Trial Outcomes & Findings for 5-Azacytidine (Azacytidine; Vidaza) in Chronic Lymphocytic Leukemia (NCT NCT00413478)

Last Updated: 2015-06-30

Results Overview

Overall response rate includes percentage of participants with complete response (CR) plus partial response (PR) responses using the National Cancer Institute (NCI) International Workshop on Chronic Lymphocytic Leukemia (IWCLL) criteria for response: Complete response defined as no palpable lymph nodes, liver or spleen and absence of symptoms. Neutrophil count \> 15,00/Mic L, and platelet count more than 100,000/MicL. Hemoglobin should be \> 11g/dl without transfusions. Lymphocyte count \<4000/micL. On bone marrow aspirate lymphocyte % should be \<30%, and biopsy showing no lymphocyte infiltrate. A partial response was defined as more than or equal to 50% decrease in lymph nodes and liver and spleen size. Neutrophils \> 1500/ micL or \>50 % improvement from baseline, platelet count \>100,000/micL or \>50 % improvement from baseline. Hemoglobin \>11g/dl or \>50% improvement from baseline. A reduction of \>50% in Leukocyte count or \<30 % lymphocytes with residual disease on biopsy for nodular PR.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

9 participants

Primary outcome timeframe

3 to 8 weeks treatment cycles, continuation up to 1 year

Results posted on

2015-06-30

Participant Flow

Recruitment Period: 09/21/2006 to 12/01/2010. All recruitment done at The University of Texas MD Anderson Cancer Center.

The study was discontinued after 9 participants enrolled because of lack of efficacy and slow accrual.

Participant milestones

Participant milestones
Measure	5-Azacytidine 5-Azacytidine 75 mg/m\^2 subcutaneously daily for 7 days, cycle repeated every 3-8 weeks.
Overall Study STARTED	9
Overall Study COMPLETED	9
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

5-Azacytidine (Azacytidine; Vidaza) in Chronic Lymphocytic Leukemia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	5-Azacytidine n=9 Participants 5-Azacytidine 75 mg/m\^2 subcutaneously daily for 7 days, cycle repeated every 3-8 weeks.
Age, Continuous	74 years n=5 Participants
Sex: Female, Male Female	3 Participants n=5 Participants
Sex: Female, Male Male	6 Participants n=5 Participants
Region of Enrollment United States	9 participants n=5 Participants

PRIMARY outcome

Timeframe: 3 to 8 weeks treatment cycles, continuation up to 1 year

Outcome measures

Outcome measures
Measure	5-Azacytidine n=9 Participants 5-Azacytidine 75 mg/m\^2 subcutaneously daily for 7 days, cycle repeated every 3-8 weeks.
Tumor Response Rate (Complete, Partial) of Azacytidine	0 percentage of participants

Adverse Events

5-Azacytidine

Serious events: 0 serious events

Other events: 9 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	5-Azacytidine n=9 participants at risk 5-Azacytidine 75 mg/m\^2 subcutaneously daily for 7 days, cycle repeated every 3-8 weeks.
Blood and lymphatic system disorders Thrombocytopenia	11.1% 1/9 • Adverse event data collected with each 7 day treatment through subsequent repeated treatment cycles every 3 to 8 weeks with continuation up to 1 year. Overall collection period: October 2006 to August 2009.
Blood and lymphatic system disorders Anemia	11.1% 1/9 • Adverse event data collected with each 7 day treatment through subsequent repeated treatment cycles every 3 to 8 weeks with continuation up to 1 year. Overall collection period: October 2006 to August 2009.
Blood and lymphatic system disorders Neutropenia	33.3% 3/9 • Adverse event data collected with each 7 day treatment through subsequent repeated treatment cycles every 3 to 8 weeks with continuation up to 1 year. Overall collection period: October 2006 to August 2009.
Gastrointestinal disorders Diarrhea	22.2% 2/9 • Adverse event data collected with each 7 day treatment through subsequent repeated treatment cycles every 3 to 8 weeks with continuation up to 1 year. Overall collection period: October 2006 to August 2009.
Skin and subcutaneous tissue disorders Skin Rash	33.3% 3/9 • Adverse event data collected with each 7 day treatment through subsequent repeated treatment cycles every 3 to 8 weeks with continuation up to 1 year. Overall collection period: October 2006 to August 2009.
Infections and infestations Infections	66.7% 6/9 • Adverse event data collected with each 7 day treatment through subsequent repeated treatment cycles every 3 to 8 weeks with continuation up to 1 year. Overall collection period: October 2006 to August 2009.
General disorders Fever	22.2% 2/9 • Adverse event data collected with each 7 day treatment through subsequent repeated treatment cycles every 3 to 8 weeks with continuation up to 1 year. Overall collection period: October 2006 to August 2009.
Infections and infestations Pneumonia	33.3% 3/9 • Adverse event data collected with each 7 day treatment through subsequent repeated treatment cycles every 3 to 8 weeks with continuation up to 1 year. Overall collection period: October 2006 to August 2009.
Gastrointestinal disorders Vomiting	22.2% 2/9 • Adverse event data collected with each 7 day treatment through subsequent repeated treatment cycles every 3 to 8 weeks with continuation up to 1 year. Overall collection period: October 2006 to August 2009.
Cardiac disorders Arrythmia	11.1% 1/9 • Adverse event data collected with each 7 day treatment through subsequent repeated treatment cycles every 3 to 8 weeks with continuation up to 1 year. Overall collection period: October 2006 to August 2009.
Nervous system disorders Bells Palsy	11.1% 1/9 • Adverse event data collected with each 7 day treatment through subsequent repeated treatment cycles every 3 to 8 weeks with continuation up to 1 year. Overall collection period: October 2006 to August 2009.
Eye disorders blurry vision	11.1% 1/9 • Adverse event data collected with each 7 day treatment through subsequent repeated treatment cycles every 3 to 8 weeks with continuation up to 1 year. Overall collection period: October 2006 to August 2009.
Infections and infestations Cellulitis	11.1% 1/9 • Adverse event data collected with each 7 day treatment through subsequent repeated treatment cycles every 3 to 8 weeks with continuation up to 1 year. Overall collection period: October 2006 to August 2009.
Gastrointestinal disorders Constipation	11.1% 1/9 • Adverse event data collected with each 7 day treatment through subsequent repeated treatment cycles every 3 to 8 weeks with continuation up to 1 year. Overall collection period: October 2006 to August 2009.
Musculoskeletal and connective tissue disorders cramps	11.1% 1/9 • Adverse event data collected with each 7 day treatment through subsequent repeated treatment cycles every 3 to 8 weeks with continuation up to 1 year. Overall collection period: October 2006 to August 2009.
Respiratory, thoracic and mediastinal disorders Dyspnea	22.2% 2/9 • Adverse event data collected with each 7 day treatment through subsequent repeated treatment cycles every 3 to 8 weeks with continuation up to 1 year. Overall collection period: October 2006 to August 2009.
Renal and urinary disorders dysuria	11.1% 1/9 • Adverse event data collected with each 7 day treatment through subsequent repeated treatment cycles every 3 to 8 weeks with continuation up to 1 year. Overall collection period: October 2006 to August 2009.
Vascular disorders Edema	11.1% 1/9 • Adverse event data collected with each 7 day treatment through subsequent repeated treatment cycles every 3 to 8 weeks with continuation up to 1 year. Overall collection period: October 2006 to August 2009.
Skin and subcutaneous tissue disorders Erythema	11.1% 1/9 • Adverse event data collected with each 7 day treatment through subsequent repeated treatment cycles every 3 to 8 weeks with continuation up to 1 year. Overall collection period: October 2006 to August 2009.
General disorders fatigue	11.1% 1/9 • Adverse event data collected with each 7 day treatment through subsequent repeated treatment cycles every 3 to 8 weeks with continuation up to 1 year. Overall collection period: October 2006 to August 2009.
General disorders headache	11.1% 1/9 • Adverse event data collected with each 7 day treatment through subsequent repeated treatment cycles every 3 to 8 weeks with continuation up to 1 year. Overall collection period: October 2006 to August 2009.
Blood and lymphatic system disorders Hematoma	11.1% 1/9 • Adverse event data collected with each 7 day treatment through subsequent repeated treatment cycles every 3 to 8 weeks with continuation up to 1 year. Overall collection period: October 2006 to August 2009.
Metabolism and nutrition disorders Hypomagnesia	11.1% 1/9 • Adverse event data collected with each 7 day treatment through subsequent repeated treatment cycles every 3 to 8 weeks with continuation up to 1 year. Overall collection period: October 2006 to August 2009.
General disorders lightheadedness	11.1% 1/9 • Adverse event data collected with each 7 day treatment through subsequent repeated treatment cycles every 3 to 8 weeks with continuation up to 1 year. Overall collection period: October 2006 to August 2009.
Infections and infestations MRSA Infection	11.1% 1/9 • Adverse event data collected with each 7 day treatment through subsequent repeated treatment cycles every 3 to 8 weeks with continuation up to 1 year. Overall collection period: October 2006 to August 2009.
Gastrointestinal disorders Nausea	33.3% 3/9 • Adverse event data collected with each 7 day treatment through subsequent repeated treatment cycles every 3 to 8 weeks with continuation up to 1 year. Overall collection period: October 2006 to August 2009.
Infections and infestations Neutropenic fever	11.1% 1/9 • Adverse event data collected with each 7 day treatment through subsequent repeated treatment cycles every 3 to 8 weeks with continuation up to 1 year. Overall collection period: October 2006 to August 2009.
General disorders Pain	11.1% 1/9 • Adverse event data collected with each 7 day treatment through subsequent repeated treatment cycles every 3 to 8 weeks with continuation up to 1 year. Overall collection period: October 2006 to August 2009.
Respiratory, thoracic and mediastinal disorders pleural effusion	11.1% 1/9 • Adverse event data collected with each 7 day treatment through subsequent repeated treatment cycles every 3 to 8 weeks with continuation up to 1 year. Overall collection period: October 2006 to August 2009.
Infections and infestations Pulmonary Infiltrates	11.1% 1/9 • Adverse event data collected with each 7 day treatment through subsequent repeated treatment cycles every 3 to 8 weeks with continuation up to 1 year. Overall collection period: October 2006 to August 2009.
Respiratory, thoracic and mediastinal disorders Shortness of breath	33.3% 3/9 • Adverse event data collected with each 7 day treatment through subsequent repeated treatment cycles every 3 to 8 weeks with continuation up to 1 year. Overall collection period: October 2006 to August 2009.
General disorders Sweats	11.1% 1/9 • Adverse event data collected with each 7 day treatment through subsequent repeated treatment cycles every 3 to 8 weeks with continuation up to 1 year. Overall collection period: October 2006 to August 2009.
General disorders swollen lips	11.1% 1/9 • Adverse event data collected with each 7 day treatment through subsequent repeated treatment cycles every 3 to 8 weeks with continuation up to 1 year. Overall collection period: October 2006 to August 2009.
Gastrointestinal disorders weight loss	11.1% 1/9 • Adverse event data collected with each 7 day treatment through subsequent repeated treatment cycles every 3 to 8 weeks with continuation up to 1 year. Overall collection period: October 2006 to August 2009.

Additional Information

Zeev Estrov, MD/Professor, Leukemia

University of Texas (UT) MD Anderson Cancer Center

Phone: 713-794-1675

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place