Trial Outcomes & Findings for Decitabine Versus Azacitidine in Myelodysplastic Syndrome Patients With Low and Intermediate-1 Risk (NCT NCT01720225)
NCT ID: NCT01720225
Last Updated: 2020-12-24
Results Overview
Overall Response = complete remission (CR) + partial remission (PR) + marrow CR (mCR) + hematologic improvement (HI). CR is normalization of peripheral blood and bone marrow with \<5% bone marrow blasts, a peripheral blood granulocyte count \> (1.0 x 10\^9/L, and platelet count \> 100 x 10\^9/L). PR is same as CR except for the presence of 6-15% marrow blasts, or 50% reduction if \<15% at start of treatment. Marrow CR is blasts \</= 5% and decreased by \>/=50% from baseline. HI is platelets increase by 50% and to above 30 x 10\^9/L untransfused (if lower than that pre-therapy; or hemoglobin increase by 2 g/dl; or transfusion independent; or splenomegaly reduction by \> 50%; or monocytosis reduction by \> 50% if pretreatment \> 5 X1 0\^9/L.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
113 participants
Primary outcome timeframe
56 days
Results posted on
2020-12-24
Participant Flow
Recruitment Period: November 2012 to February 2016
Participant milestones
| Measure |
Decitabine
Patients randomized to receive Decitabine 20 mg/m2 by vein daily for 3 days (days 1-3) every 28 days.
Decitabine: 20 mg/m2 by vein daily for 3 days (days 1-3) every 28 days.
|
Azacitidine
Patients randomized to receive Azacitidine 75 mg/m2 subcutaneously or by vein daily for 3 days (days 1-3) every 28 days.
Azacitidine: 75 mg/m2 subcutaneously or by vein daily for 3 days (days 1-3) every 28 days.
|
|---|---|---|
|
Overall Study
STARTED
|
73
|
40
|
|
Overall Study
COMPLETED
|
70
|
39
|
|
Overall Study
NOT COMPLETED
|
3
|
1
|
Reasons for withdrawal
| Measure |
Decitabine
Patients randomized to receive Decitabine 20 mg/m2 by vein daily for 3 days (days 1-3) every 28 days.
Decitabine: 20 mg/m2 by vein daily for 3 days (days 1-3) every 28 days.
|
Azacitidine
Patients randomized to receive Azacitidine 75 mg/m2 subcutaneously or by vein daily for 3 days (days 1-3) every 28 days.
Azacitidine: 75 mg/m2 subcutaneously or by vein daily for 3 days (days 1-3) every 28 days.
|
|---|---|---|
|
Overall Study
Withdrawal by Subject
|
0
|
1
|
|
Overall Study
Did not complete 2 cycles of therapy
|
3
|
0
|
Baseline Characteristics
Decitabine Versus Azacitidine in Myelodysplastic Syndrome Patients With Low and Intermediate-1 Risk
Baseline characteristics by cohort
| Measure |
Decitabine
n=73 Participants
Patients randomized to receive Decitabine 20 mg/m2 by vein daily for 3 days (days 1-3) every 28 days.
Decitabine: 20 mg/m2 by vein daily for 3 days (days 1-3) every 28 days.
|
Azacitidine
n=40 Participants
Patients randomized to receive Azacitidine 75 mg/m2 subcutaneously or by vein daily for 3 days (days 1-3) every 28 days.
Azacitidine: 75 mg/m2 subcutaneously or by vein daily for 3 days (days 1-3) every 28 days.
|
Total
n=113 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
26 Participants
n=5 Participants
|
12 Participants
n=7 Participants
|
38 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
47 Participants
n=5 Participants
|
28 Participants
n=7 Participants
|
75 Participants
n=5 Participants
|
|
Age, Continuous
|
70 years
n=5 Participants
|
70 years
n=7 Participants
|
70 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
24 Participants
n=5 Participants
|
16 Participants
n=7 Participants
|
40 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
49 Participants
n=5 Participants
|
24 Participants
n=7 Participants
|
73 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
2 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
71 Participants
n=5 Participants
|
39 Participants
n=7 Participants
|
110 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
73 participants
n=5 Participants
|
40 participants
n=7 Participants
|
113 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 56 daysOverall Response = complete remission (CR) + partial remission (PR) + marrow CR (mCR) + hematologic improvement (HI). CR is normalization of peripheral blood and bone marrow with \<5% bone marrow blasts, a peripheral blood granulocyte count \> (1.0 x 10\^9/L, and platelet count \> 100 x 10\^9/L). PR is same as CR except for the presence of 6-15% marrow blasts, or 50% reduction if \<15% at start of treatment. Marrow CR is blasts \</= 5% and decreased by \>/=50% from baseline. HI is platelets increase by 50% and to above 30 x 10\^9/L untransfused (if lower than that pre-therapy; or hemoglobin increase by 2 g/dl; or transfusion independent; or splenomegaly reduction by \> 50%; or monocytosis reduction by \> 50% if pretreatment \> 5 X1 0\^9/L.
Outcome measures
| Measure |
Decitabine
n=70 Participants
Patients randomized to receive Decitabine 20 mg/m2 by vein daily for 3 days (days 1-3) every 28 days.
Decitabine: 20 mg/m2 by vein daily for 3 days (days 1-3) every 28 days.
|
Azacitidine
n=39 Participants
Patients randomized to receive Azacitidine 75 mg/m2 subcutaneously or by vein daily for 3 days (days 1-3) every 28 days.
Azacitidine: 75 mg/m2 subcutaneously or by vein daily for 3 days (days 1-3) every 28 days.
|
|---|---|---|
|
Participants With a Response
|
49 Participants
|
19 Participants
|
SECONDARY outcome
Timeframe: 8 weeksPopulation: Participants analyzed for Transfusion Independence must have been transfusion dependent at baseline. Thirty-seven participants in the Decitabine arm and nineteen participants in the Azacitidine arm where transfusion dependent prior to treatment on this study.
Participants who were transfusion dependent at baseline prior to starting therapy on the Decitabine or Azacitidine arm will be analyzed for transfusion independence. Transfusion independence defined as being transfusion-free for ≥8 consecutive weeks between the first dose and study treatment discontinuation.
Outcome measures
| Measure |
Decitabine
n=37 Participants
Patients randomized to receive Decitabine 20 mg/m2 by vein daily for 3 days (days 1-3) every 28 days.
Decitabine: 20 mg/m2 by vein daily for 3 days (days 1-3) every 28 days.
|
Azacitidine
n=19 Participants
Patients randomized to receive Azacitidine 75 mg/m2 subcutaneously or by vein daily for 3 days (days 1-3) every 28 days.
Azacitidine: 75 mg/m2 subcutaneously or by vein daily for 3 days (days 1-3) every 28 days.
|
|---|---|---|
|
Number of Participants Who Became Transfusion Independent
|
12 Participants
|
3 Participants
|
Adverse Events
Decitabine
Serious events: 32 serious events
Other events: 20 other events
Deaths: 7 deaths
Azacitidine
Serious events: 14 serious events
Other events: 16 other events
Deaths: 2 deaths
Serious adverse events
| Measure |
Decitabine
n=73 participants at risk
Patients randomized to receive Decitabine 20 mg/m2 by vein daily for 3 days (days 1-3) every 28 days.
Decitabine: 20 mg/m2 by vein daily for 3 days (days 1-3) every 28 days.
|
Azacitidine
n=40 participants at risk
Patients randomized to receive Azacitidine 75 mg/m2 subcutaneously or by vein daily for 3 days (days 1-3) every 28 days.
Azacitidine: 75 mg/m2 subcutaneously or by vein daily for 3 days (days 1-3) every 28 days.
|
|---|---|---|
|
Renal and urinary disorders
Acute Kidney Injury
|
1.4%
1/73 • Number of events 1 • 3 years, 3 months
|
0.00%
0/40 • 3 years, 3 months
|
|
Respiratory, thoracic and mediastinal disorders
Acute Respiratory Distress Syndrome
|
1.4%
1/73 • Number of events 1 • 3 years, 3 months
|
0.00%
0/40 • 3 years, 3 months
|
|
Nervous system disorders
Altered Mental Status
|
0.00%
0/73 • 3 years, 3 months
|
2.5%
1/40 • Number of events 1 • 3 years, 3 months
|
|
Infections and infestations
Appendicitis
|
1.4%
1/73 • Number of events 1 • 3 years, 3 months
|
0.00%
0/40 • 3 years, 3 months
|
|
Gastrointestinal disorders
Ascites
|
1.4%
1/73 • Number of events 1 • 3 years, 3 months
|
0.00%
0/40 • 3 years, 3 months
|
|
General disorders
Back Pain
|
1.4%
1/73 • Number of events 1 • 3 years, 3 months
|
0.00%
0/40 • 3 years, 3 months
|
|
Respiratory, thoracic and mediastinal disorders
Bronchopulmonary Hemorrhage
|
0.00%
0/73 • 3 years, 3 months
|
2.5%
1/40 • Number of events 1 • 3 years, 3 months
|
|
Infections and infestations
Cellulitis
|
0.00%
0/73 • 3 years, 3 months
|
2.5%
1/40 • Number of events 1 • 3 years, 3 months
|
|
General disorders
Chest Pain
|
1.4%
1/73 • Number of events 1 • 3 years, 3 months
|
2.5%
1/40 • Number of events 1 • 3 years, 3 months
|
|
Surgical and medical procedures
Cholecystectomy
|
1.4%
1/73 • Number of events 1 • 3 years, 3 months
|
0.00%
0/40 • 3 years, 3 months
|
|
Gastrointestinal disorders
Diverticulitis
|
0.00%
0/73 • 3 years, 3 months
|
2.5%
1/40 • Number of events 1 • 3 years, 3 months
|
|
Infections and infestations
Ear Infection
|
1.4%
1/73 • Number of events 1 • 3 years, 3 months
|
0.00%
0/40 • 3 years, 3 months
|
|
General disorders
Edema Limbs
|
0.00%
0/73 • 3 years, 3 months
|
2.5%
1/40 • Number of events 1 • 3 years, 3 months
|
|
Injury, poisoning and procedural complications
Fall
|
0.00%
0/73 • 3 years, 3 months
|
2.5%
1/40 • Number of events 1 • 3 years, 3 months
|
|
General disorders
Fatigue
|
1.4%
1/73 • Number of events 1 • 3 years, 3 months
|
0.00%
0/40 • 3 years, 3 months
|
|
General disorders
Fever
|
2.7%
2/73 • Number of events 3 • 3 years, 3 months
|
0.00%
0/40 • 3 years, 3 months
|
|
Injury, poisoning and procedural complications
Food Poisoning
|
0.00%
0/73 • 3 years, 3 months
|
2.5%
1/40 • Number of events 1 • 3 years, 3 months
|
|
Gastrointestinal disorders
Gastroenteritis
|
1.4%
1/73 • Number of events 1 • 3 years, 3 months
|
0.00%
0/40 • 3 years, 3 months
|
|
Gastrointestinal disorders
Gastrointestinal Bleed
|
1.4%
1/73 • Number of events 1 • 3 years, 3 months
|
0.00%
0/40 • 3 years, 3 months
|
|
Skin and subcutaneous tissue disorders
Groin Abcess
|
1.4%
1/73 • Number of events 1 • 3 years, 3 months
|
0.00%
0/40 • 3 years, 3 months
|
|
General disorders
Headache
|
1.4%
1/73 • Number of events 1 • 3 years, 3 months
|
0.00%
0/40 • 3 years, 3 months
|
|
Cardiac disorders
Heart Failure
|
2.7%
2/73 • Number of events 3 • 3 years, 3 months
|
0.00%
0/40 • 3 years, 3 months
|
|
Respiratory, thoracic and mediastinal disorders
Hemoptysis
|
1.4%
1/73 • Number of events 1 • 3 years, 3 months
|
0.00%
0/40 • 3 years, 3 months
|
|
Vascular disorders
Hypertension
|
1.4%
1/73 • Number of events 1 • 3 years, 3 months
|
0.00%
0/40 • 3 years, 3 months
|
|
Infections and infestations
Tooth Infection
|
1.4%
1/73 • Number of events 1 • 3 years, 3 months
|
0.00%
0/40 • 3 years, 3 months
|
|
Nervous system disorders
Intracranial Hemorrhage
|
1.4%
1/73 • Number of events 1 • 3 years, 3 months
|
0.00%
0/40 • 3 years, 3 months
|
|
Infections and infestations
Joint Infection
|
1.4%
1/73 • Number of events 1 • 3 years, 3 months
|
0.00%
0/40 • 3 years, 3 months
|
|
Respiratory, thoracic and mediastinal disorders
Left plerual effusion
|
0.00%
0/73 • 3 years, 3 months
|
2.5%
1/40 • Number of events 1 • 3 years, 3 months
|
|
Infections and infestations
Leg Infection
|
1.4%
1/73 • Number of events 1 • 3 years, 3 months
|
0.00%
0/40 • 3 years, 3 months
|
|
Infections and infestations
Lung Infection
|
4.1%
3/73 • Number of events 3 • 3 years, 3 months
|
0.00%
0/40 • 3 years, 3 months
|
|
Cardiac disorders
Myocardial Infarction
|
1.4%
1/73 • Number of events 1 • 3 years, 3 months
|
0.00%
0/40 • 3 years, 3 months
|
|
Gastrointestinal disorders
Nausea
|
1.4%
1/73 • Number of events 1 • 3 years, 3 months
|
0.00%
0/40 • 3 years, 3 months
|
|
Blood and lymphatic system disorders
Neutropenic Fever
|
12.3%
9/73 • Number of events 17 • 3 years, 3 months
|
5.0%
2/40 • Number of events 2 • 3 years, 3 months
|
|
Ear and labyrinth disorders
Otitis Externa
|
1.4%
1/73 • Number of events 1 • 3 years, 3 months
|
0.00%
0/40 • 3 years, 3 months
|
|
General disorders
Pain
|
1.4%
1/73 • Number of events 1 • 3 years, 3 months
|
0.00%
0/40 • 3 years, 3 months
|
|
General disorders
Pain Abdominal Abcess
|
1.4%
1/73 • Number of events 1 • 3 years, 3 months
|
0.00%
0/40 • 3 years, 3 months
|
|
General disorders
Pain Right Extremity
|
0.00%
0/73 • 3 years, 3 months
|
2.5%
1/40 • Number of events 1 • 3 years, 3 months
|
|
Cardiac disorders
Pericarditis
|
1.4%
1/73 • Number of events 1 • 3 years, 3 months
|
0.00%
0/40 • 3 years, 3 months
|
|
Infections and infestations
Pneumonia
|
8.2%
6/73 • Number of events 7 • 3 years, 3 months
|
2.5%
1/40 • Number of events 4 • 3 years, 3 months
|
|
Respiratory, thoracic and mediastinal disorders
Right atelectasis
|
1.4%
1/73 • Number of events 1 • 3 years, 3 months
|
0.00%
0/40 • 3 years, 3 months
|
|
Respiratory, thoracic and mediastinal disorders
Right Pleural Effusion
|
1.4%
1/73 • Number of events 1 • 3 years, 3 months
|
0.00%
0/40 • 3 years, 3 months
|
|
Gastrointestinal disorders
Rectal Hermorrhage
|
1.4%
1/73 • Number of events 1 • 3 years, 3 months
|
0.00%
0/40 • 3 years, 3 months
|
|
Renal and urinary disorders
Renal Failure
|
1.4%
1/73 • Number of events 1 • 3 years, 3 months
|
0.00%
0/40 • 3 years, 3 months
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory Failure
|
4.1%
3/73 • Number of events 3 • 3 years, 3 months
|
0.00%
0/40 • 3 years, 3 months
|
|
General disorders
Right Hip Pain
|
0.00%
0/73 • 3 years, 3 months
|
2.5%
1/40 • Number of events 1 • 3 years, 3 months
|
|
Infections and infestations
Salmonella Bactremia
|
0.00%
0/73 • 3 years, 3 months
|
2.5%
1/40 • Number of events 1 • 3 years, 3 months
|
|
Infections and infestations
Sepsis
|
5.5%
4/73 • Number of events 4 • 3 years, 3 months
|
0.00%
0/40 • 3 years, 3 months
|
|
Respiratory, thoracic and mediastinal disorders
Shortness of Breath
|
1.4%
1/73 • Number of events 1 • 3 years, 3 months
|
0.00%
0/40 • 3 years, 3 months
|
|
Gastrointestinal disorders
Sialadenitis
|
0.00%
0/73 • 3 years, 3 months
|
2.5%
1/40 • Number of events 1 • 3 years, 3 months
|
|
Skin and subcutaneous tissue disorders
Skin Ulceration
|
1.4%
1/73 • Number of events 1 • 3 years, 3 months
|
0.00%
0/40 • 3 years, 3 months
|
|
Gastrointestinal disorders
Small Intestine Obstruction
|
0.00%
0/73 • 3 years, 3 months
|
2.5%
1/40 • Number of events 1 • 3 years, 3 months
|
|
Infections and infestations
Submandibular Cellulitis
|
1.4%
1/73 • Number of events 1 • 3 years, 3 months
|
2.5%
1/40 • Number of events 1 • 3 years, 3 months
|
|
Nervous system disorders
Syncopal episode
|
1.4%
1/73 • Number of events 1 • 3 years, 3 months
|
0.00%
0/40 • 3 years, 3 months
|
|
Infections and infestations
Urinary Tract Infection
|
2.7%
2/73 • Number of events 2 • 3 years, 3 months
|
2.5%
1/40 • Number of events 1 • 3 years, 3 months
|
|
Reproductive system and breast disorders
Vaginal Fistual
|
0.00%
0/73 • 3 years, 3 months
|
2.5%
1/40 • Number of events 1 • 3 years, 3 months
|
|
Infections and infestations
Wound Infection
|
1.4%
1/73 • Number of events 1 • 3 years, 3 months
|
0.00%
0/40 • 3 years, 3 months
|
Other adverse events
| Measure |
Decitabine
n=73 participants at risk
Patients randomized to receive Decitabine 20 mg/m2 by vein daily for 3 days (days 1-3) every 28 days.
Decitabine: 20 mg/m2 by vein daily for 3 days (days 1-3) every 28 days.
|
Azacitidine
n=40 participants at risk
Patients randomized to receive Azacitidine 75 mg/m2 subcutaneously or by vein daily for 3 days (days 1-3) every 28 days.
Azacitidine: 75 mg/m2 subcutaneously or by vein daily for 3 days (days 1-3) every 28 days.
|
|---|---|---|
|
Gastrointestinal disorders
Nausea
|
15.1%
11/73 • Number of events 11 • 3 years, 3 months
|
15.0%
6/40 • Number of events 6 • 3 years, 3 months
|
|
General disorders
Fatigue
|
8.2%
6/73 • Number of events 6 • 3 years, 3 months
|
10.0%
4/40 • Number of events 4 • 3 years, 3 months
|
|
Gastrointestinal disorders
Constipation
|
4.1%
3/73 • Number of events 3 • 3 years, 3 months
|
15.0%
6/40 • Number of events 6 • 3 years, 3 months
|
|
Gastrointestinal disorders
Diarrhea
|
2.7%
2/73 • Number of events 2 • 3 years, 3 months
|
7.5%
3/40 • Number of events 3 • 3 years, 3 months
|
Additional Information
Elias Joseph MD./Professor
The University of Texas MD Anderson Cancer Center
Phone: 713-792-4764
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place