Trial Outcomes & Findings for 2 Arm Study of Clofarabine IV in MDS Patients (NCT NCT00422032)
NCT ID: NCT00422032
Last Updated: 2012-07-27
Results Overview
Response defined as Complete Remission (CR): Normalization of blood counts with neutrophils \>/= 1 \* 10\^9/L and platelet counts \>/= 100 \* 10\^9/L, and marrow blasts \</=5%; Partial Remission: as above except for presence of 6-15% marrow blasts, or 50% reduction if \<15% at start of treatment; or Hematologic Improvement (HI): Complete Response (CR) with the exception of a lack of platelet recovery to \>/= 100 \* 10\^9/L. Repeat bone marrow samples collected every 1-3 cycles (4-8 week cycle).
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
60 participants
Primary outcome timeframe
4 weeks (minimum 1 cycle) up to 24 weeks (maximum 3 cycles of 8 weeks)
Results posted on
2012-07-27
Participant Flow
Recruitment Period 1/31/06 - 9/21/09. All patients were registered at The University of Texas M.D. Anderson Cancer Center.
Of the 60 participants registered on this study, two (2) were excluded prior to receiving treatment.
Participant milestones
| Measure |
15 mg/m^2 Clofarabine
Lower Dose Clofarabine Group A: 15 mg/m\^2 intravenous (IV) over 1 hour daily for 5 days
|
30 mg/m^2 Clofarabine
Higher Dose Clofarabine Group B: 30 mg/m\^2 IV over 1 hour daily for 5 days
|
|---|---|---|
|
Overall Study
STARTED
|
37
|
21
|
|
Overall Study
COMPLETED
|
37
|
21
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
2 Arm Study of Clofarabine IV in MDS Patients
Baseline characteristics by cohort
| Measure |
15 mg/m^2 Clofarabine
n=37 Participants
Lower Dose Clofarabine Group A: 15 mg/m\^2 intravenous (IV) over 1 hour daily for 5 days
|
30 mg/m^2 Clofarabine
n=21 Participants
Higher Dose Clofarabine Group B: 30 mg/m\^2 IV over 1 hour daily for 5 days
|
Total
n=58 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age Continuous
|
68 Years
n=5 Participants
|
65 Years
n=7 Participants
|
68 Years
n=5 Participants
|
|
Sex: Female, Male
Female
|
13 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
20 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
24 Participants
n=5 Participants
|
14 Participants
n=7 Participants
|
38 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
37 participants
n=5 Participants
|
21 participants
n=7 Participants
|
58 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 4 weeks (minimum 1 cycle) up to 24 weeks (maximum 3 cycles of 8 weeks)Response defined as Complete Remission (CR): Normalization of blood counts with neutrophils \>/= 1 \* 10\^9/L and platelet counts \>/= 100 \* 10\^9/L, and marrow blasts \</=5%; Partial Remission: as above except for presence of 6-15% marrow blasts, or 50% reduction if \<15% at start of treatment; or Hematologic Improvement (HI): Complete Response (CR) with the exception of a lack of platelet recovery to \>/= 100 \* 10\^9/L. Repeat bone marrow samples collected every 1-3 cycles (4-8 week cycle).
Outcome measures
| Measure |
15 mg/m^2 Clofarabine
n=37 Participants
Lower Dose Clofarabine Group A: 15 mg/m\^2 intravenous (IV) over 1 hour daily for 5 days
|
30 mg/m^2 Clofarabine
n=21 Participants
Higher Dose Clofarabine Group B: 30 mg/m\^2 IV over 1 hour daily for 5 days
|
|---|---|---|
|
Number of Participants With Response for Two Dose Schedules of Clofarabine
Hematologic Improvement
|
5 Participants
|
1 Participants
|
|
Number of Participants With Response for Two Dose Schedules of Clofarabine
Complete Response
|
10 Participants
|
5 Participants
|
Adverse Events
15 mg/m^2 Clofarabine
Serious events: 7 serious events
Other events: 35 other events
Deaths: 0 deaths
30 mg/m^2 Clofarabine
Serious events: 8 serious events
Other events: 20 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
15 mg/m^2 Clofarabine
n=37 participants at risk
Lower Dose Clofarabine Group A: 15 mg/m\^2 intravenous (IV) over 1 hour daily for 5 days
|
30 mg/m^2 Clofarabine
n=21 participants at risk
Higher Dose Clofarabine Group B: 30 mg/m\^2 IV over 1 hour daily for 5 days
|
|---|---|---|
|
Renal and urinary disorders
Acute Renal Failure
|
5.4%
2/37 • Number of events 2 • 4 years, 10 months.
|
4.8%
1/21 • Number of events 1 • 4 years, 10 months.
|
|
Nervous system disorders
Hemorhage
|
2.7%
1/37 • Number of events 1 • 4 years, 10 months.
|
0.00%
0/21 • 4 years, 10 months.
|
|
Infections and infestations
Infection
|
5.4%
2/37 • Number of events 2 • 4 years, 10 months.
|
23.8%
5/21 • Number of events 5 • 4 years, 10 months.
|
|
Infections and infestations
Febrile Neutropenia
|
2.7%
1/37 • Number of events 1 • 4 years, 10 months.
|
0.00%
0/21 • 4 years, 10 months.
|
|
General disorders
Death
|
5.4%
2/37 • Number of events 2 • 4 years, 10 months.
|
4.8%
1/21 • Number of events 1 • 4 years, 10 months.
|
|
Cardiac disorders
Thrombosis
|
0.00%
0/37 • 4 years, 10 months.
|
4.8%
1/21 • Number of events 1 • 4 years, 10 months.
|
|
General disorders
Pain
|
0.00%
0/37 • 4 years, 10 months.
|
4.8%
1/21 • Number of events 1 • 4 years, 10 months.
|
Other adverse events
| Measure |
15 mg/m^2 Clofarabine
n=37 participants at risk
Lower Dose Clofarabine Group A: 15 mg/m\^2 intravenous (IV) over 1 hour daily for 5 days
|
30 mg/m^2 Clofarabine
n=21 participants at risk
Higher Dose Clofarabine Group B: 30 mg/m\^2 IV over 1 hour daily for 5 days
|
|---|---|---|
|
Gastrointestinal disorders
Nausea
|
62.2%
23/37 • Number of events 23 • 4 years, 10 months.
|
61.9%
13/21 • Number of events 13 • 4 years, 10 months.
|
|
Skin and subcutaneous tissue disorders
Skin Rash
|
43.2%
16/37 • Number of events 16 • 4 years, 10 months.
|
38.1%
8/21 • Number of events 8 • 4 years, 10 months.
|
|
Metabolism and nutrition disorders
Transaminase elevations
|
35.1%
13/37 • Number of events 13 • 4 years, 10 months.
|
66.7%
14/21 • Number of events 14 • 4 years, 10 months.
|
|
Gastrointestinal disorders
Diarrhea
|
27.0%
10/37 • Number of events 10 • 4 years, 10 months.
|
28.6%
6/21 • Number of events 6 • 4 years, 10 months.
|
|
Gastrointestinal disorders
Vomiting
|
24.3%
9/37 • Number of events 9 • 4 years, 10 months.
|
38.1%
8/21 • Number of events 8 • 4 years, 10 months.
|
|
Metabolism and nutrition disorders
Hyperbilirubinemia
|
18.9%
7/37 • Number of events 7 • 4 years, 10 months.
|
28.6%
6/21 • Number of events 6 • 4 years, 10 months.
|
|
Nervous system disorders
Headache
|
13.5%
5/37 • Number of events 5 • 4 years, 10 months.
|
19.0%
4/21 • Number of events 4 • 4 years, 10 months.
|
|
Metabolism and nutrition disorders
Elevated Creatinine
|
5.4%
2/37 • Number of events 2 • 4 years, 10 months.
|
28.6%
6/21 • Number of events 6 • 4 years, 10 months.
|
|
Gastrointestinal disorders
Mucositis
|
10.8%
4/37 • Number of events 4 • 4 years, 10 months.
|
4.8%
1/21 • Number of events 1 • 4 years, 10 months.
|
|
Gastrointestinal disorders
Constipation
|
8.1%
3/37 • Number of events 3 • 4 years, 10 months.
|
9.5%
2/21 • Number of events 2 • 4 years, 10 months.
|
|
Skin and subcutaneous tissue disorders
Pruritis
|
8.1%
3/37 • Number of events 3 • 4 years, 10 months.
|
4.8%
1/21 • Number of events 1 • 4 years, 10 months.
|
|
General disorders
Myalgia/bone pain
|
5.4%
2/37 • Number of events 2 • 4 years, 10 months.
|
9.5%
2/21 • Number of events 2 • 4 years, 10 months.
|
|
General disorders
Fatigue
|
5.4%
2/37 • Number of events 2 • 4 years, 10 months.
|
4.8%
1/21 • Number of events 1 • 4 years, 10 months.
|
|
General disorders
Edema
|
5.4%
2/37 • Number of events 2 • 4 years, 10 months.
|
4.8%
1/21 • Number of events 1 • 4 years, 10 months.
|
|
Renal and urinary disorders
Acute Renal Failure
|
0.00%
0/37 • 4 years, 10 months.
|
14.3%
3/21 • Number of events 3 • 4 years, 10 months.
|
|
Cardiac disorders
Congestive Heart Failure
|
0.00%
0/37 • 4 years, 10 months.
|
4.8%
1/21 • Number of events 1 • 4 years, 10 months.
|
|
General disorders
Weakness
|
0.00%
0/37 • 4 years, 10 months.
|
4.8%
1/21 • Number of events 1 • 4 years, 10 months.
|
|
Blood and lymphatic system disorders
Prolonged Myelosuppression
|
0.00%
0/37 • 4 years, 10 months.
|
4.8%
1/21 • Number of events 1 • 4 years, 10 months.
|
Additional Information
Hagop Kantarjian, MD / Professor
The University of Texas M. D. Anderson Cancer Center
Phone: 713-792-7026
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place