Trial Outcomes & Findings for Phase II 5-Azacytidine Plus VPA Plus ATRA (NCT NCT00326170)
NCT ID: NCT00326170
Last Updated: 2012-06-15
Results Overview
Clinical activity of combination defined as: Complete Response (CR), bone marrow with 5% or fewer blasts and peripheral blood count with an absolute neutrophil count of 10\^9/L or more and platelet count of 100x10\^9 or more; Complete response without platelets (CRp), a complete response except for a platelet count less than 100x10\^9 and transfusion independent; and Bone Marrow (BM) Response, bone marrow blast of 5% or less but without meeting the peripheral blood count criteria for (CR) or (CRp).
COMPLETED
PHASE2
34 participants
Up to 12 cycles of treatment (28 day cycles)
2012-06-15
Participant Flow
Recruitment Period: 06/10/05 through 11/17/06. All participants recruited at UT MD Anderson Cancer Center.
Participant milestones
| Measure |
VPA + 5-aza + ATRA
Daily for 7 days, Valproic acid (VPA) starting dose 75 mg/m\^2 subcutaneously in combination with 5-azacytidine (5-aza) 50 mg/kg orally; and all-trans retinoic acid (ATRA) 45 mg/m\^2 orally daily (in two divided doses) for 5 days starting on day 3.
|
|---|---|
|
Overall Study
STARTED
|
34
|
|
Overall Study
COMPLETED
|
34
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Phase II 5-Azacytidine Plus VPA Plus ATRA
Baseline characteristics by cohort
| Measure |
VPA + 5-aza + ATRA
n=34 Participants
Daily for 7 days, Valproic acid (VPA) starting dose 75 mg/m\^2 subcutaneously in combination with 5-azacytidine (5-aza) 50 mg/kg orally; and all-trans retinoic acid (ATRA) 45 mg/m\^2 orally daily (in two divided doses) for 5 days starting on day 3.
|
|---|---|
|
Age Continuous
|
70 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
11 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
23 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
34 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Up to 12 cycles of treatment (28 day cycles)Clinical activity of combination defined as: Complete Response (CR), bone marrow with 5% or fewer blasts and peripheral blood count with an absolute neutrophil count of 10\^9/L or more and platelet count of 100x10\^9 or more; Complete response without platelets (CRp), a complete response except for a platelet count less than 100x10\^9 and transfusion independent; and Bone Marrow (BM) Response, bone marrow blast of 5% or less but without meeting the peripheral blood count criteria for (CR) or (CRp).
Outcome measures
| Measure |
VPA + 5-aza + ATRA
n=34 Participants
Daily for 7 days, Valproic acid (VPA) starting dose 75 mg/m\^2 subcutaneously in combination with 5-azacytidine (5-aza) 50 mg/kg orally; and all-trans retinoic acid (ATRA) 45 mg/m\^2 orally daily (in two divided doses) for 5 days starting on day 3.
|
|---|---|
|
Number of Participants With Response
CR
|
12 Participants
|
|
Number of Participants With Response
CRp
|
3 Participants
|
|
Number of Participants With Response
BM
|
7 Participants
|
Adverse Events
VPA + 5-aza + ATRA
Serious adverse events
| Measure |
VPA + 5-aza + ATRA
n=34 participants at risk
Daily for 7 days, Valproic acid (VPA) starting dose 75 mg/m\^2 subcutaneously in combination with 5-azacytidine (5-aza) 50 mg/kg orally; and all-trans retinoic acid (ATRA) 45 mg/m\^2 orally daily (in two divided doses) for 5 days starting on day 3.
|
|---|---|
|
Infections and infestations
Infection
|
23.5%
8/34 • Number of events 8 • 2 Years
|
|
General disorders
Death
|
32.4%
11/34 • Number of events 11 • 2 Years
|
|
General disorders
Fatigue
|
20.6%
7/34 • Number of events 8 • 2 Years
|
|
Gastrointestinal disorders
Diarrhea
|
11.8%
4/34 • Number of events 4 • 2 Years
|
|
Nervous system disorders
Somnolence
|
29.4%
10/34 • Number of events 11 • 2 Years
|
|
Nervous system disorders
Syncope
|
5.9%
2/34 • Number of events 2 • 2 Years
|
|
Nervous system disorders
Confusion
|
11.8%
4/34 • Number of events 4 • 2 Years
|
|
General disorders
Pain
|
11.8%
4/34 • Number of events 4 • 2 Years
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
11.8%
4/34 • Number of events 4 • 2 Years
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
2.9%
1/34 • Number of events 1 • 2 Years
|
|
Skin and subcutaneous tissue disorders
Rash
|
5.9%
2/34 • Number of events 2 • 2 Years
|
|
Gastrointestinal disorders
Dehydration
|
5.9%
2/34 • Number of events 2 • 2 Years
|
|
Gastrointestinal disorders
Constipation
|
2.9%
1/34 • Number of events 1 • 2 Years
|
|
Blood and lymphatic system disorders
Pancytopenia
|
5.9%
2/34 • Number of events 2 • 2 Years
|
|
Cardiac disorders
Atrial Fibrillation
|
2.9%
1/34 • Number of events 1 • 2 Years
|
|
Vascular disorders
Hemorrhage
|
5.9%
2/34 • Number of events 2 • 2 Years
|
|
General disorders
Fever
|
2.9%
1/34 • Number of events 1 • 2 Years
|
|
Blood and lymphatic system disorders
Edema
|
2.9%
1/34 • Number of events 2 • 2 Years
|
Other adverse events
| Measure |
VPA + 5-aza + ATRA
n=34 participants at risk
Daily for 7 days, Valproic acid (VPA) starting dose 75 mg/m\^2 subcutaneously in combination with 5-azacytidine (5-aza) 50 mg/kg orally; and all-trans retinoic acid (ATRA) 45 mg/m\^2 orally daily (in two divided doses) for 5 days starting on day 3.
|
|---|---|
|
General disorders
Weakness
|
14.7%
5/34 • Number of events 5 • 2 Years
|
|
Gastrointestinal disorders
Vomiting
|
2.9%
1/34 • Number of events 1 • 2 Years
|
|
General disorders
Fatigue
|
2.9%
1/34 • Number of events 1 • 2 Years
|
|
Nervous system disorders
Confusion
|
11.8%
4/34 • Number of events 4 • 2 Years
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
2.9%
1/34 • Number of events 1 • 2 Years
|
|
Nervous system disorders
Somnolence
|
5.9%
2/34 • Number of events 2 • 2 Years
|
|
General disorders
Fever
|
2.9%
1/34 • Number of events 1 • 2 Years
|
|
Renal and urinary disorders
Nocturia
|
2.9%
1/34 • Number of events 1 • 2 Years
|
Additional Information
Guillermo Garcia-Manero, MD / Associate Professor
UT MD Anderson Cancer Center
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place