Trial Outcomes & Findings for Decitabine for Older or Unfit Patients With Acute Myeloid Leukemia (AML) (NCT NCT01786343)
NCT ID: NCT01786343
Last Updated: 2020-04-15
Results Overview
Response is defined as Complete Response (CR) + Partial Remission (PR) + Complete Remission with incomplete recovery (CRi) + Clinical Benefit. CR is the normalization of the peripheral blood and bone marrow with \</= 5% bone marrow blasts, a peripheral blood granulocyte count \>/= (1.0 x 10\^9/L, and a platelet count \>/= 100 x 10\^9/L). PR is the same as CR except for the presence of 6-15% marrow blasts, or 50% reduction if \<15% at start of treatment. CRi meets all criteria for CR except for platelet recovery to \>100 x 10\^9/L and/or granulocyte count \> (1.0 x 10\^9/L). Clinical benefit is platelets increase by 50% and to above 30 x 10\^9/L untransfused (if lower than that pretherapy); or granulocytes increase by 100% and to above 10\^9/L (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 x 109/L.
COMPLETED
PHASE2
74 participants
Up to 3 months
2020-04-15
Participant Flow
Recruitment Period: February 2013 - April 2018
74 participants were enrolled in the study, all participants completed the study.
Participant milestones
| Measure |
Decitabine - 5 Day Regimen
Decitabine 20 mg/m2 by vein daily for 5 days.
Decitabine: 20 mg/m2 by vein daily for either 5 or 10 days.
|
Decitabine - 10 Day Regimen
Decitabine 20 mg/m2 by vein daily for 10 days.
Decitabine: 20 mg/m2 by vein daily for either 5 or 10 days.
|
|---|---|---|
|
Overall Study
STARTED
|
28
|
46
|
|
Overall Study
COMPLETED
|
28
|
46
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Decitabine for Older or Unfit Patients With Acute Myeloid Leukemia (AML)
Baseline characteristics by cohort
| Measure |
Decitabine - 5 Day Regimen
n=28 Participants
Decitabine 20 mg/m2 by vein daily for 5 days.
Decitabine: 20 mg/m2 by vein daily for either 5 or 10 days.
|
Decitabine - 10 Day Regimen
n=46 Participants
Decitabine 20 mg/m2 by vein daily for 10 days.
Decitabine: 20 mg/m2 by vein daily for either 5 or 10 days.
|
Total
n=74 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
|
3 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
25 Participants
n=5 Participants
|
46 Participants
n=7 Participants
|
71 Participants
n=5 Participants
|
|
Age, Continuous
|
77 years
n=5 Participants
|
78 years
n=7 Participants
|
78 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
12 Participants
n=5 Participants
|
14 Participants
n=7 Participants
|
26 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
16 Participants
n=5 Participants
|
32 Participants
n=7 Participants
|
48 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
|
2 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
4 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
|
7 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
22 Participants
n=5 Participants
|
36 Participants
n=7 Participants
|
58 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
|
2 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
28 participants
n=5 Participants
|
46 participants
n=7 Participants
|
74 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Up to 3 monthsResponse is defined as Complete Response (CR) + Partial Remission (PR) + Complete Remission with incomplete recovery (CRi) + Clinical Benefit. CR is the normalization of the peripheral blood and bone marrow with \</= 5% bone marrow blasts, a peripheral blood granulocyte count \>/= (1.0 x 10\^9/L, and a platelet count \>/= 100 x 10\^9/L). PR is the same as CR except for the presence of 6-15% marrow blasts, or 50% reduction if \<15% at start of treatment. CRi meets all criteria for CR except for platelet recovery to \>100 x 10\^9/L and/or granulocyte count \> (1.0 x 10\^9/L). Clinical benefit is platelets increase by 50% and to above 30 x 10\^9/L untransfused (if lower than that pretherapy); or granulocytes increase by 100% and to above 10\^9/L (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 x 109/L.
Outcome measures
| Measure |
Decitabine - 5 Day Regimen
n=28 Participants
Decitabine 20 mg/m2 by vein daily for 5 days.
Decitabine: 20 mg/m2 by vein daily for either 5 or 10 days.
|
Decitabine - 10 Day Regimen
n=46 Participants
Decitabine 20 mg/m2 by vein daily for 10 days.
Decitabine: 20 mg/m2 by vein daily for either 5 or 10 days.
|
|---|---|---|
|
Participants With a Response
|
12 Participants
|
19 Participants
|
SECONDARY outcome
Timeframe: Up to 5 yearsTime from date of treatment start until date of death due to any cause or last Follow-up.
Outcome measures
| Measure |
Decitabine - 5 Day Regimen
n=28 Participants
Decitabine 20 mg/m2 by vein daily for 5 days.
Decitabine: 20 mg/m2 by vein daily for either 5 or 10 days.
|
Decitabine - 10 Day Regimen
n=46 Participants
Decitabine 20 mg/m2 by vein daily for 10 days.
Decitabine: 20 mg/m2 by vein daily for either 5 or 10 days.
|
|---|---|---|
|
Overall Survival
|
5.5 Months
Interval 2.1 to 11.7
|
6.0 Months
Interval 1.9 to 11.7
|
SECONDARY outcome
Timeframe: Up to 5 yearsThe date of response to date of loss of response or last follow-up. Response is defined as Complete Response (CR) + Partial Remission (PR) + Complete Remission with incomplete recovery (CRi) + Clinical Benefit. CR is the normalization of the peripheral blood and bone marrow with \</= 5% bone marrow blasts, a peripheral blood granulocyte count \>/= (1.0 x 10\^9/L, and a platelet count \>/= 100 x 10\^9/L). PR is the same as CR except for the presence of 6-15% marrow blasts, or 50% reduction if \<15% at start of treatment. CRi meets all criteria for CR except for platelet recovery to \>100 x 10\^9/L and/or granulocyte count \> (1.0 x 10\^9/L). Clinical benefit is platelets increase by 50% and to above 30 x 10\^9/L untransfused (if lower than that pretherapy); or granulocytes increase by 100% and to above 10\^9/L (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
Outcome measures
| Measure |
Decitabine - 5 Day Regimen
n=28 Participants
Decitabine 20 mg/m2 by vein daily for 5 days.
Decitabine: 20 mg/m2 by vein daily for either 5 or 10 days.
|
Decitabine - 10 Day Regimen
n=46 Participants
Decitabine 20 mg/m2 by vein daily for 10 days.
Decitabine: 20 mg/m2 by vein daily for either 5 or 10 days.
|
|---|---|---|
|
Response Duration
|
9.4 Months
Interval 5.6 to 17.9
|
6.4 Months
Interval 2.8 to 12.4
|
Adverse Events
Decitabine - 5 Day Regimen
Decitabine - 10 Day Regimen
Serious adverse events
| Measure |
Decitabine - 5 Day Regimen
n=28 participants at risk
Decitabine 20 mg/m2 by vein daily for 5 days.
Decitabine: 20 mg/m2 by vein daily for either 5 or 10 days.
|
Decitabine - 10 Day Regimen
n=46 participants at risk
Decitabine 20 mg/m2 by vein daily for 10 days.
Decitabine: 20 mg/m2 by vein daily for either 5 or 10 days.
|
|---|---|---|
|
Renal and urinary disorders
Acute Kidney Injury
|
0.00%
0/28 • Up to 5 years
|
2.2%
1/46 • Number of events 1 • Up to 5 years
|
|
Gastrointestinal disorders
Anorexia
|
0.00%
0/28 • Up to 5 years
|
2.2%
1/46 • Number of events 1 • Up to 5 years
|
|
Musculoskeletal and connective tissue disorders
Arthritis
|
0.00%
0/28 • Up to 5 years
|
4.3%
2/46 • Number of events 2 • Up to 5 years
|
|
Cardiac disorders
Atrial Flutter
|
0.00%
0/28 • Up to 5 years
|
2.2%
1/46 • Number of events 2 • Up to 5 years
|
|
Infections and infestations
Bactreamia
|
7.1%
2/28 • Number of events 2 • Up to 5 years
|
0.00%
0/46 • Up to 5 years
|
|
Vascular disorders
Blood Clot
|
0.00%
0/28 • Up to 5 years
|
2.2%
1/46 • Number of events 1 • Up to 5 years
|
|
Infections and infestations
Cellulitis
|
3.6%
1/28 • Number of events 1 • Up to 5 years
|
6.5%
3/46 • Number of events 4 • Up to 5 years
|
|
General disorders
Chest Pain
|
0.00%
0/28 • Up to 5 years
|
2.2%
1/46 • Number of events 1 • Up to 5 years
|
|
Hepatobiliary disorders
Cholecystitis
|
0.00%
0/28 • Up to 5 years
|
2.2%
1/46 • Number of events 2 • Up to 5 years
|
|
Nervous system disorders
Confusion
|
0.00%
0/28 • Up to 5 years
|
2.2%
1/46 • Number of events 1 • Up to 5 years
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/28 • Up to 5 years
|
2.2%
1/46 • Number of events 1 • Up to 5 years
|
|
Cardiac disorders
CVA Left Hemiparesis
|
3.6%
1/28 • Number of events 1 • Up to 5 years
|
0.00%
0/46 • Up to 5 years
|
|
General disorders
Death
|
10.7%
3/28 • Number of events 3 • Up to 5 years
|
34.8%
16/46 • Number of events 16 • Up to 5 years
|
|
Vascular disorders
Deep Vein Thrombosis
|
3.6%
1/28 • Number of events 1 • Up to 5 years
|
2.2%
1/46 • Number of events 1 • Up to 5 years
|
|
Metabolism and nutrition disorders
Dehydration
|
3.6%
1/28 • Number of events 1 • Up to 5 years
|
0.00%
0/46 • Up to 5 years
|
|
Infections and infestations
Device Related Infection
|
0.00%
0/28 • Up to 5 years
|
2.2%
1/46 • Number of events 1 • Up to 5 years
|
|
Gastrointestinal disorders
Diarrhea
|
10.7%
3/28 • Number of events 3 • Up to 5 years
|
2.2%
1/46 • Number of events 1 • Up to 5 years
|
|
General disorders
Edema
|
0.00%
0/28 • Up to 5 years
|
6.5%
3/46 • Number of events 3 • Up to 5 years
|
|
Investigations
Elevated Troponins
|
0.00%
0/28 • Up to 5 years
|
2.2%
1/46 • Number of events 1 • Up to 5 years
|
|
Gastrointestinal disorders
Enterocolitis
|
3.6%
1/28 • Number of events 1 • Up to 5 years
|
2.2%
1/46 • Number of events 1 • Up to 5 years
|
|
Metabolism and nutrition disorders
Failure to Thrive
|
0.00%
0/28 • Up to 5 years
|
2.2%
1/46 • Number of events 1 • Up to 5 years
|
|
General disorders
Fall
|
0.00%
0/28 • Up to 5 years
|
2.2%
1/46 • Number of events 1 • Up to 5 years
|
|
General disorders
Fatigue
|
0.00%
0/28 • Up to 5 years
|
2.2%
1/46 • Number of events 1 • Up to 5 years
|
|
General disorders
Fever
|
7.1%
2/28 • Number of events 2 • Up to 5 years
|
0.00%
0/46 • Up to 5 years
|
|
Musculoskeletal and connective tissue disorders
Generalized Weakness
|
0.00%
0/28 • Up to 5 years
|
2.2%
1/46 • Number of events 1 • Up to 5 years
|
|
Nervous system disorders
Hemorrhage CNS
|
3.6%
1/28 • Number of events 1 • Up to 5 years
|
0.00%
0/46 • Up to 5 years
|
|
Musculoskeletal and connective tissue disorders
Hip Fracture
|
0.00%
0/28 • Up to 5 years
|
2.2%
1/46 • Number of events 1 • Up to 5 years
|
|
Metabolism and nutrition disorders
Hypoglycemia
|
0.00%
0/28 • Up to 5 years
|
2.2%
1/46 • Number of events 1 • Up to 5 years
|
|
Infections and infestations
Infection
|
0.00%
0/28 • Up to 5 years
|
4.3%
2/46 • Number of events 2 • Up to 5 years
|
|
Blood and lymphatic system disorders
Left Neck Mass - Lymph nodes
|
0.00%
0/28 • Up to 5 years
|
2.2%
1/46 • Number of events 1 • Up to 5 years
|
|
Blood and lymphatic system disorders
Leukocytosis
|
3.6%
1/28 • Number of events 1 • Up to 5 years
|
0.00%
0/46 • Up to 5 years
|
|
Infections and infestations
Lung Infection
|
10.7%
3/28 • Number of events 3 • Up to 5 years
|
15.2%
7/46 • Number of events 8 • Up to 5 years
|
|
Gastrointestinal disorders
Nausea
|
3.6%
1/28 • Number of events 1 • Up to 5 years
|
0.00%
0/46 • Up to 5 years
|
|
Blood and lymphatic system disorders
Neutropenia
|
3.6%
1/28 • Number of events 1 • Up to 5 years
|
0.00%
0/46 • Up to 5 years
|
|
Infections and infestations
Neutropenic Fever
|
28.6%
8/28 • Number of events 10 • Up to 5 years
|
28.3%
13/46 • Number of events 21 • Up to 5 years
|
|
Infections and infestations
Pneumonia
|
17.9%
5/28 • Number of events 5 • Up to 5 years
|
13.0%
6/46 • Number of events 6 • Up to 5 years
|
|
Infections and infestations
Pseudomonas
|
0.00%
0/28 • Up to 5 years
|
2.2%
1/46 • Number of events 1 • Up to 5 years
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary Edema
|
0.00%
0/28 • Up to 5 years
|
2.2%
1/46 • Number of events 1 • Up to 5 years
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary Insufficiency
|
0.00%
0/28 • Up to 5 years
|
6.5%
3/46 • Number of events 3 • Up to 5 years
|
|
Renal and urinary disorders
Renal Insufficiency
|
0.00%
0/28 • Up to 5 years
|
2.2%
1/46 • Number of events 1 • Up to 5 years
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory Failure
|
3.6%
1/28 • Number of events 1 • Up to 5 years
|
0.00%
0/46 • Up to 5 years
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory Other
|
3.6%
1/28 • Number of events 1 • Up to 5 years
|
0.00%
0/46 • Up to 5 years
|
|
Infections and infestations
Sepsis
|
3.6%
1/28 • Number of events 1 • Up to 5 years
|
4.3%
2/46 • Number of events 2 • Up to 5 years
|
|
Nervous system disorders
Syncope
|
3.6%
1/28 • Number of events 1 • Up to 5 years
|
0.00%
0/46 • Up to 5 years
|
|
Gastrointestinal disorders
Upper Gastrointestinal Hemorrhage
|
3.6%
1/28 • Number of events 2 • Up to 5 years
|
0.00%
0/46 • Up to 5 years
|
|
Respiratory, thoracic and mediastinal disorders
Upper Respiratory Infection
|
3.6%
1/28 • Number of events 1 • Up to 5 years
|
0.00%
0/46 • Up to 5 years
|
|
Infections and infestations
Urinary Tract Infection
|
0.00%
0/28 • Up to 5 years
|
2.2%
1/46 • Number of events 1 • Up to 5 years
|
Other adverse events
| Measure |
Decitabine - 5 Day Regimen
n=28 participants at risk
Decitabine 20 mg/m2 by vein daily for 5 days.
Decitabine: 20 mg/m2 by vein daily for either 5 or 10 days.
|
Decitabine - 10 Day Regimen
n=46 participants at risk
Decitabine 20 mg/m2 by vein daily for 10 days.
Decitabine: 20 mg/m2 by vein daily for either 5 or 10 days.
|
|---|---|---|
|
Investigations
Hyperbilirubinemia
|
7.1%
2/28 • Number of events 2 • Up to 5 years
|
0.00%
0/46 • Up to 5 years
|
|
Nervous system disorders
Confusion
|
0.00%
0/28 • Up to 5 years
|
6.5%
3/46 • Number of events 3 • Up to 5 years
|
|
Investigations
Elevated Creatinine
|
17.9%
5/28 • Number of events 6 • Up to 5 years
|
2.2%
1/46 • Number of events 2 • Up to 5 years
|
|
Gastrointestinal disorders
Diarrhea
|
10.7%
3/28 • Number of events 3 • Up to 5 years
|
2.2%
1/46 • Number of events 1 • Up to 5 years
|
|
General disorders
Edema limb
|
7.1%
2/28 • Number of events 2 • Up to 5 years
|
6.5%
3/46 • Number of events 3 • Up to 5 years
|
|
General disorders
Fatigue
|
21.4%
6/28 • Number of events 7 • Up to 5 years
|
13.0%
6/46 • Number of events 12 • Up to 5 years
|
|
Infections and infestations
Neutropenic Fever
|
28.6%
8/28 • Number of events 9 • Up to 5 years
|
30.4%
14/46 • Number of events 20 • Up to 5 years
|
|
Cardiac disorders
Hypotension
|
3.6%
1/28 • Number of events 1 • Up to 5 years
|
6.5%
3/46 • Number of events 3 • Up to 5 years
|
|
Infections and infestations
Infection
|
7.1%
2/28 • Number of events 3 • Up to 5 years
|
15.2%
7/46 • Number of events 8 • Up to 5 years
|
|
Gastrointestinal disorders
Mucositis
|
7.1%
2/28 • Number of events 2 • Up to 5 years
|
0.00%
0/46 • Up to 5 years
|
|
Musculoskeletal and connective tissue disorders
Muscle Weakness
|
3.6%
1/28 • Number of events 1 • Up to 5 years
|
13.0%
6/46 • Number of events 8 • Up to 5 years
|
|
Gastrointestinal disorders
Nausea
|
7.1%
2/28 • Number of events 3 • Up to 5 years
|
8.7%
4/46 • Number of events 4 • Up to 5 years
|
|
Infections and infestations
Opportunistic Infection
|
25.0%
7/28 • Number of events 8 • Up to 5 years
|
28.3%
13/46 • Number of events 13 • Up to 5 years
|
|
General disorders
Pain
|
7.1%
2/28 • Number of events 2 • Up to 5 years
|
21.7%
10/46 • Number of events 11 • Up to 5 years
|
|
Respiratory, thoracic and mediastinal disorders
Pleural Effusion
|
7.1%
2/28 • Number of events 2 • Up to 5 years
|
0.00%
0/46 • Up to 5 years
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
0.00%
0/28 • Up to 5 years
|
6.5%
3/46 • Number of events 3 • Up to 5 years
|
Additional Information
Farhad Ravandi-Kashani MD/Professor
The University of Texas MD Anderson Cancer Center
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place