Trial Outcomes & Findings for Study of Azacitidine to Evaluate Safety and Effectiveness for Chinese Patients With Higher Risk Myelodysplastic Syndrome (NCT NCT01599325)

Last Updated: 2019-03-19

Results Overview

Hematologic Response is defined by those participants who experienced a Complete Response, Partial Response and Stable Disease (SD) based on IWG 2000 response criteria for MDS. CR = repeat bone marrow (BM) shows \<5% myeloblasts, and peripheral blood values lasting ≥ 2 months of hemoglobin (hgb) (\>110 g/L), neutrophils (≥1.5x10\^9/L), platelets (≥100x10\^9/L), blasts (0%) and no dysplasia • PR = same as CR for peripheral blood: BM shows blasts decrease by ≥ 50% or a less advanced FAB classification from pretreatment • Stable disease (SD): failure to achieve a PR, no evidence of progression for at least 2 months. • Failure: death during treatment or disease progression • Relapse After CR or PR: return to pretreatment BM blast percent or decrement of ≥ 50% from remission/response levels in granulocytes or platelets; or reduction in hgb by ≥20 g/L or transfusion dependence • Disease Progression: change in blast levels • Disease Transformation to Acute Myelogenous Leukemia.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

72 participants

Primary outcome timeframe

Response initially assessed at end of cycle 6, then every 4 cycles; Up to 29 January 2015; 894 days

Results posted on

2019-03-19

Participant Flow

Participant milestones

Participant milestones
Measure	Azacitidine Azacitidine 75 mg/m\^2/day subcutaneously (SC) for 7 days every 28 days until disease progression (DP), adverse events (AE), withdrawal of consent from further treatment, withdrawal of consent, death, lost to follow-up, or protocol violation.
Primary Parent Phase STARTED	72
Primary Parent Phase Intent to Treat (ITT) Population	72
Primary Parent Phase Pharmacokinetic (PK) Population	12
Primary Parent Phase COMPLETED	15
Primary Parent Phase NOT COMPLETED	57
Extension Phase STARTED	15
Extension Phase COMPLETED	0
Extension Phase NOT COMPLETED	15

Reasons for withdrawal

Reasons for withdrawal
Measure	Azacitidine Azacitidine 75 mg/m\^2/day subcutaneously (SC) for 7 days every 28 days until disease progression (DP), adverse events (AE), withdrawal of consent from further treatment, withdrawal of consent, death, lost to follow-up, or protocol violation.
Primary Parent Phase Adverse Event	5
Primary Parent Phase Disease Progression	7
Primary Parent Phase Withdrawal by Subject	20
Primary Parent Phase Death	13
Primary Parent Phase Protocol Violation	1
Primary Parent Phase Lack of Therapeutic Effect	9
Primary Parent Phase Other	2
Extension Phase Study Closure	1
Extension Phase Stopped to Receive Stem Cell Transplant	1
Extension Phase Lack of Therapeutic Effect	4
Extension Phase Disease Progression	1
Extension Phase Withdrawal by Subject	8

Baseline Characteristics

Study of Azacitidine to Evaluate Safety and Effectiveness for Chinese Patients With Higher Risk Myelodysplastic Syndrome

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Azacitidine n=72 Participants Azacitidine 75 mg/m\^2/day subcutaneously (SC) for 7 days every 28 days until disease progression (DP), adverse events (AE), withdrawal of consent from further treatment, withdrawal of consent, death, lost to follow-up, or protocol violation.
Age, Continuous	54.3 years STANDARD_DEVIATION 11.78 • n=5 Participants
Sex: Female, Male Female	34 Participants n=5 Participants
Sex: Female, Male Male	38 Participants n=5 Participants
International Prognostic Scoring System (IPSS) Risk Classification Low (0)	0 Participants n=5 Participants
International Prognostic Scoring System (IPSS) Risk Classification Intermediate 1 (0.5-1.0)	0 Participants n=5 Participants
International Prognostic Scoring System (IPSS) Risk Classification Intermediate 2 (1.5-2.0)	47 Participants n=5 Participants
International Prognostic Scoring System (IPSS) Risk Classification High (>=2.5)	25 Participants n=5 Participants
French-American-British classification (FAB) RAEB=Refractory anemia with excess blasts	63 Participants n=5 Participants
French-American-British classification (FAB) RAEB in transformation	8 Participants n=5 Participants
French-American-British classification (FAB) Modified CMML=Chronic myelomonocytic leukemia	1 Participants n=5 Participants
MDS World Health Organization (WHO) Classification RAEB-1=Refractory anemia with excess blasts	14 Participants n=5 Participants
MDS World Health Organization (WHO) Classification RAEB -2 with MPD=Myeloproliferative disease	50 Participants n=5 Participants
MDS World Health Organization (WHO) Classification Acute Myelogenous Leukemia (AML)	7 Participants n=5 Participants
MDS World Health Organization (WHO) Classification CMML	1 Participants n=5 Participants
MDS World Health Organization (WHO) Classification MDS and myeloproliferative disease (MPD)	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: Response initially assessed at end of cycle 6, then every 4 cycles; Up to 29 January 2015; 894 days

Population: Intent to Treat (ITT) includes all participants who were enrolled into the study.

Outcome measures

Outcome measures
Measure	Azacitidine n=72 Participants Azacitidine 75 mg/m\^2/day subcutaneously (SC) for 7 days every 28 days until disease progression (DP), adverse events (AE), withdrawal of consent from further treatment, withdrawal of consent, death, lost to follow-up, or protocol violation.
Percentage of Participants With a Hematologic Response Based on the International Working Group (IWG) Criteria for Myelodysplastic Syndrome (MDS) and Programmatically Assessed by the Sponsor Using Clinically Relevant Data. Overall CR+PR	1.4 percentage of participants Interval 0.0 to 7.5
Percentage of Participants With a Hematologic Response Based on the International Working Group (IWG) Criteria for Myelodysplastic Syndrome (MDS) and Programmatically Assessed by the Sponsor Using Clinically Relevant Data. Overall CR+PR+SD	95.8 percentage of participants Interval 88.3 to 99.1
Percentage of Participants With a Hematologic Response Based on the International Working Group (IWG) Criteria for Myelodysplastic Syndrome (MDS) and Programmatically Assessed by the Sponsor Using Clinically Relevant Data. Complete Remission (CR)	1.4 percentage of participants Interval 0.0 to 7.5
Percentage of Participants With a Hematologic Response Based on the International Working Group (IWG) Criteria for Myelodysplastic Syndrome (MDS) and Programmatically Assessed by the Sponsor Using Clinically Relevant Data. Partial Remission (PR)	0 percentage of participants Interval 0.0 to 5.0

PRIMARY outcome

Timeframe: Response initially assessed at end of cycle 6, then every 4 cycles; Up to 29 January 2015; 894 days

Population: Intent to Treat (ITT) includes all participants who were enrolled into the study.

Outcome measures

Outcome measures
Measure	Azacitidine n=72 Participants Azacitidine 75 mg/m\^2/day subcutaneously (SC) for 7 days every 28 days until disease progression (DP), adverse events (AE), withdrawal of consent from further treatment, withdrawal of consent, death, lost to follow-up, or protocol violation.
Percentage of Participants With a Hematologic Response Using IWG Criteria for MDS and Assessed by the Investigator Overall CR+PR	12.5 percentage of participants Interval 5.9 to 22.4
Percentage of Participants With a Hematologic Response Using IWG Criteria for MDS and Assessed by the Investigator Overall CR+PR+SD	70.8 percentage of participants Interval 58.9 to 81.0
Percentage of Participants With a Hematologic Response Using IWG Criteria for MDS and Assessed by the Investigator CR	6.9 percentage of participants Interval 2.3 to 15.5
Percentage of Participants With a Hematologic Response Using IWG Criteria for MDS and Assessed by the Investigator PR	5.6 percentage of participants Interval 1.5 to 13.6

PRIMARY outcome

Timeframe: Up to 29 January 2015; 894 days

Population: Intent to Treat (ITT) includes all participants who were enrolled into the study.

Hematologic improvements (HI) have 4 categories: 1. Erythroid response (HI-E): Major \>20g/L increase or transfusion independent. Minor: 10-20g/L increase or ≥50% decrease in transfusion requirements. 2. Platelet response (HI-P): Major absolute increase of ≥30x10\^9/L or platelet transfusion independence. Minor: ≥50% increase. 3. Neutrophil response (HI-N): Major 100% increase or an absolute increase of \>0.5x10\^9/L. Minor: ≥100% increase and absolute increase of \<0.5x10\^9/L 4. Progression or relapse after HI Hematological improvement (HI) was defined as any type (major or minor) of improvement of HI-E, HI-P, or HI-N. 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. Sponsor's determination was derived using clinically relevant data. Denominator for progression/relapse after HI included participants who had achieved HI.

Outcome measures

Outcome measures
Measure	Azacitidine n=72 Participants Azacitidine 75 mg/m\^2/day subcutaneously (SC) for 7 days every 28 days until disease progression (DP), adverse events (AE), withdrawal of consent from further treatment, withdrawal of consent, death, lost to follow-up, or protocol violation.
Percentage of Participants Achieving a Hematologic Improvement (HI) Based on 2000 IWG Response Criteria for MDS and Programmatically Assessed by the Sponsor Using Clinically Relevant Data. Hematologic Improvement-P (minor)	0.0 percentage of participants Interval 0.0 to 6.2
Percentage of Participants Achieving a Hematologic Improvement (HI) Based on 2000 IWG Response Criteria for MDS and Programmatically Assessed by the Sponsor Using Clinically Relevant Data. Hematologic Improvement-N (major)	21.8 percentage of participants Interval 11.8 to 35.0
Percentage of Participants Achieving a Hematologic Improvement (HI) Based on 2000 IWG Response Criteria for MDS and Programmatically Assessed by the Sponsor Using Clinically Relevant Data. Any improvement	52.8 percentage of participants Interval 40.7 to 64.7
Percentage of Participants Achieving a Hematologic Improvement (HI) Based on 2000 IWG Response Criteria for MDS and Programmatically Assessed by the Sponsor Using Clinically Relevant Data. Hematologic Improvement-E (major)	35.8 percentage of participants Interval 24.5 to 48.5
Percentage of Participants Achieving a Hematologic Improvement (HI) Based on 2000 IWG Response Criteria for MDS and Programmatically Assessed by the Sponsor Using Clinically Relevant Data. Hematologic Improvement-E (minor)	9.0 percentage of participants Interval 3.4 to 18.5
Percentage of Participants Achieving a Hematologic Improvement (HI) Based on 2000 IWG Response Criteria for MDS and Programmatically Assessed by the Sponsor Using Clinically Relevant Data. Hematologic Improvement-P (major)	37.9 percentage of participants Interval 25.5 to 51.6
Percentage of Participants Achieving a Hematologic Improvement (HI) Based on 2000 IWG Response Criteria for MDS and Programmatically Assessed by the Sponsor Using Clinically Relevant Data. Hematologic Improvement-N (minor)	0.0 percentage of participants Interval 0.0 to 6.5

SECONDARY outcome

Timeframe: Up to cycle 27; The on-treatment period was considered the period from the date of first dose to the last treatment study visit; Up to 29 January 2015; 894 days

Population: Intent to Treat (ITT) includes all participants who were enrolled into the study.

The number of units of platelet transfusions received 56 days prior to treatment, considered the baseline period, (baseline period defined as the screening period before the date of the first dose of azacitidine; any laboratory and blood transfusion data reported on the day of the first dose of azacitidine was considered to be baseline data) and during study was standardized per 28 days and summarized by cycle for platelets. The formula for standardizing per 28 days was: \[(# of transfusions in the measurement period / length of the measurement period (days)) x 28\], where the measurement period was either baseline or the relevant cycle length.

Outcome measures

Outcome measures
Measure	Azacitidine n=72 Participants Azacitidine 75 mg/m\^2/day subcutaneously (SC) for 7 days every 28 days until disease progression (DP), adverse events (AE), withdrawal of consent from further treatment, withdrawal of consent, death, lost to follow-up, or protocol violation.
The Number of Units of Platelet Transfusions by Cycle Cycle 23	0.0 Units of platelet transfusions Standard Deviation NA Not applicable = Null value
The Number of Units of Platelet Transfusions by Cycle Cycle 25	0.0 Units of platelet transfusions Standard Deviation NA Not applicable = Null value
The Number of Units of Platelet Transfusions by Cycle Baseline N = 72	1.9 Units of platelet transfusions Standard Deviation 4.91
The Number of Units of Platelet Transfusions by Cycle Overall Change from Baseline N = 72	2.8 Units of platelet transfusions Standard Deviation 17.89
The Number of Units of Platelet Transfusions by Cycle Cycle 1 N = 72	5.2 Units of platelet transfusions Standard Deviation 18.83
The Number of Units of Platelet Transfusions by Cycle Cycle 2	2.4 Units of platelet transfusions Standard Deviation 5.86
The Number of Units of Platelet Transfusions by Cycle Cycle 5	2.5 Units of platelet transfusions Standard Deviation 7.69
The Number of Units of Platelet Transfusions by Cycle Cycle 14	0.1 Units of platelet transfusions Standard Deviation 0.35
The Number of Units of Platelet Transfusions by Cycle Cycle 20	0.2 Units of platelet transfusions Standard Deviation 0.37
The Number of Units of Platelet Transfusions by Cycle Cycle 21	0.0 Units of platelet transfusions Standard Deviation 0.00
The Number of Units of Platelet Transfusions by Cycle Cycle 4	2.7 Units of platelet transfusions Standard Deviation 6.13
The Number of Units of Platelet Transfusions by Cycle Cycle 6	1.5 Units of platelet transfusions Standard Deviation 4.15
The Number of Units of Platelet Transfusions by Cycle Cycle 7	1.0 Units of platelet transfusions Standard Deviation 2.84
The Number of Units of Platelet Transfusions by Cycle Cycle 8	1.1 Units of platelet transfusions Standard Deviation 2.05
The Number of Units of Platelet Transfusions by Cycle Cycle 9	0.8 Units of platelet transfusions Standard Deviation 2.46
The Number of Units of Platelet Transfusions by Cycle Cycle 10	0.8 Units of platelet transfusions Standard Deviation 2.08
The Number of Units of Platelet Transfusions by Cycle Cycle 11	0.5 Units of platelet transfusions Standard Deviation 1.00
The Number of Units of Platelet Transfusions by Cycle Cycle 12	0.3 Units of platelet transfusions Standard Deviation 0.78
The Number of Units of Platelet Transfusions by Cycle Cycle 13	0.3 Units of platelet transfusions Standard Deviation 0.72
The Number of Units of Platelet Transfusions by Cycle Cycle 15	0.3 Units of platelet transfusions Standard Deviation 0.90
The Number of Units of Platelet Transfusions by Cycle Cycle 16	0.3 Units of platelet transfusions Standard Deviation 0.90
The Number of Units of Platelet Transfusions by Cycle Cycle 17	0.3 Units of platelet transfusions Standard Deviation 0.73
The Number of Units of Platelet Transfusions by Cycle Cycle 18	0.6 Units of platelet transfusions Standard Deviation 1.51
The Number of Units of Platelet Transfusions by Cycle Cycle 19	0.3 Units of platelet transfusions Standard Deviation 0.64
The Number of Units of Platelet Transfusions by Cycle Cycle 22	0.0 Units of platelet transfusions Standard Deviation NA Not applicable = Null value
The Number of Units of Platelet Transfusions by Cycle Cycle 24	0.0 Units of platelet transfusions Standard Deviation NA Not applicable = Null value
The Number of Units of Platelet Transfusions by Cycle Cycle 26	0.0 Units of platelet transfusions Standard Deviation NA Not applicable = Null value
The Number of Units of Platelet Transfusions by Cycle Cycle 27	0.0 Units of platelet transfusions Standard Deviation NA Not applicable = Null value
The Number of Units of Platelet Transfusions by Cycle Overall Post-Baseline Average N = 72	4.7 Units of platelet transfusions Standard Deviation 17.79
The Number of Units of Platelet Transfusions by Cycle Cycle 3	3.3 Units of platelet transfusions Standard Deviation 11.44

SECONDARY outcome

Timeframe: Up to cycle 27; The on-treatment period was considered the period from the date of first dose to the last treatment study visit; Up to 29 January 2015; 894 days

Population: Intent to Treat (ITT) includes all participants who were enrolled into the study.

The number of platelet transfusions received 56 days prior to treatment, considered the baseline period (baseline period defined as the screening period before the date of the first dose of azacitidine; any laboratory and blood transfusion data reported on the day of the first dose of azacitidine was considered to be baseline data) and during study was standardized per 28 days and summarized by cycle for platelets. The formula for standardizing per 28 days was: \[(# of transfusions in the measurement period / length of the measurement period (days)) x 28\], where the measurement period was either baseline or the relevant cycle length.

Outcome measures

Outcome measures
Measure	Azacitidine n=72 Participants Azacitidine 75 mg/m\^2/day subcutaneously (SC) for 7 days every 28 days until disease progression (DP), adverse events (AE), withdrawal of consent from further treatment, withdrawal of consent, death, lost to follow-up, or protocol violation.
The Number of Platelet Transfusions by Cycle Cycle 2	1.3 Platelet transfusions Standard Deviation 2.59
The Number of Platelet Transfusions by Cycle Cycle 14	0.1 Platelet transfusions Standard Deviation 0.35
The Number of Platelet Transfusions by Cycle Cycle 22	0.0 Platelet transfusions Standard Deviation NA Not applicable = Null value
The Number of Platelet Transfusions by Cycle Baseline N = 72	0.8 Platelet transfusions Standard Deviation 2.36
The Number of Platelet Transfusions by Cycle Overall Post-Baseline Average N = 72	1.6 Platelet transfusions Standard Deviation 2.83
The Number of Platelet Transfusions by Cycle Overall Change from Baseline N = 72	0.8 Platelet transfusions Standard Deviation 3.29
The Number of Platelet Transfusions by Cycle Cycle 1 N = 72	1.8 Platelet transfusions Standard Deviation 2.89
The Number of Platelet Transfusions by Cycle Cycle 3	1.2 Platelet transfusions Standard Deviation 2.28
The Number of Platelet Transfusions by Cycle Cycle 4	1.3 Platelet transfusions Standard Deviation 2.18
The Number of Platelet Transfusions by Cycle Cycle 5	0.9 Platelet transfusions Standard Deviation 1.58
The Number of Platelet Transfusions by Cycle Cycle 6	0.7 Platelet transfusions Standard Deviation 1.34
The Number of Platelet Transfusions by Cycle Cycle 7	0.8 Platelet transfusions Standard Deviation 2.50
The Number of Platelet Transfusions by Cycle Cycle 8	0.8 Platelet transfusions Standard Deviation 1.66
The Number of Platelet Transfusions by Cycle Cycle 9	0.8 Platelet transfusions Standard Deviation 2.46
The Number of Platelet Transfusions by Cycle Cycle 10	0.8 Platelet transfusions Standard Deviation 2.08
The Number of Platelet Transfusions by Cycle Cycle 11	0.5 Platelet transfusions Standard Deviation 1.00
The Number of Platelet Transfusions by Cycle Cycle 12	0.3 Platelet transfusions Standard Deviation 0.78
The Number of Platelet Transfusions by Cycle Cycle 13	0.3 Platelet transfusions Standard Deviation 0.72
The Number of Platelet Transfusions by Cycle Cycle 15	0.3 Platelet transfusions Standard Deviation 0.90
The Number of Platelet Transfusions by Cycle Cycle 16	0.3 Platelet transfusions Standard Deviation 0.90
The Number of Platelet Transfusions by Cycle Cycle 17	0.3 Platelet transfusions Standard Deviation 0.73
The Number of Platelet Transfusions by Cycle Cycle 18	0.6 Platelet transfusions Standard Deviation 1.51
The Number of Platelet Transfusions by Cycle Cycle 19	0.3 Platelet transfusions Standard Deviation 0.64
The Number of Platelet Transfusions by Cycle Cycle 20	0.2 Platelet transfusions Standard Deviation 0.37
The Number of Platelet Transfusions by Cycle Cycle 21	0.0 Platelet transfusions Standard Deviation 0.00
The Number of Platelet Transfusions by Cycle Cycle 23	0.0 Platelet transfusions Standard Deviation NA Not applicable = Null value
The Number of Platelet Transfusions by Cycle Cycle 24	0.0 Platelet transfusions Standard Deviation NA Not applicable = Null value
The Number of Platelet Transfusions by Cycle Cycle 25	0.0 Platelet transfusions Standard Deviation NA Not applicable = Null value
The Number of Platelet Transfusions by Cycle Cycle 26	0.0 Platelet transfusions Standard Deviation NA Not applicable = Null value
The Number of Platelet Transfusions by Cycle Cycle 27	0.0 Platelet transfusions Standard Deviation NA Not applicable = Null value

SECONDARY outcome

Timeframe: Up to cycle 27; The on-treatment period was considered the period from the date of first dose to the last treatment study visit; Up to 29 January 2015; 894 days

Population: ITT includes all participants who were enrolled into the study

The number of units of RBC received 56 days prior to treatment, considered the baseline period (baseline period defined as the screening period before the date of the first dose of azacitidine; any laboratory and blood transfusion data reported on the day of the first dose of azacitidine was considered to be baseline data) and during study was standardized per 28 days and summarized by cycle for RBC. The formula for standardizing per 28 days was: \[(# of transfusions in the measurement period / length of the measurement period (days)) x 28\], where the measurement period was either baseline or the relevant cycle length.

Outcome measures

Outcome measures
Measure	Azacitidine n=72 Participants Azacitidine 75 mg/m\^2/day subcutaneously (SC) for 7 days every 28 days until disease progression (DP), adverse events (AE), withdrawal of consent from further treatment, withdrawal of consent, death, lost to follow-up, or protocol violation.
The Number of Units of Red Blood Cell (RBC) Transfusions by Cycle Cycle 22 N	0 Units of RBC Transfusions Standard Deviation NA Not applicable= Null value
The Number of Units of Red Blood Cell (RBC) Transfusions by Cycle Cycle 23	0.0 Units of RBC Transfusions Standard Deviation NA Not applicable= Null value
The Number of Units of Red Blood Cell (RBC) Transfusions by Cycle Baseline N = 72	2.3 Units of RBC Transfusions Standard Deviation 3.55
The Number of Units of Red Blood Cell (RBC) Transfusions by Cycle Overall Post-Baseline Average N = 72	3.5 Units of RBC Transfusions Standard Deviation 3.88
The Number of Units of Red Blood Cell (RBC) Transfusions by Cycle Overall Change from Baseline N = 72	1.2 Units of RBC Transfusions Standard Deviation 4.34
The Number of Units of Red Blood Cell (RBC) Transfusions by Cycle Cycle 1 N = 72	3.6 Units of RBC Transfusions Standard Deviation 3.41
The Number of Units of Red Blood Cell (RBC) Transfusions by Cycle Cycle 2	3.6 Units of RBC Transfusions Standard Deviation 4.88
The Number of Units of Red Blood Cell (RBC) Transfusions by Cycle Cycle 3	3.0 Units of RBC Transfusions Standard Deviation 3.68
The Number of Units of Red Blood Cell (RBC) Transfusions by Cycle Cycle 4	2.7 Units of RBC Transfusions Standard Deviation 3.73
The Number of Units of Red Blood Cell (RBC) Transfusions by Cycle Cycle 5	2.5 Units of RBC Transfusions Standard Deviation 3.94
The Number of Units of Red Blood Cell (RBC) Transfusions by Cycle Cycle 6	2.0 Units of RBC Transfusions Standard Deviation 3.46
The Number of Units of Red Blood Cell (RBC) Transfusions by Cycle Cycle 7	1.7 Units of RBC Transfusions Standard Deviation 3.84
The Number of Units of Red Blood Cell (RBC) Transfusions by Cycle Cycle 8	1.7 Units of RBC Transfusions Standard Deviation 2.68
The Number of Units of Red Blood Cell (RBC) Transfusions by Cycle Cycle 9	0.9 Units of RBC Transfusions Standard Deviation 1.56
The Number of Units of Red Blood Cell (RBC) Transfusions by Cycle Cycle 10	1.2 Units of RBC Transfusions Standard Deviation 2.25
The Number of Units of Red Blood Cell (RBC) Transfusions by Cycle Cycle 11	1.3 Units of RBC Transfusions Standard Deviation 1.97
The Number of Units of Red Blood Cell (RBC) Transfusions by Cycle Cycle 12	1.1 Units of RBC Transfusions Standard Deviation 2.21
The Number of Units of Red Blood Cell (RBC) Transfusions by Cycle Cycle 13	1.2 Units of RBC Transfusions Standard Deviation 2.62
The Number of Units of Red Blood Cell (RBC) Transfusions by Cycle Cycle 14	0.4 Units of RBC Transfusions Standard Deviation 1.05
The Number of Units of Red Blood Cell (RBC) Transfusions by Cycle Cycle 15	1.1 Units of RBC Transfusions Standard Deviation 2.07
The Number of Units of Red Blood Cell (RBC) Transfusions by Cycle Cycle 16	0.9 Units of RBC Transfusions Standard Deviation 1.61
The Number of Units of Red Blood Cell (RBC) Transfusions by Cycle Cycle 17	1.2 Units of RBC Transfusions Standard Deviation 3.28
The Number of Units of Red Blood Cell (RBC) Transfusions by Cycle Cycle 18	1.6 Units of RBC Transfusions Standard Deviation 3.07
The Number of Units of Red Blood Cell (RBC) Transfusions by Cycle Cycle 19	0.8 Units of RBC Transfusions Standard Deviation 1.37
The Number of Units of Red Blood Cell (RBC) Transfusions by Cycle Cycle 20	1.1 Units of RBC Transfusions Standard Deviation 1.42
The Number of Units of Red Blood Cell (RBC) Transfusions by Cycle Cycle 21	0.4 Units of RBC Transfusions Standard Deviation 0.62
The Number of Units of Red Blood Cell (RBC) Transfusions by Cycle Cycle 24	0.0 Units of RBC Transfusions Standard Deviation NA Not applicable= Null value
The Number of Units of Red Blood Cell (RBC) Transfusions by Cycle Cycle 25	0.0 Units of RBC Transfusions Standard Deviation NA Not applicable= Null value
The Number of Units of Red Blood Cell (RBC) Transfusions by Cycle Cycle 26	0.0 Units of RBC Transfusions Standard Deviation NA Not applicable= Null value
The Number of Units of Red Blood Cell (RBC) Transfusions by Cycle Cycle 27	0.0 Units of RBC Transfusions Standard Deviation NA Not applicable= Null value

SECONDARY outcome

Timeframe: Up to cycle 27; The on-treatment period was considered the period from the date of first dose to the last treatment study visit; Up to 29 January 2015; 894 days

Population: Intent to Treat (ITT) includes all participants who were enrolled into the study.

The number of RBC transfusions received 56 days prior to treatment, considered the baseline period (baseline period defined as the screening period before the date of the first dose of azacitidine; any laboratory and blood transfusion data reported on the day of the first dose of azacitidine was considered to be baseline data) and during study was standardized per 28 days and summarized by cycle for platelets. The formula for standardizing per 28 days was: \[(# of transfusions in the measurement period / length of the measurement period (days)) x 28\], where the measurement period was either baseline or the relevant cycle length.

Outcome measures

Outcome measures
Measure	Azacitidine n=72 Participants Azacitidine 75 mg/m\^2/day subcutaneously (SC) for 7 days every 28 days until disease progression (DP), adverse events (AE), withdrawal of consent from further treatment, withdrawal of consent, death, lost to follow-up, or protocol violation.
The Number of RBC Transfusions by Cycle Cycle 4	1.2 RBC Transfusions Standard Deviation 1.73
The Number of RBC Transfusions by Cycle Cycle 16	0.4 RBC Transfusions Standard Deviation 0.57
The Number of RBC Transfusions by Cycle Baseline N = 72	1.1 RBC Transfusions Standard Deviation 1.79
The Number of RBC Transfusions by Cycle Overall Post-Baseline Average N = 72	1.6 RBC Transfusions Standard Deviation 1.97
The Number of RBC Transfusions by Cycle Overall Change from Baseline N = 72	0.5 RBC Transfusions Standard Deviation 2.12
The Number of RBC Transfusions by Cycle Cycle 1 N = 72	1.7 RBC Transfusions Standard Deviation 1.64
The Number of RBC Transfusions by Cycle Cycle 2	1.7 RBC Transfusions Standard Deviation 2.56
The Number of RBC Transfusions by Cycle Cycle 3	1.4 RBC Transfusions Standard Deviation 1.81
The Number of RBC Transfusions by Cycle Cycle 5	1.1 RBC Transfusions Standard Deviation 1.81
The Number of RBC Transfusions by Cycle Cycle 6	0.9 RBC Transfusions Standard Deviation 1.63
The Number of RBC Transfusions by Cycle Cycle 7	0.8 RBC Transfusions Standard Deviation 1.97
The Number of RBC Transfusions by Cycle Cycle 8	0.8 RBC Transfusions Standard Deviation 1.23
The Number of RBC Transfusions by Cycle Cycle 9	0.4 RBC Transfusions Standard Deviation 0.74
The Number of RBC Transfusions by Cycle Cycle 10	0.5 RBC Transfusions Standard Deviation 0.94
The Number of RBC Transfusions by Cycle Cycle 11	0.5 RBC Transfusions Standard Deviation 0.78
The Number of RBC Transfusions by Cycle Cycle 12	0.4 RBC Transfusions Standard Deviation 0.87
The Number of RBC Transfusions by Cycle Cycle 13	0.5 RBC Transfusions Standard Deviation 0.94
The Number of RBC Transfusions by Cycle Cycle 14	0.2 RBC Transfusions Standard Deviation 0.53
The Number of RBC Transfusions by Cycle Cycle 15	0.4 RBC Transfusions Standard Deviation 0.77
The Number of RBC Transfusions by Cycle Cycle 17	0.3 RBC Transfusions Standard Deviation 0.91
The Number of RBC Transfusions by Cycle Cycle 18	0.7 RBC Transfusions Standard Deviation 1.17
The Number of RBC Transfusions by Cycle Cycle 19	0.4 RBC Transfusions Standard Deviation 0.64
The Number of RBC Transfusions by Cycle Cycle 20	0.6 RBC Transfusions Standard Deviation 0.71
The Number of RBC Transfusions by Cycle Cycle 21	0.2 RBC Transfusions Standard Deviation 0.31
The Number of RBC Transfusions by Cycle Cycle 22	0.0 RBC Transfusions Standard Deviation NA Not applicable= Null value
The Number of RBC Transfusions by Cycle Cycle 23	0.0 RBC Transfusions Standard Deviation NA Not applicable= Null value
The Number of RBC Transfusions by Cycle Cycle 24	0.0 RBC Transfusions Standard Deviation NA Not applicable= Null value
The Number of RBC Transfusions by Cycle Cycle 25	0.0 RBC Transfusions Standard Deviation NA Not applicable= Null value
The Number of RBC Transfusions by Cycle Cycle 26	0.0 RBC Transfusions Standard Deviation NA Not applicable= Null value
The Number of RBC Transfusions by Cycle Cycle 27	0.0 RBC Transfusions Standard Deviation NA Not applicable= Null value

SECONDARY outcome

Timeframe: Up to cycle 27; The on-treatment period was considered the period from the date of first dose to the last treatment study visit; Up to 29 January 2015; 894 days

Population: Intent to Treat (ITT) includes all participants who were enrolled into the study.

The on-treatment adverse event of infection requiring IV antibiotics, antifungals, or antivirals per 28 days/cycle. The overall post-baseline average is the average of number of infections requiring IV antibiotics or IV antiviral per 28 days/cycle.

Outcome measures

Outcome measures
Measure	Azacitidine n=72 Participants Azacitidine 75 mg/m\^2/day subcutaneously (SC) for 7 days every 28 days until disease progression (DP), adverse events (AE), withdrawal of consent from further treatment, withdrawal of consent, death, lost to follow-up, or protocol violation.
The Number of Infections (Post-baseline Average) Requiring Intravenous (IV) Antibiotics, Anti-fungals, or Antivirals by Cycle Cycle 1 N = 72	0.6 Infections Standard Deviation 1.32
The Number of Infections (Post-baseline Average) Requiring Intravenous (IV) Antibiotics, Anti-fungals, or Antivirals by Cycle Cycle 2	0.5 Infections Standard Deviation 1.67
The Number of Infections (Post-baseline Average) Requiring Intravenous (IV) Antibiotics, Anti-fungals, or Antivirals by Cycle Cycle 11	0.1 Infections Standard Deviation 0.31
The Number of Infections (Post-baseline Average) Requiring Intravenous (IV) Antibiotics, Anti-fungals, or Antivirals by Cycle Cycle12	0.1 Infections Standard Deviation 0.27
The Number of Infections (Post-baseline Average) Requiring Intravenous (IV) Antibiotics, Anti-fungals, or Antivirals by Cycle Cycle 16	0.3 Infections Standard Deviation 0.42
The Number of Infections (Post-baseline Average) Requiring Intravenous (IV) Antibiotics, Anti-fungals, or Antivirals by Cycle Cycle 17	0.4 Infections Standard Deviation 0.75
The Number of Infections (Post-baseline Average) Requiring Intravenous (IV) Antibiotics, Anti-fungals, or Antivirals by Cycle Cycle 24	0.0 Infections Standard Deviation NA Not Applicable = Null value
The Number of Infections (Post-baseline Average) Requiring Intravenous (IV) Antibiotics, Anti-fungals, or Antivirals by Cycle Cycle 25	0.0 Infections Standard Deviation NA Not Applicable = Null value
The Number of Infections (Post-baseline Average) Requiring Intravenous (IV) Antibiotics, Anti-fungals, or Antivirals by Cycle Cycle 27	0.0 Infections Standard Deviation NA Not Applicable = Null value
The Number of Infections (Post-baseline Average) Requiring Intravenous (IV) Antibiotics, Anti-fungals, or Antivirals by Cycle Baseline N = 72	0.0 Infections Standard Deviation 0.20
The Number of Infections (Post-baseline Average) Requiring Intravenous (IV) Antibiotics, Anti-fungals, or Antivirals by Cycle Overall Post Baseline Average N = 72	0.6 Infections Standard Deviation 1.43
The Number of Infections (Post-baseline Average) Requiring Intravenous (IV) Antibiotics, Anti-fungals, or Antivirals by Cycle Overall Change from Baseline N =72	0.5 Infections Standard Deviation 1.45
The Number of Infections (Post-baseline Average) Requiring Intravenous (IV) Antibiotics, Anti-fungals, or Antivirals by Cycle Cycle 3	0.4 Infections Standard Deviation 0.86
The Number of Infections (Post-baseline Average) Requiring Intravenous (IV) Antibiotics, Anti-fungals, or Antivirals by Cycle Cycle 4	0.5 Infections Standard Deviation 1.03
The Number of Infections (Post-baseline Average) Requiring Intravenous (IV) Antibiotics, Anti-fungals, or Antivirals by Cycle Cycle 5	0.4 Infections Standard Deviation 0.72
The Number of Infections (Post-baseline Average) Requiring Intravenous (IV) Antibiotics, Anti-fungals, or Antivirals by Cycle Cycle 6	0.2 Infections Standard Deviation 0.61
The Number of Infections (Post-baseline Average) Requiring Intravenous (IV) Antibiotics, Anti-fungals, or Antivirals by Cycle Cycle 7	0.3 Infections Standard Deviation 1.01
The Number of Infections (Post-baseline Average) Requiring Intravenous (IV) Antibiotics, Anti-fungals, or Antivirals by Cycle Cycle 8	0.2 Infections Standard Deviation 0.47
The Number of Infections (Post-baseline Average) Requiring Intravenous (IV) Antibiotics, Anti-fungals, or Antivirals by Cycle Cycle 9	0.1 Infections Standard Deviation 0.32
The Number of Infections (Post-baseline Average) Requiring Intravenous (IV) Antibiotics, Anti-fungals, or Antivirals by Cycle Cycle 10	0.2 Infections Standard Deviation 0.44
The Number of Infections (Post-baseline Average) Requiring Intravenous (IV) Antibiotics, Anti-fungals, or Antivirals by Cycle Cycle 13	0.1 Infections Standard Deviation 0.24
The Number of Infections (Post-baseline Average) Requiring Intravenous (IV) Antibiotics, Anti-fungals, or Antivirals by Cycle Cycle 14 15	0.1 Infections Standard Deviation 0.32
The Number of Infections (Post-baseline Average) Requiring Intravenous (IV) Antibiotics, Anti-fungals, or Antivirals by Cycle Cycle 15	0.3 Infections Standard Deviation 0.70
The Number of Infections (Post-baseline Average) Requiring Intravenous (IV) Antibiotics, Anti-fungals, or Antivirals by Cycle Cycle 18	0.4 Infections Standard Deviation 0.74
The Number of Infections (Post-baseline Average) Requiring Intravenous (IV) Antibiotics, Anti-fungals, or Antivirals by Cycle Cycle 19	0.2 Infections Standard Deviation 0.41
The Number of Infections (Post-baseline Average) Requiring Intravenous (IV) Antibiotics, Anti-fungals, or Antivirals by Cycle Cycle 20	0.0 Infections Standard Deviation 0.00
The Number of Infections (Post-baseline Average) Requiring Intravenous (IV) Antibiotics, Anti-fungals, or Antivirals by Cycle Cycle 21	0.0 Infections Standard Deviation 0.00
The Number of Infections (Post-baseline Average) Requiring Intravenous (IV) Antibiotics, Anti-fungals, or Antivirals by Cycle Cycle 22	0.9 Infections Standard Deviation NA Not Applicable = Null value
The Number of Infections (Post-baseline Average) Requiring Intravenous (IV) Antibiotics, Anti-fungals, or Antivirals by Cycle Cycle 23	1.0 Infections Standard Deviation NA Not Applicable = Null value
The Number of Infections (Post-baseline Average) Requiring Intravenous (IV) Antibiotics, Anti-fungals, or Antivirals by Cycle Cycle 26 N	0.0 Infections Standard Deviation NA Not Applicable = Null value

SECONDARY outcome

Timeframe: Until the end of the survival follow-up period; Up to data cut-off of 29 January 2015; 894 days

Population: Intent to Treat (ITT) includes all participants who were enrolled into the study.

Overall survival is defined as time to death from any cause, is calculated using date of first dose and date of death, or date of last follow-up for censored participants. Those, who die regardless of the cause of death, will be considered to have an event.

Outcome measures

Outcome measures
Measure	Azacitidine n=72 Participants Azacitidine 75 mg/m\^2/day subcutaneously (SC) for 7 days every 28 days until disease progression (DP), adverse events (AE), withdrawal of consent from further treatment, withdrawal of consent, death, lost to follow-up, or protocol violation.
Kaplan Meier Estimates for Overall Survival (OS) Median OS	22.0 months Interval 15.1 to The data for overall survival has not matured and does not exist as of data cut- off date of 29 Jan 2015 as the study is on-going
Kaplan Meier Estimates for Overall Survival (OS) 25th percentile OS	8.5 months Interval 6.1 to 13.8
Kaplan Meier Estimates for Overall Survival (OS) 75th Percentile OS	NA months Interval 24.2 to The data for overall survival among the 75th percentile has not matured and does not exist as of data cut-off date of 29 Jan 2015 as the study is on-going. This also applies to the upper limit.

SECONDARY outcome

Timeframe: Up to 29 January 2015; from the first dose of study drug to 28 days after the date of the last dose of study drug (maximum time on study was 244 days)

Population: Safety population included all participants who received at least one dose of azacitidine and had at least one post-dose safety assessment

A treatment-emergent adverse events (TEAE) was defined as AEs with an onset date on or after the date of first dose and within 28 days after the date of the last dose. Any AE that occurred beyond this timeframe and was assessed by the investigator as possibly related to study drug was considered treatment-emergent. The intensity and severity of AEs was assessed by the investigator according to the Common Terminology Criteria for Adverse Event (CTCAE) Version 4.0. For any AEs not listed in the CTCAE grading system, the intensities of these events was assessed by the Investigator using the 5-point scale: Grade 1 = Mild, Grade 2 = Moderate; Grade 3 = Severe, Grade 4 = Life threatening, Grade 5 = Death. An SAE is any AE occurring that results in death, is life-threatening, requires hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, and constitutes an important medical event.

Outcome measures

Outcome measures
Measure	Azacitidine n=72 Participants Azacitidine 75 mg/m\^2/day subcutaneously (SC) for 7 days every 28 days until disease progression (DP), adverse events (AE), withdrawal of consent from further treatment, withdrawal of consent, death, lost to follow-up, or protocol violation.
Number of Participants With Treatment Emergent Adverse Events (TEAE) During the Parent Phase ≥ 1 TEAE	72 participants
Number of Participants With Treatment Emergent Adverse Events (TEAE) During the Parent Phase ≥ 1 Grade 3 or 4 TEAE	70 participants
Number of Participants With Treatment Emergent Adverse Events (TEAE) During the Parent Phase ≥ 1 TEAE related to study drug	68 participants
Number of Participants With Treatment Emergent Adverse Events (TEAE) During the Parent Phase ≥ 1 Grade 3 or 4 TEAE related to study drug	59 participants
Number of Participants With Treatment Emergent Adverse Events (TEAE) During the Parent Phase ≥ 1 serious TEAE	38 participants
Number of Participants With Treatment Emergent Adverse Events (TEAE) During the Parent Phase ≥ 1 Grade 3 or 4 serious TEAE	33 participants
Number of Participants With Treatment Emergent Adverse Events (TEAE) During the Parent Phase ≥ 1 serious TEAE related to study drug	28 participants
Number of Participants With Treatment Emergent Adverse Events (TEAE) During the Parent Phase ≥ 1 TEAE leading to discontinuation of study drug	14 participants
Number of Participants With Treatment Emergent Adverse Events (TEAE) During the Parent Phase ≥ 1 TEAE leading to dose reduction of study drug	19 participants
Number of Participants With Treatment Emergent Adverse Events (TEAE) During the Parent Phase ≥1 TEAE leading to dose interruption of study drug	49 participants
Number of Participants With Treatment Emergent Adverse Events (TEAE) During the Parent Phase ≥1 TEAE leading to dose reduction/interruption	50 participants
Number of Participants With Treatment Emergent Adverse Events (TEAE) During the Parent Phase ≥1 TEAE with outcome of death	11 participants

SECONDARY outcome

Timeframe: PK blood samples collected at 0.25, 0.5, 1,2,3,4, 6 and 8 hours after azacitidine administration on Day 7

Population: The PK population includes up to 12 participants with evaluable azacitidine plasma PK profile.

Area under the plasma concentration-time curve from time zero to infinity (AUC∞) following multiple doses of Azacitidine on Day 7; if possible, the area under the concentration-time curve from time zero to infinity, calculated by the linear trapezoidal rule and extrapolated to infinity was calculated according to the following equation: AUC∞ = AUCt + (Ct/ λz ), where Ct is the last quantifiable concentration. No AUC extrapolation will be performed with unreliable λz. If % AUC extrapolated is ≥ 25%, AUC∞ will not be reported

Outcome measures

Outcome measures
Measure	Azacitidine n=12 Participants Azacitidine 75 mg/m\^2/day subcutaneously (SC) for 7 days every 28 days until disease progression (DP), adverse events (AE), withdrawal of consent from further treatment, withdrawal of consent, death, lost to follow-up, or protocol violation.
Area Under the Plasma Concentration-time Curve From Time Zero to Infinity (AUC∞) of Azacitidine	1172.6 ng*h/mL Geometric Coefficient of Variation 18.0

SECONDARY outcome

Timeframe: PK blood samples collected at 0.25, 0.5, 1,2,3,4, 6 and 8 hours after azacitidine administration on Day 7

Population: The PK population includes up to 12 participants with evaluable azacitidine plasma PK profile.

Area under the plasma concentration-time curve from time zero to the last quantifiable time point, calculated by the linear trapezoidal rule when concentrations are increasing and the logarithmic trapezoidal method when concentrations are decreasing.

Outcome measures

Outcome measures
Measure	Azacitidine n=12 Participants Azacitidine 75 mg/m\^2/day subcutaneously (SC) for 7 days every 28 days until disease progression (DP), adverse events (AE), withdrawal of consent from further treatment, withdrawal of consent, death, lost to follow-up, or protocol violation.
Area Under the Plasma Concentration-time Curve From Time Zero to the Last Measurable Concentration (AUCt) of Azacitidine	1161.9 ng*h/mL Geometric Coefficient of Variation 17.7

SECONDARY outcome

Timeframe: PK blood samples collected at 0.25, 0.5, 1,2,3,4, 6 and 8 hours after azacitidine administration on Day 7

Population: The PK population includes up to 12 participants with evaluable azacitidine plasma PK profile.

The observed maximum plasma concentration obtained directly from the observed concentration versus time data.

Outcome measures

Outcome measures
Measure	Azacitidine n=12 Participants Azacitidine 75 mg/m\^2/day subcutaneously (SC) for 7 days every 28 days until disease progression (DP), adverse events (AE), withdrawal of consent from further treatment, withdrawal of consent, death, lost to follow-up, or protocol violation.
Maximum Observed Plasma Concentration (Cmax) of Azacitidine	1264.6 ng/mL Geometric Coefficient of Variation 30.7

SECONDARY outcome

Timeframe: PK blood samples collected at 0.25, 0.5, 1,2,3,4, 6 and 8 hours after azacitidine administration on Day 7

Population: The PK population includes up to 12 participants with evaluable azacitidine plasma PK profile.

Time to maximum observed plasma concentration obtained directly from the observed concentration versus time data.

Outcome measures

Outcome measures
Measure	Azacitidine n=12 Participants Azacitidine 75 mg/m\^2/day subcutaneously (SC) for 7 days every 28 days until disease progression (DP), adverse events (AE), withdrawal of consent from further treatment, withdrawal of consent, death, lost to follow-up, or protocol violation.
Time to Maximum Plasma Concentration (Tmax) of Azacitidine	0.25 hours Full Range 27.66 • Interval 0.23 to 0.5

SECONDARY outcome

Timeframe: PK blood samples collected at 0.25, 0.5, 1,2,3,4, 6 and 8 hours after azacitidine administration on Day 7

Population: The PK population includes up to 12 participants with evaluable azacitidine plasma PK profile.

The apparent terminal half-life was calculated according to the following equation t½ = 0.693/λz.

Outcome measures

Outcome measures
Measure	Azacitidine n=12 Participants Azacitidine 75 mg/m\^2/day subcutaneously (SC) for 7 days every 28 days until disease progression (DP), adverse events (AE), withdrawal of consent from further treatment, withdrawal of consent, death, lost to follow-up, or protocol violation.
Terminal Phase of Half-life (T1/2) of Azacitidine	0.8 hours Geometric Coefficient of Variation 69.0

SECONDARY outcome

Timeframe: PK blood samples collected at 0.25, 0.5, 1,2,3,4, 6 and 8 hours after azacitidine administration on Day 7

Population: The PK population includes up to 12 participants with evaluable azacitidine plasma PK profile.

Apparent total plasma clearance (CL/F) of Azacitidine was calculated as Dose/AUC∞

Outcome measures

Outcome measures
Measure	Azacitidine n=12 Participants Azacitidine 75 mg/m\^2/day subcutaneously (SC) for 7 days every 28 days until disease progression (DP), adverse events (AE), withdrawal of consent from further treatment, withdrawal of consent, death, lost to follow-up, or protocol violation.
Apparent Total Plasma Clearance (CL/F) of Azacitidine	107.2 Liters/hours Geometric Coefficient of Variation 19.1

SECONDARY outcome

Timeframe: PK blood samples collected at 0.25, 0.5, 1,2,3,4, 6 and 8 hours after azacitidine administration on Day 7

Population: The PK population includes up to 12 participants with evaluable azacitidine plasma PK profile.

Apparent volume of distribution, was calculated according to the equation: Vd/F = (CL/F)/λz

Outcome measures

Outcome measures
Measure	Azacitidine n=12 Participants Azacitidine 75 mg/m\^2/day subcutaneously (SC) for 7 days every 28 days until disease progression (DP), adverse events (AE), withdrawal of consent from further treatment, withdrawal of consent, death, lost to follow-up, or protocol violation.
Apparent Volume of Distribution (Vd/F) of Azacitidine	121.5 Liters Geometric Coefficient of Variation 65.1

SECONDARY outcome

Timeframe: Up to final data cut off date of 25 April 2018; from the first dose of study drug extesnion of 29 December 2014 to 28 days after the date of the last dose of study drug; median duration of any dose of study drug was 169 days

Population: Safety population included all participants who received at least one dose of azacitidine and had at least one post-dose safety assessment

Outcome measures

Outcome measures
Measure	Azacitidine n=15 Participants Azacitidine 75 mg/m\^2/day subcutaneously (SC) for 7 days every 28 days until disease progression (DP), adverse events (AE), withdrawal of consent from further treatment, withdrawal of consent, death, lost to follow-up, or protocol violation.
Number of Participants With Treatment Emergent Adverse Events (TEAE) During the Extension Phase ≥ 1 TEAE	14 participants
Number of Participants With Treatment Emergent Adverse Events (TEAE) During the Extension Phase ≥ 1 TEAE related to study drug	8 participants
Number of Participants With Treatment Emergent Adverse Events (TEAE) During the Extension Phase ≥ 1 Grade 3 or 4 TEAE	13 participants
Number of Participants With Treatment Emergent Adverse Events (TEAE) During the Extension Phase ≥ 1 Grade 3 or 4 TEAE related to study drug	6 participants
Number of Participants With Treatment Emergent Adverse Events (TEAE) During the Extension Phase ≥ 1 serious TEAE	5 participants
Number of Participants With Treatment Emergent Adverse Events (TEAE) During the Extension Phase ≥ 1 serious TEAE related to study drug	1 participants
Number of Participants With Treatment Emergent Adverse Events (TEAE) During the Extension Phase ≥ 1 Grade 3 or 4 serious TEAE	5 participants
Number of Participants With Treatment Emergent Adverse Events (TEAE) During the Extension Phase ≥1 TEAE leading to dose interruption of study drug	4 participants
Number of Participants With Treatment Emergent Adverse Events (TEAE) During the Extension Phase ≥1 TEAE leading to dose reduction/interruption	4 participants

Adverse Events

Azacitidine - Parent Phase

Serious events: 38 serious events

Other events: 72 other events

Deaths: 13 deaths

Azacitdine - Extension Phase

Serious events: 5 serious events

Other events: 14 other events

Deaths: 0 deaths

Serious adverse events

Other adverse events

Additional Information

Anne McClain, Senior Manager, Clinical Trial Disclosure

Celgene Corporation

Phone: 1-888-260-1599

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee The investigator shall have the right to publish and/or present study data provided that the investigator shall (i) furnish the sponsor a copy of any proposed publication or presentation generally sixty (60) days in advance of the submission, (ii) delete any confidential information of the sponsor, and (iii) delay submission for generally up to ninety (90) days to permit the preparation and filing of intellectual property applications or until sponsor gives its consent in a timely manner.
Publication restrictions are in place

Restriction type: OTHER