Trial Outcomes & Findings for Clofarabine vs Clofarabine in Plus With Low-Dose Ara-C in Previously Untreated Patients With Acute Myeloid Leukemia (AML) and High-Risk Myelodysplastic Syndromes (MDS). (NCT NCT00088218)
NCT ID: NCT00088218
Last Updated: 2012-08-07
Results Overview
Participant responses are categorized as 'Complete Remission,' Complete Remission, No Platelet Recovery,' 'No Response.' Complete Remission: Disappearance of all clinical and/or radiologic evidence of disease. Neutrophil count \> 1.0 x 109/L and platelet count \> 100 x 109/L, and normal bone marrow differential (\< 5% blasts); Complete Remission, No Platelet Recovery: Peripheral blood and bone marrow results as for CR, but with platelet counts of \< 100 x 109/L. Blood draws once a week until remission then every 2 to 8 weeks during therapy.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
95 participants
Primary outcome timeframe
Every 2 to 8 weeks
Results posted on
2012-08-07
Participant Flow
Recruitment Period 7/21/04 - 2/15/08; all patients were registered at The University of Texas M.D. Anderson Cancer Center.
Participant milestones
| Measure |
Clofarabine
Clofarabine intravenous (IV) 30 mg/m\^2 daily times 5 days
|
Clofarabine Plus Ara-C
Clofarabine IV 30 mg/m\^2 daily times 5 days + Ara-C 20 mg/m\^2 subcutaneously daily times 14 days.
|
|---|---|---|
|
Overall Study
STARTED
|
16
|
79
|
|
Overall Study
COMPLETED
|
16
|
79
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Clofarabine vs Clofarabine in Plus With Low-Dose Ara-C in Previously Untreated Patients With Acute Myeloid Leukemia (AML) and High-Risk Myelodysplastic Syndromes (MDS).
Baseline characteristics by cohort
| Measure |
Clofarabine
n=16 Participants
Clofarabine intravenous (IV) 30 mg/m\^2 daily times 5 days
|
Clofarabine Plus Ara-C
n=79 Participants
Clofarabine IV 30 mg/m\^2 daily times 5 days + Ara-C 20 mg/m\^2 subcutaneously daily times 14 days.
|
Total
n=95 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
|
1 Participants
n=5 Participants
|
12 Participants
n=7 Participants
|
13 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
15 Participants
n=5 Participants
|
67 Participants
n=7 Participants
|
82 Participants
n=5 Participants
|
|
Age Continuous
|
72 years
n=5 Participants
|
70 years
n=7 Participants
|
70 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=5 Participants
|
34 Participants
n=7 Participants
|
38 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
12 Participants
n=5 Participants
|
45 Participants
n=7 Participants
|
57 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
16 participants
n=5 Participants
|
79 participants
n=7 Participants
|
95 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Every 2 to 8 weeksPopulation: All treated subjects.
Participant responses are categorized as 'Complete Remission,' Complete Remission, No Platelet Recovery,' 'No Response.' Complete Remission: Disappearance of all clinical and/or radiologic evidence of disease. Neutrophil count \> 1.0 x 109/L and platelet count \> 100 x 109/L, and normal bone marrow differential (\< 5% blasts); Complete Remission, No Platelet Recovery: Peripheral blood and bone marrow results as for CR, but with platelet counts of \< 100 x 109/L. Blood draws once a week until remission then every 2 to 8 weeks during therapy.
Outcome measures
| Measure |
Clofarabine
n=16 Participants
Clofarabine intravenous (IV) 30 mg/m\^2 daily times 5 days
|
Clofarabine Plus Ara-C
n=79 Participants
Clofarabine IV 30 mg/m\^2 daily times 5 days + Ara-C 20 mg/m\^2 subcutaneously daily times 14 days.
|
|---|---|---|
|
Number of Participants With Response
Complete Remission
|
5 Participants
|
49 Participants
|
|
Number of Participants With Response
Complete Remission, No Platelet Recovery
|
0 Participants
|
4 Participants
|
|
Number of Participants With Response
No Response
|
11 Participants
|
26 Participants
|
Adverse Events
Clofarabine
Serious events: 12 serious events
Other events: 16 other events
Deaths: 0 deaths
Clofarabine Plus Ara-C
Serious events: 39 serious events
Other events: 54 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Clofarabine
n=16 participants at risk
Clofarabine intravenous (IV) 30 mg/m\^2 daily times 5 days
|
Clofarabine Plus Ara-C
n=79 participants at risk
Clofarabine IV 30 mg/m\^2 daily times 5 days + Ara-C 20 mg/m\^2 subcutaneously daily times 14 days.
|
|---|---|---|
|
Gastrointestinal disorders
GI Perforation
|
6.2%
1/16 • Number of events 1 • 3 years 7 months
|
0.00%
0/79 • 3 years 7 months
|
|
General disorders
Death
|
6.2%
1/16 • Number of events 1 • 3 years 7 months
|
6.3%
5/79 • Number of events 5 • 3 years 7 months
|
|
Cardiac disorders
Atrial Fibrillation
|
6.2%
1/16 • Number of events 1 • 3 years 7 months
|
0.00%
0/79 • 3 years 7 months
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
0.00%
0/16 • 3 years 7 months
|
10.1%
8/79 • Number of events 8 • 3 years 7 months
|
|
Respiratory, thoracic and mediastinal disorders
Adult Respiratiory Distress Syndrome
|
6.2%
1/16 • Number of events 1 • 3 years 7 months
|
0.00%
0/79 • 3 years 7 months
|
|
Renal and urinary disorders
Renal Failure
|
6.2%
1/16 • Number of events 1 • 3 years 7 months
|
7.6%
6/79 • Number of events 6 • 3 years 7 months
|
|
Infections and infestations
Pneumonia
|
12.5%
2/16 • Number of events 2 • 3 years 7 months
|
10.1%
8/79 • Number of events 8 • 3 years 7 months
|
|
Blood and lymphatic system disorders
Prolonged Myelosuppression
|
6.2%
1/16 • Number of events 1 • 3 years 7 months
|
7.6%
6/79 • Number of events 8 • 3 years 7 months
|
|
Infections and infestations
Febrile Neutropenia
|
6.2%
1/16 • Number of events 1 • 3 years 7 months
|
1.3%
1/79 • Number of events 1 • 3 years 7 months
|
|
Infections and infestations
Staphylococcus Infection
|
0.00%
0/16 • 3 years 7 months
|
2.5%
2/79 • Number of events 2 • 3 years 7 months
|
|
Infections and infestations
Sepsis Infection
|
6.2%
1/16 • Number of events 1 • 3 years 7 months
|
0.00%
0/79 • 3 years 7 months
|
|
Infections and infestations
Sinus Infection
|
0.00%
0/16 • 3 years 7 months
|
1.3%
1/79 • Number of events 1 • 3 years 7 months
|
|
Cardiac disorders
Elevated Triponin
|
0.00%
0/16 • 3 years 7 months
|
1.3%
1/79 • Number of events 1 • 3 years 7 months
|
|
Blood and lymphatic system disorders
Neutropenia
|
6.2%
1/16 • Number of events 1 • 3 years 7 months
|
1.3%
1/79 • Number of events 1 • 3 years 7 months
|
|
General disorders
Syncope
|
0.00%
0/16 • 3 years 7 months
|
2.5%
2/79 • Number of events 2 • 3 years 7 months
|
|
Infections and infestations
Infection
|
6.2%
1/16 • Number of events 1 • 3 years 7 months
|
3.8%
3/79 • Number of events 4 • 3 years 7 months
|
|
Infections and infestations
Fungal Infection
|
6.2%
1/16 • Number of events 1 • 3 years 7 months
|
0.00%
0/79 • 3 years 7 months
|
|
Gastrointestinal disorders
Diarrhea
|
6.2%
1/16 • Number of events 1 • 3 years 7 months
|
0.00%
0/79 • 3 years 7 months
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/16 • 3 years 7 months
|
1.3%
1/79 • Number of events 1 • 3 years 7 months
|
|
Cardiac disorders
Left Ventricular Systolic Dysfunction
|
0.00%
0/16 • 3 years 7 months
|
1.3%
1/79 • Number of events 1 • 3 years 7 months
|
|
Cardiac disorders
Tachycardia
|
0.00%
0/16 • 3 years 7 months
|
1.3%
1/79 • Number of events 1 • 3 years 7 months
|
|
Gastrointestinal disorders
Secondary Malignancy Colon Cancer
|
0.00%
0/16 • 3 years 7 months
|
1.3%
1/79 • Number of events 1 • 3 years 7 months
|
|
Infections and infestations
Bactremia Infection
|
0.00%
0/16 • 3 years 7 months
|
1.3%
1/79 • Number of events 1 • 3 years 7 months
|
|
Skin and subcutaneous tissue disorders
Secondary Malignancy Squamous Cell Carcinoma
|
0.00%
0/16 • 3 years 7 months
|
1.3%
1/79 • Number of events 1 • 3 years 7 months
|
|
General disorders
Pain
|
0.00%
0/16 • 3 years 7 months
|
1.3%
1/79 • Number of events 1 • 3 years 7 months
|
|
Gastrointestinal disorders
Cholecystitis
|
0.00%
0/16 • 3 years 7 months
|
1.3%
1/79 • Number of events 1 • 3 years 7 months
|
Other adverse events
| Measure |
Clofarabine
n=16 participants at risk
Clofarabine intravenous (IV) 30 mg/m\^2 daily times 5 days
|
Clofarabine Plus Ara-C
n=79 participants at risk
Clofarabine IV 30 mg/m\^2 daily times 5 days + Ara-C 20 mg/m\^2 subcutaneously daily times 14 days.
|
|---|---|---|
|
Gastrointestinal disorders
Mucositis
|
18.8%
3/16 • Number of events 3 • 3 years 7 months
|
30.4%
24/79 • Number of events 24 • 3 years 7 months
|
|
Hepatobiliary disorders
Elevated alanine aminotransferase
|
62.5%
10/16 • Number of events 10 • 3 years 7 months
|
44.3%
35/79 • Number of events 35 • 3 years 7 months
|
|
Skin and subcutaneous tissue disorders
Skin rash
|
56.2%
9/16 • Number of events 9 • 3 years 7 months
|
46.8%
37/79 • Number of events 37 • 3 years 7 months
|
|
Nervous system disorders
Headache
|
37.5%
6/16 • Number of events 6 • 3 years 7 months
|
34.2%
27/79 • Number of events 27 • 3 years 7 months
|
|
Gastrointestinal disorders
Emesis
|
56.2%
9/16 • Number of events 9 • 3 years 7 months
|
34.2%
27/79 • Number of events 27 • 3 years 7 months
|
|
General disorders
Edema
|
37.5%
6/16 • Number of events 6 • 3 years 7 months
|
36.7%
29/79 • Number of events 29 • 3 years 7 months
|
|
Hepatobiliary disorders
Elevated aspartate transaminase
|
31.2%
5/16 • Number of events 5 • 3 years 7 months
|
31.6%
25/79 • Number of events 25 • 3 years 7 months
|
|
Gastrointestinal disorders
Anorexia
|
18.8%
3/16 • Number of events 3 • 3 years 7 months
|
20.3%
16/79 • Number of events 16 • 3 years 7 months
|
|
Hepatobiliary disorders
Elevated alkaline phosphatase
|
18.8%
3/16 • Number of events 3 • 3 years 7 months
|
19.0%
15/79 • Number of events 15 • 3 years 7 months
|
|
Blood and lymphatic system disorders
Elevated Creatinine
|
31.2%
5/16 • Number of events 5 • 3 years 7 months
|
17.7%
14/79 • Number of events 14 • 3 years 7 months
|
|
Renal and urinary disorders
Acute renal failure
|
12.5%
2/16 • Number of events 2 • 3 years 7 months
|
3.8%
3/79 • Number of events 3 • 3 years 7 months
|
|
General disorders
Fatigue
|
18.8%
3/16 • Number of events 3 • 3 years 7 months
|
11.4%
9/79 • Number of events 9 • 3 years 7 months
|
|
Cardiac disorders
Atrial fibrillation
|
12.5%
2/16 • Number of events 2 • 3 years 7 months
|
10.1%
8/79 • Number of events 8 • 3 years 7 months
|
|
Nervous system disorders
Hand-Foot syndrome
|
12.5%
2/16 • Number of events 2 • 3 years 7 months
|
8.9%
7/79 • Number of events 7 • 3 years 7 months
|
|
General disorders
Facial flushing
|
6.2%
1/16 • Number of events 1 • 3 years 7 months
|
7.6%
6/79 • Number of events 6 • 3 years 7 months
|
|
Skin and subcutaneous tissue disorders
Puritis
|
12.5%
2/16 • Number of events 2 • 3 years 7 months
|
6.3%
5/79 • Number of events 5 • 3 years 7 months
|
|
Cardiac disorders
Chest tightness
|
0.00%
0/16 • 3 years 7 months
|
7.6%
6/79 • Number of events 6 • 3 years 7 months
|
|
Gastrointestinal disorders
Diarrhea
|
75.0%
12/16 • Number of events 12 • 3 years 7 months
|
60.8%
48/79 • Number of events 48 • 3 years 7 months
|
|
Gastrointestinal disorders
Nausea
|
75.0%
12/16 • Number of events 12 • 3 years 7 months
|
49.4%
39/79 • Number of events 39 • 3 years 7 months
|
|
Hepatobiliary disorders
Hyperbilirubinemia
|
62.5%
10/16 • Number of events 10 • 3 years 7 months
|
62.0%
49/79 • Number of events 49 • 3 years 7 months
|
Additional Information
Stefan Fader, M.D./Associate Professor
The University of Texas M. D. Anderson Cancer Center
Phone: 713/745-4613
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place