Trial Outcomes & Findings for Clofarabine and Ara-C for the Treatment of Relapsed AML and Untreated MDS (NCT NCT00334074)
NCT ID: NCT00334074
Last Updated: 2013-07-17
Results Overview
Based on International working group for diagnosis, standardization of response criteria, and treatment outcomes for reporting standards for therapeutic trials in Acute myeloid Leukemia: Complete Response (CR) was defined as normalization of marrow blasts (\< 5%), recovery of normal heamtopoiesis (absolute neutrophil count \>1 X 10\^9/l, platelet count ≥100 X10\^9/l, and absence of peripheral blood blasts, independent of transfusions and growth factor support. Partial response was defined as blood count recovery as for complete response with the exception of leukemic marrow blasts in the range of 6%-25% or a ≥50% decrease in bone marrow blasts. Treatment failure was defined as a \<25% change in marrow blasts within 30 days of starting therapy
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
30 participants
Primary outcome timeframe
Proportion of confirmed responses was estimated by the number of patients who achieved a CR or PR, defined as two consecutive evaluations at least 4 weeks apart, divided by the number of eligible participants in the study.
Results posted on
2013-07-17
Participant Flow
Patients were recruited after the initial IRB approval in september 2004 at Baylor University Medical Center. Enrollment was closed in october 2006. The study was completed Including follow up in February 2007.
Participant milestones
| Measure |
Clofarabine and Cytarabine
5 consecutive days of Clofarabine 40 mg/m\^2 intravenous infusion over 1 hour followed 4 hours later by cytarabine 1000mg/m\^2 intravenous infusion over 2 hours.Next cycle will start approximately 4 weeks after Day 1 of previous cycle. Patients will receive a maximum of 4 cycles of study treatment.
|
|---|---|
|
Overall Study
STARTED
|
30
|
|
Overall Study
COMPLETED
|
30
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Clofarabine and Ara-C for the Treatment of Relapsed AML and Untreated MDS
Baseline characteristics by cohort
| Measure |
Clofarabine and Cytarabine
n=30 Participants
Clofarabine 40 mg/m2 IV infusion over 1 hour for 5 days; Cytarabine 1000 mg/m2 IV infusion 4 hours post clofarabine IVI.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
10 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
20 Participants
n=5 Participants
|
|
Age Continuous
|
64 years
STANDARD_DEVIATION 21 • n=5 Participants
|
|
Sex: Female, Male
Female
|
13 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
17 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
30 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Proportion of confirmed responses was estimated by the number of patients who achieved a CR or PR, defined as two consecutive evaluations at least 4 weeks apart, divided by the number of eligible participants in the study.Population: Intent to Treat analysis; per eligible participants enrolled in the study.
Based on International working group for diagnosis, standardization of response criteria, and treatment outcomes for reporting standards for therapeutic trials in Acute myeloid Leukemia: Complete Response (CR) was defined as normalization of marrow blasts (\< 5%), recovery of normal heamtopoiesis (absolute neutrophil count \>1 X 10\^9/l, platelet count ≥100 X10\^9/l, and absence of peripheral blood blasts, independent of transfusions and growth factor support. Partial response was defined as blood count recovery as for complete response with the exception of leukemic marrow blasts in the range of 6%-25% or a ≥50% decrease in bone marrow blasts. Treatment failure was defined as a \<25% change in marrow blasts within 30 days of starting therapy
Outcome measures
| Measure |
Clofarabine Plus Cytarabine
n=30 Participants
Clofarabine 40 mg/m2 IV infusion over 1 hour for 5 days; Four hours post clofarabine infusion,Give cytarabine 1000 mg/m2 IV infusion over 2 hours.Patients will receive a maximum upto 4 cycles of study treatment.Next cycle will start approximately 4 weeks after Day 1 of previous cycle.
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|---|---|
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Response Rate (Complete Response [CR] Plus Partial Response [PR]) of Clofarabine Plus Cytarabine in Patients With Relapsed/Refractory AML, Untreated MDS, CML in Blast Phase, or in Selected Untreated Patients With High Risk of Anthracycline Toxicity
Overall Response Rate
|
16 participants
|
|
Response Rate (Complete Response [CR] Plus Partial Response [PR]) of Clofarabine Plus Cytarabine in Patients With Relapsed/Refractory AML, Untreated MDS, CML in Blast Phase, or in Selected Untreated Patients With High Risk of Anthracycline Toxicity
Complete Response
|
14 participants
|
|
Response Rate (Complete Response [CR] Plus Partial Response [PR]) of Clofarabine Plus Cytarabine in Patients With Relapsed/Refractory AML, Untreated MDS, CML in Blast Phase, or in Selected Untreated Patients With High Risk of Anthracycline Toxicity
Partial Response
|
2 participants
|
|
Response Rate (Complete Response [CR] Plus Partial Response [PR]) of Clofarabine Plus Cytarabine in Patients With Relapsed/Refractory AML, Untreated MDS, CML in Blast Phase, or in Selected Untreated Patients With High Risk of Anthracycline Toxicity
Not Responding
|
8 participants
|
|
Response Rate (Complete Response [CR] Plus Partial Response [PR]) of Clofarabine Plus Cytarabine in Patients With Relapsed/Refractory AML, Untreated MDS, CML in Blast Phase, or in Selected Untreated Patients With High Risk of Anthracycline Toxicity
Not evaluable
|
6 participants
|
SECONDARY outcome
Timeframe: Up to five months (includes follow up period of 30 days) from the day patient received their first dose of study drugPopulation: Intention To Treat
Patients will be monitored clinically and diagnostically using measures including blood test, bone marrow aspiration and MUGA. Toxicity assessment every week using the NCI Common Terminology Criteria for Adverse Events (CTCAE) version 3.0 will be performed.
Outcome measures
| Measure |
Clofarabine Plus Cytarabine
n=30 Participants
Clofarabine 40 mg/m2 IV infusion over 1 hour for 5 days; Four hours post clofarabine infusion,Give cytarabine 1000 mg/m2 IV infusion over 2 hours.Patients will receive a maximum upto 4 cycles of study treatment.Next cycle will start approximately 4 weeks after Day 1 of previous cycle.
|
|---|---|
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Number of Participants Who Had an Adverse Event While on Treatment With Clofarabine Plus Cytarabine
|
30 participants; with adverse events
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Adverse Events
Clofarabine and Cytarabine
Serious events: 30 serious events
Other events: 29 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Clofarabine and Cytarabine
n=30 participants at risk
Clofarabine 40 mg/m2 intravenous infusion over 1 hour for 5 days; Cytarabine 1000 mg/m2 intravenous infusion 4 hours post clofarabine IVI.
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|---|---|
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Cardiac disorders
Atrial Fibrillation
|
10.0%
3/30 • Number of events 3 • 1 year 6 months. Initial IRB approval in 9/2004. Enrollment start date - 8/2005. Study completion including follow up - Feb 2007.
|
|
Blood and lymphatic system disorders
Edema/Weight Gain
|
10.0%
3/30 • Number of events 3 • 1 year 6 months. Initial IRB approval in 9/2004. Enrollment start date - 8/2005. Study completion including follow up - Feb 2007.
|
|
Investigations
Elevated LFT
|
3.3%
1/30 • Number of events 1 • 1 year 6 months. Initial IRB approval in 9/2004. Enrollment start date - 8/2005. Study completion including follow up - Feb 2007.
|
|
Infections and infestations
Febrile Neutropenia
|
6.7%
2/30 • Number of events 2 • 1 year 6 months. Initial IRB approval in 9/2004. Enrollment start date - 8/2005. Study completion including follow up - Feb 2007.
|
|
Infections and infestations
Fever
|
10.0%
3/30 • Number of events 3 • 1 year 6 months. Initial IRB approval in 9/2004. Enrollment start date - 8/2005. Study completion including follow up - Feb 2007.
|
|
Infections and infestations
Fungal Pneumonia
|
6.7%
2/30 • Number of events 2 • 1 year 6 months. Initial IRB approval in 9/2004. Enrollment start date - 8/2005. Study completion including follow up - Feb 2007.
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|
Skin and subcutaneous tissue disorders
Hand and Foot Syndrome
|
10.0%
3/30 • Number of events 3 • 1 year 6 months. Initial IRB approval in 9/2004. Enrollment start date - 8/2005. Study completion including follow up - Feb 2007.
|
|
Cardiac disorders
Myocardial Infarction
|
3.3%
1/30 • Number of events 1 • 1 year 6 months. Initial IRB approval in 9/2004. Enrollment start date - 8/2005. Study completion including follow up - Feb 2007.
|
|
Gastrointestinal disorders
Nausea
|
3.3%
1/30 • Number of events 1 • 1 year 6 months. Initial IRB approval in 9/2004. Enrollment start date - 8/2005. Study completion including follow up - Feb 2007.
|
|
Blood and lymphatic system disorders
Pancytopenia
|
80.0%
24/30 • Number of events 24 • 1 year 6 months. Initial IRB approval in 9/2004. Enrollment start date - 8/2005. Study completion including follow up - Feb 2007.
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|
Blood and lymphatic system disorders
Platelet Allosensitization
|
3.3%
1/30 • Number of events 1 • 1 year 6 months. Initial IRB approval in 9/2004. Enrollment start date - 8/2005. Study completion including follow up - Feb 2007.
|
|
Infections and infestations
Sepsis
|
16.7%
5/30 • Number of events 5 • 1 year 6 months. Initial IRB approval in 9/2004. Enrollment start date - 8/2005. Study completion including follow up - Feb 2007.
|
|
Gastrointestinal disorders
Small Bowell Obstruction
|
3.3%
1/30 • Number of events 1 • 1 year 6 months. Initial IRB approval in 9/2004. Enrollment start date - 8/2005. Study completion including follow up - Feb 2007.
|
|
Infections and infestations
Staph Bacteremia
|
3.3%
1/30 • Number of events 1 • 1 year 6 months. Initial IRB approval in 9/2004. Enrollment start date - 8/2005. Study completion including follow up - Feb 2007.
|
|
Infections and infestations
Staph epidermitis
|
3.3%
1/30 • Number of events 1 • 1 year 6 months. Initial IRB approval in 9/2004. Enrollment start date - 8/2005. Study completion including follow up - Feb 2007.
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
3.3%
1/30 • Number of events 1 • 1 year 6 months. Initial IRB approval in 9/2004. Enrollment start date - 8/2005. Study completion including follow up - Feb 2007.
|
|
Blood and lymphatic system disorders
Volume Overload
|
3.3%
1/30 • Number of events 1 • 1 year 6 months. Initial IRB approval in 9/2004. Enrollment start date - 8/2005. Study completion including follow up - Feb 2007.
|
|
Infections and infestations
Bilateral External Otitis
|
3.3%
1/30 • Number of events 1 • 1 year 6 months. Initial IRB approval in 9/2004. Enrollment start date - 8/2005. Study completion including follow up - Feb 2007.
|
|
Nervous system disorders
Delirium
|
3.3%
1/30 • Number of events 1 • 1 year 6 months. Initial IRB approval in 9/2004. Enrollment start date - 8/2005. Study completion including follow up - Feb 2007.
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
3.3%
1/30 • Number of events 1 • 1 year 6 months. Initial IRB approval in 9/2004. Enrollment start date - 8/2005. Study completion including follow up - Feb 2007.
|
|
Infections and infestations
Multiple System Organ Failure
|
20.0%
6/30 • Number of events 6 • 1 year 6 months. Initial IRB approval in 9/2004. Enrollment start date - 8/2005. Study completion including follow up - Feb 2007.
|
|
Gastrointestinal disorders
Perianal Abcess
|
3.3%
1/30 • Number of events 1 • 1 year 6 months. Initial IRB approval in 9/2004. Enrollment start date - 8/2005. Study completion including follow up - Feb 2007.
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary Infiltrate
|
3.3%
1/30 • Number of events 1 • 1 year 6 months. Initial IRB approval in 9/2004. Enrollment start date - 8/2005. Study completion including follow up - Feb 2007.
|
|
Renal and urinary disorders
Renal Insufficiency / Failure
|
10.0%
3/30 • Number of events 3 • 1 year 6 months. Initial IRB approval in 9/2004. Enrollment start date - 8/2005. Study completion including follow up - Feb 2007.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory Failure
|
10.0%
3/30 • Number of events 3 • 1 year 6 months. Initial IRB approval in 9/2004. Enrollment start date - 8/2005. Study completion including follow up - Feb 2007.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory Distress
|
6.7%
2/30 • Number of events 2 • 1 year 6 months. Initial IRB approval in 9/2004. Enrollment start date - 8/2005. Study completion including follow up - Feb 2007.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia
|
6.7%
2/30 • Number of events 2 • 1 year 6 months. Initial IRB approval in 9/2004. Enrollment start date - 8/2005. Study completion including follow up - Feb 2007.
|
Other adverse events
| Measure |
Clofarabine and Cytarabine
n=30 participants at risk
Clofarabine 40 mg/m2 intravenous infusion over 1 hour for 5 days; Cytarabine 1000 mg/m2 intravenous infusion 4 hours post clofarabine IVI.
|
|---|---|
|
Blood and lymphatic system disorders
Edema/Weight Gain
|
46.7%
14/30 • Number of events 14 • 1 year 6 months. Initial IRB approval in 9/2004. Enrollment start date - 8/2005. Study completion including follow up - Feb 2007.
|
|
Gastrointestinal disorders
Diarrhea
|
56.7%
17/30 • Number of events 17 • 1 year 6 months. Initial IRB approval in 9/2004. Enrollment start date - 8/2005. Study completion including follow up - Feb 2007.
|
|
Infections and infestations
Fever
|
43.3%
13/30 • Number of events 13 • 1 year 6 months. Initial IRB approval in 9/2004. Enrollment start date - 8/2005. Study completion including follow up - Feb 2007.
|
|
Gastrointestinal disorders
Nausea
|
40.0%
12/30 • Number of events 12 • 1 year 6 months. Initial IRB approval in 9/2004. Enrollment start date - 8/2005. Study completion including follow up - Feb 2007.
|
|
Skin and subcutaneous tissue disorders
Rash
|
43.3%
13/30 • Number of events 13 • 1 year 6 months. Initial IRB approval in 9/2004. Enrollment start date - 8/2005. Study completion including follow up - Feb 2007.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60