An Efficacy and Safety Study of Decitabine in Participants With Myelodysplastic Syndrome (MDS)

• Sponsor: Johnson & Johnson Taiwan Ltd (Industry)
• Overall Status: Completed
• CT.gov ID: NCT00744757
• Collaborator: (none)
• Enrollment: 37
• Locations: 6
• Arm: 1
• Duration (Months): 48
• Patients Per Site: 6.2
• Patients Per Site Per Month: 0.1

Study Details

Study Description

Brief Summary

The purpose of this study is to evaluate the response rate of decitabine in previously treated and untreated Taiwanese participants with Myelodysplastic Syndrome (MDS - a disease associated with decreased production of blood cells, blood cells are produced but do not mature normally).

Condition or Disease	Intervention/Treatment	Phase
Myelodysplastic Syndrome	Drug: Decitabine	Phase 2

Detailed Description

This is an open-label (a medical research study in which participants and researchers are told which treatments the participants are receiving, "unblinded"), multi-center (when more than 1 hospital or medical school team work on a medical research study), single-arm study of decitabine. The study will consist of 5 phases: Pre-treatment phase (before 30 days of first dose), Treatment phase (consist of 8 cycles, each cycle of 28 days), End-of-treatment phase (consist of 30-42 days after the last dose of cycle 8, or at time of discontinuation), Extension phase (1 cycle of 4 weeks) and Post-study phase or follow-up phase (every 2 months until 1 year, lost to follow-up or death). Participants who achieve a complete remission (when a medical problem gets better or goes away at least for a while) will be treated for at least 2 more cycles after first documentation of complete response (CR), after which treatment can be discontinued. Decitabine in a dose of 20 milligram per square meter (mg per m^2) will be administered intravenously over 1 hour infusion, 1 hour, once daily for 5 consecutive days of a 28 days cycle up to 8 cycles or continued until disease progression or unacceptable toxicity. The primary objective is to evaluate the best response rate (complete response and partial response). Participants' safety will be monitored throughout the study.

Study Design

• Study Type: Interventional
• Actual Enrollment: 37 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A Phase II Multi-center Study of 5-AZA-2'-Deoxycytidine (Decitabine) Single Agent in Taiwanese Patients With Myelodysplastic Syndrome (MDS)
• Study Start Date: Aug 1, 2008
• Actual Primary Completion Date: Sep 1, 2010
• Actual Study Completion Date: Aug 1, 2012

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Decitabine Decitabine 20 milligram per square meter (mg per m^2) will be administered intravenous (into a vein) infusion (a fluid or a medicine delivered into a vein by way of a needle) over 1 hour, once daily for 5 consecutive days of a 28 days cycle up to 8 cycles or continued until disease progression or unacceptable toxicity.	Drug: Decitabine; Decitabine 20 mg per m^2 will be administered intravenous infusion over 1 hour, once daily for 5 consecutive days of a 28 days cycle up to 8 cycles or continued until disease progression or unacceptable toxicity.; Other Names: Dacogen 5-aza-2'-deoxycytidine

Outcome Measures

Primary Outcome Measures

1. Percentage of Participants With Response [Day 1 of Cycle 2, 4, 6, 8; each Cycle of 28 days and End of treatment (30-42 days after Cycle 8 or early withdrawal)]

   Percentage of participants with response: complete response (CR) or partial response (PR) according to International Working Group (IWG) 2006 criteria was evaluated. CR in bone marrow is defined as<=to 5% myeloblasts with normal maturation of all cell lines and persistent dysplasia was noted in peripheral blood hemoglobin >=11 gram (g) per deciliter (dl), platelets >=100*10^9 liter (l), neutrophils >=1.0*10^9 l, Blasts 0%. Partial response is defined as all CR criteria if abnormal before treatment except: bone marrow blasts decreased by >=50% over pre-treatment but still >=5%.

Secondary Outcome Measures

1. Percentage of Participants With Hematologic Treatment Response [Day 1 of Cycle 1, 2, 3, 4, 5, 6, 7 and 8, each Cycle of 28 days and End of treatment (30-42 days after Cycle 8 or early withdrawal)]

   Hematologic treatment response was assessed as per IWG 2006 criteria. This is measured in erythroid(HI-E), platelet(HI-P) and neutrophil(HI-N) lineages. HI-E response(pre-treatment<11 gram per deciliter [g/dl]):hemoglobin increase by>=1.5 g/dl and relevant reduction in RBC transfusions by 4 RBC transfusions/8week. HI-P response (pre-treatment<100*109/l):absolute increase of >=30*10^9/l for participants starting with>20*10^9/l and increase from <20*10^9/l to>20*10^9/l and by at least 100%. HI-N response (pre-treatment<1.0*10^9/l): at least 100% increase and an absolute increase >0.5*10^9/l.

2. Percentage of Participants With Cytogenetic Response [Day 1 of Cycle 2, 4, 6, 8; each Cycle of 28 days and End of treatment (30-42 days after Cycle 8 or early withdrawal)]

   Cytogenetic responses was assessed as per IWG 2006 criteria which define complete response as disappearance of the chromosomal abnormality without appearance of new ones and partial response as at least 50 percent reduction of the chromosomal abnormality.

3. Time to Acute Myeloid Leukemia (AML) Progression or Death [Start of treatment until disease progression or death (whichever occur first) or up to 736 days]

   Time to AML is defined as greater than 30 percent of blasts in bone marrow or the time to death was calculated from the date of treatment start until disease progression to AML or until death, which ever occurred first. Participants who were still alive and did not progress to AML were censored at the moment of last visit.

4. Overall Survival [Start of treatment until disease progression or death (whichever occur first) or up to 736 days]

   Overall survival was defined as the time from the date of treatment start until death (whatever the cause). It was calculated from Kaplan-Meier estimates. Participants still alive were censored at the moment of last visit or contact.

5. Percentage of Participants With Transfusion Dependency [8 weeks before first dose until disease progression or death (whichever occur first) or up to 736 days]

   Transfusion requirements for both red blood cells as well as platelets were recorded for each participant.

6. Percentage of Participants With Transfusion Independency [8 weeks before first dose and 736 days of treatment]

   Transfusion independent participants were calculated from all the participants who required transfusion in the duration of 8 weeks before first dose until disease progression or death or up to 736 days. Transfusion independence was defined as lack of requirement for transfusions for at least 8 weeks.

7. Duration for Hospitalization [Cycle 1 up to Cycle 8, each cycle of 28 days.]

   Duration of hospitalization was calculated for each participant, using the sum of all hospital days by subtracting the date of discharge from the date of admission.

8. Number of Events Which Led to Hospitalization [Start of treatment until disease progression or death or up to Cycle 8, each cycle of 28 days]

   The events (reasons) for hospitalizations such as infection, transfusion, acute choleycystitis, allergic transfusion reaction, dyspnoea with right pleural effusion, febrile neutropenia, fever, for decitabine, Myelodysplastic Syndrome (MDS) hematuria, paronychia, pneumonia, heart failure, peri-anal abscess (PAA), pancytopenia,fluctuated neutropenia fever, right dorsal foot cellulitis, Right lower (Rt.Lw) lung pneumonia with impending respiratory (resp) failure, Serious adverse event (SAE)+schedule hospitalization for decitabine, septic shock and not available were reported.

9. Quality of Life Assessment [Day 1 of Cycle 1 and Cycle 8 (each cycle of 28 days)]

   The quality of life was assessed by the questionnaire QLQ C-30 designed by European Organization for the Research and Treatment of Cancer (EORTC). EORTC QLQ-C30 is a 30-item questionnaire to assess the overall quality of life in cancer participants. Most questions used 4-point scale (1=Not at All' to 4=Very Much') and 2 questions: Q29 on overall health and Q30 on overall quality of life uses 7-point scale ranging from 1=Very Poor to 7=Excellent. Higher score indicates better quality of life.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Participants with documented pathological (bone marrow, no longer than 30 days before first dosing in study) evidence of Myelodysplastic syndromes (MDS) or of chronic myelomonocytic leukemia (CMML) by World health organisation classification

• Participants with international prognostic scoring system (IPSS) score equal to 0.5 or more (only for participants for whom IPSS is applicable)

• Participants with an Eastern oncology cooperative group (ECOG) performance status of 0-2

• Participants with adequate hepatic (liver) and renal (kidney) function as measured by pre-treatment laboratory criteria within 21 days of starting treatment with decitabine

• Participants must have recovered from toxic effects of previous therapy and not receiving any chemotherapy for a minimum of 4 weeks (6 weeks if the participants has been treated with a nitrosoureas) before to the first dose of study drug

Exclusion Criteria:

• Participants with a diagnosis of acute myeloid leukemia (AML) (greater than 30 percent bone marrow blasts)

• Participants with AML with multilineage dysplasia (abnormal development or cell growth) following MDS (20-30 percent bone marrow blasts) can be enrolled. For these latter participants an observation period of 1 month is necessary to exclude those participants with rapid progression to full blown AML

• Participants with previous treatment with azacitadine or decitabine or hematopoietic stem cell transplantation less than 1 year prior to study enrollment

• Participants with past history of malignancy and received any treatment for this before malignancy within the last 3 years, except for superficial bladder cancer, basal cell or squamous cell carcinoma of the skin, cervical intraepithelial neoplasia (CIN) or prostate intraepithelial neoplasia (PIN)

• Participants with known hepatitis B (surface antigen-positive) or active hepatitis C infection

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1		Changhua		Taiwan
2		Kaohsiung		Taiwan
3		Taichung		Taiwan
4		Tainan		Taiwan
5		Taipei		Taiwan
6		Tau-Yuan County 333		Taiwan

Sponsors and Collaborators

• Johnson & Johnson Taiwan Ltd

Investigators

• Study Director: Johnson & Johnson Taiwan, Ltd. Clinical Trial, Johnson & Johnson Taiwan Ltd

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Johnson & Johnson Taiwan Ltd
• ClinicalTrials.gov Identifier: NCT00744757
• Other Study ID Numbers: CR014785; DACOGENMDS2001
• First Posted: Sep 1, 2008
• Last Update Posted: Sep 9, 2013
• Last Verified: Aug 1, 2013

• Keywords provided by Johnson & Johnson Taiwan Ltd: 5-aza-2'-deoxycytidine; decitabine; dacogen; Myelodysplastic Syndrome
• Additional relevant MeSH terms: Preleukemia; Myelodysplastic Syndromes; Syndrome; Decitabine

Study Results

Participant Flow

Arm/Group Title	Decitabine
Arm/Group Description	Decitabine 20 milligram per square meter (mg per m^2) will be administered intravenous (into a vein) infusion (a fluid or a medicine delivered into a vein by way of a needle) over 1 hour, once daily for 5 consecutive days of a 28 days cycle up to 8 cycles or continued until disease progression or unacceptable toxicity.
Period Title: Overall Study
STARTED	37
COMPLETED	16
NOT COMPLETED	21

Baseline Characteristics

Arm/Group Title	Decitabine
Arm/Group Description	Decitabine 20 milligram per square meter (mg per m^2) will be administered intravenous (into a vein) infusion (a fluid or a medicine delivered into a vein by way of a needle) over 1 hour, once daily for 5 consecutive days of a 28 days cycle up to 8 cycles or continued until disease progression or unacceptable toxicity.
Overall Participants	37
Age (Years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [Years]	68.0 (14.1)
Sex: Female, Male (Count of Participants)
Female	11 29.7%
Male	26 70.3%

Outcome Measures

1. Primary Outcome

Title	Percentage of Participants With Response
Description	Percentage of participants with response: complete response (CR) or partial response (PR) according to International Working Group (IWG) 2006 criteria was evaluated. CR in bone marrow is defined as<=to 5% myeloblasts with normal maturation of all cell lines and persistent dysplasia was noted in peripheral blood hemoglobin >=11 gram (g) per deciliter (dl), platelets >=100*10^9 liter (l), neutrophils >=1.0*10^9 l, Blasts 0%. Partial response is defined as all CR criteria if abnormal before treatment except: bone marrow blasts decreased by >=50% over pre-treatment but still >=5%.
Time Frame	Day 1 of Cycle 2, 4, 6, 8; each Cycle of 28 days and End of treatment (30-42 days after Cycle 8 or early withdrawal)

Outcome Measure Data

Analysis Population Description

The efficacy-evaluable (EE) population included all participants who received at least 2 cycles of treatment. Participants who died before receiving 2 complete cycles or were taken off study due to progressive disease were included. Here 'N' specifies those participants who were evaluable for this outcome measure.

Arm/Group Title	Decitabine
Arm/Group Description	Decitabine 20 milligram per square meter (mg per m^2) will be administered intravenous (into a vein) infusion (a fluid or a medicine delivered into a vein by way of a needle) over 1 hour, once daily for 5 consecutive days of a 28 days cycle up to 8 cycles or continued until disease progression or unacceptable toxicity.
Measure Participants	34
Number [Percentage of Participants]	23.5 63.5%

2. Secondary Outcome

Title	Percentage of Participants With Hematologic Treatment Response
Description	Hematologic treatment response was assessed as per IWG 2006 criteria. This is measured in erythroid(HI-E), platelet(HI-P) and neutrophil(HI-N) lineages. HI-E response(pre-treatment<11 gram per deciliter [g/dl]):hemoglobin increase by>=1.5 g/dl and relevant reduction in RBC transfusions by 4 RBC transfusions/8week. HI-P response (pre-treatment<100*109/l):absolute increase of >=30*10^9/l for participants starting with>20*10^9/l and increase from <20*10^9/l to>20*10^9/l and by at least 100%. HI-N response (pre-treatment<1.0*10^9/l): at least 100% increase and an absolute increase >0.5*10^9/l.
Time Frame	Day 1 of Cycle 1, 2, 3, 4, 5, 6, 7 and 8, each Cycle of 28 days and End of treatment (30-42 days after Cycle 8 or early withdrawal)

Outcome Measure Data

Analysis Population Description

Intent-to-treat (ITT) population was defined as all participants who had received at least one dose of treatment. Here 'N' specifies those participants who were evaluable for this outcome measure and 'n' specifies those participants who were evaluable for this outcome measure at given time point.

Arm/Group Title	Decitabine
Arm/Group Description	Decitabine 20 milligram per square meter (mg per m^2) will be administered intravenous (into a vein) infusion (a fluid or a medicine delivered into a vein by way of a needle) over 1 hour, once daily for 5 consecutive days of a 28 days cycle up to 8 cycles or continued until disease progression or unacceptable toxicity.
Measure Participants	34
Cycle 1: HI-E	5.9 15.9%
Cycle 1: HI-P	8.8 23.8%
Cycle 1: HI-N	2.9 7.8%
Cycle 2: HI-E	17.2 46.5%
Cycle 2: HI-P	27.6 74.6%
Cycle 2: HI-N	6.9 18.6%
Cycle 3: HI-E	28.6 77.3%
Cycle 3: HI-P	39.3 106.2%
Cycle 3: HI-N	17.9 48.4%
Cycle 4: HI-E	37.0 100%
Cycle 4: HI-P	33.3 90%
Cycle 4: HI-N	7.4 20%
Cycle 5: HI-E	40.9 110.5%
Cycle 5: HI-P	40.9 110.5%
Cycle 5: HI-N	13.6 36.8%
Cycle 6: HI-E	52.4 141.6%
Cycle 6: HI-P	61.9 167.3%
Cycle 6: HI-N	19.1 51.6%
Cycle 7: HI-E	47.1 127.3%
Cycle 7: HI-P	52.9 143%
Cycle 7: HI-N	5.9 15.9%
Cycle 8: HI-E	43.8 118.4%
Cycle 8: HI-P	56.3 152.2%
Cycle 8: HI-N	12.5 33.8%
End of treatment: HI-E	27.6 74.6%
End of treatment: HI-P	34.5 93.2%
End of treatment: HI-N	6.9 18.6%

3. Secondary Outcome

Title	Percentage of Participants With Cytogenetic Response
Description	Cytogenetic responses was assessed as per IWG 2006 criteria which define complete response as disappearance of the chromosomal abnormality without appearance of new ones and partial response as at least 50 percent reduction of the chromosomal abnormality.
Time Frame	Day 1 of Cycle 2, 4, 6, 8; each Cycle of 28 days and End of treatment (30-42 days after Cycle 8 or early withdrawal)

Outcome Measure Data

Analysis Population Description

ITT population was defined as all participants who had received at least one dose of treatment. Here 'N' specifies those participants who were evaluated for this outcome measure and 'n' specifies those participants who were evaluated at given time point.

Arm/Group Title	Decitabine
Arm/Group Description	Decitabine 20 milligram per square meter (mg per m^2) will be administered intravenous (into a vein) infusion (a fluid or a medicine delivered into a vein by way of a needle) over 1 hour, once daily for 5 consecutive days of a 28 days cycle up to 8 cycles or continued until disease progression or unacceptable toxicity.
Measure Participants	20
Cycle 2, Complete Response (n=18)	22.2 60%
Cycle 2, Partial Response (n=18)	0.0 0%
Cycle 4, Complete Response (n=20)	20.0 54.1%
Cycle 4, Partial Response (n=20)	25.0 67.6%
Cycle 6, Complete Response (n=11)	18.2 49.2%
Cycle 6, Partial Response (n=11)	27.3 73.8%
Cycle 8, Complete Response (n=6)	0.0 0%
Cycle 8, Partial Response (n=6)	0.0 0%
End of treatment, Complete Response (n=17)	11.8 31.9%
End of treatment, Partial Response (n=17)	5.9 15.9%

4. Secondary Outcome

Title	Time to Acute Myeloid Leukemia (AML) Progression or Death
Description	Time to AML is defined as greater than 30 percent of blasts in bone marrow or the time to death was calculated from the date of treatment start until disease progression to AML or until death, which ever occurred first. Participants who were still alive and did not progress to AML were censored at the moment of last visit.
Time Frame	Start of treatment until disease progression or death (whichever occur first) or up to 736 days

Outcome Measure Data

Analysis Population Description

ITT population was defined as all participants who had received at least 1 dose of study medication.

Arm/Group Title	Decitabine
Arm/Group Description	Decitabine 20 milligram per square meter (mg per m^2) will be administered intravenous (into a vein) infusion (a fluid or a medicine delivered into a vein by way of a needle) over 1 hour, once daily for 5 consecutive days of a 28 days cycle up to 8 cycles or continued until disease progression or unacceptable toxicity.
Measure Participants	37
Median (Full Range) [Months]	22.8

5. Secondary Outcome

Title	Overall Survival
Description	Overall survival was defined as the time from the date of treatment start until death (whatever the cause). It was calculated from Kaplan-Meier estimates. Participants still alive were censored at the moment of last visit or contact.
Time Frame	Start of treatment until disease progression or death (whichever occur first) or up to 736 days

Outcome Measure Data

Analysis Population Description

ITT population was defined as all participants who had received at least 1 dose of study medication.

Arm/Group Title	Decitabine
Arm/Group Description	Decitabine 20 milligram per square meter (mg per m^2) will be administered intravenous (into a vein) infusion (a fluid or a medicine delivered into a vein by way of a needle) over 1 hour, once daily for 5 consecutive days of a 28 days cycle up to 8 cycles or continued until disease progression or unacceptable toxicity.
Measure Participants	37
Median (Full Range) [Months]	22.8

6. Secondary Outcome

Title	Percentage of Participants With Transfusion Dependency
Description	Transfusion requirements for both red blood cells as well as platelets were recorded for each participant.
Time Frame	8 weeks before first dose until disease progression or death (whichever occur first) or up to 736 days

Outcome Measure Data

Analysis Population Description

ITT population was defined as all participants who had received at least 1 dose of study medication.

Arm/Group Title	Decitabine
Arm/Group Description	Decitabine 20 milligram per square meter (mg per m^2) will be administered intravenous (into a vein) infusion (a fluid or a medicine delivered into a vein by way of a needle) over 1 hour, once daily for 5 consecutive days of a 28 days cycle up to 8 cycles or continued until disease progression or unacceptable toxicity.
Measure Participants	37
Number [Percentage of participants]	100 270.3%

7. Secondary Outcome

Title	Percentage of Participants With Transfusion Independency
Description	Transfusion independent participants were calculated from all the participants who required transfusion in the duration of 8 weeks before first dose until disease progression or death or up to 736 days. Transfusion independence was defined as lack of requirement for transfusions for at least 8 weeks.
Time Frame	8 weeks before first dose and 736 days of treatment

Outcome Measure Data

Analysis Population Description

ITT population was defined as all participants who had received at least 1 dose of study medication.

Arm/Group Title	Decitabine
Arm/Group Description	Decitabine 20 milligram per square meter (mg per m^2) will be administered intravenous (into a vein) infusion (a fluid or a medicine delivered into a vein by way of a needle) over 1 hour, once daily for 5 consecutive days of a 28 days cycle up to 8 cycles or continued until disease progression or unacceptable toxicity.
Measure Participants	37
Number [percentage of participants]	27 73%

8. Secondary Outcome

Title	Duration for Hospitalization
Description	Duration of hospitalization was calculated for each participant, using the sum of all hospital days by subtracting the date of discharge from the date of admission.
Time Frame	Cycle 1 up to Cycle 8, each cycle of 28 days.

Outcome Measure Data

Analysis Population Description

ITT population was defined as all participants who had received at least one dose of treatment. Here 'n' specifies those participants who were evaluated for this outcome measure at given time point.

Arm/Group Title	Decitabine
Arm/Group Description	Decitabine 20 milligram per square meter (mg per m^2) will be administered intravenous (into a vein) infusion (a fluid or a medicine delivered into a vein by way of a needle) over 1 hour, once daily for 5 consecutive days of a 28 days cycle up to 8 cycles or continued until disease progression or unacceptable toxicity.
Measure Participants	37
Cycle 1 (n=37)	9.0 (9.4)
Cycle 2 (n=30)	15.4 (16.2)
Cycle 3 (n=28)	13.3 (11.5)
Cycle 4 (n=28)	27.1 (52.9)
Cycle 5 (n=22)	22.5 (38.6)
Cycle 6 (n=21)	7.5 (5.0)
Cycle 7 (n=17)	33.3 (56.5)
Cycle 8 (n=16)	5.0 (0.0)

9. Secondary Outcome

Title	Number of Events Which Led to Hospitalization
Description	The events (reasons) for hospitalizations such as infection, transfusion, acute choleycystitis, allergic transfusion reaction, dyspnoea with right pleural effusion, febrile neutropenia, fever, for decitabine, Myelodysplastic Syndrome (MDS) hematuria, paronychia, pneumonia, heart failure, peri-anal abscess (PAA), pancytopenia,fluctuated neutropenia fever, right dorsal foot cellulitis, Right lower (Rt.Lw) lung pneumonia with impending respiratory (resp) failure, Serious adverse event (SAE)+schedule hospitalization for decitabine, septic shock and not available were reported.
Time Frame	Start of treatment until disease progression or death or up to Cycle 8, each cycle of 28 days

Outcome Measure Data

Analysis Population Description

ITT population was defined as all participants who had received at least 1 dose of study medication.

Arm/Group Title	Decitabine
Arm/Group Description	Decitabine 20 milligram per square meter (mg per m^2) will be administered intravenous (into a vein) infusion (a fluid or a medicine delivered into a vein by way of a needle) over 1 hour, once daily for 5 consecutive days of a 28 days cycle up to 8 cycles or continued until disease progression or unacceptable toxicity.
Measure Participants	37
Infection	10
Transfusion	4
Acute choleycystitis	1
Allergic transfusion reaction	1
Dyspnoea with right pleural effusion	4
Febrile neutropenia	16
Fever	4
For Decitabine	33
MDS hematuria	2
Paronychia, pneumonia, heart failure	1
PAA,pancytopenia,fluctuated neutropenia fever	1
Right dorsal foot cellulitis	1
Rt Lw lung pneumonia with impending resp. failure	1
SAE+Schedule hospitalization for Decitabine	4
Septic shock	1
Not available	36

10. Secondary Outcome

Title	Quality of Life Assessment
Description	The quality of life was assessed by the questionnaire QLQ C-30 designed by European Organization for the Research and Treatment of Cancer (EORTC). EORTC QLQ-C30 is a 30-item questionnaire to assess the overall quality of life in cancer participants. Most questions used 4-point scale (1=Not at All' to 4=Very Much') and 2 questions: Q29 on overall health and Q30 on overall quality of life uses 7-point scale ranging from 1=Very Poor to 7=Excellent. Higher score indicates better quality of life.
Time Frame	Day 1 of Cycle 1 and Cycle 8 (each cycle of 28 days)

Outcome Measure Data

Analysis Population Description

ITT population was defined as all participants who had received at least 1 dose of study medication.

Arm/Group Title	Decitabine
Arm/Group Description	Decitabine 20 milligram per square meter (mg per m^2) will be administered intravenous (into a vein) infusion (a fluid or a medicine delivered into a vein by way of a needle) over 1 hour, once daily for 5 consecutive days of a 28 days cycle up to 8 cycles or continued until disease progression or unacceptable toxicity.
Measure Participants	37
Baseline, Q29	4.4 (1.5)
Baseline, Q30	4.9 (1.3)
End of treatment, Q29	4.3 (1.7)
End of treatment, Q30	4.5 (1.8)

Adverse Events

Time Frame	Baseline up to 30 days after last medication
Arm/Group Title	Decitabine
Arm/Group Description	Decitabine 20 milligram per square meter (mg per m^2) will be administered intravenous (into a vein) infusion (a fluid or a medicine delivered into a vein by way of a needle) over 1 hour, once daily for 5 consecutive days of a 28 days cycle up to 8 cycles or continued until disease progression or unacceptable toxicity.
All Cause Mortality
	Decitabine
	Affected / at Risk (%)	# Events
Total	/ (NaN)
Serious Adverse Events
	Decitabine
	Affected / at Risk (%)	# Events
Total	28/37 (75.7%)
Blood and lymphatic system disorders
Febrile neutropenia	19/37 (51.4%)
Anaemia	3/37 (8.1%)
Pancytopenia	3/37 (8.1%)
Neutropenia	2/37 (5.4%)
Thrombocytopenia	2/37 (5.4%)
Bone marrow failure	1/37 (2.7%)
Leukopenia	1/37 (2.7%)
Lymphadenopathy	1/37 (2.7%)
Cardiac disorders
Cardiac failure	1/37 (2.7%)
Cardiac failure congestive	1/37 (2.7%)
Cardiac valve vegetation	1/37 (2.7%)
Gastrointestinal disorders
Abdominal pain	1/37 (2.7%)
Anal fistula	1/37 (2.7%)
Constipation	1/37 (2.7%)
Gastrointestinal haemorrhage	1/37 (2.7%)
Ileus	1/37 (2.7%)
General disorders
Pyrexia	5/37 (13.5%)
Multi-organ failure	3/37 (8.1%)
Induration	1/37 (2.7%)
Hepatobiliary disorders
Cholecystitis acute	1/37 (2.7%)
Hepatitis acute	1/37 (2.7%)
Immune system disorders
Anaphylactic shock	1/37 (2.7%)
Infections and infestations
Pneumonia	7/37 (18.9%)
Septic shock	4/37 (10.8%)
Anal abscess	2/37 (5.4%)
Cellulitis	2/37 (5.4%)
Pneumonia fungal	2/37 (5.4%)
Upper respiratory tract infection	2/37 (5.4%)
Bronchopneumonia	1/37 (2.7%)
Escherichia bacterial	1/37 (2.7%)
Escherichia bacteraemia	1/37 (2.7%)
Escherichia infection	1/37 (2.7%)
Fungaemia	1/37 (2.7%)
Hepatic infection	1/37 (2.7%)
Liver abscess	1/37 (2.7%)
Paronychia	1/37 (2.7%)
Pneumonia klebsiella	1/37 (2.7%)
Splenic abscess	1/37 (2.7%)
Splenic infection	1/37 (2.7%)
Urinary tract infection	1/37 (2.7%)
Metabolism and nutrition disorders
Hypercalcaemia	1/37 (2.7%)
Hypernatraemia	1/37 (2.7%)
Metabolic acidosis	1/37 (2.7%)
Renal and urinary disorders
Renal failure acute	2/37 (5.4%)
Urinary retention	1/37 (2.7%)
Respiratory, thoracic and mediastinal disorders
Acute respiratory failure	3/37 (8.1%)
Dyspnoea	1/37 (2.7%)
Oropharyngeal pain	1/37 (2.7%)
Pleural effusion	1/37 (2.7%)
Pneumonia aspiration	1/37 (2.7%)
Pulmonary embolism	1/37 (2.7%)
Pulmonary oedema	1/37 (2.7%)
Skin and subcutaneous tissue disorders
Ecchymosis	1/37 (2.7%)
Vascular disorders
Deep vein thrombosis	2/37 (5.4%)
Shock	1/37 (2.7%)
Other (Not Including Serious) Adverse Events
	Decitabine
	Affected / at Risk (%)	# Events
Total	37/37 (100%)
Blood and lymphatic system disorders
Leucopenia	28/37 (75.7%)
Anaemia	23/37 (62.2%)
Neutropenia	22/37 (59.5%)
Thrombocytopenia	20/37 (54.1%)
Febrile neutropenia	2/37 (5.4%)
Lymphopenia	7/37 (18.9%)
White blood cell disorder	5/37 (13.5%)
Pancytopenia	4/37 (10.8%)
Lymphadenopathy	2/37 (5.4%)
Platelet disorder	3/37 (8.1%)
Lymphadenitis	2/37 (5.4%)
Splenomegaly	2/37 (5.4%)
Cardiac disorders
Cardiomegaly	3/37 (8.1%)
Palpitations	3/37 (8.1%)
Bradycardia	2/37 (5.4%)
Ear and labyrinth disorders
Ear pain	2/37 (5.4%)
Tinnitus	2/37 (5.4%)
Eye disorders
Conjunctival haemorrhage	4/37 (10.8%)
Conjunctivitis	3/37 (8.1%)
Gastrointestinal disorders
Nausea	14/37 (37.8%)
Constipation	11/37 (29.7%)
Diarrhoea	12/37 (32.4%)
Mouth ulceration	12/37 (32.4%)
Vomiting	10/37 (27%)
Abdominal distension	8/37 (21.6%)
Gingival bleeding	7/37 (18.9%)
Abdominal pain	6/37 (16.2%)
Haemorrhoids	6/37 (16.2%)
Abdominal pain upper	5/37 (13.5%)
Mouth haemorrhage	5/37 (13.5%)
Melaena	4/37 (10.8%)
Gastrointestinal haemorrhage	2/37 (5.4%)
Gingival pain	3/37 (8.1%)
Haematochezia	3/37 (8.1%)
Stomatitis	3/37 (8.1%)
Abdominal discomfort	2/37 (5.4%)
Dysgeusia	2/37 (5.4%)
Reflux oesophagitis	2/37 (5.4%)
Dyspepsia	2/37 (5.4%)
Gingivitis	2/37 (5.4%)
Tongue haemorrhage	2/37 (5.4%)
Periodontitis	2/37 (5.4%)
General disorders
Pyrexia	13/37 (35.1%)
Fatigue	13/37 (35.1%)
Malaise	13/37 (35.1%)
Oedema peripheral	13/37 (35.1%)
Asthenia	5/37 (13.5%)
Chest discomfort	4/37 (10.8%)
Chest pain	4/37 (10.8%)
Oedema	4/37 (10.8%)
Chills	3/37 (8.1%)
Pain	3/37 (8.1%)
Hyperhidrosis	2/37 (5.4%)
Ulcer	2/37 (5.4%)
Hepatobiliary disorders
Hepatic cyst	3/37 (8.1%)
Jaundice	2/37 (5.4%)
Immune system disorders
Hypersensitivity	2/37 (5.4%)
Infections and infestations
Pneumonia	3/37 (8.1%)
Upper respiratory tract infection	8/37 (21.6%)
Cellulitis	3/37 (8.1%)
Septic shock	5/37 (13.5%)
Herpes simplex	4/37 (10.8%)
Urinary tract infection	3/37 (8.1%)
Oral Candidiasis	3/37 (8.1%)
Paronychia	2/37 (5.4%)
Sepsis	3/37 (8.1%)
Tinea pedis	3/37 (8.1%)
Onychomycosis	2/37 (5.4%)
Injury, poisoning and procedural complications
Transfusion reaction	7/37 (18.9%)
Wound complication	4/37 (10.8%)
Contusion	3/37 (8.1%)
Allergic transfusion reaction	2/37 (5.4%)
Procedural pain	2/37 (5.4%)
Investigations
Weight decreased	6/37 (16.2%)
Alanine aminotransferase increased	5/37 (13.5%)
Aspartate aminotransferase increased	4/37 (10.8%)
Haemoglobin	4/37 (10.8%)
Monocyte count decreased	3/37 (8.1%)
Basophil count increased	2/37 (5.4%)
Blood alkaline phosphatase increased	2/37 (5.4%)
Blood creatinine increased	2/37 (5.4%)
Cardiac murmur	2/37 (5.4%)
Eosinophil count increased	2/37 (5.4%)
Lymphocyte count increased	2/37 (5.4%)
Neutrophil count	2/37 (5.4%)
Urine output decrease	2/37 (5.4%)
Weight increased	2/37 (5.4%)
Metabolism and nutrition disorders
Decreased appetite	14/37 (37.8%)
Hypokalaemia	10/37 (27%)
Hyperglycaemia	7/37 (18.9%)
Hypoalbuminaemia	6/37 (16.2%)
Hyponatraemia	5/37 (13.5%)
Hyperbilirubinaemia	4/37 (10.8%)
Hyperkalaemia	3/37 (8.1%)
Hypocalcaemia	2/37 (5.4%)
Increased appetite	2/37 (5.4%)
Musculoskeletal and connective tissue disorders
Arthralgia	10/37 (27%)
Myalgia	6/37 (16.2%)
Back pain	5/37 (13.5%)
Osteoarthritis	4/37 (10.8%)
Pain in extremity	4/37 (10.8%)
Muscle spasms	3/37 (8.1%)
Neck pain	3/37 (8.1%)
Spinal osteoarthritis	3/37 (8.1%)
Flank pain	2/37 (5.4%)
Nervous system disorders
Dizziness	14/37 (37.8%)
Headache	7/37 (18.9%)
Hypoaesthesia	3/37 (8.1%)
Poor quality sleep	2/37 (5.4%)
Vertigo	2/37 (5.4%)
Psychiatric disorders
Insomnia	8/37 (21.6%)
Anxiety	5/37 (13.5%)
Depressed mood	3/37 (8.1%)
Renal and urinary disorders
Renal failure acute	2/37 (5.4%)
Dysuria	3/37 (8.1%)
Respiratory, thoracic and mediastinal disorders
Cough	14/37 (37.8%)
Oropharyngeal pain	9/37 (24.3%)
Rhinorrhoea	9/37 (24.3%)
Dyspnoea	6/37 (16.2%)
Haemoptysis	5/37 (13.5%)
Dyspnoea exertional	4/37 (10.8%)
Pleural effusion	4/37 (10.8%)
Acute tonsillitis	3/37 (8.1%)
Nasal congestion	3/37 (8.1%)
Epistaxis	2/37 (5.4%)
Productive cough	2/37 (5.4%)
Sneezing	2/37 (5.4%)
Skin and subcutaneous tissue disorders
Rash	11/37 (29.7%)
Ecchymosis	5/37 (13.5%)
Pruritus	6/37 (16.2%)
Erythema	3/37 (8.1%)
Alopecia	2/37 (5.4%)
Decubitus ulcer	2/37 (5.4%)
Vascular disorders
Petechiae	5/37 (13.5%)
Haematoma	3/37 (8.1%)
Hypertension	2/37 (5.4%)
Hypotension	2/37 (5.4%)

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.