Institution - 351
Leipzig, , Germany
Local Institution - 352
München, , Germany
Local Institution - 357
Tübingen, , Germany
Local Institution - 360
Ulm, , Germany
Local Institution - 853
Heraklion, Irakleio, Greece
Local Institution - 851
Alexandroupoli, , Greece
Local Institution - 852
Athens, , Greece
Local Institution - 855
Athens, , Greece
Local Institution - 850
Athens, , Greece
Local Institution - 854
Pátrai, , Greece
Local Institution - 452
Tel Aviv, Tel Aviv, Israel
Local Institution - 453
Haifa, , Israel
Local Institution - 451
Petah Tikva, , Israel
Local Institution - 454
Tel Litwinsky, , Israel
Local Institution - 482
Alessandria, , Italy
Local Institution - 492
Bari, , Italy
Local Institution - 489
Bologna, , Italy
Local Institution - 494
Cagliari, , Italy
Local Institution - 486
Florence, , Italy
Local Institution - 479
Lecce, , Italy
Local Institution - 498
Milan, , Italy
Local Institution - 488
Milan, , Italy
Local Institution - 497
Novara, , Italy
Local Institution - 480
Palermo, , Italy
Local Institution - 495
Pavia, , Italy
Local Institution - 496
Pisa, , Italy
Local Institution - 485
Reggio Calabria, , Italy
Local Institution - 484
Rionero in Vulture, , Italy
Local Institution - 487
Roma, , Italy
Local Institution - 490
Roma, , Italy
Local Institution - 481
Roma, , Italy
Local Institution - 499
Terni, , Italy
Local Institution - 477
Torino, , Italy
Local Institution - 493
Torrette Di Ancona, , Italy
Local Institution - 478
Udine, , Italy
Local Institution - 476
Venezia - Mestre, , Italy
Local Institution - 830
Huixquilucan de Degollado, , Mexico
Local Institution - 828
Monterrey, , Mexico
Local Institution - 829
Monterrey, , Mexico
Local Institution - 827
Tlalpan, , Mexico
Local Institution - 528
Amsterdam, , Netherlands
Local Institution - 527
Groningen, , Netherlands
Local Institution - 526
Nijmegen, , Netherlands
Local Institution - 529
Rotterdam, , Netherlands
Local Institution - 552
Førde, , Norway
Local Institution - 551
Oslo, , Norway
Local Institution - 582
Bydgoszcz, , Poland
Local Institution - 576
Gdansk, , Poland
Local Institution - 579
Krakow, , Poland
Local Institution - 581
Lodz, , Poland
Local Institution - 580
Torun, , Poland
Local Institution - 583
Warsaw, , Poland
Local Institution - 577
Warsaw, , Poland
Local Institution - 578
Wroclaw, , Poland
Local Institution - 604
Beja, , Portugal
Local Institution - 600
Coimbra, , Portugal
Local Institution - 601
Lisbon, , Portugal
Local Institution - 603
Lisbon, , Portugal
Local Institution - 602
Porto, , Portugal
Local Institution - 704
Busan, , South Korea
Local Institution - 703
Daegu, , South Korea
Local Institution - 701
Seoul, , South Korea
Local Institution - 702
Seoul, , South Korea
Local Institution - 700
Seoul, , South Korea
Local Institution - 626
Badalona (Barcelona), , Spain
Local Institution - 635
Barcelona, , Spain
Local Institution - 625
Barcelona, , Spain
Local Institution - 636
Granada, , Spain
Local Institution - 629
Madrid, , Spain
Local Institution - 634
Madrid, , Spain
Local Institution - 631
Madrid, , Spain
Local Institution - 632
Málaga, , Spain
Local Institution - 628
Oviedo, , Spain
Local Institution - 630
Salamanca, , Spain
Local Institution - 627
Seville, , Spain
Local Institution - 633
Vitoria-Gasteiz, Álava, , Spain
Local Institution - 653
Gothenburg, , Sweden
Local Institution - 652
Lund, , Sweden
Local Institution - 651
Stockholm, , Sweden
Local Institution - 401
Adana, , Turkey (Türkiye)
Local Institution - 405
Antalya, , Turkey (Türkiye)
Local Institution - 403
Istanbul, , Turkey (Türkiye)
Local Institution - 400
Istanbul, , Turkey (Türkiye)
Local Institution - 404
Mersin, , Turkey (Türkiye)
Local Institution - 402
Trabzon, , Turkey (Türkiye)
Local Institution - 685
Oxford, Oxfordshire, United Kingdom
Local Institution - 687
Aberdeen, , United Kingdom
Local Institution - 691
Birmingham, , United Kingdom
Local Institution - 686
Bristol, , United Kingdom
Local Institution - 689
Cambridge, , United Kingdom
Local Institution - 678
Cardiff, , United Kingdom
Local Institution - 688
Harrow Middlesex, , United Kingdom
Local Institution - 692
Hull, , United Kingdom
Local Institution - 677
Leicester, , United Kingdom
Local Institution - 683
Liverpool, , United Kingdom
Local Institution - 676
London, , United Kingdom
Local Institution - 684
London, , United Kingdom
Local Institution - 679
Manchester, , United Kingdom
Local Institution - 681
Nottingham, , United Kingdom
Local Institution - 690
Sutton in Ashfield, , United Kingdom
Local Institution - 680
Wolverhampton, , United Kingdom
Countries
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References
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Garcia-Manero G, Almeida A, Giagounidis A, Platzbecker U, Garcia R, Voso MT, Larsen SR, Valcarcel D, Silverman LR, Skikne B, Santini V. Design and rationale of the QUAZAR Lower-Risk MDS (AZA-MDS-003) trial: a randomized phase 3 study of CC-486 (oral azacitidine) plus best supportive care vs placebo plus best supportive care in patients with IPSS lower-risk myelodysplastic syndromes and poor prognosis due to red blood cell transfusion-dependent anemia and thrombocytopenia. BMC Hematol. 2016 May 3;16:12. doi: 10.1186/s12878-016-0049-5. eCollection 2016.
Garcia-Manero G, Santini V, Almeida A, Platzbecker U, Jonasova A, Silverman LR, Falantes J, Reda G, Buccisano F, Fenaux P, Buckstein R, Diez Campelo M, Larsen S, Valcarcel D, Vyas P, Giai V, Oliva EN, Shortt J, Niederwieser D, Mittelman M, Fianchi L, La Torre I, Zhong J, Laille E, Lopes de Menezes D, Skikne B, Beach CL, Giagounidis A. Phase III, Randomized, Placebo-Controlled Trial of CC-486 (Oral Azacitidine) in Patients With Lower-Risk Myelodysplastic Syndromes. J Clin Oncol. 2021 May 1;39(13):1426-1436. doi: 10.1200/JCO.20.02619. Epub 2021 Mar 25.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Related Links
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BMS Clinical Trial Information
BMS Clinical Trial Patient Recruiting
Other Identifiers
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2012-002471-34
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
AZA-MDS-003
Identifier Type: -
Identifier Source: org_study_id
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