Trial Outcomes & Findings for Azacytidine With Valproic Acid Versus Ara-C in Acute Myeloid Leukemia (AML)/ Myelodysplastic Syndrome (MDS) Patients (NCT NCT00382590)
NCT ID: NCT00382590
Last Updated: 2012-08-07
Results Overview
Patient response defined by: Death, Resistant to Therapy \[no major hematologic improvement using International Myelodysplastic Syndromes (MDS) Working Group (Cheson B, Bennett J, Kantarjian H et al, Blood 2006) criteria after a maximum of 4 courses\], or Relapse.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
11 participants
Primary outcome timeframe
Evaluated every 3 weeks, following 4 courses (16/24 weeks ) and till study end
Results posted on
2012-08-07
Participant Flow
Recruitment Period: 09/20/05 through 11/01/06. All participants recruited at UT MD Anderson Cancer Center.
Eleven (11) patients were registered, one (1) patient was not eligible and was taken off study prior to receiving study drug.
Participant milestones
| Measure |
5-Aza + VPA
5-Azacytidine (5-Aza) 75 mg/m\^2 subcutaneously daily + Valproic Acid (VPA) 50 mg/m\^2 orally daily, each for 7 days
|
Ara-C
Low-Dose Cytarabine (Ara-C) 20 mg twice daily subcutaneously for 10 days.
|
|---|---|---|
|
Overall Study
STARTED
|
4
|
6
|
|
Overall Study
COMPLETED
|
4
|
6
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Azacytidine With Valproic Acid Versus Ara-C in Acute Myeloid Leukemia (AML)/ Myelodysplastic Syndrome (MDS) Patients
Baseline characteristics by cohort
| Measure |
5-Aza + VPA
n=4 Participants
5-Azacytidine (5-Aza) 75 mg/m\^2 subcutaneously daily + Valproic Acid (VPA) 50 mg/m\^2 orally daily, each for 7 days
|
Ara-C
n=6 Participants
Low-Dose Cytarabine (Ara-C) 20 mg twice daily subcutaneously for 10 days.
|
Total
n=10 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age Continuous
|
75 years
n=5 Participants
|
76.5 years
n=7 Participants
|
75.5 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
4 participants
n=5 Participants
|
6 participants
n=7 Participants
|
10 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Evaluated every 3 weeks, following 4 courses (16/24 weeks ) and till study endPopulation: The analysis was per intention to treat. One participant was inevaluable for response.
Patient response defined by: Death, Resistant to Therapy \[no major hematologic improvement using International Myelodysplastic Syndromes (MDS) Working Group (Cheson B, Bennett J, Kantarjian H et al, Blood 2006) criteria after a maximum of 4 courses\], or Relapse.
Outcome measures
| Measure |
5-Aza + VPA
n=4 Participants
5-Azacytidine (5-Aza) 75 mg/m\^2 subcutaneously daily + Valproic Acid (VPA) 50 mg/m\^2 orally daily, each for 7 days
|
Ara-C
n=5 Participants
Low-Dose Cytarabine (Ara-C) 20 mg twice daily subcutaneously for 10 days.
|
|---|---|---|
|
Number of Participants With Response
Death
|
0 Participants
|
0 Participants
|
|
Number of Participants With Response
Resistant to Therapy
|
4 Participants
|
5 Participants
|
|
Number of Participants With Response
Relapse
|
0 Participants
|
0 Participants
|
Adverse Events
5-Aza + VPA
Serious events: 4 serious events
Other events: 2 other events
Deaths: 0 deaths
Ara-C
Serious events: 6 serious events
Other events: 4 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
5-Aza + VPA
n=4 participants at risk
5-Azacytidine (5-Aza) 75 mg/m\^2 subcutaneously daily + Valproic Acid (VPA) 50 mg/m\^2 orally daily, each for 7 days
|
Ara-C
n=6 participants at risk
Low-Dose Cytarabine (Ara-C) 20 mg twice daily subcutaneously for 10 days.
|
|---|---|---|
|
Blood and lymphatic system disorders
Elevated liver function test
|
0.00%
0/4 • 1 Year 2 months
|
16.7%
1/6 • Number of events 1 • 1 Year 2 months
|
|
Infections and infestations
Infection
|
25.0%
1/4 • Number of events 1 • 1 Year 2 months
|
16.7%
1/6 • Number of events 1 • 1 Year 2 months
|
|
Infections and infestations
Pneumonia
|
0.00%
0/4 • 1 Year 2 months
|
16.7%
1/6 • Number of events 1 • 1 Year 2 months
|
|
Cardiac disorders
Congestive Heart Failure
|
0.00%
0/4 • 1 Year 2 months
|
16.7%
1/6 • Number of events 1 • 1 Year 2 months
|
|
Vascular disorders
Right periocular hemorrhage
|
0.00%
0/4 • 1 Year 2 months
|
16.7%
1/6 • Number of events 1 • 1 Year 2 months
|
|
Vascular disorders
Left ocular hemorrhage
|
0.00%
0/4 • 1 Year 2 months
|
16.7%
1/6 • Number of events 1 • 1 Year 2 months
|
|
Blood and lymphatic system disorders
Anemia
|
0.00%
0/4 • 1 Year 2 months
|
33.3%
2/6 • Number of events 2 • 1 Year 2 months
|
|
Nervous system disorders
Confusion
|
25.0%
1/4 • Number of events 1 • 1 Year 2 months
|
16.7%
1/6 • Number of events 1 • 1 Year 2 months
|
|
General disorders
Death
|
25.0%
1/4 • Number of events 1 • 1 Year 2 months
|
33.3%
2/6 • Number of events 2 • 1 Year 2 months
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
0.00%
0/4 • 1 Year 2 months
|
16.7%
1/6 • Number of events 1 • 1 Year 2 months
|
|
Nervous system disorders
Lethargic fall
|
25.0%
1/4 • Number of events 1 • 1 Year 2 months
|
0.00%
0/6 • 1 Year 2 months
|
|
Blood and lymphatic system disorders
Pancytopenia
|
25.0%
1/4 • Number of events 1 • 1 Year 2 months
|
0.00%
0/6 • 1 Year 2 months
|
|
Skin and subcutaneous tissue disorders
Rash
|
25.0%
1/4 • Number of events 1 • 1 Year 2 months
|
0.00%
0/6 • 1 Year 2 months
|
|
Cardiac disorders
Atrail fibrillation
|
0.00%
0/4 • 1 Year 2 months
|
16.7%
1/6 • Number of events 1 • 1 Year 2 months
|
Other adverse events
| Measure |
5-Aza + VPA
n=4 participants at risk
5-Azacytidine (5-Aza) 75 mg/m\^2 subcutaneously daily + Valproic Acid (VPA) 50 mg/m\^2 orally daily, each for 7 days
|
Ara-C
n=6 participants at risk
Low-Dose Cytarabine (Ara-C) 20 mg twice daily subcutaneously for 10 days.
|
|---|---|---|
|
Cardiac disorders
Arrythmia
|
0.00%
0/4 • 1 Year 2 months
|
16.7%
1/6 • Number of events 1 • 1 Year 2 months
|
|
Renal and urinary disorders
Acute Renal Failure
|
25.0%
1/4 • Number of events 1 • 1 Year 2 months
|
0.00%
0/6 • 1 Year 2 months
|
|
General disorders
Tumor lysis syndrome
|
25.0%
1/4 • Number of events 1 • 1 Year 2 months
|
0.00%
0/6 • 1 Year 2 months
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
25.0%
1/4 • Number of events 1 • 1 Year 2 months
|
0.00%
0/6 • 1 Year 2 months
|
|
Blood and lymphatic system disorders
Anemia
|
25.0%
1/4 • Number of events 1 • 1 Year 2 months
|
16.7%
1/6 • Number of events 1 • 1 Year 2 months
|
|
Blood and lymphatic system disorders
Leukopenia
|
25.0%
1/4 • Number of events 1 • 1 Year 2 months
|
0.00%
0/6 • 1 Year 2 months
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
25.0%
1/4 • Number of events 1 • 1 Year 2 months
|
0.00%
0/6 • 1 Year 2 months
|
|
Gastrointestinal disorders
fatigue
|
0.00%
0/4 • 1 Year 2 months
|
16.7%
1/6 • Number of events 1 • 1 Year 2 months
|
|
General disorders
edema
|
0.00%
0/4 • 1 Year 2 months
|
16.7%
1/6 • Number of events 1 • 1 Year 2 months
|
|
Cardiac disorders
chest pain
|
0.00%
0/4 • 1 Year 2 months
|
16.7%
1/6 • Number of events 1 • 1 Year 2 months
|
|
Nervous system disorders
depression
|
0.00%
0/4 • 1 Year 2 months
|
16.7%
1/6 • Number of events 1 • 1 Year 2 months
|
|
Infections and infestations
Infection
|
0.00%
0/4 • 1 Year 2 months
|
16.7%
1/6 • Number of events 1 • 1 Year 2 months
|
Additional Information
Guillermo Garcia-Manero, MD / Associate Professor
UT MD Anderson Cancer Center
Phone: 713-745-3428
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place