Trial Outcomes & Findings for Azacitidine, Darbepoetin Alfa, and Erythropoietin and Filgastrim (G-CSF) in Treating Patients With Myelodysplastic Syndromes (NCT NCT00398047)

Results Overview

Complete response is normalization of abnormal blood counts, and disappearance of signs of morphological changes in the bone marrow. If the previously present cytogenetic abnormalities are absent then it is referred also as a cytogenetic complete remission.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

3 participants

Primary outcome timeframe

Approximately 112 days

Results posted on

2018-09-06

Participant Flow

Three patients were enrolled between 09/14/06 and 01/07/2008. The study was closed for slow accrual on 09/02/2009.

Participant milestones

Participant milestones
Measure	Combination of Azacitadine and Hematopoietic Growth Factors azacitidine 100 miligrams/meter squares subcutaneous for 5 days every 28 day cycle,o If the patient had a major hematological improvement; or the patient had grade 3 or 4 hematological toxicities during the first two cycles, and/or there is \>=50% reduction in bone marrow cellularity compared to the baseline bone marrow, filgastrim will be administered at dose of 300 µg (if weight is less then 100 kilogram) or 450 µg (if weight is ≥100 kilogram) subcutaneous three times a week on week 2, 3, 4 along with darbopoietin 500 µg subcutaneous on day 8. Patients not meeting the above criteria will have a dose escalation of azacitidine to 125 miligrams/meter subcutaneous for 5 days, beginning on day 57 with growth factor support and filgastrim will be administered at dose of 300 µg (if weight is less then 100 kilogram) or 450 µg (if weight is ≥100 kilogra,) sq three times a week on week 2, 3, 4 along with darbopoietin 500 µg subcutaneous on day 8).
Overall Study STARTED	3
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Reasons for withdrawal
Measure	Combination of Azacitadine and Hematopoietic Growth Factors azacitidine 100 miligrams/meter squares subcutaneous for 5 days every 28 day cycle,o If the patient had a major hematological improvement; or the patient had grade 3 or 4 hematological toxicities during the first two cycles, and/or there is \>=50% reduction in bone marrow cellularity compared to the baseline bone marrow, filgastrim will be administered at dose of 300 µg (if weight is less then 100 kilogram) or 450 µg (if weight is ≥100 kilogram) subcutaneous three times a week on week 2, 3, 4 along with darbopoietin 500 µg subcutaneous on day 8. Patients not meeting the above criteria will have a dose escalation of azacitidine to 125 miligrams/meter subcutaneous for 5 days, beginning on day 57 with growth factor support and filgastrim will be administered at dose of 300 µg (if weight is less then 100 kilogram) or 450 µg (if weight is ≥100 kilogra,) sq three times a week on week 2, 3, 4 along with darbopoietin 500 µg subcutaneous on day 8).
Overall Study all patient progressed before completion	3

Baseline Characteristics

Azacitidine, Darbepoetin Alfa, and Erythropoietin and Filgastrim (G-CSF) in Treating Patients With Myelodysplastic Syndromes

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Azacitidine and Darbopoietin and G-CSF n=3 Participants
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	3 Participants n=5 Participants
Age, Continuous	69.407 years STANDARD_DEVIATION 3.691 • n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants
Region of Enrollment United States	3 participants n=5 Participants

PRIMARY outcome

Timeframe: Approximately 112 days

Population: There were a total of 3 patients accrued on this trial. All were eligible and evaluable for response and evaluable for toxicity, as per protocol.

Complete response is normalization of abnormal blood counts, and disappearance of signs of morphological changes in the bone marrow. If the previously present cytogenetic abnormalities are absent then it is referred also as a cytogenetic complete remission.

Outcome measures

Outcome measures
Measure	Azacitidine and Darbopoietin and G-CSF n=3 Participants
Number of Participants With Complete Response	0 Participants

PRIMARY outcome

Timeframe: Approximately 112 days

Population: Because no patients completed therapy, no analysis was possible

For patients with pretreatment hemoglobin less than 11 g/dL, greater than 2 g/dL increase in hemoglobin; for red cell transfusion-dependent patients, transfusion independence.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Approximately 112 days

Population: Because no patients completed therapy, no analysis was possible

For patients with pretreatment platelet count less than 100,000/mm3, a 50% or more increase in platelet count with a net increase greater than 10,000/mm3 but less than 30,000/mm3

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Approximately 12 months

Population: Because no patients completed therapy, no analysis was possible

Death during treatment or disease progression characterized by worsening of cytopenias, increase in the percentage of the blasts, reduction of hemoglobin concentration by at least 2 g/dl or transfusion dependence in the absence of another explanation, such as acute infection, gastrointestinal bleeding, hemolysis.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Approximately 12 months

Population: Because no patients completed therapy, and the protocol was closed early, analysis was not performed

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline and approximately 12 months

Population: Because no patients completed therapy, no analysis was done

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Approximately 12 months

Population: Because no patients completed therapy, no analysis was possible

Outcome measures

Outcome data not reported

Adverse Events

Azacitidine and Darbopoietin and G-CSF

Serious events: 1 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Azacitidine and Darbopoietin and G-CSF n=3 participants at risk
Gastrointestinal disorders GI Bleed	33.3% 1/3 • Number of events 1 • Adverse event data collected for the 3 patients on study for a total of 14 months.
Infections and infestations Cellulitis	33.3% 1/3 • Number of events 1 • Adverse event data collected for the 3 patients on study for a total of 14 months.
Blood and lymphatic system disorders Neutropenia	33.3% 1/3 • Number of events 1 • Adverse event data collected for the 3 patients on study for a total of 14 months.

Other adverse events

Other adverse events
Measure	Azacitidine and Darbopoietin and G-CSF n=3 participants at risk
Blood and lymphatic system disorders Platelets	100.0% 3/3 • Number of events 3 • Adverse event data collected for the 3 patients on study for a total of 14 months.
Blood and lymphatic system disorders Hemoglobin (gender based)	100.0% 3/3 • Number of events 3 • Adverse event data collected for the 3 patients on study for a total of 14 months.
Blood and lymphatic system disorders Neutrophils/granulocytes (ANC/AGC)	66.7% 2/3 • Number of events 2 • Adverse event data collected for the 3 patients on study for a total of 14 months.
Respiratory, thoracic and mediastinal disorders Pleural effusion (non-malignant)	33.3% 1/3 • Number of events 1 • Adverse event data collected for the 3 patients on study for a total of 14 months.
Respiratory, thoracic and mediastinal disorders Hypoxia	33.3% 1/3 • Number of events 1 • Adverse event data collected for the 3 patients on study for a total of 14 months.
Gastrointestinal disorders Hemmorrhage, GI: Rectum	33.3% 1/3 • Number of events 1 • Adverse event data collected for the 3 patients on study for a total of 14 months.
Gastrointestinal disorders Diarrhea	33.3% 1/3 • Number of events 1 • Adverse event data collected for the 3 patients on study for a total of 14 months.
Infections and infestations Infection (Doc.) with Grade 3/4 ANC: Skin (cellulitis)	33.3% 1/3 • Number of events 1 • Adverse event data collected for the 3 patients on study for a total of 14 months.
Blood and lymphatic system disorders Leukocytes (total WVC)	33.3% 1/3 • Number of events 1 • Adverse event data collected for the 3 patients on study for a total of 14 months.

Additional Information

Ralph D'Agostino Jr., Ph.D.

Wake Forest University Health Sciences

Phone: 113-716-3483

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place