Trial Outcomes & Findings for Study of Three Different Schedules of Low-Dose Decitabine in Myelodysplastic Syndrome (MDS) (NCT NCT00067808)
NCT ID: NCT00067808
Last Updated: 2012-08-07
Results Overview
Objective responses by International Working Group criteria: 'Complete Response' (CR) defined as Normalization of the peripheral blood and bone marrow with \<5% bone marrow blasts, a peripheral blood granulocyte count \> (1.0 x 109/ L, and a platelet count \> 100 x 109/L); 'Other Response' including Partial Remission (PR) defined as above, except for the presence of 6-15% marrow blasts, or 50% reduction if \<15% at start of treatment combined with participants who meet all criteria for CR except for platelet recovery to \>100 x 109/L; and 'No Response'.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
128 participants
Primary outcome timeframe
Response to treatment after 8 weeks of therapy
Results posted on
2012-08-07
Participant Flow
Recruitment Period 6/4/03 - 5/18/09; all patients were registered at The University of Texas M.D. Anderson Cancer Center.
Enrollment of 128 patients: 1 patient withdrew consent prior to treatment; 3 did not meet eligibility requirement for a total of 124 evaluable patients.
Participant milestones
| Measure |
Decitabine 10 mg/m2 IV
10 mg/m2 by vein (IV) over 1 hour daily for 10 days
|
Decitabine 20 mg/m2 IV
20 mg/m2 IV over 1 hour daily for 5 days
|
Decitabine 20 mg/m2 SQ
20 mg/m2 subcutaneous (SQ) daily for 5 days
|
|---|---|---|---|
|
Overall Study
STARTED
|
17
|
93
|
14
|
|
Overall Study
COMPLETED
|
17
|
93
|
14
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Study of Three Different Schedules of Low-Dose Decitabine in Myelodysplastic Syndrome (MDS)
Baseline characteristics by cohort
| Measure |
Decitabine 10 mg/m2 IV
n=17 Participants
10 mg/m2 by vein (IV) over 1 hour daily for 10 days
|
Decitabine 20 mg/m2 IV
n=93 Participants
20 mg/m2 IV over 1 hour daily for 5 days
|
Decitabine 20 mg/m2 SQ
n=14 Participants
20 mg/m2 subcutaneous (SQ) daily for 5 days
|
Total
n=124 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
|
0 Participants
n=4 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
7 Participants
n=5 Participants
|
45 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
59 Participants
n=4 Participants
|
|
Age, Categorical
>=65 years
|
10 Participants
n=5 Participants
|
48 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
65 Participants
n=4 Participants
|
|
Age Continuous
|
67 years
n=5 Participants
|
65 years
n=7 Participants
|
64 years
n=5 Participants
|
65 years
n=4 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=5 Participants
|
33 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
40 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
14 Participants
n=5 Participants
|
60 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
84 Participants
n=4 Participants
|
|
Region of Enrollment
United States
|
17 participants
n=5 Participants
|
93 participants
n=7 Participants
|
14 participants
n=5 Participants
|
124 participants
n=4 Participants
|
PRIMARY outcome
Timeframe: Response to treatment after 8 weeks of therapyPopulation: As treated: 124 patients completed treatment.
Objective responses by International Working Group criteria: 'Complete Response' (CR) defined as Normalization of the peripheral blood and bone marrow with \<5% bone marrow blasts, a peripheral blood granulocyte count \> (1.0 x 109/ L, and a platelet count \> 100 x 109/L); 'Other Response' including Partial Remission (PR) defined as above, except for the presence of 6-15% marrow blasts, or 50% reduction if \<15% at start of treatment combined with participants who meet all criteria for CR except for platelet recovery to \>100 x 109/L; and 'No Response'.
Outcome measures
| Measure |
Decitabine 10 mg/m2 IV
n=17 Participants
10 mg/m2 by vein (IV) over 1 hour daily for 10 days
|
Decitabine 20 mg/m2 IV
n=93 Participants
20 mg/m2 IV over 1 hour daily for 5 days
|
Decitabine 20 mg/m2 SQ
n=14 Participants
20 mg/m2 subcutaneous (SQ) daily for 5 days
|
|---|---|---|---|
|
Participant Responses
Complete Response
|
4 Participants
|
47 Participants
|
4 Participants
|
|
Participant Responses
Other Response
|
6 Participants
|
21 Participants
|
4 Participants
|
|
Participant Responses
No Response
|
7 Participants
|
25 Participants
|
6 Participants
|
Adverse Events
Decitabine 10 mg/m2 IV
Serious events: 5 serious events
Other events: 4 other events
Deaths: 0 deaths
Decitabine 20 mg/m2 IV
Serious events: 32 serious events
Other events: 32 other events
Deaths: 0 deaths
Decitabine 20 mg/m2 SQ
Serious events: 7 serious events
Other events: 8 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Decitabine 10 mg/m2 IV
n=17 participants at risk
10 mg/m2 by vein (IV) over 1 hour daily for 10 days
|
Decitabine 20 mg/m2 IV
n=93 participants at risk
20 mg/m2 IV over 1 hour daily for 5 days
|
Decitabine 20 mg/m2 SQ
n=14 participants at risk
20 mg/m2 subcutaneous (SQ) daily for 5 days
|
|---|---|---|---|
|
Renal and urinary disorders
Renal failure
|
11.8%
2/17 • Number of events 2 • 6 Years
|
1.1%
1/93 • Number of events 1 • 6 Years
|
0.00%
0/14 • 6 Years
|
|
General disorders
Adrenal Insufficiency
|
0.00%
0/17 • 6 Years
|
0.00%
0/93 • 6 Years
|
7.1%
1/14 • Number of events 1 • 6 Years
|
|
General disorders
Allergic Reaction
|
0.00%
0/17 • 6 Years
|
2.2%
2/93 • Number of events 2 • 6 Years
|
0.00%
0/14 • 6 Years
|
|
Cardiac disorders
Cardiac arrythmia
|
0.00%
0/17 • 6 Years
|
1.1%
1/93 • Number of events 1 • 6 Years
|
0.00%
0/14 • 6 Years
|
|
Cardiac disorders
Cardiac Ischemia
|
5.9%
1/17 • Number of events 1 • 6 Years
|
2.2%
2/93 • Number of events 2 • 6 Years
|
0.00%
0/14 • 6 Years
|
|
Cardiac disorders
Cardipulmonary arrest
|
0.00%
0/17 • 6 Years
|
0.00%
0/93 • 6 Years
|
7.1%
1/14 • Number of events 1 • 6 Years
|
|
Gastrointestinal disorders
Cholecystitis
|
0.00%
0/17 • 6 Years
|
1.1%
1/93 • Number of events 1 • 6 Years
|
0.00%
0/14 • 6 Years
|
|
General disorders
Death
|
0.00%
0/17 • 6 Years
|
6.5%
6/93 • Number of events 6 • 6 Years
|
7.1%
1/14 • Number of events 1 • 6 Years
|
|
Gastrointestinal disorders
Diarrhea
|
0.00%
0/17 • 6 Years
|
1.1%
1/93 • Number of events 1 • 6 Years
|
0.00%
0/14 • 6 Years
|
|
Gastrointestinal disorders
diverticulitis
|
5.9%
1/17 • Number of events 1 • 6 Years
|
0.00%
0/93 • 6 Years
|
0.00%
0/14 • 6 Years
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
11.8%
2/17 • Number of events 2 • 6 Years
|
1.1%
1/93 • Number of events 1 • 6 Years
|
0.00%
0/14 • 6 Years
|
|
Cardiac disorders
Elevated cardiac triponin
|
0.00%
0/17 • 6 Years
|
1.1%
1/93 • Number of events 1 • 6 Years
|
7.1%
1/14 • Number of events 1 • 6 Years
|
|
General disorders
Encephalopathy
|
0.00%
0/17 • 6 Years
|
1.1%
1/93 • Number of events 1 • 6 Years
|
0.00%
0/14 • 6 Years
|
|
Infections and infestations
Febrile neutropenia
|
0.00%
0/17 • 6 Years
|
1.1%
1/93 • Number of events 1 • 6 Years
|
7.1%
1/14 • Number of events 1 • 6 Years
|
|
Gastrointestinal disorders
Hemorrhage
|
0.00%
0/17 • 6 Years
|
1.1%
1/93 • Number of events 1 • 6 Years
|
0.00%
0/14 • 6 Years
|
|
Cardiac disorders
Hypotension
|
5.9%
1/17 • Number of events 1 • 6 Years
|
1.1%
1/93 • Number of events 1 • 6 Years
|
0.00%
0/14 • 6 Years
|
|
Gastrointestinal disorders
Ileus
|
0.00%
0/17 • 6 Years
|
0.00%
0/93 • 6 Years
|
7.1%
1/14 • Number of events 1 • 6 Years
|
|
Infections and infestations
Infection
|
11.8%
2/17 • Number of events 2 • 6 Years
|
3.2%
3/93 • Number of events 3 • 6 Years
|
0.00%
0/14 • 6 Years
|
|
Musculoskeletal and connective tissue disorders
Knee effusion
|
0.00%
0/17 • 6 Years
|
1.1%
1/93 • Number of events 1 • 6 Years
|
0.00%
0/14 • 6 Years
|
|
Nervous system disorders
Neuropathy
|
0.00%
0/17 • 6 Years
|
0.00%
0/93 • 6 Years
|
7.1%
1/14 • Number of events 1 • 6 Years
|
|
General disorders
Pain
|
23.5%
4/17 • Number of events 5 • 6 Years
|
14.0%
13/93 • Number of events 17 • 6 Years
|
21.4%
3/14 • Number of events 3 • 6 Years
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
0.00%
0/17 • 6 Years
|
1.1%
1/93 • Number of events 1 • 6 Years
|
0.00%
0/14 • 6 Years
|
|
Infections and infestations
Pneumonia
|
11.8%
2/17 • Number of events 2 • 6 Years
|
2.2%
2/93 • Number of events 2 • 6 Years
|
0.00%
0/14 • 6 Years
|
|
General disorders
Prolonged myelosuppression
|
0.00%
0/17 • 6 Years
|
0.00%
0/93 • 6 Years
|
7.1%
1/14 • Number of events 1 • 6 Years
|
|
Gastrointestinal disorders
Small bowel obstruction
|
0.00%
0/17 • 6 Years
|
0.00%
0/93 • 6 Years
|
7.1%
1/14 • Number of events 1 • 6 Years
|
|
Cardiac disorders
Supraventricular arrhythmia
|
5.9%
1/17 • Number of events 1 • 6 Years
|
0.00%
0/93 • 6 Years
|
0.00%
0/14 • 6 Years
|
|
Cardiac disorders
Syncope
|
0.00%
0/17 • 6 Years
|
0.00%
0/93 • 6 Years
|
7.1%
1/14 • Number of events 1 • 6 Years
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
5.9%
1/17 • Number of events 1 • 6 Years
|
0.00%
0/93 • 6 Years
|
0.00%
0/14 • 6 Years
|
|
General disorders
Vertigo
|
5.9%
1/17 • Number of events 1 • 6 Years
|
0.00%
0/93 • 6 Years
|
0.00%
0/14 • 6 Years
|
Other adverse events
| Measure |
Decitabine 10 mg/m2 IV
n=17 participants at risk
10 mg/m2 by vein (IV) over 1 hour daily for 10 days
|
Decitabine 20 mg/m2 IV
n=93 participants at risk
20 mg/m2 IV over 1 hour daily for 5 days
|
Decitabine 20 mg/m2 SQ
n=14 participants at risk
20 mg/m2 subcutaneous (SQ) daily for 5 days
|
|---|---|---|---|
|
General disorders
Allergic reaction
|
0.00%
0/17 • 6 Years
|
0.00%
0/93 • 6 Years
|
7.1%
1/14 • Number of events 1 • 6 Years
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
0.00%
0/17 • 6 Years
|
1.1%
1/93 • Number of events 1 • 6 Years
|
7.1%
1/14 • Number of events 1 • 6 Years
|
|
Hepatobiliary disorders
Elevated alanine aminotransferase
|
5.9%
1/17 • Number of events 1 • 6 Years
|
9.7%
9/93 • Number of events 9 • 6 Years
|
0.00%
0/14 • 6 Years
|
|
Gastrointestinal disorders
Anorexia
|
5.9%
1/17 • Number of events 1 • 6 Years
|
4.3%
4/93 • Number of events 4 • 6 Years
|
0.00%
0/14 • 6 Years
|
|
Hepatobiliary disorders
Elevated aspartate aminotransferase
|
5.9%
1/17 • Number of events 1 • 6 Years
|
7.5%
7/93 • Number of events 7 • 6 Years
|
0.00%
0/14 • 6 Years
|
|
Hepatobiliary disorders
Hyperbilirubinemia
|
5.9%
1/17 • Number of events 1 • 6 Years
|
8.6%
8/93 • Number of events 8 • 6 Years
|
7.1%
1/14 • Number of events 1 • 6 Years
|
|
Renal and urinary disorders
Elevated Creatinine
|
0.00%
0/17 • 6 Years
|
1.1%
1/93 • Number of events 1 • 6 Years
|
0.00%
0/14 • 6 Years
|
|
Gastrointestinal disorders
Diarrhea
|
0.00%
0/17 • 6 Years
|
1.1%
1/93 • Number of events 1 • 6 Years
|
7.1%
1/14 • Number of events 1 • 6 Years
|
|
General disorders
Fatigue
|
0.00%
0/17 • 6 Years
|
5.4%
5/93 • Number of events 5 • 6 Years
|
7.1%
1/14 • Number of events 1 • 6 Years
|
|
Skin and subcutaneous tissue disorders
Injection site reaction
|
5.9%
1/17 • Number of events 1 • 6 Years
|
0.00%
0/93 • 6 Years
|
7.1%
1/14 • Number of events 1 • 6 Years
|
|
Infections and infestations
Mucositis
|
0.00%
0/17 • 6 Years
|
1.1%
1/93 • Number of events 1 • 6 Years
|
0.00%
0/14 • 6 Years
|
|
Gastrointestinal disorders
Nausea
|
5.9%
1/17 • Number of events 1 • 6 Years
|
12.9%
12/93 • Number of events 13 • 6 Years
|
7.1%
1/14 • Number of events 1 • 6 Years
|
|
General disorders
Pain
|
0.00%
0/17 • 6 Years
|
3.2%
3/93 • Number of events 3 • 6 Years
|
7.1%
1/14 • Number of events 1 • 6 Years
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/17 • 6 Years
|
3.2%
3/93 • Number of events 3 • 6 Years
|
0.00%
0/14 • 6 Years
|
Additional Information
Hagop Kantarjian, MD / Professor
The University of Texas M. D. Anderson Cancer Center
Phone: 713-792-7026
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place