Trial Outcomes & Findings for Decitabine in Treating Patients With Myelofibrosis (NCT NCT00095784)

NCT ID: NCT00095784

Last Updated: 2025-08-24

Results Overview

Complete response is normalization of counts and transfusion-independence. Partial response is hemoglobin increase to normal levels, multilineage improvement including absolute neutrophil count (ANC) and/or platelets. Hematologic improvement is red cell transfusion-independence or \>50% increase in platelet levels.

• Recruitment status: ACTIVE_NOT_RECRUITING
• Study phase: PHASE2
• Target enrollment: 21 participants
• Primary outcome timeframe: Up to 36 weeks (6 cycles)
• Results posted on: 2025-08-24

Participant Flow

Participant milestones

Measure	Decitabine Patients receive 0.3 mg/kg/day decitabine subcutaneously on days 1-5 and 8-12. decitabine : Given SC
Overall Study STARTED	21
Overall Study COMPLETED	21
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Decitabine in Treating Patients With Myelofibrosis

Baseline characteristics by cohort

Measure	Arm I n=21 Participants Patients receive 0.3 mg/kg/day decitabine subcutaneously on days 1-5 and 8-12. decitabine : Given SC
Age, Continuous	67 years n=5 Participants
Sex: Female, Male Female	9 Participants n=5 Participants
Sex: Female, Male Male	12 Participants n=5 Participants
Region of Enrollment United States	21 participants n=5 Participants

PRIMARY outcome

Timeframe: Up to 36 weeks (6 cycles)

Population: Two patients were non-evaluable for response.

Complete response is normalization of counts and transfusion-independence.

Partial response is hemoglobin increase to normal levels, multilineage improvement including absolute neutrophil count (ANC) and/or platelets.

Hematologic improvement is red cell transfusion-independence or >50% increase in platelet levels.

Outcome measures

Measure	Arm I n=19 Participants Patients receive 0.3 mg/kg/day decitabine subcutaneously on days 1-5 and 8-12. decitabine : Given SC
Response Rate (Complete Response, Partial Response, or Hematologic Improvement.	37 percentage of participants Interval 17.0 to 54.0

PRIMARY outcome

Timeframe: Up to 30 days of last dose of decitabine

Percentage of patients experiencing any toxicity, any grade level. Additional details on adverse events are reported in Adverse Events section.

Outcome measures

Measure	Arm I n=21 Participants Patients receive 0.3 mg/kg/day decitabine subcutaneously on days 1-5 and 8-12. decitabine : Given SC
Incidence of Toxicities, Graded According to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) v3.0	100 percentage of patients Interval 84.0 to 100.0

SECONDARY outcome

Timeframe: Cycle 1, Day 1

Population: One patient had missing data.

CD34 positive(+) cells are determined by flow immunostaining and light scatter in peripheral blood. Samples are then analyzed by flow cytometry, and CD34+ cells quantitated after 75,000 CD45 events are studied. (At least 75,000 CD45 events must be studied to ensure accuracy of the assay). Absolute numbers are determined by multiplying the % CD34+ cells by the total white blood cell count obtained on a CBC that is processed simultaneously.

Outcome measures

Measure	Arm I n=20 Participants Patients receive 0.3 mg/kg/day decitabine subcutaneously on days 1-5 and 8-12. decitabine : Given SC
CD34+ Cells	117 cells x 10^6/L Interval 44.0 to 310.0

SECONDARY outcome

Timeframe: Cycle 1, Day 5

Population: One patient had missing data.

CD34 positive(+) cells are determined by flow immunostaining and light scatter in peripheral blood. Samples are then analyzed by flow cytometry, and CD34+ cells quantitated after 75,000 CD45 events are studied. (At least 75,000 CD45 events must be studied to ensure accuracy of the assay). Absolute numbers are determined by multiplying the % CD34+ cells by the total white blood cell count obtained on a CBC that is processed simultaneously.

Outcome measures

Measure	Arm I n=20 Participants Patients receive 0.3 mg/kg/day decitabine subcutaneously on days 1-5 and 8-12. decitabine : Given SC
CD34+ Cells	71 cells x 10^6/L Interval 31.0 to 159.0

SECONDARY outcome

Timeframe: Cycle 1, Day 12

Population: Three patients had missing data.

CD34 positive(+) cells are determined by flow immunostaining and light scatter in peripheral blood. Samples are then analyzed by flow cytometry, and CD34+ cells quantitated after 75,000 CD45 events are studied. (At least 75,000 CD45 events must be studied to ensure accuracy of the assay). Absolute numbers are determined by multiplying the % CD34+ cells by the total white blood cell count obtained on a CBC that is processed simultaneously.

Outcome measures

Measure	Arm I n=18 Participants Patients receive 0.3 mg/kg/day decitabine subcutaneously on days 1-5 and 8-12. decitabine : Given SC
CD34+ Cells	35 cells x 10^6/L Interval 14.0 to 88.0

SECONDARY outcome

Timeframe: Cycle 2, Day 1

Population: Five patients had missing data.

CD34 positive(+) cells are determined by flow immunostaining and light scatter in peripheral blood. Samples are then analyzed by flow cytometry, and CD34+ cells quantitated after 75,000 CD45 events are studied. (At least 75,000 CD45 events must be studied to ensure accuracy of the assay). Absolute numbers are determined by multiplying the % CD34+ cells by the total white blood cell count obtained on a CBC that is processed simultaneously.

Outcome measures

Measure	Arm I n=16 Participants Patients receive 0.3 mg/kg/day decitabine subcutaneously on days 1-5 and 8-12. decitabine : Given SC
CD34+ Cells	76 cells x 10^6/L Interval 23.0 to 243.0

SECONDARY outcome

Timeframe: Cycle 2, Day 5

Population: Six patients had missing data.

CD34 positive(+) cells are determined by flow immunostaining and light scatter in peripheral blood. Samples are then analyzed by flow cytometry, and CD34+ cells quantitated after 75,000 CD45 events are studied. (At least 75,000 CD45 events must be studied to ensure accuracy of the assay). Absolute numbers are determined by multiplying the % CD34+ cells by the total white blood cell count obtained on a CBC that is processed simultaneously.

Outcome measures

Measure	Arm I n=15 Participants Patients receive 0.3 mg/kg/day decitabine subcutaneously on days 1-5 and 8-12. decitabine : Given SC
CD34+ Cells	35 cells x 10^6/L Interval 14.0 to 87.0

SECONDARY outcome

Timeframe: Cycle 2, Day 12

Population: Six patients had missing data.

CD34 positive(+) cells are determined by flow immunostaining and light scatter in peripheral blood. Samples are then analyzed by flow cytometry, and CD34+ cells quantitated after 75,000 CD45 events are studied. (At least 75,000 CD45 events must be studied to ensure accuracy of the assay). Absolute numbers are determined by multiplying the % CD34+ cells by the total white blood cell count obtained on a CBC that is processed simultaneously.

Outcome measures

Measure	Arm I n=15 Participants Patients receive 0.3 mg/kg/day decitabine subcutaneously on days 1-5 and 8-12. decitabine : Given SC
CD34+ Cells	21 cells x 10^6/L Interval 8.0 to 58.0

SECONDARY outcome

Timeframe: Cycle 1, Day 1

Population: Four patients had missing data.

CXCR4 gene expression level measured by real-time RT_PCR. Ratio of CXCR4 vs a housekeeping gene (i.e., ABL)

Outcome measures

Measure	Arm I n=17 Participants Patients receive 0.3 mg/kg/day decitabine subcutaneously on days 1-5 and 8-12. decitabine : Given SC
CXCR4	4.4 ratio Interval 2.6 to 7.3

SECONDARY outcome

Timeframe: Cycle 1, Day 5

Population: Three patients had missing data.

CXCR4 gene expression level measured by real-time RT_PCR. Ratio of CXCR4 vs a housekeeping gene (i.e., ABL)

Outcome measures

Measure	Arm I n=18 Participants Patients receive 0.3 mg/kg/day decitabine subcutaneously on days 1-5 and 8-12. decitabine : Given SC
CXCR4	5.7 ratio Interval 4.1 to 8.0

SECONDARY outcome

Timeframe: Cycle 1, Day 12

Population: Three patients had missing data.

CXCR4 gene expression level measured by real-time RT_PCR. Ratio of CXCR4 vs a housekeeping gene (i.e., ABL)

Outcome measures

Measure	Arm I n=18 Participants Patients receive 0.3 mg/kg/day decitabine subcutaneously on days 1-5 and 8-12. decitabine : Given SC
CXCR4	6.4 ratio Interval 4.0 to 10.1

SECONDARY outcome

Timeframe: Cycle 2, Day 1

Population: Seven patients had missing data.

CXCR4 gene expression level measured by real-time RT_PCR. Ratio of CXCR4 vs a housekeeping gene (i.e., ABL)

Outcome measures

Measure	Arm I n=14 Participants Patients receive 0.3 mg/kg/day decitabine subcutaneously on days 1-5 and 8-12. decitabine : Given SC
CXCR4	4.0 ratio Interval 2.4 to 6.8

SECONDARY outcome

Timeframe: Cycle 2, Day 5

Population: Seven patients had missing data.

CXCR4 gene expression level measured by real-time RT_PCR. Ratio of CXCR4 vs a housekeeping gene (i.e., ABL)

Outcome measures

Measure	Arm I n=14 Participants Patients receive 0.3 mg/kg/day decitabine subcutaneously on days 1-5 and 8-12. decitabine : Given SC
CXCR4	6.0 ratio Interval 3.7 to 9.6

SECONDARY outcome

Timeframe: Cycle 2, Day 12

Population: Eleven patients had missing data.

CXCR4 gene expression level measured by real-time RT_PCR. Ratio of CXCR4 vs a housekeeping gene (i.e., ABL)

Outcome measures

Measure	Arm I n=10 Participants Patients receive 0.3 mg/kg/day decitabine subcutaneously on days 1-5 and 8-12. decitabine : Given SC
CXCR4	7.1 ratio Interval 3.8 to 13.1

SECONDARY outcome

Timeframe: Cycle 1, Day 1

Population: Four patients had missing data.

Percentage of hemoglobin F as a proportion of total Hb (%HbF) measured by HPLC on peripheral blood samples.

Midpoint of 0.5% imputed for values reported as below limit of detection (1.0%). Hemoglobin F has been previously shown to be upregulated by decitabine in other hematologic disorders- sickle cell disease specifically. The rationale for exploring it in this study was to evaluate its potential utility as a biomarker of drug effect/PD marker.

Outcome measures

Measure	Arm I n=17 Participants Patients receive 0.3 mg/kg/day decitabine subcutaneously on days 1-5 and 8-12. decitabine : Given SC
Hemoglobin F	1.6 percentage of HbF Interval 0.8 to 2.5

SECONDARY outcome

Timeframe: Cycle 1, Day 5

Population: Three patients had missing data.

Percentage of hemoglobin F as a proportion of total Hb (%HbF) measured by HPLC on peripheral blood samples.

Midpoint of 0.5% imputed for values reported as below limit of detection (1.0%). Hemoglobin F has been previously shown to be upregulated by decitabine in other hematologic disorders- sickle cell disease specifically. The rationale for exploring it in this study was to evaluate its potential utility as a biomarker of drug effect/PD marker.

Outcome measures

Measure	Arm I n=18 Participants Patients receive 0.3 mg/kg/day decitabine subcutaneously on days 1-5 and 8-12. decitabine : Given SC
Hemoglobin F	1.6 percentage of HbF Interval 0.7 to 2.5

SECONDARY outcome

Timeframe: Cycle 1, Day 12

Population: Three patients had missing data.

Percentage of hemoglobin F as a proportion of total Hb (%HbF) measured by HPLC on peripheral blood samples.

Midpoint of 0.5% imputed for values reported as below limit of detection (1.0%). Hemoglobin F has been previously shown to be upregulated by decitabine in other hematologic disorders- sickle cell disease specifically. The rationale for exploring it in this study was to evaluate its potential utility as a biomarker of drug effect/PD marker.

Outcome measures

Measure	Arm I n=18 Participants Patients receive 0.3 mg/kg/day decitabine subcutaneously on days 1-5 and 8-12. decitabine : Given SC
Hemoglobin F	1.4 percentage of HbF Interval 0.6 to 2.2

SECONDARY outcome

Timeframe: Cycle 2, Day 1

Population: Nine patients had missing data.

Percentage of hemoglobin F as a proportion of total Hb (%HbF) measured by HPLC on peripheral blood samples.

Midpoint of 0.5% imputed for values reported as below limit of detection (1.0%). Hemoglobin F has been previously shown to be upregulated by decitabine in other hematologic disorders- sickle cell disease specifically. The rationale for exploring it in this study was to evaluate its potential utility as a biomarker of drug effect/PD marker.

Outcome measures

Measure	Arm I n=12 Participants Patients receive 0.3 mg/kg/day decitabine subcutaneously on days 1-5 and 8-12. decitabine : Given SC
Hemoglobin F	1.5 percentage of HbF Interval 0.5 to 2.5

SECONDARY outcome

Timeframe: Cycle 2, Day 5

Population: Seven patients had missing data.

Percentage of hemoglobin F as a proportion of total Hb (%HbF) measured by HPLC on peripheral blood samples.

Midpoint of 0.5% imputed for values reported as below limit of detection (1.0%). Hemoglobin F has been previously shown to be upregulated by decitabine in other hematologic disorders- sickle cell disease specifically. The rationale for exploring it in this study was to evaluate its potential utility as a biomarker of drug effect/PD marker.

Outcome measures

Measure	Arm I n=14 Participants Patients receive 0.3 mg/kg/day decitabine subcutaneously on days 1-5 and 8-12. decitabine : Given SC
Hemoglobin F	1.5 percentage of HbF Interval 0.6 to 2.4

SECONDARY outcome

Timeframe: Cycle 2, Day 12

Population: Seven patients had missing data.

Percentage of hemoglobin F as a proportion of total Hb (%HbF) measured by HPLC on peripheral blood samples.

Midpoint of 0.5% imputed for values reported as below limit of detection (1.0%). Hemoglobin F has been previously shown to be upregulated by decitabine in other hematologic disorders- sickle cell disease specifically. The rationale for exploring it in this study was to evaluate its potential utility as a biomarker of drug effect/PD marker.

Outcome measures

Measure	Arm I n=14 Participants Patients receive 0.3 mg/kg/day decitabine subcutaneously on days 1-5 and 8-12. decitabine : Given SC
Hemoglobin F	1.5 percentage of HbF Interval 0.8 to 2.2

Adverse Events

Arm I

Serious events: 15 serious events

Other events: 19 other events

Deaths: 0 deaths

Serious adverse events

Measure	Arm I n=21 participants at risk Patients receive 0.3 mg/kg/day decitabine subcutaneously on days 1-5 and 8-12. decitabine : Given SC
Infections and infestations Infection with Grade 3 or 4 neutrophils (ANC <1.0 x 10e9/L): Blood	14.3% 3/21 • Up to 30 days of last dose of decitabine
Infections and infestations Infection with Grade 3 or 4 neutrophils (ANC <1.0 x 10e9/L): Catheter-related	4.8% 1/21 • Up to 30 days of last dose of decitabine
Infections and infestations Infection with Grade 3 or 4 neutrophils (ANC <1.0 x 10e9/L): Lung (pneumonia)	9.5% 2/21 • Up to 30 days of last dose of decitabine
Vascular disorders Splenic infarct vs hemorrhage/rupture	4.8% 1/21 • Up to 30 days of last dose of decitabine
General disorders Sweet's Syndrome	4.8% 1/21 • Up to 30 days of last dose of decitabine
Gastrointestinal disorders Abdominal pain	4.8% 1/21 • Up to 30 days of last dose of decitabine
Renal and urinary disorders Acute kidney injury	4.8% 1/21 • Up to 30 days of last dose of decitabine
Blood and lymphatic system disorders Anemia	19.0% 4/21 • Up to 30 days of last dose of decitabine
Psychiatric disorders Confusion	9.5% 2/21 • Up to 30 days of last dose of decitabine
Respiratory, thoracic and mediastinal disorders Dyspnea	4.8% 1/21 • Up to 30 days of last dose of decitabine
Gastrointestinal disorders Esophageal varices hemorrhage	4.8% 1/21 • Up to 30 days of last dose of decitabine
Blood and lymphatic system disorders Febrile neutropenia	38.1% 8/21 • Up to 30 days of last dose of decitabine
General disorders Fever	4.8% 1/21 • Up to 30 days of last dose of decitabine
General disorders General disorders and administration site conditions - Other	4.8% 1/21 • Up to 30 days of last dose of decitabine
Vascular disorders Hematoma	9.5% 2/21 • Up to 30 days of last dose of decitabine
Metabolism and nutrition disorders Hypoglycemia	4.8% 1/21 • Up to 30 days of last dose of decitabine
Vascular disorders Hypotension	9.5% 2/21 • Up to 30 days of last dose of decitabine
Respiratory, thoracic and mediastinal disorders Hypoxia	4.8% 1/21 • Up to 30 days of last dose of decitabine
Infections and infestations Infections and infestations - Other	19.0% 4/21 • Up to 30 days of last dose of decitabine
Infections and infestations Lung infection	4.8% 1/21 • Up to 30 days of last dose of decitabine
Investigations Neutrophil count decreased	19.0% 4/21 • Up to 30 days of last dose of decitabine
Gastrointestinal disorders Oesophageal varices	4.8% 1/21 • Up to 30 days of last dose of decitabine
Investigations Platelet count decreased	14.3% 3/21 • Up to 30 days of last dose of decitabine
Renal and urinary disorders Urinary retention	4.8% 1/21 • Up to 30 days of last dose of decitabine
Vascular disorders Vascular disorders - Other	4.8% 1/21 • Up to 30 days of last dose of decitabine
Gastrointestinal disorders Vomiting	4.8% 1/21 • Up to 30 days of last dose of decitabine
Investigations White blood cell decreased	4.8% 1/21 • Up to 30 days of last dose of decitabine

Other adverse events

Measure	Arm I n=21 participants at risk Patients receive 0.3 mg/kg/day decitabine subcutaneously on days 1-5 and 8-12. decitabine : Given SC
Gastrointestinal disorders Abdominal distension	9.5% 2/21 • Up to 30 days of last dose of decitabine
Gastrointestinal disorders Abdominal pain	19.0% 4/21 • Up to 30 days of last dose of decitabine
Investigations Alanine aminotransferase increased	28.6% 6/21 • Up to 30 days of last dose of decitabine
Investigations Alkaline phosphatase increased	38.1% 8/21 • Up to 30 days of last dose of decitabine
Blood and lymphatic system disorders Anemia	76.2% 16/21 • Up to 30 days of last dose of decitabine
Metabolism and nutrition disorders Anorexia	42.9% 9/21 • Up to 30 days of last dose of decitabine
Musculoskeletal and connective tissue disorders Arthralgia	14.3% 3/21 • Up to 30 days of last dose of decitabine
Investigations Aspartate aminotransferase increased	23.8% 5/21 • Up to 30 days of last dose of decitabine
Investigations Blood bilirubin increased	28.6% 6/21 • Up to 30 days of last dose of decitabine
Musculoskeletal and connective tissue disorders Bone pain	9.5% 2/21 • Up to 30 days of last dose of decitabine
Injury, poisoning and procedural complications Bruising	9.5% 2/21 • Up to 30 days of last dose of decitabine
Investigations CD4 lymphocytes decreased	9.5% 2/21 • Up to 30 days of last dose of decitabine
General disorders Chills	9.5% 2/21 • Up to 30 days of last dose of decitabine
Gastrointestinal disorders Constipation	33.3% 7/21 • Up to 30 days of last dose of decitabine
Respiratory, thoracic and mediastinal disorders Cough	14.3% 3/21 • Up to 30 days of last dose of decitabine
Investigations Creatinine increased	23.8% 5/21 • Up to 30 days of last dose of decitabine
Gastrointestinal disorders Diarrhea	33.3% 7/21 • Up to 30 days of last dose of decitabine
Nervous system disorders Dizziness	14.3% 3/21 • Up to 30 days of last dose of decitabine
Gastrointestinal disorders Dysphagia	9.5% 2/21 • Up to 30 days of last dose of decitabine
Respiratory, thoracic and mediastinal disorders Dyspnea	23.8% 5/21 • Up to 30 days of last dose of decitabine
General disorders Edema limbs	23.8% 5/21 • Up to 30 days of last dose of decitabine
General disorders Fatigue	66.7% 14/21 • Up to 30 days of last dose of decitabine
Blood and lymphatic system disorders Febrile neutropenia	33.3% 7/21 • Up to 30 days of last dose of decitabine
General disorders Fever	19.0% 4/21 • Up to 30 days of last dose of decitabine
Musculoskeletal and connective tissue disorders Generalized muscle weakness	14.3% 3/21 • Up to 30 days of last dose of decitabine
Nervous system disorders Headache	9.5% 2/21 • Up to 30 days of last dose of decitabine
Metabolism and nutrition disorders Hypercalcemia	9.5% 2/21 • Up to 30 days of last dose of decitabine
Metabolism and nutrition disorders Hyperglycemia	66.7% 14/21 • Up to 30 days of last dose of decitabine
Skin and subcutaneous tissue disorders Hyperhidrosis	14.3% 3/21 • Up to 30 days of last dose of decitabine
Metabolism and nutrition disorders Hyperkalemia	23.8% 5/21 • Up to 30 days of last dose of decitabine
Metabolism and nutrition disorders Hypermagnesemia	14.3% 3/21 • Up to 30 days of last dose of decitabine
Metabolism and nutrition disorders Hypoalbuminemia	47.6% 10/21 • Up to 30 days of last dose of decitabine
Metabolism and nutrition disorders Hypocalcemia	52.4% 11/21 • Up to 30 days of last dose of decitabine
Metabolism and nutrition disorders Hypokalemia	19.0% 4/21 • Up to 30 days of last dose of decitabine
Metabolism and nutrition disorders Hyponatremia	28.6% 6/21 • Up to 30 days of last dose of decitabine
Metabolism and nutrition disorders Hypophosphatemia	19.0% 4/21 • Up to 30 days of last dose of decitabine
Investigations INR increased	19.0% 4/21 • Up to 30 days of last dose of decitabine
Infections and infestations Infections and infestations - Other	23.8% 5/21 • Up to 30 days of last dose of decitabine
General disorders Injection site reaction	9.5% 2/21 • Up to 30 days of last dose of decitabine
Psychiatric disorders Insomnia	9.5% 2/21 • Up to 30 days of last dose of decitabine
Investigations Lymphocyte count decreased	14.3% 3/21 • Up to 30 days of last dose of decitabine
Gastrointestinal disorders Mucositis oral	23.8% 5/21 • Up to 30 days of last dose of decitabine
Musculoskeletal and connective tissue disorders Myalgia	23.8% 5/21 • Up to 30 days of last dose of decitabine
Gastrointestinal disorders Nausea	33.3% 7/21 • Up to 30 days of last dose of decitabine
Investigations Neutrophil count decreased	71.4% 15/21 • Up to 30 days of last dose of decitabine
Gastrointestinal disorders Oral hemorrhage	9.5% 2/21 • Up to 30 days of last dose of decitabine
Musculoskeletal and connective tissue disorders Pain in extremity	9.5% 2/21 • Up to 30 days of last dose of decitabine
Respiratory, thoracic and mediastinal disorders Pharyngolaryngeal pain	19.0% 4/21 • Up to 30 days of last dose of decitabine
Investigations Platelet count decreased	76.2% 16/21 • Up to 30 days of last dose of decitabine
Renal and urinary disorders Proteinuria	9.5% 2/21 • Up to 30 days of last dose of decitabine
Skin and subcutaneous tissue disorders Rash maculo-papular	23.8% 5/21 • Up to 30 days of last dose of decitabine
Renal and urinary disorders Urinary frequency	9.5% 2/21 • Up to 30 days of last dose of decitabine
Renal and urinary disorders Urinary retention	9.5% 2/21 • Up to 30 days of last dose of decitabine
Gastrointestinal disorders Vomiting	9.5% 2/21 • Up to 30 days of last dose of decitabine
Investigations Weight loss	14.3% 3/21 • Up to 30 days of last dose of decitabine
Investigations White blood cell decreased	81.0% 17/21 • Up to 30 days of last dose of decitabine

Additional Information

Olatoyosi Odenike, MD

University of Chicago

Phone: 773-702-3354

Email: todenike@medicine.bsd.uchicago.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place

Restriction type: LTE60