Trial Outcomes & Findings for Clofarabine, Cytarabine, and G-CSF in Treating Patients With Myelodysplastic Syndromes (NCT NCT00503880)

Last Updated: 2023-09-18

Results Overview

Maximum Tolerated Dose (MTD) is defined to be the dose cohort below which 2 out of 6 patients experience dose limiting toxicities or the highest dose cohort, if 2 limiting toxicities are not observed at any dose cohort. These will be presented as actual rates. Dose limiting toxicity (DLT) will be defined according to oncology standards based on NCI CTC version 2 grading criteria (DLT = \> grade 3 non-hematological toxicity or any \> 4 hematological toxicity that persists for more than 4 weeks and in the opinion of the investigator is felt not to be due to disease).

Recruitment status

TERMINATED

Study phase

PHASE1/PHASE2

Target enrollment

2 participants

Primary outcome timeframe

7 months

Results posted on

2023-09-18

Participant Flow

Participant milestones

Participant milestones
Measure	Phase I: Cohort #1 Clofarabine Dose Level 0: 10mg/m2	Phase I: Cohort #2 Clofarabine Dose Level +1: 15 mg/m2	Phase 1: Dose Cohort #3 Clofarabine Level +2: 20mg/m2	Phase I: Cohort #4 Clofarabine Level +3: 30 mg/m2	Phase II: Clinical Response The presence of hematologic response is the outcome of interest in the Phase II component of the study. Clinical Response will include complete response and partial response.
Overall Study STARTED	2	0	0	0	0
Overall Study COMPLETED	0	0	0	0	0
Overall Study NOT COMPLETED	2	0	0	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Clofarabine, Cytarabine, and G-CSF in Treating Patients With Myelodysplastic Syndromes

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treatment n=2 Participants clofarabine: single IV dose over 1 hour daily for 5 days cytarabine: subcutaneously daily for 5 days 2-4 hours following the end of the Clofarabine infusion microarray analysis: Both standard cytogenetic testing and FISH (fluorescent in situ hybridization) are adequate to assess responses. biopsy: bone marrow biopsy
Age, Continuous	69 years n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants
Sex: Female, Male Male	1 Participants n=5 Participants
Region of Enrollment United States	2 participants n=5 Participants

PRIMARY outcome

Timeframe: 7 months

Population: Not done - Genzyme discontinued Funding

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: Following phase I, responses must last at least 8 weeks.

Population: The study only enrolled 2 patients of the planned 30. The two patients enrolled did not complete the research as planned and were not evaluable. The study was closed due to lack of funding support. Data were not collected.

These are measured in patients with pretreatment abnormalities defined as: Hemoglobin \< 11 g/dL or transfusion dependence \[erythroid- E\] Platelets less than 100 x 109/L or platelet-transfusion dependence \[platelet- P\] Absolute neutrophil count (ANC) less than 1.0 x 109/L \[neutrophil- N\] Pretreatment baseline measures of cytopenias are averages of at least 2 measurements (not influenced by transfusions)- at least 1 week apart.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 7 months

Population: Not done - Genzyme discontinued Funding

To assess effects on quality of life of this patient population (questionnaire).

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 7months

Population: Not done - Genzyme discontinued Funding

To assess the time to acute myeloid leukemia transformation or death.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 7 months

Population: Not done - Genzyme discontinued Funding

To assess cytogenetic response rates.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 7 months

Population: Not done - Genzyme discontinued Funding

To assess changes in flow cytometric patterns.

Outcome measures

Outcome data not reported

Adverse Events

Treatment

Serious events: 1 serious events

Other events: 2 other events

Deaths: 1 deaths

Serious adverse events

Serious adverse events
Measure	Treatment n=2 participants at risk clofarabine: single IV dose over 1 hour daily for 5 days cytarabine: subcutaneously daily for 5 days 2-4 hours following the end of the Clofarabine infusion microarray analysis: Both standard cytogenetic testing and FISH (fluorescent in situ hybridization) are adequate to assess responses. biopsy: bone marrow biopsy
Blood and lymphatic system disorders Neutropenic Fever	50.0% 1/2 • Number of events 1 • 6 months

Other adverse events

Other adverse events
Measure	Treatment n=2 participants at risk clofarabine: single IV dose over 1 hour daily for 5 days cytarabine: subcutaneously daily for 5 days 2-4 hours following the end of the Clofarabine infusion microarray analysis: Both standard cytogenetic testing and FISH (fluorescent in situ hybridization) are adequate to assess responses. biopsy: bone marrow biopsy
Blood and lymphatic system disorders ANC	50.0% 1/2 • Number of events 2 • 6 months
Blood and lymphatic system disorders Bacteremia	50.0% 1/2 • Number of events 1 • 6 months
Blood and lymphatic system disorders Low Hemoglobin	50.0% 1/2 • Number of events 2 • 6 months
Blood and lymphatic system disorders Low Platelets	50.0% 1/2 • Number of events 1 • 6 months
Blood and lymphatic system disorders Low WBC	50.0% 1/2 • Number of events 1 • 6 months
Blood and lymphatic system disorders Neutropenia	100.0% 2/2 • Number of events 3 • 6 months
Blood and lymphatic system disorders Thrombocytopenia	50.0% 1/2 • Number of events 3 • 6 months
Gastrointestinal disorders Nausea	100.0% 2/2 • Number of events 4 • 6 months
General disorders Fatigue	50.0% 1/2 • Number of events 1 • 6 months
Metabolism and nutrition disorders Hypokalemia	50.0% 1/2 • Number of events 1 • 6 months
Gastrointestinal disorders Vomiting	100.0% 2/2 • Number of events 2 • 6 months
Metabolism and nutrition disorders Anorexia	50.0% 1/2 • Number of events 1 • 6 months
Respiratory, thoracic and mediastinal disorders Cough	100.0% 2/2 • Number of events 2 • 6 months
General disorders Chills	100.0% 2/2 • Number of events 2 • 6 months
Eye disorders Eye disorders - Other, specify	50.0% 1/2 • Number of events 1 • 6 months
Skin and subcutaneous tissue disorders Skin and subcutaneous tissue disorders - Other, specify	50.0% 1/2 • Number of events 2 • 6 months
General disorders General disorders and administration site conditions - Other, specify	50.0% 1/2 • Number of events 1 • 6 months
General disorders General disorders and administration site conditions	50.0% 1/2 • Number of events 3 • 6 months
Musculoskeletal and connective tissue disorders Bone pain	50.0% 1/2 • Number of events 2 • 6 months
Gastrointestinal disorders Constipation	50.0% 1/2 • Number of events 1 • 6 months
Metabolism and nutrition disorders Hypophosphatemia	50.0% 1/2 • Number of events 1 • 6 months
Respiratory, thoracic and mediastinal disorders Epistaxis	50.0% 1/2 • Number of events 2 • 6 months
Investigations White blood cell decreased	50.0% 1/2 • Number of events 2 • 6 months
Metabolism and nutrition disorders Dehydration	50.0% 1/2 • Number of events 1 • 6 months
Cardiac disorders Cardiac disorders - Other, specify	50.0% 1/2 • Number of events 1 • 6 months
Gastrointestinal disorders Gastrointestinal disorders - Other, specify	50.0% 1/2 • Number of events 1 • 6 months

Additional Information

Lori Maness-Harris, MD

University of Nebraska Medical Center

Phone: 402-559-3848

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place