Trial Outcomes & Findings for Clofarabine, Cytarabine, and Filgrastim in Treating Patients With Newly Diagnosed Acute Myeloid Leukemia, Advanced Myelodysplastic Syndrome, and/or Advanced Myeloproliferative Neoplasm (NCT NCT01101880)

Last Updated: 2017-10-19

Results Overview

With 50 patients, the rates of these endpoints will be estimated with a standard error of 5 to 7 percentage points, depending on the observed rates. Complete remission is defined as less than 5% blast cells present in the bone marrow and count recovery (absolute neutrophil count greater than 1000/microL and platelet count greater than 100,000/microL). Complete remission with incomplete recovery of counts is defined as less than 5% blast cells present in the bone marrow without compete count recovery (absolute neutrophil count less than 1000/microL and platelet count less than 100,000/microL).

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

50 participants

Primary outcome timeframe

Up to 5 years

Results posted on

2017-10-19

Participant Flow

Participant milestones

Participant milestones
Measure	Treatment (Chemotherapy and Colony Stimulating Factor) INDUCTION THERAPY: Patients receive filgrastim SC daily beginning the day prior to chemotherapy and continuing until blood counts recover. Patients receive clofarabine IV over 1 hour followed by cytarabine IV over 2 hours daily for 5 days. CONSOLIDATION THERAPY: Patients receive filgrastim SC daily for 5 days beginning the day prior to chemotherapy. Patients receive clofarabine IV over 1 hour followed by cytarabine IV over 2 hours daily for 4 days. Treatment with induction therapy may continue for up to 2 courses and treatment with consolidation therapy may continue for up to 3 courses in the absence of disease progression or unacceptable toxicity. filgrastim: Given SC clofarabine: Given IV cytarabine: Given IV
Overall Study STARTED	50
Overall Study COMPLETED	50
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Clofarabine, Cytarabine, and Filgrastim in Treating Patients With Newly Diagnosed Acute Myeloid Leukemia, Advanced Myelodysplastic Syndrome, and/or Advanced Myeloproliferative Neoplasm

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treatment (Chemotherapy and Colony Stimulating Factor) n=50 Participants INDUCTION THERAPY: Patients receive filgrastim SC daily beginning the day prior to chemotherapy and continuing until blood counts recover. Patients receive clofarabine IV over 1 hour followed by cytarabine IV over 2 hours daily for 5 days. CONSOLIDATION THERAPY: Patients receive filgrastim SC daily for 5 days beginning the day prior to chemotherapy. Patients receive clofarabine IV over 1 hour followed by cytarabine IV over 2 hours daily for 4 days. Treatment with induction therapy may continue for up to 2 courses and treatment with consolidation therapy may continue for up to 3 courses in the absence of disease progression or unacceptable toxicity. filgrastim: Given SC clofarabine: Given IV cytarabine: Given IV
Age, Continuous	53 years n=5 Participants
Sex: Female, Male Female	23 Participants n=5 Participants
Sex: Female, Male Male	27 Participants n=5 Participants
Cytogenetic Risk Factor Favorable	4 Participants n=5 Participants
Cytogenetic Risk Factor Intermediate	32 Participants n=5 Participants
Cytogenetic Risk Factor Unfavorable	13 Participants n=5 Participants
Cytogenetic Risk Factor Indeterminate	1 Participants n=5 Participants
Antecedent hematological disorder (AHD)	23 Participants n=5 Participants
FLT3 ITD mutation status	10 Participants n=5 Participants
WBC	12 cells x 10^9/L n=5 Participants
Peripheral Blast	16 percent of white blood cells n=5 Participants

PRIMARY outcome

Timeframe: Up to 5 years

Population: CR achieved with no AHD only applies to those who did not have AHD.

Outcome measures

Outcome measures
Measure	Treatment (Chemotherapy and Colony Stimulating Factor) n=50 Participants INDUCTION THERAPY: Patients receive filgrastim SC daily beginning the day prior to chemotherapy and continuing until blood counts recover. Patients receive clofarabine IV over 1 hour followed by cytarabine IV over 2 hours daily for 5 days. CONSOLIDATION THERAPY: Patients receive filgrastim SC daily for 5 days beginning the day prior to chemotherapy. Patients receive clofarabine IV over 1 hour followed by cytarabine IV over 2 hours daily for 4 days. Treatment with induction therapy may continue for up to 2 courses and treatment with consolidation therapy may continue for up to 3 courses in the absence of disease progression or unacceptable toxicity. filgrastim: Given SC clofarabine: Given IV cytarabine: Given IV
Rates of Complete Remission and Complete Remission With Incomplete Recovery of Counts CR achieved	38 Participants
Rates of Complete Remission and Complete Remission With Incomplete Recovery of Counts CR achieved with first course of induction	33 Participants
Rates of Complete Remission and Complete Remission With Incomplete Recovery of Counts CR + CRp achieved	41 Participants
Rates of Complete Remission and Complete Remission With Incomplete Recovery of Counts CR achieved with no AHD	23 Participants
Rates of Complete Remission and Complete Remission With Incomplete Recovery of Counts CR achieved with AHD	15 Participants
Rates of Complete Remission and Complete Remission With Incomplete Recovery of Counts CR + CRp achieved with AHD	18 Participants
Rates of Complete Remission and Complete Remission With Incomplete Recovery of Counts CR achieved with FLT3 positive	7 Participants
Rates of Complete Remission and Complete Remission With Incomplete Recovery of Counts CR achieved with favorable risk cytogenetics	4 Participants
Rates of Complete Remission and Complete Remission With Incomplete Recovery of Counts CR achieved with intermediate risk cytogenetics	26 Participants
Rates of Complete Remission and Complete Remission With Incomplete Recovery of Counts CR + CRp achieved with intermediate risk cyto.	27 Participants
Rates of Complete Remission and Complete Remission With Incomplete Recovery of Counts CR achieved with unfavorable risk cytogenetics	8 Participants
Rates of Complete Remission and Complete Remission With Incomplete Recovery of Counts CR + CRp achieved with unfavorable risk cyto.	10 Participants

PRIMARY outcome

Timeframe: Up to 5 years

With 50 patients, the rates of these endpoints will be estimated with a standard error of 5 to 7 percentage points, depending on the observed rates. Remission is defined as less than 5% blasts in the bone marrow, no appearance of blasts in the peripheral blood, and no extramedullary disease (appearance of leukemic cells in other tissues).

Outcome measures

Outcome measures
Measure	Treatment (Chemotherapy and Colony Stimulating Factor) n=9 Participants INDUCTION THERAPY: Patients receive filgrastim SC daily beginning the day prior to chemotherapy and continuing until blood counts recover. Patients receive clofarabine IV over 1 hour followed by cytarabine IV over 2 hours daily for 5 days. CONSOLIDATION THERAPY: Patients receive filgrastim SC daily for 5 days beginning the day prior to chemotherapy. Patients receive clofarabine IV over 1 hour followed by cytarabine IV over 2 hours daily for 4 days. Treatment with induction therapy may continue for up to 2 courses and treatment with consolidation therapy may continue for up to 3 courses in the absence of disease progression or unacceptable toxicity. filgrastim: Given SC clofarabine: Given IV cytarabine: Given IV
Duration of Remission	7 weeks Interval 4.0 to 84.0

PRIMARY outcome

Timeframe: Up to 5 years

With 50 patients, the rates of these endpoints will be estimated with a standard error of 5 to 7 percentage points, depending on the observed rates.

Outcome measures

Outcome measures
Measure	Treatment (Chemotherapy and Colony Stimulating Factor) n=9 Participants INDUCTION THERAPY: Patients receive filgrastim SC daily beginning the day prior to chemotherapy and continuing until blood counts recover. Patients receive clofarabine IV over 1 hour followed by cytarabine IV over 2 hours daily for 5 days. CONSOLIDATION THERAPY: Patients receive filgrastim SC daily for 5 days beginning the day prior to chemotherapy. Patients receive clofarabine IV over 1 hour followed by cytarabine IV over 2 hours daily for 4 days. Treatment with induction therapy may continue for up to 2 courses and treatment with consolidation therapy may continue for up to 3 courses in the absence of disease progression or unacceptable toxicity. filgrastim: Given SC clofarabine: Given IV cytarabine: Given IV
Time to Progression	7 weeks Interval 4.0 to 84.0

PRIMARY outcome

Timeframe: Up to 5 years

Number of patients in remission at a median follow up of 15 months.

Outcome measures

Outcome measures
Measure	Treatment (Chemotherapy and Colony Stimulating Factor) n=50 Participants INDUCTION THERAPY: Patients receive filgrastim SC daily beginning the day prior to chemotherapy and continuing until blood counts recover. Patients receive clofarabine IV over 1 hour followed by cytarabine IV over 2 hours daily for 5 days. CONSOLIDATION THERAPY: Patients receive filgrastim SC daily for 5 days beginning the day prior to chemotherapy. Patients receive clofarabine IV over 1 hour followed by cytarabine IV over 2 hours daily for 4 days. Treatment with induction therapy may continue for up to 2 courses and treatment with consolidation therapy may continue for up to 3 courses in the absence of disease progression or unacceptable toxicity. filgrastim: Given SC clofarabine: Given IV cytarabine: Given IV
Event Free Survival	21 Participants

PRIMARY outcome

Timeframe: Up to 5 years

Population: Treatment-related mortality (TRM) data was not collected.

Treatment-related mortality (TRM) data was not collected.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: Up to 5 years

With 50 patients, the rates of these endpoints will be estimated with a standard error of 5 to 7 percentage points, depending on the observed rates.

Outcome measures

Outcome measures
Measure	Treatment (Chemotherapy and Colony Stimulating Factor) n=50 Participants INDUCTION THERAPY: Patients receive filgrastim SC daily beginning the day prior to chemotherapy and continuing until blood counts recover. Patients receive clofarabine IV over 1 hour followed by cytarabine IV over 2 hours daily for 5 days. CONSOLIDATION THERAPY: Patients receive filgrastim SC daily for 5 days beginning the day prior to chemotherapy. Patients receive clofarabine IV over 1 hour followed by cytarabine IV over 2 hours daily for 4 days. Treatment with induction therapy may continue for up to 2 courses and treatment with consolidation therapy may continue for up to 3 courses in the absence of disease progression or unacceptable toxicity. filgrastim: Given SC clofarabine: Given IV cytarabine: Given IV
Overall Survival	24.3 months Interval 15.0 to 60.0

Adverse Events

Treatment (Chemotherapy and Colony Stimulating Factor)

Serious events: 50 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Treatment (Chemotherapy and Colony Stimulating Factor) n=50 participants at risk INDUCTION THERAPY: Patients receive filgrastim SC daily beginning the day prior to chemotherapy and continuing until blood counts recover. Patients receive clofarabine IV over 1 hour followed by cytarabine IV over 2 hours daily for 5 days. CONSOLIDATION THERAPY: Patients receive filgrastim SC daily for 5 days beginning the day prior to chemotherapy. Patients receive clofarabine IV over 1 hour followed by cytarabine IV over 2 hours daily for 4 days. Treatment with induction therapy may continue for up to 2 courses and treatment with consolidation therapy may continue for up to 3 courses in the absence of disease progression or unacceptable toxicity. filgrastim: Given SC clofarabine: Given IV cytarabine: Given IV
Infections and infestations infection	100.0% 50/50 • Number of events 95 Other \[Not including Serious\] Adverse Events were not monitored/assessed.
Respiratory, thoracic and mediastinal disorders Pulmonary	62.0% 31/50 • Number of events 31 Other \[Not including Serious\] Adverse Events were not monitored/assessed.
Hepatobiliary disorders Liver enzymes	40.0% 20/50 • Number of events 20 Other \[Not including Serious\] Adverse Events were not monitored/assessed.
Gastrointestinal disorders Gastrointestinal	20.0% 10/50 • Number of events 10 Other \[Not including Serious\] Adverse Events were not monitored/assessed.
Metabolism and nutrition disorders Metabolic	20.0% 10/50 • Number of events 10 Other \[Not including Serious\] Adverse Events were not monitored/assessed.
Skin and subcutaneous tissue disorders Dermatologic	20.0% 10/50 • Number of events 10 Other \[Not including Serious\] Adverse Events were not monitored/assessed.
Nervous system disorders Neurologic	12.0% 6/50 • Number of events 6 Other \[Not including Serious\] Adverse Events were not monitored/assessed.
Blood and lymphatic system disorders Coagulation	8.0% 4/50 • Number of events 4 Other \[Not including Serious\] Adverse Events were not monitored/assessed.
Cardiac disorders Cardiovascular	4.0% 2/50 • Number of events 2 Other \[Not including Serious\] Adverse Events were not monitored/assessed.
Eye disorders Ocular	2.0% 1/50 • Number of events 1 Other \[Not including Serious\] Adverse Events were not monitored/assessed.
General disorders Pain	2.0% 1/50 • Number of events 1 Other \[Not including Serious\] Adverse Events were not monitored/assessed.
Hepatobiliary disorders Hepatobiliary	2.0% 1/50 • Number of events 1 Other \[Not including Serious\] Adverse Events were not monitored/assessed.
Renal and urinary disorders Urinary	2.0% 1/50 • Number of events 1 Other \[Not including Serious\] Adverse Events were not monitored/assessed.

Other adverse events

Adverse event data not reported

Additional Information

Pamela Becker, MD, PhD

University of Washington

Phone: 206-288-7273

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place