Trial Review

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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT01566695




Registration number
NCT01566695
Ethics application status
Date submitted
27/03/2012
Date registered
29/03/2012
Date last updated
26/01/2024

Titles & IDs
Public title
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)
Scientific title
A Phase 3, Multicenter, Randomized, Double-blind Study to Compare the Efficacy and Safety of Oral Azacitidine Plus Best Supportive Care Versus Placebo Plus Best Supportive Care in Subjects With Red Blood Cell Transfusion-dependent Anemia and Thrombocytopenia Due to IPSS Lower-risk Myelodysplastic Syndromes.
Secondary ID [1] 0 0
2012-002471-34
Secondary ID [2] 0 0
AZA-MDS-003
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Myelodysplastic Syndrome 0 0
Condition category
Condition code
Blood 0 0 0 0
Haematological diseases
Blood 0 0 0 0
Other blood disorders
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Oral Azacitidine
Treatment: Drugs - Placebo
Other interventions - Best Supportiv Care (BSC)

Experimental: 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.

Placebo Comparator: Placebo - Arm 2: Identically matching placebo tablets plus best supportive care on days 1 to 21 of each 28-day treatment cycle.


Treatment: Drugs: Oral Azacitidine
300 mg daily, days 1 to 21 of each 28-day treatment cycle

Treatment: Drugs: Placebo
Identically matching placebo tablets on day 1 to 21 of each 28-day treatment cycle.

Other interventions: 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.
Intervention code [1] 0 0
Treatment: Drugs
Intervention code [2] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Percentage of Participants Who Achieved Red Blood Cell (RBC) Transfusion Independence for = 56 Days
Timepoint [1] 0 0
Each participant was assessed for at least 56 days or more; from the date of randomization of study drug up to the data cut-off date of 25 January 2019, approximately 5 months.
Secondary outcome [1] 0 0
Duration of RBC Transfusion Independence Among Participants Who Achieved RBC Transfusion Independence for at Least 56 Days
Timepoint [1] 0 0
From the date of randomization of study drug up to the data cut-off date of 25 January 2019; median duration of treatment to oral azacitidine was 5.29 months and 5.36 months for placebo
Secondary outcome [2] 0 0
Time to RBC Transfusion Independence for at Least 56 Days Among Participants Who Achieved RBC Transfusion Independence for at Least 56 Days
Timepoint [2] 0 0
From the date of randomization of study drug up to the data cut-off date of 25 January 2019; median duration of treatment to oral azacitidine was 5.29 months and 5.36 months for placebo
Secondary outcome [3] 0 0
Duration of RBC Transfusion Reduction for Participants Who Achieved RBC Transfusion Reduction of at Least 4 Units of RBCs for at Least 8 Weeks
Timepoint [3] 0 0
From the date of randomization of study drug up to the treatment period; up to the data cut-off date of 25 January 2019; median duration of treatment to oral azacitidine was 5.29 months and 5.36 months for placebo
Secondary outcome [4] 0 0
Percentage of Participants Who Achieved Red Blood Cell Transfusion Independence for = 84 Days
Timepoint [4] 0 0
From the date of randomization of study drug up to the treatment period; up to the data cut-off date of 25 January 2019; median duration of treatment to oral azacitidine was 5.29 months and 5.36 months for placebo
Secondary outcome [5] 0 0
Duration of RBC Transfusion Independence Among Participants Who Achieved RBC Transfusion Independence for at Least 84 Days
Timepoint [5] 0 0
From the date of randomization of study drug up to the treatment period; up to the data cut-off date of 25 January 2019; median duration of treatment to oral azacitidine was 5.29 months and 5.36 months for placebo
Secondary outcome [6] 0 0
Time to RBC Transfusion Independence for at Least 84 Days Among Participants Who Achieved RBC Transfusion Independence for at Least 84 Days
Timepoint [6] 0 0
From the date of randomization of study drug up to the treatment period; up to the data cut-off date of 25 January 2019; median duration of treatment to oral azacitidine was 5.29 months and 5.36 months for placebo
Secondary outcome [7] 0 0
Percentage of Participants With an Erythroid Hematological Improvement (HI-E) Response According to 2006 IWG Criteria
Timepoint [7] 0 0
From the date of randomization of study drug up to the treatment period; up to the data cut-off date of 25 January 2019; median duration of treatment to oral azacitidine was 5.29 months and 5.36 months for placebo
Secondary outcome [8] 0 0
Percentage of Participants With a Hematological Improvement Response in Platelets (HI-P) According to 2006 IWG Criteria
Timepoint [8] 0 0
From the date of randomization of study drug up to the treatment period; up to the data cut-off date of 25 January 2019; median duration of treatment to oral azacitidine was 5.29 months and 5.36 months for placebo
Secondary outcome [9] 0 0
Percentage of Participants Who Achieved Platelet Transfusion Independence With a Duration of = 8 Weeks (56 Days)
Timepoint [9] 0 0
From the date of randomization of study drug up to the treatment period; up to the data cut-off date of 25 January 2019; median duration of treatment to oral azacitidine was 5.29 months and 5.36 months for placebo
Secondary outcome [10] 0 0
Time to Platelet Transfusion Independence
Timepoint [10] 0 0
From the date of randomization of study drug up to the treatment period; up to the data cut-off date of 25 January 2019; median duration of treatment to oral azacitidine was 5.29 months and 5.36 months for placebo
Secondary outcome [11] 0 0
Kaplan-Meier Estimate of Overall Survival (OS)
Timepoint [11] 0 0
From randomization to death from any cause; up to the data cut-off of date of 25 January 2019; maximum follow-up time for all participants was 67.9 months for the oral azacitidine arm and 64.8 months for placebo arm
Secondary outcome [12] 0 0
Percentage of Participants With a Hematologic Response According to the 2006 IWG Criteria for MDS
Timepoint [12] 0 0
Response was assessed every 3 cycles; up to the data cut-off date of 25 Jan 2019; median duration of exposure to oral azacitidine was 86.0 days and 119.0 days for placebo
Secondary outcome [13] 0 0
Percentage of Participants Who Progressed to Acute Myeloid Leukemia (AML)
Timepoint [13] 0 0
From randomization of study drug to the end up to final data cut-off date of 25 January 2019; maximum follow-up time was 67.9 months for azacitidine and 64.8 months for placebo group
Secondary outcome [14] 0 0
Time to Progression to Acute Myeloid Leukemia (AML) Among Participants Who Progressed to AML
Timepoint [14] 0 0
From randomization of study drug to progression of AML; up to final data cut-off date of 25 January 2019; median duration of treatment to oral azacitidine was 5.29 months and 5.36 months for placebo
Secondary outcome [15] 0 0
Percentage of Participants With Significant Bleeding Events
Timepoint [15] 0 0
From date of randomization until 28 days after the last dose of IP; up to data cut off date of 25 January 2019; median duration of treatment to oral azacitidine was 5.29 months and 5.36 months for placebo
Secondary outcome [16] 0 0
Number of Participants With Treatment Emergent Adverse Events (TEAE)
Timepoint [16] 0 0
From first dose of IP up to 28 days after the last dose of IP; up to data cut-off date of 25 Jan 2019; median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Secondary outcome [17] 0 0
Mean Change From Baseline in the Physical Well-Being Component of the Functional Assessment of Cancer Therapy-Anemia (FACT-An) Endpoints at Cycle 6
Timepoint [17] 0 0
Baseline to Cycle 6 Day 1
Secondary outcome [18] 0 0
Mean Change From Baseline in the Social Well-Being Component of the Functional Assessment of Cancer Therapy-Anemia Instrument at Cycle 6
Timepoint [18] 0 0
Baseline to Cycle 6 Day 1
Secondary outcome [19] 0 0
Mean Change From Baseline in the Emotional Well-Being Component of the Functional Assessment of Cancer Therapy-Anemia Instrument at Cycle 6
Timepoint [19] 0 0
Baseline to Cycle 6 Day 1
Secondary outcome [20] 0 0
Mean Change From Baseline in the Functional Well-Being Component of the FACT-An Instrument at Cycle 6
Timepoint [20] 0 0
Baseline to Cycle 6 Day 1
Secondary outcome [21] 0 0
Mean Change From Baseline in the Anemia Subscale Within FACT-An Instrument at Cycle 6
Timepoint [21] 0 0
Baseline to Cycle 6 Day 1
Secondary outcome [22] 0 0
Mean Change From Baseline in the Fatigue-Related Subscale Within the FACT-An Instrument at Cycle 6
Timepoint [22] 0 0
Baseline to Cycle 6 Day 1
Secondary outcome [23] 0 0
Mean Change From Baseline in the Functional Assessment of Cancer Therapy-Anemia Trial Outcome Index (FACT-An TOI) Summary Scale Within the FACT-An Instrument at Cycle 6
Timepoint [23] 0 0
Baseline to Cycle 6 Day 1
Secondary outcome [24] 0 0
Mean Change From Baseline in the Functional Assessment of Cancer Therapy-Anemia-General (FACT-G) Summary Scale Within the FACT-An Instrument at Cycle 6
Timepoint [24] 0 0
Baseline to Cycle 6 Day 1
Secondary outcome [25] 0 0
Mean Change From Baseline in the Functional Assessment of Cancer Therapy-Anemia-Total Score at Cycle 6
Timepoint [25] 0 0
Baseline to Cycle 6 Day 1
Secondary outcome [26] 0 0
Percentage of Participants With a Clinically Meaningful Improvment (CMI) From Baseline on the Physical Well-Being Domain Within the FACT-An Instrument at Cycle 6
Timepoint [26] 0 0
Cycle 6 Day 1
Secondary outcome [27] 0 0
Percentage of Participants With a Clinically Meaningful Improvment (CMI) From Baseline on the Social Well-Being Domain Within the FACT-An Instrument at Cycle 6
Timepoint [27] 0 0
Cycle 6 Day 1
Secondary outcome [28] 0 0
Percentage of Participants With a Clinically Meaningful Improvment (CMI) From Baseline on the Emotional Well-Being Domain Within the FACT-An Instrument at Cycle 6
Timepoint [28] 0 0
Cycle 6 Day 1
Secondary outcome [29] 0 0
Percentage of Participants With a Clinically Meaningful Improvment (CMI) From Baseline on the Functional Well-Being Domain Within the FACT-An Instrument at Cycle 6
Timepoint [29] 0 0
Cycle 6 Day 1
Secondary outcome [30] 0 0
Percentage of Participants With a Clinically Meaningful Improvment (CMI) From Baseline on the Anemia Subscale Domain Within the FACT-An Instrument at Cycle 6
Timepoint [30] 0 0
Cycle 6 Day 1
Secondary outcome [31] 0 0
Percentage of Participants With a Clinically Meaningful Improvment (CMI) From Baseline in the Fatigue Related Symptoms Subscale Domain Within the FACT-An Instrument at Cycle 6
Timepoint [31] 0 0
Cycle 6 Day 1
Secondary outcome [32] 0 0
Percentage of Participants With a Clinically Meaningful Improvment (CMI) From Baseline in the Functional Assessment of Cancer Therapy-Anemia Trial Outcome Index Subscale Domain Within the FACT-An Instrument at Cycle 6
Timepoint [32] 0 0
Cycle 6 Day 1
Secondary outcome [33] 0 0
Percentage of Participants With a Clinically Meaningful Improvment (CMI) From Baseline in the Functional Assessment of Cancer Therapy-Anemia-General Subscale Domain Within the FACT-An Instrument at Cycle 6
Timepoint [33] 0 0
Cycle 6 Day 1
Secondary outcome [34] 0 0
Percentage of Participants With a Clinically Meaningful Improvement (CMI) From Baseline in the Functional Assessment of Cancer Therapy Anemia-Total Score Domain Within the FACT-An Instrument at Cycle 6
Timepoint [34] 0 0
Cycle 6 Day 1
Secondary outcome [35] 0 0
Percentage of Participants With Change From Baseline in Responses to the Fact-Anemia Item GP-5
Timepoint [35] 0 0
From Baseline to Cycle 2 Day 1 (C2D1)
Secondary outcome [36] 0 0
Percentage of Participants With Change From Baseline in Responses to the Fact-Anemia Item GP-5
Timepoint [36] 0 0
From Baseline to Cycle 3 Day 1 (C3D1)
Secondary outcome [37] 0 0
Percentage of Participants With Change From Baseline in Responses to the Fact-Anemia Item GP-5
Timepoint [37] 0 0
From Baseline to Cycle 4 Day 1 (C4D1)
Secondary outcome [38] 0 0
Percentage of Participants With Change From Baseline in Responses to the Fact-Anemia Item GP-5
Timepoint [38] 0 0
From Baseline to Cycle 5 Day 1 (C5D1)
Secondary outcome [39] 0 0
Percentage of Participants With Change From Baseline in Responses to the Fact-Anemia Item GP-5
Timepoint [39] 0 0
From Baseline to Cycle 6 Day 1 (C6 D1)
Secondary outcome [40] 0 0
Percentage of Participants With Change From Baseline in Responses to the Fact-Anemia Item GP-5
Timepoint [40] 0 0
From Baseline to Cycle 7 Day 1 (C7D1)
Secondary outcome [41] 0 0
Percentage of Participants With Change From Baseline in Responses to the Fact-Anemia Item GP-5
Timepoint [41] 0 0
From Baseline to End of Treatment
Secondary outcome [42] 0 0
Percentage of Participants With Improved, Worsened, or No Change in the European Quality of Life-Five Dimension-Three Level (EQ-5D-3L) Mobility Dimension Responses at Cycle 6
Timepoint [42] 0 0
From Baseline to Cycle 6 Day 1
Secondary outcome [43] 0 0
Percentage of Participants With Improved, Worsened, or No Change in the European Quality of Life-Five Dimension-Three Level of Self-Care Dimension Responses at Cycle 6
Timepoint [43] 0 0
From Baseline to Cycle 6 Day 1
Secondary outcome [44] 0 0
Percentage of Participants With Improved, Worsened, or No Change in the European Quality of Life-Five Dimension-Three Level Usual Activities Dimension Responses at Cycle 6
Timepoint [44] 0 0
From Baseline to Cycle 6 Day 1
Secondary outcome [45] 0 0
Percentage of Participants With Improved, Worsened, or No Change in the European Quality of Life-Five Dimension-Three Level in the Pain/Discomfort Dimension Responses at Cycle 6
Timepoint [45] 0 0
From Baseline to Cycle 6 Day 1
Secondary outcome [46] 0 0
Percentage of Participants With Improved, Worsened, or No Change in the European Quality of Life-Five Dimension-Three Level in the Anxiety/Depression Dimension Responses at Cycle 6
Timepoint [46] 0 0
From Baseline to Cycle 6 Day 1
Secondary outcome [47] 0 0
Healthcare Resource Utilization (HRU): Number of Participants Who Were Hospitalized During the Treatment Period
Timepoint [47] 0 0
From date of randomization up to 28 days after the last dose of study drug; up to data cut off date of 25 January 2019; median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Secondary outcome [48] 0 0
Healthcare Resource Utilization (HRU): Total Number of Days Hospitalized Due to Any Reason During the Treatment Period
Timepoint [48] 0 0
From date of randomization up to 28 days after the last dose of study drug; up to data cut off date of 25 January 2019; median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo
Secondary outcome [49] 0 0
Healthcare Resource Utilization (HRU): Total Number of Days Hospitalized Per Total Patient-Years
Timepoint [49] 0 0
From date of randomization up to 28 days after the last dose of study drug; up to data cut off date of 25 January 2019; median duration of treatment to oral azacitaidine was 5.29 months and 5.36 months for placebo

Eligibility
Key inclusion criteria
- 18 years or older

- 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
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Secondary or hypoplastic MDS or other subtype with eligibility for treatment with
immunotherapy

- 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
26/04/2013
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
21/12/2023
Sample size
Target
Accrual to date
Final
216
Recruitment in Australia
Recruitment state(s)
ACT,SA,VIC
Recruitment hospital [1] 0 0
Local Institution - 137 - Garran
Recruitment hospital [2] 0 0
Local Institution - 129 - Adelaide
Recruitment hospital [3] 0 0
Local Institution - 134 - Clayton
Recruitment hospital [4] 0 0
Local Institution - 132 - Frankston
Recruitment hospital [5] 0 0
Local Institution - 131 - Camperdown
Recruitment hospital [6] 0 0
Local Institution - 127 - Epping, VIC
Recruitment hospital [7] 0 0
Local Institution - 133 - Fitzroy
Recruitment hospital [8] 0 0
Local Institution - 135 - Kogarah
Recruitment hospital [9] 0 0
Local Institution - 130 - Malvern
Recruitment hospital [10] 0 0
Local Institution - 126 - Milton, Brisbane
Recruitment hospital [11] 0 0
Local Institution - 138 - Waratah
Recruitment hospital [12] 0 0
Local Institution - 136 - Woolloongabba
Recruitment postcode(s) [1] 0 0
2605 - Garran
Recruitment postcode(s) [2] 0 0
SA 5000 - Adelaide
Recruitment postcode(s) [3] 0 0
3168 - Clayton
Recruitment postcode(s) [4] 0 0
3199 - Frankston
Recruitment postcode(s) [5] 0 0
2050 - Camperdown
Recruitment postcode(s) [6] 0 0
3076 - Epping, VIC
Recruitment postcode(s) [7] 0 0
3065 - Fitzroy
Recruitment postcode(s) [8] 0 0
2217 - Kogarah
Recruitment postcode(s) [9] 0 0
3144 - Malvern
Recruitment postcode(s) [10] 0 0
4064 - Milton, Brisbane
Recruitment postcode(s) [11] 0 0
NSW - Waratah
Recruitment postcode(s) [12] 0 0
QLD 4102 - Woolloongabba
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Connecticut
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United States of America
State/province [3] 0 0
Florida
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United States of America
State/province [4] 0 0
Georgia
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United States of America
State/province [5] 0 0
Illinois
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United States of America
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Kansas
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United States of America
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Kentucky
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United States of America
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Louisiana
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United States of America
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Maryland
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Massachusetts
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Minnesota
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United States of America
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Mississippi
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United States of America
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Missouri
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United States of America
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Nebraska
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United States of America
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New York
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United States of America
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North Carolina
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United States of America
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Ohio
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Oregon
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United States of America
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Pennsylvania
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Texas
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Virginia
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United States of America
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Washington
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United States of America
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Wisconsin
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Belgium
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Brasschaat
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Belgium
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Brugge
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Belgium
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Charleroi
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Belgium
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Leuven
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Brazil
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Ceará
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Brazil
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Paraná
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Brazil
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Rio Grande Do Sul
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Brazil
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Rio De Janeiro
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Brazil
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Sao Paulo
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Canada
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Alberta
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Canada
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British Columbia
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Canada
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Ontario
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Canada
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Quebec
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Czechia
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Jihomoravský Kraj
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Czechia
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Hradec Králové
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Czechia
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Olomouc
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Czechia
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Praha
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Denmark
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Aarhus
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Denmark
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Odense C
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Denmark
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Roskilde
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Finland
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Helsinki
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Turku
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Lille
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Marseille Cedex 9
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France
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Nantes
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Paris
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France
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Pierre-Bénite Cedex
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Rennes Cedex
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Rouen Cedex
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Strasbourg
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France
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Toulouse Cedex 9
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Tours
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Germany
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Dresden
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Duesseldorf
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Germany
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Dusseldorf
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Germany
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Hamburg
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Germany
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Keil
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Germany
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Leipzig
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Germany
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München
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Germany
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Tubingen
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Germany
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Ulm
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Greece
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Irakleio
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Greece
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Alexandroupolis
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Greece
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Athens
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Greece
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Patras
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Israel
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Tel Aviv
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Israel
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Haifa
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Israel
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Petach Tikva
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Israel
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Tel Hashomer
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Italy
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Alessandria
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Italy
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Bari
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Italy
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Bologna
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Italy
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Cagliari
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Italy
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Firenze
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Italy
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Lecce
Country [79] 0 0
Italy
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Celgene
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Evaluation of 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) lower risk
myelodysplastic syndromes (MDS).
Trial website
https://clinicaltrials.gov/ct2/show/NCT01566695
Trial related presentations / publications
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.
Public notes

Contacts
Principal investigator
Name 0 0
Bristol-Myers Squibb
Address 0 0
Bristol-Myers Squibb
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT01566695