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Genomic Predictors of Decitabine Response in Patients With Acute Myeloid Leukemia or Myelodysplastic Syndromes

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01687400
Recruitment Status : Completed
First Posted : September 18, 2012
Results First Posted : October 2, 2018
Last Update Posted : October 2, 2018
Sponsor:
Information provided by (Responsible Party):
Washington University School of Medicine

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Leukemia, Myeloid, Acute
Myelodysplastic Syndromes
Intervention Drug: decitabine
Enrollment 114
Recruitment Details The study opened to participant enrollment on 02/12/2013 and closed to participant enrollment on 06/19/2017.
Pre-assignment Details  
Arm/Group Title Decitabine
Hide Arm/Group Description Patients receive decitabine IV over 1 hour on days 1-10 of a 28-day cycle. Treatment continues for 2 cycles. Patients then receive decitabine IV over 1 hour on days 1-10, 1-5, or 1-3 (depending on response). Treatment continues in the absence of disease progression or unacceptable toxicity.
Period Title: Overall Study
Started 114
Completed 114
Not Completed 0
Arm/Group Title Decitabine
Hide Arm/Group Description Patients receive decitabine IV over 1 hour on days 1-10 of a 28-day cycle. Treatment continues for 2 cycles. Patients then receive decitabine IV over 1 hour on days 1-10, 1-5, or 1-3 (depending on response). Treatment continues in the absence of disease progression or unacceptable toxicity.
Overall Number of Baseline Participants 114
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 114 participants
73
(31 to 88)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 114 participants
Female
48
  42.1%
Male
66
  57.9%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 114 participants
Hispanic or Latino
1
   0.9%
Not Hispanic or Latino
90
  78.9%
Unknown or Not Reported
23
  20.2%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 114 participants
American Indian or Alaska Native
0
   0.0%
Asian
2
   1.8%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
6
   5.3%
White
105
  92.1%
More than one race
0
   0.0%
Unknown or Not Reported
1
   0.9%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 114 participants
114
1.Primary Outcome
Title Correlation of Patient Specific Mutations With Overall Response Rate
Hide Description

-Best response after 4 treatment cycles as assessed according to International Working Group (IWG) criteria; bone marrow for gene sequencing will be collected at baseline; mutations will be correlated with overall response rate

--Complete remission (CR), Complete remission with incomplete hematologic recovery (CRi), Marrow complete remission (mCR), Partial remission (PR), Stable disease (SD), Progressive disease (PD)

Time Frame 4 months (4 treatment cycles)
Hide Outcome Measure Data
Hide Analysis Population Description
-Some participants were not evaluable for this outcome measure due to sample collection quality issues and if they had repeat bone marrow biopsies and could be evaluated for responses.
Arm/Group Title Decitabine (Participants With Response of CR/CRi/mCR/PR) Decitabine (Participants With Response of SD/PD)
Hide Arm/Group Description:
Patients receive decitabine IV over 1 hour on days 1-10 of a 28-day cycle. Treatment continues for 2 cycles. Patients then receive decitabine IV over 1 hour on days 1-10, 1-5, or 1-3 (depending on response). Treatment continues in the absence of disease progression or unacceptable toxicity.
Patients receive decitabine IV over 1 hour on days 1-10 of a 28-day cycle. Treatment continues for 2 cycles. Patients then receive decitabine IV over 1 hour on days 1-10, 1-5, or 1-3 (depending on response). Treatment continues in the absence of disease progression or unacceptable toxicity.
Overall Number of Participants Analyzed 64 22
Measure Type: Count of Participants
Unit of Measure: Participants
TP53 Number Analyzed 64 participants 17 participants
26
  40.6%
3
  17.6%
ASXL1 Number Analyzed 64 participants 17 participants
9
  14.1%
2
  11.8%
SRSF2 Number Analyzed 64 participants 17 participants
7
  10.9%
5
  29.4%
IDH2 Number Analyzed 64 participants 17 participants
6
   9.4%
5
  29.4%
DNMT3A Number Analyzed 64 participants 17 participants
8
  12.5%
5
  29.4%
SF3B1 Number Analyzed 64 participants 17 participants
7
  10.9%
0
   0.0%
RUNX1 Number Analyzed 45 participants 15 participants
5
  11.1%
3
  20.0%
TET2 Number Analyzed 45 participants 15 participants
4
   8.9%
3
  20.0%
IDH1 Number Analyzed 64 participants 17 participants
2
   3.1%
3
  17.6%
NPM1 Number Analyzed 45 participants 15 participants
4
   8.9%
1
   6.7%
NRAS Number Analyzed 45 participants 15 participants
3
   6.7%
3
  20.0%
U2AF1 Number Analyzed 64 participants 17 participants
4
   6.3%
1
   5.9%
MY05B Number Analyzed 45 participants 15 participants
3
   6.7%
2
  13.3%
WT1 Number Analyzed 45 participants 15 participants
5
  11.1%
1
   6.7%
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Decitabine (Participants With Response of CR/CRi/mCR/PR), Decitabine (Participants With Response of SD/PD)
Comments Statistical analysis #1 is for the genetic mutation TP53.
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.035
Comments [Not Specified]
Method Fisher Exact
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Decitabine (Participants With Response of CR/CRi/mCR/PR), Decitabine (Participants With Response of SD/PD)
Comments Statistical analysis #2 is for ASXL1 genetic mutation
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.72
Comments [Not Specified]
Method Fisher Exact
Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Decitabine (Participants With Response of CR/CRi/mCR/PR), Decitabine (Participants With Response of SD/PD)
Comments Statistical analysis #3 is for SRSF2 genetic mutation
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.28
Comments [Not Specified]
Method Fisher Exact
Comments [Not Specified]
Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Decitabine (Participants With Response of CR/CRi/mCR/PR), Decitabine (Participants With Response of SD/PD)
Comments Statistical analysis #4 is for IDH2 genetic mutation
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.14
Comments [Not Specified]
Method Fisher Exact
Comments [Not Specified]
Hide Statistical Analysis 5
Statistical Analysis Overview Comparison Group Selection Decitabine (Participants With Response of CR/CRi/mCR/PR), Decitabine (Participants With Response of SD/PD)
Comments Statistical analysis #5 is for DNMT3A genetic mutation
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.3
Comments [Not Specified]
Method Fisher Exact
Comments [Not Specified]
Hide Statistical Analysis 6
Statistical Analysis Overview Comparison Group Selection Decitabine (Participants With Response of CR/CRi/mCR/PR), Decitabine (Participants With Response of SD/PD)
Comments Statistical analysis #6 is for SF3B1 genetic mutation
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.183
Comments [Not Specified]
Method Fisher Exact
Comments [Not Specified]
Hide Statistical Analysis 7
Statistical Analysis Overview Comparison Group Selection Decitabine (Participants With Response of CR/CRi/mCR/PR), Decitabine (Participants With Response of SD/PD)
Comments Statistical analysis #7 is for RUNX1 genetic mutation
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.42
Comments [Not Specified]
Method Fisher Exact
Comments [Not Specified]
Hide Statistical Analysis 8
Statistical Analysis Overview Comparison Group Selection Decitabine (Participants With Response of CR/CRi/mCR/PR), Decitabine (Participants With Response of SD/PD)
Comments Statistical analysis #8 is for TET2 genetic mutation
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.36
Comments [Not Specified]
Method Fisher Exact
Comments [Not Specified]
Hide Statistical Analysis 9
Statistical Analysis Overview Comparison Group Selection Decitabine (Participants With Response of CR/CRi/mCR/PR), Decitabine (Participants With Response of SD/PD)
Comments Statistical analysis #9 is for IDH1 genetic mutation
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.1
Comments [Not Specified]
Method Fisher Exact
Comments [Not Specified]
Hide Statistical Analysis 10
Statistical Analysis Overview Comparison Group Selection Decitabine (Participants With Response of CR/CRi/mCR/PR), Decitabine (Participants With Response of SD/PD)
Comments Statistical analysis #10 is for NPM1 genetic mutation
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 1
Comments [Not Specified]
Method Fisher Exact
Comments [Not Specified]
Hide Statistical Analysis 11
Statistical Analysis Overview Comparison Group Selection Decitabine (Participants With Response of CR/CRi/mCR/PR), Decitabine (Participants With Response of SD/PD)
Comments Statistical analysis #11 is for NRAS genetic mutation
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.17
Comments [Not Specified]
Method Fisher Exact
Comments [Not Specified]
Hide Statistical Analysis 12
Statistical Analysis Overview Comparison Group Selection Decitabine (Participants With Response of CR/CRi/mCR/PR), Decitabine (Participants With Response of SD/PD)
Comments -Statistical analysis #12 is for U2AF1 genetic mutation
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 1
Comments [Not Specified]
Method Fisher Exact
Comments [Not Specified]
Hide Statistical Analysis 13
Statistical Analysis Overview Comparison Group Selection Decitabine (Participants With Response of CR/CRi/mCR/PR), Decitabine (Participants With Response of SD/PD)
Comments Statistical analysis #13 is for MY05B genetic mutation
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.6
Comments [Not Specified]
Method Fisher Exact
Comments [Not Specified]
Hide Statistical Analysis 14
Statistical Analysis Overview Comparison Group Selection Decitabine (Participants With Response of CR/CRi/mCR/PR), Decitabine (Participants With Response of SD/PD)
Comments Statistical analysis #14 is for WT1 genetic mutation
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 1
Comments [Not Specified]
Method Fisher Exact
Comments [Not Specified]
2.Secondary Outcome
Title Compare Outcomes of a 10-day Decitabine Per Cycle Regimen to a 5-day Regimen (Historical Controls)
Hide Description The overall response rate (CR/CRi/mCR/PR) and complete response rate (CR/CRi/mCR) will be compared with historical controls. Response assessed according to IWG criteria.
Time Frame 4 months (4 treatment cycles)
Hide Outcome Measure Data
Hide Analysis Population Description
-Participants were evaluable for this outcome measure if they completed at least one cycle of treatment and the cycle 1 day 28 bone marrow biopsy to assess response
Arm/Group Title Decitabine
Hide Arm/Group Description:
Patients receive decitabine IV over 1 hour on days 1-10 of a 28-day cycle. Treatment continues for 2 cycles. Patients then receive decitabine IV over 1 hour on days 1-10, 1-5, or 1-3 (depending on response). Treatment continues in the absence of disease progression or unacceptable toxicity.
Overall Number of Participants Analyzed 98
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
Overall response rate (CR, CRi/mCR/PR)
74.42
(65.20 to 83.64)
Complete response rate (CR, CRi, mCR)
63.95
(53.81 to 74.10)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Decitabine
Comments -The historical control of a 5-day regimen (Cashen et al 2010 JCO = NCT00358644) showed 25% (14 out of 55 participants) in overall response rate
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Chi-squared
Comments 1-sample Chi-square test to compare the overall response rate (ORR) to historical control (with ORR=0.25)
Method of Estimation Estimation Parameter Overall Response Rate-for current study
Estimated Value 0.744
Confidence Interval (2-Sided) 95%
0.652 to 0.836
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Decitabine
Comments -The historical control of a 5-day regimen (Cashen et al 2010 JCO = NCT00358644) showed 24% (13 out of 55 participants) in complete response rate.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Chi-squared
Comments 1-sample Chi-square test to compare the complete response rate to historical control (with CR=0.24)
Method of Estimation Estimation Parameter Complete response rate-for current study
Estimated Value 0.640
Confidence Interval (2-Sided) 95%
0.538 to 0.741
Estimation Comments [Not Specified]
3.Secondary Outcome
Title Rate of Mutation Clearance During Treatment
Hide Description Samples collected at baseline and after 10, 28 and 56 days of therapy; the rate of mutation clearance was measured as mean VAF change per day of treatment and was estimated using linear mixed model for repeated measurement data .
Time Frame Up to Day 56
Hide Outcome Measure Data
Hide Analysis Population Description
-The first 39 cases with adequate samples were serially evaluated with enhanced exome sequencing. The investigators evaluated 15 additional cases using gene panel sequencing.
Arm/Group Title Decitabine
Hide Arm/Group Description:
Patients receive decitabine IV over 1 hour on days 1-10 of a 28-day cycle. Treatment continues for 2 cycles. Patients then receive decitabine IV over 1 hour on days 1-10, 1-5, or 1-3 (depending on response). Treatment continues in the absence of disease progression or unacceptable toxicity.
Overall Number of Participants Analyzed 54
Mean (Standard Deviation)
Unit of Measure: proportion of variant alleles per day
TP53 Number Analyzed 16 participants
-0.781  (0.4423)
SRSF2 Number Analyzed 10 participants
-0.2214  (0.2705)
DNMT3A Number Analyzed 10 participants
-0.2122  (0.2817)
IDH2 Number Analyzed 9 participants
-0.08344  (0.2841)
RUNX1 Number Analyzed 8 participants
-0.2641  (0.6536)
TET2 Number Analyzed 9 participants
-0.07478  (0.3164)
ASXL1 Number Analyzed 7 participants
-0.3898  (0.3791)
IDH1 Number Analyzed 5 participants
-0.1103  (0.196)
NRAS Number Analyzed 5 participants
0.04544  (0.2554)
SF3B1 Number Analyzed 5 participants
-0.719  (0.3481)
4.Secondary Outcome
Title Peripheral Blood Decitabine Plasma Levels
Hide Description
  • To determine whether steady state serum concentrations of decitabine correlated with responses
  • Complete remission (CR), Complete remission with incomplete hematologic recovery (CRi), Partial remission (PR), Stable disease (SD), Progressive disease (PD), Not applicable (NA) - assessed according to International Working Group (IWG) criteria
Time Frame Day 4
Hide Outcome Measure Data
Hide Analysis Population Description
The first 45 participants enrolled with adequate samples were analyzed using GC-MS quantification of serum decitabine levels. Sufficient funds were lacking to complete the analysis of additional participants and all participants with data analyzed are reported.
Arm/Group Title Decitabine
Hide Arm/Group Description:
Patients receive decitabine IV over 1 hour on days 1-10 of a 28-day cycle. Treatment continues for 2 cycles. Patients then receive decitabine IV over 1 hour on days 1-10, 1-5, or 1-3 (depending on response). Treatment continues in the absence of disease progression or unacceptable toxicity.
Overall Number of Participants Analyzed 45
Mean (Standard Deviation)
Unit of Measure: ng/ml
CR Number Analyzed 3 participants
140.5  (53.41)
CRi Number Analyzed 16 participants
117.2  (54.6)
PR Number Analyzed 5 participants
67.71  (56.18)
SD Number Analyzed 12 participants
145  (73.06)
PD Number Analyzed 3 participants
298.5  (213.3)
NA Number Analyzed 6 participants
99.64  (58.4)
5.Secondary Outcome
Title Change in Bone Marrow Methylcytosine
Hide Description -Change of total bone marrow deoxyribonucleic acid (DNA) methylcytosine from baseline to Day 10
Time Frame Baseline and Day 10
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Decitabine (CR/CRi/mCR) Decitabine (PR/SD/PD)
Hide Arm/Group Description:
Patients receive decitabine IV over 1 hour on days 1-10 of a 28-day cycle. Treatment continues for 2 cycles. Patients then receive decitabine IV over 1 hour on days 1-10, 1-5, or 1-3 (depending on response). Treatment continues in the absence of disease progression or unacceptable toxicity.
Patients receive decitabine IV over 1 hour on days 1-10 of a 28-day cycle. Treatment continues for 2 cycles. Patients then receive decitabine IV over 1 hour on days 1-10, 1-5, or 1-3 (depending on response). Treatment continues in the absence of disease progression or unacceptable toxicity.
Overall Number of Participants Analyzed 14 11
Mean (Standard Deviation)
Unit of Measure: proportion of methylcytosine
Day 0 0.53  (0) 0.5374  (0)
Day 10 0.4416  (0.0247) 0.4639  (0.035)
Time Frame -Adverse events were collected from the start of treatment until 30 days following the last day of study treatment (average 5 months) -All deaths were collected from enrollment through study completion date.
Adverse Event Reporting Description Grades 1-5 adverse events will be recorded for the first 50 enrolled participants. Grades 3-5 will be recorded for the remainder of enrolled participants (n=64).
 
Arm/Group Title Decitabine (First 50 Enrolled Patients) Decitabine (Remainder of Enrolled Patients n=64)
Hide Arm/Group Description Patients receive decitabine IV over 1 hour on days 1-10 of a 28-day cycle. Treatment continues for 2 cycles. Patients then receive decitabine IV over 1 hour on days 1-10, 1-5, or 1-3 (depending on response). Treatment continues in the absence of disease progression or unacceptable toxicity. Patients receive decitabine IV over 1 hour on days 1-10 of a 28-day cycle. Treatment continues for 2 cycles. Patients then receive decitabine IV over 1 hour on days 1-10, 1-5, or 1-3 (depending on response). Treatment continues in the absence of disease progression or unacceptable toxicity.
All-Cause Mortality
Decitabine (First 50 Enrolled Patients) Decitabine (Remainder of Enrolled Patients n=64)
Affected / at Risk (%) Affected / at Risk (%)
Total   38/50 (76.00%)   43/64 (67.19%) 
Hide Serious Adverse Events
Decitabine (First 50 Enrolled Patients) Decitabine (Remainder of Enrolled Patients n=64)
Affected / at Risk (%) Affected / at Risk (%)
Total   42/50 (84.00%)   47/64 (73.44%) 
Blood and lymphatic system disorders     
Febrile neutropenia  1  23/50 (46.00%)  30/64 (46.88%) 
Cardiac disorders     
Atrial fibrillation  1  1/50 (2.00%)  1/64 (1.56%) 
Atrial flutter  1  0/50 (0.00%)  1/64 (1.56%) 
Cardiac arrest  1  1/50 (2.00%)  0/64 (0.00%) 
Heart failure  1  0/50 (0.00%)  1/64 (1.56%) 
NSTEMI  1  0/50 (0.00%)  1/64 (1.56%) 
Pericardial effusion  1  1/50 (2.00%)  0/64 (0.00%) 
STEMI  1  0/50 (0.00%)  2/64 (3.13%) 
Eye disorders     
Central retinal venous occlusion  1  1/50 (2.00%)  0/64 (0.00%) 
Gastrointestinal disorders     
Colitis  1  0/50 (0.00%)  3/64 (4.69%) 
Constipation  1  0/50 (0.00%)  2/64 (3.13%) 
Diarrhea  1  1/50 (2.00%)  2/64 (3.13%) 
Hematemesis  1  1/50 (2.00%)  1/64 (1.56%) 
Ileus  1  1/50 (2.00%)  0/64 (0.00%) 
Mucositis - oral  1  0/50 (0.00%)  1/64 (1.56%) 
General disorders     
Edema  1  1/50 (2.00%)  0/64 (0.00%) 
Failure to thrive  1  0/50 (0.00%)  1/64 (1.56%) 
Fever  1  1/50 (2.00%)  0/64 (0.00%) 
Infusion related reaction  1  2/50 (4.00%)  1/64 (1.56%) 
Malaise  1  1/50 (2.00%)  0/64 (0.00%) 
Pain  1  1/50 (2.00%)  0/64 (0.00%) 
Progressive disease/death  1  5/50 (10.00%)  2/64 (3.13%) 
Infections and infestations     
Bacteremia - Coagulase negative staphylococcus  1  0/50 (0.00%)  2/64 (3.13%) 
Bacteremia - E. coli  1  0/50 (0.00%)  2/64 (3.13%) 
Bacteremia - ESBL E. coli  1  1/50 (2.00%)  0/64 (0.00%) 
Bacteremia - Enterococcus faecium  1  1/50 (2.00%)  0/64 (0.00%) 
Bacteremia - Klebsiella pneumoniae  1  0/50 (0.00%)  1/64 (1.56%) 
Bacteremia - Micrococcus  1  0/50 (0.00%)  1/64 (1.56%) 
Bacteremia - Staphylococcus capitis  1  0/50 (0.00%)  1/64 (1.56%) 
Bacteremia - Staphylococcus epidermis  1  0/50 (0.00%)  1/64 (1.56%) 
Bacteremia - Staphylococcus infection  1  0/50 (0.00%)  1/64 (1.56%) 
Bacteremia - Streptococcus  1  0/50 (0.00%)  2/64 (3.13%) 
Bacteremia - Streptococcus mitis  1  0/50 (0.00%)  4/64 (6.25%) 
Bacteremia - VRE  1  0/50 (0.00%)  1/64 (1.56%) 
Bacteremia - gram negative  1  1/50 (2.00%)  0/64 (0.00%) 
Bone infection - Osteomyelitis  1  1/50 (2.00%)  0/64 (0.00%) 
C. difficile  1  0/50 (0.00%)  2/64 (3.13%) 
Catheter related infection  1  4/50 (8.00%)  2/64 (3.13%) 
Cellulitis infection/pneumonia infection  1  0/50 (0.00%)  1/64 (1.56%) 
Dental infection  1  1/50 (2.00%)  0/64 (0.00%) 
Diverticulitis  1  0/50 (0.00%)  1/64 (1.56%) 
Diverticulitis infection  1  0/50 (0.00%)  2/64 (3.13%) 
Endovascular infection  1  0/50 (0.00%)  1/64 (1.56%) 
Fusarium fungemia infection  1  0/50 (0.00%)  1/64 (1.56%) 
Iletitis  1  0/50 (0.00%)  1/64 (1.56%) 
Lung infection  1  4/50 (8.00%)  14/64 (21.88%) 
Parainfluenza infection  1  1/50 (2.00%)  0/64 (0.00%) 
Sepsis  1  3/50 (6.00%)  7/64 (10.94%) 
Septic arthritis  1  1/50 (2.00%)  0/64 (0.00%) 
Sinusitis  1  0/50 (0.00%)  2/64 (3.13%) 
Skin infection  1  3/50 (6.00%)  5/64 (7.81%) 
Submandicular sialadenitis infection  1  0/50 (0.00%)  1/64 (1.56%) 
Tooth infection  1  2/50 (4.00%)  0/64 (0.00%) 
Upper respiratory infection  1  0/50 (0.00%)  2/64 (3.13%) 
Upper respiratory infection - parainfluenza  1  1/50 (2.00%)  0/64 (0.00%) 
Urinary tract infection  1  1/50 (2.00%)  3/64 (4.69%) 
Bacteremia - Streptococcus mitis/oralis  1  0/50 (0.00%)  1/64 (1.56%) 
Injury, poisoning and procedural complications     
Fall  1  2/50 (4.00%)  1/64 (1.56%) 
Investigations     
White blood cell count decreased  1  0/50 (0.00%)  1/64 (1.56%) 
Metabolism and nutrition disorders     
Hyponatremia  1  1/50 (2.00%)  0/64 (0.00%) 
Musculoskeletal and connective tissue disorders     
Arthralgia  1  0/50 (0.00%)  1/64 (1.56%) 
Back pain  1  1/50 (2.00%)  0/64 (0.00%) 
Myositis  1  0/50 (0.00%)  1/64 (1.56%) 
Nervous system disorders     
Intracranial hemorrhage  1  0/50 (0.00%)  1/64 (1.56%) 
Stroke  1  1/50 (2.00%)  1/64 (1.56%) 
Psychiatric disorders     
Confusion  1  1/50 (2.00%)  0/64 (0.00%) 
Renal and urinary disorders     
Acute kidney injury  1  2/50 (4.00%)  0/64 (0.00%) 
Hematuria  1  1/50 (2.00%)  1/64 (1.56%) 
Respiratory, thoracic and mediastinal disorders     
Acute hypoxemic respiratory failure  1  0/50 (0.00%)  1/64 (1.56%) 
Dyspnea  1  1/50 (2.00%)  0/64 (0.00%) 
Epistaxis  1  0/50 (0.00%)  5/64 (7.81%) 
Pleural effusion  1  1/50 (2.00%)  1/64 (1.56%) 
Pulmonary embolism  1  0/50 (0.00%)  2/64 (3.13%) 
Respiratory failure  1  1/50 (2.00%)  1/64 (1.56%) 
Respiratory syncytial virus  1  0/50 (0.00%)  1/64 (1.56%) 
Skin and subcutaneous tissue disorders     
Rash maculo-papular  1  1/50 (2.00%)  2/64 (3.13%) 
Sweet's syndrome  1  1/50 (2.00%)  1/64 (1.56%) 
Vascular disorders     
Hematoma  1  0/50 (0.00%)  1/64 (1.56%) 
Hypotension  1  1/50 (2.00%)  1/64 (1.56%) 
Thromboembolic event  1  3/50 (6.00%)  2/64 (3.13%) 
1
Term from vocabulary, CTCAE (4.0)
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Decitabine (First 50 Enrolled Patients) Decitabine (Remainder of Enrolled Patients n=64)
Affected / at Risk (%) Affected / at Risk (%)
Total   50/50 (100.00%)   64/64 (100.00%) 
Blood and lymphatic system disorders     
Anemia  1  14/50 (28.00%)  22/64 (34.38%) 
Febrile neutropenia  1  16/50 (32.00%)  13/64 (20.31%) 
Lymph node pain  1  1/50 (2.00%)  0/64 (0.00%) 
Submandibular lymphadenopathy  1  1/50 (2.00%)  0/64 (0.00%) 
Cardiac disorders     
Atrial fibrillation  1  4/50 (8.00%)  4/64 (6.25%) 
Atrial flutter  1  1/50 (2.00%)  0/64 (0.00%) 
Chest pain  1  2/50 (4.00%)  0/64 (0.00%) 
Electrocardiogram QT corrected interval prolonged  1  1/50 (2.00%)  0/64 (0.00%) 
Heart failure  1  1/50 (2.00%)  2/64 (3.13%) 
Left ventricular systolic dysfunction  1  1/50 (2.00%)  1/64 (1.56%) 
Myocardial infarction  1  2/50 (4.00%)  1/64 (1.56%) 
Palpitations  1  2/50 (4.00%)  0/64 (0.00%) 
Pericardial effusion  1  4/50 (8.00%)  0/64 (0.00%) 
Pericardial tamponade  1  1/50 (2.00%)  0/64 (0.00%) 
Sinus bradycardia  1  5/50 (10.00%)  0/64 (0.00%) 
Sinus tachycardia  1  18/50 (36.00%)  0/64 (0.00%) 
Supraventricular tachycardia  1  2/50 (4.00%)  1/64 (1.56%) 
Ventricular ectopy  1  0/50 (0.00%)  1/64 (1.56%) 
Ear and labyrinth disorders     
Ear congestion  1  2/50 (4.00%)  0/64 (0.00%) 
Ear pain  1  1/50 (2.00%)  0/64 (0.00%) 
Hearing impaired  1  1/50 (2.00%)  0/64 (0.00%) 
Eye disorders     
Blurred vision  1  4/50 (8.00%)  0/64 (0.00%) 
Conjunctivitis  1  1/50 (2.00%)  0/64 (0.00%) 
Diplopia  1  1/50 (2.00%)  0/64 (0.00%) 
Dry eye  1  1/50 (2.00%)  0/64 (0.00%) 
Floaters  1  1/50 (2.00%)  0/64 (0.00%) 
Photophobia  1  1/50 (2.00%)  0/64 (0.00%) 
Vision changes unspecified  1  1/50 (2.00%)  0/64 (0.00%) 
Vision loss partial (temporary)  1  1/50 (2.00%)  0/64 (0.00%) 
Gastrointestinal disorders     
Abdominal pain  1  12/50 (24.00%)  0/64 (0.00%) 
Anal mucositis  1  1/50 (2.00%)  0/64 (0.00%) 
Bloating  1  1/50 (2.00%)  0/64 (0.00%) 
Burping  1  1/50 (2.00%)  0/64 (0.00%) 
Constipation  1  25/50 (50.00%)  0/64 (0.00%) 
Dental caries  1  1/50 (2.00%)  0/64 (0.00%) 
Diarrhea  1  26/50 (52.00%)  0/64 (0.00%) 
Diverticulitis  1  0/50 (0.00%)  2/64 (3.13%) 
Diverticulosis  1  1/50 (2.00%)  0/64 (0.00%) 
Dry mouth  1  2/50 (4.00%)  0/64 (0.00%) 
Dyspepsia  1  3/50 (6.00%)  0/64 (0.00%) 
Dysphagia  1  5/50 (10.00%)  1/64 (1.56%) 
Esophagitis  1  1/50 (2.00%)  0/64 (0.00%) 
Fecal incontinence  1  4/50 (8.00%)  0/64 (0.00%) 
Gastric hemorrhage  1  1/50 (2.00%)  0/64 (0.00%) 
Gastrointestinal pain  1  1/50 (2.00%)  0/64 (0.00%) 
Hiatal hernia  1  3/50 (6.00%)  0/64 (0.00%) 
Hypothermia  1  1/50 (2.00%)  0/64 (0.00%) 
Ileus  1  1/50 (2.00%)  0/64 (0.00%) 
Lower gastrointestinal hemorrhage  1  1/50 (2.00%)  0/64 (0.00%) 
Mucositis - oral  1  33/50 (66.00%)  2/64 (3.13%) 
Nausea  1  28/50 (56.00%)  1/64 (1.56%) 
Oral hemorrhage  1  3/50 (6.00%)  1/64 (1.56%) 
Oral pain  1  5/50 (10.00%)  0/64 (0.00%) 
Pancreatic cyst  1  1/50 (2.00%)  0/64 (0.00%) 
Pancreatitis  1  0/50 (0.00%)  1/64 (1.56%) 
Small bowel obstruction  1  1/50 (2.00%)  0/64 (0.00%) 
Toothache  1  3/50 (6.00%)  1/64 (1.56%) 
Typhlitis  1  0/50 (0.00%)  1/64 (1.56%) 
Vomiting  1  19/50 (38.00%)  0/64 (0.00%) 
General disorders     
Chills  1  31/50 (62.00%)  0/64 (0.00%) 
Edema face  1  2/50 (4.00%)  0/64 (0.00%) 
Edema limbs  1  31/50 (62.00%)  2/64 (3.13%) 
Facial pain  1  1/50 (2.00%)  0/64 (0.00%) 
Fatigue  1  26/50 (52.00%)  7/64 (10.94%) 
Fever  1  2/50 (4.00%)  0/64 (0.00%) 
Gait disturbance  1  1/50 (2.00%)  0/64 (0.00%) 
Infusion related reaction  1  9/50 (18.00%)  1/64 (1.56%) 
Irritability  1  1/50 (2.00%)  0/64 (0.00%) 
Localized edema  1  1/50 (2.00%)  0/64 (0.00%) 
Malaise  1  3/50 (6.00%)  0/64 (0.00%) 
Non-cardiac chest pain  1  7/50 (14.00%)  0/64 (0.00%) 
Pain  1  15/50 (30.00%)  1/64 (1.56%) 
Immune system disorders     
Allergic reaction  1  5/50 (10.00%)  0/64 (0.00%) 
Infections and infestations     
Bacteremia (Gram negative)  1  1/50 (2.00%)  0/64 (0.00%) 
Bacteremia - Escherichia coli/Pseudomonus aeruginosa  1  1/50 (2.00%)  0/64 (0.00%) 
Bacteremia - Myobacterium abscessus  1  1/50 (2.00%)  0/64 (0.00%) 
Bacteremia - Pseudomonas aeruginosa  1  1/50 (2.00%)  1/64 (1.56%) 
Bacteremia - Staphylococcus epidermidis  1  2/50 (4.00%)  1/64 (1.56%) 
Bacteremia - Streptococcus mitis  1  0/50 (0.00%)  3/64 (4.69%) 
Bacteremia - Streptococcus mitis/Coagulase negative staphylococcus  1  0/50 (0.00%)  1/64 (1.56%) 
C. difficile  1  2/50 (4.00%)  0/64 (0.00%) 
Candidal intertrigo (genital)  1  1/50 (2.00%)  0/64 (0.00%) 
Catheter related infection  1  4/50 (8.00%)  6/64 (9.38%) 
Fungal abscess brain  1  0/50 (0.00%)  1/64 (1.56%) 
Fungal infection - spleen and liver  1  0/50 (0.00%)  1/64 (1.56%) 
Gastroenteritis (viral)  1  2/50 (4.00%)  0/64 (0.00%) 
Gum infection  1  1/50 (2.00%)  0/64 (0.00%) 
HSV infection  1  1/50 (2.00%)  0/64 (0.00%) 
Infection mouth lesion  1  1/50 (2.00%)  0/64 (0.00%) 
Infective myositis  1  0/50 (0.00%)  1/64 (1.56%) 
Joint infection  1  0/50 (0.00%)  1/64 (1.56%) 
Lung infection  1  17/50 (34.00%)  10/64 (15.63%) 
Oral buccosal infection  1  1/50 (2.00%)  0/64 (0.00%) 
Oral thrush  1  13/50 (26.00%)  0/64 (0.00%) 
Otitis externa  1  1/50 (2.00%)  0/64 (0.00%) 
Perineal folliculitis  1  0/50 (0.00%)  1/64 (1.56%) 
Preseptal cellulitis  1  0/50 (0.00%)  2/64 (3.13%) 
Salivary gland infection  1  0/50 (0.00%)  1/64 (1.56%) 
Sepsis  1  9/50 (18.00%)  1/64 (1.56%) 
Septic arthritis  1  1/50 (2.00%)  0/64 (0.00%) 
Sinusitis  1  4/50 (8.00%)  1/64 (1.56%) 
Skin infection  1  17/50 (34.00%)  1/64 (1.56%) 
Tooth infection  1  4/50 (8.00%)  0/64 (0.00%) 
Upper respiratory infection  1  4/50 (8.00%)  0/64 (0.00%) 
Urinary tract infection  1  2/50 (4.00%)  0/64 (0.00%) 
Vaginal infection  1  1/50 (2.00%)  0/64 (0.00%) 
Injury, poisoning and procedural complications     
Bruising  1  21/50 (42.00%)  0/64 (0.00%) 
Burn  1  1/50 (2.00%)  0/64 (0.00%) 
Fall  1  9/50 (18.00%)  2/64 (3.13%) 
Fracture  1  2/50 (4.00%)  0/64 (0.00%) 
Hemorrhage from bronchoscopy  1  0/50 (0.00%)  1/64 (1.56%) 
Hip pain following bone marrow biopsy  1  0/50 (0.00%)  1/64 (1.56%) 
Postoperative hemmorhage  1  3/50 (6.00%)  0/64 (0.00%) 
Skin abrasion  1  2/50 (4.00%)  0/64 (0.00%) 
Investigations     
Activated partial thromboplastin time prolonged  1  19/50 (38.00%)  5/64 (7.81%) 
Alanine aminotransferase increased  1  30/50 (60.00%)  2/64 (3.13%) 
Alkaline phosphatase increased  1  19/50 (38.00%)  1/64 (1.56%) 
Aspartate aminotransferase increased  1  26/50 (52.00%)  2/64 (3.13%) 
Blood bilirubin increased  1  19/50 (38.00%)  0/64 (0.00%) 
Cardiac troponin I increased  1  3/50 (6.00%)  0/64 (0.00%) 
Cardiac troponin T increased  1  1/50 (2.00%)  0/64 (0.00%) 
Creatinine increased  1  8/50 (16.00%)  0/64 (0.00%) 
Electrocardiogram QT corrected interval prolonged  1  6/50 (12.00%)  3/64 (4.69%) 
INR increased  1  28/50 (56.00%)  0/64 (0.00%) 
Lymphocyte count decreased  1  44/50 (88.00%)  45/64 (70.31%) 
Lymphocyte count increased  1  0/50 (0.00%)  1/64 (1.56%) 
Neutrophil count decreased  1  20/50 (40.00%)  38/64 (59.38%) 
Platelet count decreased  1  27/50 (54.00%)  47/64 (73.44%) 
Weight gain  1  1/50 (2.00%)  0/64 (0.00%) 
Weight loss  1  21/50 (42.00%)  0/64 (0.00%) 
White blood cell decreased  1  43/50 (86.00%)  54/64 (84.38%) 
Metabolism and nutrition disorders     
Acidosis  1  1/50 (2.00%)  0/64 (0.00%) 
Anorexia  1  22/50 (44.00%)  2/64 (3.13%) 
Dehydration  1  15/50 (30.00%)  6/64 (9.38%) 
Hypercalcemia  1  3/50 (6.00%)  0/64 (0.00%) 
Hyperglycemia  1  8/50 (16.00%)  13/64 (20.31%) 
Hyperkalemia  1  9/50 (18.00%)  0/64 (0.00%) 
Hypermagnesemia  1  5/50 (10.00%)  1/64 (1.56%) 
Hypernatremia  1  7/50 (14.00%)  1/64 (1.56%) 
Hyperuricemia  1  1/50 (2.00%)  1/64 (1.56%) 
Hypoalbuminemia  1  39/50 (78.00%)  2/64 (3.13%) 
Hypocalcemia  1  29/50 (58.00%)  1/64 (1.56%) 
Hypokalemia  1  29/50 (58.00%)  14/64 (21.88%) 
Hypomagnesemia  1  7/50 (14.00%)  0/64 (0.00%) 
Hyponatremia  1  37/50 (74.00%)  12/64 (18.75%) 
Hypophosphatemia  1  28/50 (56.00%)  13/64 (20.31%) 
Musculoskeletal and connective tissue disorders     
Arthralgia  1  10/50 (20.00%)  0/64 (0.00%) 
Back pain  1  10/50 (20.00%)  1/64 (1.56%) 
Bone pain  1  2/50 (4.00%)  0/64 (0.00%) 
Bone spur  1  1/50 (2.00%)  0/64 (0.00%) 
Degenerative disc disease  1  1/50 (2.00%)  0/64 (0.00%) 
Flank pain  1  0/50 (0.00%)  1/64 (1.56%) 
Generalized muscle weakness  1  24/50 (48.00%)  6/64 (9.38%) 
Hand cramps  1  2/50 (4.00%)  0/64 (0.00%) 
Joint effusion  1  0/50 (0.00%)  1/64 (1.56%) 
Muscle cramps  1  1/50 (2.00%)  0/64 (0.00%) 
Myalgia  1  3/50 (6.00%)  0/64 (0.00%) 
Neck pain  1  2/50 (4.00%)  0/64 (0.00%) 
Pain in extremity  1  10/50 (20.00%)  1/64 (1.56%) 
Nervous system disorders     
Burning sensation  1  1/50 (2.00%)  0/64 (0.00%) 
Cognitive impairment acute worsening from baseline  1  0/50 (0.00%)  1/64 (1.56%) 
Dizziness  1  15/50 (30.00%)  1/64 (1.56%) 
Dysarthria  1  1/50 (2.00%)  0/64 (0.00%) 
Dysgeusia  1  3/50 (6.00%)  0/64 (0.00%) 
Encephalopathy  1  3/50 (6.00%)  1/64 (1.56%) 
Facial muscle weakness  1  1/50 (2.00%)  0/64 (0.00%) 
Headache  1  16/50 (32.00%)  0/64 (0.00%) 
Lethargy  1  13/50 (26.00%)  0/64 (0.00%) 
Neuralgia  1  1/50 (2.00%)  0/64 (0.00%) 
Paresthesia  1  2/50 (4.00%)  0/64 (0.00%) 
Peripheral motor neuropathy  1  0/50 (0.00%)  1/64 (1.56%) 
Peripheral sensory neuropathy  1  3/50 (6.00%)  0/64 (0.00%) 
Presyncope  1  1/50 (2.00%)  0/64 (0.00%) 
Restless leg syndrome  1  2/50 (4.00%)  0/64 (0.00%) 
Seizure  1  1/50 (2.00%)  0/64 (0.00%) 
Sinus pain  1  2/50 (4.00%)  0/64 (0.00%) 
Somnolence  1  7/50 (14.00%)  0/64 (0.00%) 
Stroke  1  1/50 (2.00%)  1/64 (1.56%) 
Syncope  1  0/50 (0.00%)  2/64 (3.13%) 
Tremor  1  2/50 (4.00%)  0/64 (0.00%) 
Psychiatric disorders     
Agitation  1  2/50 (4.00%)  0/64 (0.00%) 
Altered mental status  1  1/50 (2.00%)  0/64 (0.00%) 
Anxiety  1  3/50 (6.00%)  0/64 (0.00%) 
Confusion  1  17/50 (34.00%)  1/64 (1.56%) 
Delirium  1  2/50 (4.00%)  0/64 (0.00%) 
Depression  1  5/50 (10.00%)  0/64 (0.00%) 
Hallucinations  1  3/50 (6.00%)  0/64 (0.00%) 
Insomnia  1  9/50 (18.00%)  0/64 (0.00%) 
Restlessness  1  1/50 (2.00%)  0/64 (0.00%) 
Renal and urinary disorders     
Acute kidney injury  1  7/50 (14.00%)  0/64 (0.00%) 
Acute renal failure  1  0/50 (0.00%)  2/64 (3.13%) 
Bladder spasm  1  1/50 (2.00%)  0/64 (0.00%) 
Hematuria  1  28/50 (56.00%)  0/64 (0.00%) 
Proteinuria  1  22/50 (44.00%)  0/64 (0.00%) 
Urinary frequency  1  6/50 (12.00%)  0/64 (0.00%) 
Urinary incontinence  1  4/50 (8.00%)  0/64 (0.00%) 
Urinary retention  1  5/50 (10.00%)  0/64 (0.00%) 
Urinary tract obstruction  1  1/50 (2.00%)  0/64 (0.00%) 
Urinary tract pain  1  2/50 (4.00%)  0/64 (0.00%) 
Reproductive system and breast disorders     
Penile pain  1  1/50 (2.00%)  0/64 (0.00%) 
Vaginal hemorrhage  1  1/50 (2.00%)  0/64 (0.00%) 
Respiratory, thoracic and mediastinal disorders     
Allergic rhinitis  1  1/50 (2.00%)  0/64 (0.00%) 
Aspiration  1  2/50 (4.00%)  0/64 (0.00%) 
Atelectasis  1  25/50 (50.00%)  0/64 (0.00%) 
Bronchopulmonary hemorrhage  1  1/50 (2.00%)  0/64 (0.00%) 
Cough  1  22/50 (44.00%)  0/64 (0.00%) 
Diffuse aveolar hemorrhage  1  0/50 (0.00%)  1/64 (1.56%) 
Dyspnea  1  28/50 (56.00%)  4/64 (6.25%) 
Epistaxis  1  8/50 (16.00%)  4/64 (6.25%) 
Hemoptysis  1  3/50 (6.00%)  0/64 (0.00%) 
Hiccups  1  1/50 (2.00%)  0/64 (0.00%) 
Hoarseness  1  1/50 (2.00%)  1/64 (1.56%) 
Hypoxia  1  22/50 (44.00%)  4/64 (6.25%) 
Nasal congestion  1  8/50 (16.00%)  0/64 (0.00%) 
Pleural effusion  1  17/50 (34.00%)  1/64 (1.56%) 
Pleuritic pain  1  5/50 (10.00%)  0/64 (0.00%) 
Postnasal drip  1  5/50 (10.00%)  0/64 (0.00%) 
Productive cough  1  6/50 (12.00%)  0/64 (0.00%) 
Pulmonary edema  1  10/50 (20.00%)  0/64 (0.00%) 
Pulmonary hypertension  1  1/50 (2.00%)  0/64 (0.00%) 
Respiratory failure  1  8/50 (16.00%)  3/64 (4.69%) 
Rhinorrhea  1  1/50 (2.00%)  0/64 (0.00%) 
Sneezing  1  1/50 (2.00%)  0/64 (0.00%) 
Sore throat  1  12/50 (24.00%)  0/64 (0.00%) 
Wheezing  1  6/50 (12.00%)  1/64 (1.56%) 
Skin and subcutaneous tissue disorders     
Alopecia  1  2/50 (4.00%)  0/64 (0.00%) 
Dry skin  1  7/50 (14.00%)  0/64 (0.00%) 
Hematoma  1  5/50 (10.00%)  0/64 (0.00%) 
Hot flashes  1  2/50 (4.00%)  0/64 (0.00%) 
Papulopustular rash  1  2/50 (4.00%)  0/64 (0.00%) 
Pruritus  1  7/50 (14.00%)  1/64 (1.56%) 
Purpura  1  17/50 (34.00%)  0/64 (0.00%) 
Rash acneiform  1  1/50 (2.00%)  0/64 (0.00%) 
Rash maculo-papular  1  12/50 (24.00%)  2/64 (3.13%) 
Skin nodules  1  1/50 (2.00%)  0/64 (0.00%) 
Skin ulceration  1  17/50 (34.00%)  0/64 (0.00%) 
Vascular disorders     
Flushing  1  3/50 (6.00%)  0/64 (0.00%) 
Hypertension  1  2/50 (4.00%)  3/64 (4.69%) 
Hypotension  1  14/50 (28.00%)  7/64 (10.94%) 
Superficial thrombophlebitis  1  1/50 (2.00%)  0/64 (0.00%) 
Thromboembolic event  1  6/50 (12.00%)  2/64 (3.13%) 
1
Term from vocabulary, CTCAE (4.0)
Indicates events were collected by systematic assessment
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: John Welch, M.D., Ph.D.
Organization: Washington University School of Medicine
Phone: 314-362-2626
EMail: jwelch@wustl.edu
Layout table for additonal information
Responsible Party: Washington University School of Medicine
ClinicalTrials.gov Identifier: NCT01687400    
Other Study ID Numbers: 201210102
First Submitted: September 13, 2012
First Posted: September 18, 2012
Results First Submitted: June 13, 2018
Results First Posted: October 2, 2018
Last Update Posted: October 2, 2018