Trial Outcomes & Findings for Maintenance Azacitidine in Elderly Patients With Acute Myeloid Leukemia (AML) in CR After Induction Chemotherapy (NCT NCT00387647)

Last Updated: 2014-08-22

Results Overview

The primary efficacy variable is disease free survival measured at one year, which is the percentage of patients who remain alive and disease free one year after the confirmation of remission by bone marrow biopsy. Relapse is defined by a bone marrow specimen with \>5% blasts or the presence of Auer rods.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

24 participants

Primary outcome timeframe

1 year

Results posted on

2014-08-22

Participant Flow

Researchers consented 29 potential participants between 08/02/0206 and 11/01/2011. 24 eligible participants began treatment, one at Duke University Medical Center and 23 at Moffitt Cancer Center.

Participant milestones

Participant milestones
Measure	Azacitidine Treatment Azacitidine 50 mg/m\^2 subcutaneously daily for 5 days (Monday through Friday) on days 1 through 5, every 28 days for 6-12 cycles. Patients were to be followed for 1 year following completion of study drug treatment.
Overall Study STARTED	24
Overall Study COMPLETED	6
Overall Study NOT COMPLETED	18

Reasons for withdrawal

Reasons for withdrawal
Measure	Azacitidine Treatment Azacitidine 50 mg/m\^2 subcutaneously daily for 5 days (Monday through Friday) on days 1 through 5, every 28 days for 6-12 cycles. Patients were to be followed for 1 year following completion of study drug treatment.
Overall Study Death	18

Baseline Characteristics

Maintenance Azacitidine in Elderly Patients With Acute Myeloid Leukemia (AML) in CR After Induction Chemotherapy

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Azacitidine Treatment n=24 Participants Azacitidine 50 mg/m\^2 subcutaneously daily for 5 days (Monday through Friday) on days 1 through 5, every 28 days for 6-12 cycles. Patients were to be followed for 1 year following completion of study drug treatment.
Age, Continuous	68.7 years n=5 Participants
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	6 Participants n=5 Participants
Age, Categorical >=65 years	18 Participants n=5 Participants
Sex: Female, Male Female	4 Participants n=5 Participants
Sex: Female, Male Male	20 Participants n=5 Participants
Region of Enrollment United States	24 participants n=5 Participants

PRIMARY outcome

Timeframe: 1 year

Population: All participants

Outcome measures

Outcome measures
Measure	Azacitidine Treatment n=24 Participants Azacitidine 50 mg/m\^2 subcutaneously daily for 5 days (Monday through Friday) on days 1 through 5, every 28 days for 6-12 cycles. Patients were to be followed for 1 year following completion of study drug treatment.
Rate of Disease Free Survival at One Year	50 percentage of participants Interval 7.3 to

SECONDARY outcome

Timeframe: 48 months

Population: All participants

The secondary efficacy variable is overall survival measured as time to death, which is the time from remission until death from any cause.

Outcome measures

Outcome measures
Measure	Azacitidine Treatment n=24 Participants Azacitidine 50 mg/m\^2 subcutaneously daily for 5 days (Monday through Friday) on days 1 through 5, every 28 days for 6-12 cycles. Patients were to be followed for 1 year following completion of study drug treatment.
Overall Survival (OS)	20.4 months Interval 12.2 to 26.0

SECONDARY outcome

Timeframe: 48 months

Population: All participants

Safety and tolerability of treatment as measured by NCI Common Terminology Criteria for Adverse Events (CTCAE) v3.0

Outcome measures

Outcome measures
Measure	Azacitidine Treatment n=24 Participants Azacitidine 50 mg/m\^2 subcutaneously daily for 5 days (Monday through Friday) on days 1 through 5, every 28 days for 6-12 cycles. Patients were to be followed for 1 year following completion of study drug treatment.
Number of Participants With Adverse Events	24 participants

Adverse Events

Azacitidine Treatment

Serious events: 2 serious events

Other events: 24 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Azacitidine Treatment n=24 participants at risk Azacitidine 50 mg/m\^2 subcutaneously daily for 5 days (Monday through Friday) on days 1 through 5, every 28 days for 6-12 cycles. Patients were to be followed for 1 year following completion of study drug treatment.
Infections and infestations Colitis, infectious (e.g., Clostridium difficile) - possibly related	4.2% 1/24 • Number of events 1 • 6 years, 1 month overall Adverse event observation from after study drug administration on cycle 1 /day 1, through 28 days following the last dose of azacitidine.
Respiratory, thoracic and mediastinal disorders Dyspnea (shortness of breath), unrelated	4.2% 1/24 • Number of events 1 • 6 years, 1 month overall Adverse event observation from after study drug administration on cycle 1 /day 1, through 28 days following the last dose of azacitidine.

Other adverse events

Other adverse events
Measure	Azacitidine Treatment n=24 participants at risk Azacitidine 50 mg/m\^2 subcutaneously daily for 5 days (Monday through Friday) on days 1 through 5, every 28 days for 6-12 cycles. Patients were to be followed for 1 year following completion of study drug treatment.
Blood and lymphatic system disorders Platelets - low	79.2% 19/24 • Number of events 81 • 6 years, 1 month overall Adverse event observation from after study drug administration on cycle 1 /day 1, through 28 days following the last dose of azacitidine.
Blood and lymphatic system disorders Neutrophils/granulocytes (ANC/AGC) - low	75.0% 18/24 • Number of events 73 • 6 years, 1 month overall Adverse event observation from after study drug administration on cycle 1 /day 1, through 28 days following the last dose of azacitidine.
Blood and lymphatic system disorders Leukocytes (total WBC) - low	62.5% 15/24 • Number of events 57 • 6 years, 1 month overall Adverse event observation from after study drug administration on cycle 1 /day 1, through 28 days following the last dose of azacitidine.
Blood and lymphatic system disorders Hemoglobin - low	12.5% 3/24 • Number of events 9 • 6 years, 1 month overall Adverse event observation from after study drug administration on cycle 1 /day 1, through 28 days following the last dose of azacitidine.
Gastrointestinal disorders Constipation	45.8% 11/24 • Number of events 20 • 6 years, 1 month overall Adverse event observation from after study drug administration on cycle 1 /day 1, through 28 days following the last dose of azacitidine.
Gastrointestinal disorders Nausea	29.2% 7/24 • Number of events 7 • 6 years, 1 month overall Adverse event observation from after study drug administration on cycle 1 /day 1, through 28 days following the last dose of azacitidine.
Gastrointestinal disorders Anorexia	20.8% 5/24 • Number of events 5 • 6 years, 1 month overall Adverse event observation from after study drug administration on cycle 1 /day 1, through 28 days following the last dose of azacitidine.
Gastrointestinal disorders Diarrhea	12.5% 3/24 • Number of events 5 • 6 years, 1 month overall Adverse event observation from after study drug administration on cycle 1 /day 1, through 28 days following the last dose of azacitidine.
Gastrointestinal disorders Heartburn/dyspepsia	8.3% 2/24 • Number of events 2 • 6 years, 1 month overall Adverse event observation from after study drug administration on cycle 1 /day 1, through 28 days following the last dose of azacitidine.
Gastrointestinal disorders Taste alteration (dysgeusia)	8.3% 2/24 • Number of events 2 • 6 years, 1 month overall Adverse event observation from after study drug administration on cycle 1 /day 1, through 28 days following the last dose of azacitidine.
Skin and subcutaneous tissue disorders Injection site reaction/extravasation changes	41.7% 10/24 • Number of events 23 • 6 years, 1 month overall Adverse event observation from after study drug administration on cycle 1 /day 1, through 28 days following the last dose of azacitidine.
Skin and subcutaneous tissue disorders Rash/desquamation	25.0% 6/24 • Number of events 11 • 6 years, 1 month overall Adverse event observation from after study drug administration on cycle 1 /day 1, through 28 days following the last dose of azacitidine.
Skin and subcutaneous tissue disorders Bruising (in absence of Grade 3 or 4 thrombocytopenia)	12.5% 3/24 • Number of events 3 • 6 years, 1 month overall Adverse event observation from after study drug administration on cycle 1 /day 1, through 28 days following the last dose of azacitidine.
Skin and subcutaneous tissue disorders Dermatology/Skin - Other	12.5% 3/24 • Number of events 11 • 6 years, 1 month overall Adverse event observation from after study drug administration on cycle 1 /day 1, through 28 days following the last dose of azacitidine.
Skin and subcutaneous tissue disorders Pruritus/itching	12.5% 3/24 • Number of events 5 • 6 years, 1 month overall Adverse event observation from after study drug administration on cycle 1 /day 1, through 28 days following the last dose of azacitidine.
Skin and subcutaneous tissue disorders Hair loss/alopecia (scalp or body)	8.3% 2/24 • Number of events 2 • 6 years, 1 month overall Adverse event observation from after study drug administration on cycle 1 /day 1, through 28 days following the last dose of azacitidine.
General disorders Pain - Head/headache	16.7% 4/24 • Number of events 7 • 6 years, 1 month overall Adverse event observation from after study drug administration on cycle 1 /day 1, through 28 days following the last dose of azacitidine.
General disorders Pain - Abdomen NOS	12.5% 3/24 • Number of events 4 • 6 years, 1 month overall Adverse event observation from after study drug administration on cycle 1 /day 1, through 28 days following the last dose of azacitidine.
General disorders Pain - Back	8.3% 2/24 • Number of events 2 • 6 years, 1 month overall Adverse event observation from after study drug administration on cycle 1 /day 1, through 28 days following the last dose of azacitidine.
General disorders Pain - Dental/teeth/peridontal	8.3% 2/24 • Number of events 2 • 6 years, 1 month overall Adverse event observation from after study drug administration on cycle 1 /day 1, through 28 days following the last dose of azacitidine.
General disorders Pain - Extremity-limb	8.3% 2/24 • Number of events 2 • 6 years, 1 month overall Adverse event observation from after study drug administration on cycle 1 /day 1, through 28 days following the last dose of azacitidine.
General disorders Pain - Other	8.3% 2/24 • Number of events 2 • 6 years, 1 month overall Adverse event observation from after study drug administration on cycle 1 /day 1, through 28 days following the last dose of azacitidine.
General disorders Fatigue (asthenia, lethargy, malaise)	33.3% 8/24 • Number of events 11 • 6 years, 1 month overall Adverse event observation from after study drug administration on cycle 1 /day 1, through 28 days following the last dose of azacitidine.
General disorders Constitutional Symptoms - Other	8.3% 2/24 • Number of events 2 • 6 years, 1 month overall Adverse event observation from after study drug administration on cycle 1 /day 1, through 28 days following the last dose of azacitidine.
General disorders Fever (in the absence of neutropenia)	8.3% 2/24 • Number of events 4 • 6 years, 1 month overall Adverse event observation from after study drug administration on cycle 1 /day 1, through 28 days following the last dose of azacitidine.
General disorders Sweating (diaphoresis)	8.3% 2/24 • Number of events 2 • 6 years, 1 month overall Adverse event observation from after study drug administration on cycle 1 /day 1, through 28 days following the last dose of azacitidine.
General disorders Weight loss	8.3% 2/24 • Number of events 2 • 6 years, 1 month overall Adverse event observation from after study drug administration on cycle 1 /day 1, through 28 days following the last dose of azacitidine.
Nervous system disorders Dizziness	25.0% 6/24 • Number of events 8 • 6 years, 1 month overall Adverse event observation from after study drug administration on cycle 1 /day 1, through 28 days following the last dose of azacitidine.
Nervous system disorders Neuropathy: sensory	8.3% 2/24 • Number of events 2 • 6 years, 1 month overall Adverse event observation from after study drug administration on cycle 1 /day 1, through 28 days following the last dose of azacitidine.
Respiratory, thoracic and mediastinal disorders Pulmonary/Upper Respiratory - Other	16.7% 4/24 • Number of events 5 • 6 years, 1 month overall Adverse event observation from after study drug administration on cycle 1 /day 1, through 28 days following the last dose of azacitidine.
Respiratory, thoracic and mediastinal disorders Cough	8.3% 2/24 • Number of events 2 • 6 years, 1 month overall Adverse event observation from after study drug administration on cycle 1 /day 1, through 28 days following the last dose of azacitidine.
Respiratory, thoracic and mediastinal disorders Nasal cavity/paranasal sinus reactions	8.3% 2/24 • Number of events 2 • 6 years, 1 month overall Adverse event observation from after study drug administration on cycle 1 /day 1, through 28 days following the last dose of azacitidine.
Infections and infestations Infection with unknown ANC - Skin (cellulitis)	12.5% 3/24 • Number of events 3 • 6 years, 1 month overall Adverse event observation from after study drug administration on cycle 1 /day 1, through 28 days following the last dose of azacitidine.
Musculoskeletal and connective tissue disorders Muscle weakness, generalized or specific area (not due to neuropathy) - Whole body/generalized	12.5% 3/24 • Number of events 3 • 6 years, 1 month overall Adverse event observation from after study drug administration on cycle 1 /day 1, through 28 days following the last dose of azacitidine.
Eye disorders Ocular/Visual - Other	16.7% 4/24 • Number of events 4 • 6 years, 1 month overall Adverse event observation from after study drug administration on cycle 1 /day 1, through 28 days following the last dose of azacitidine.
Cardiac disorders Hypotension	8.3% 2/24 • Number of events 3 • 6 years, 1 month overall Adverse event observation from after study drug administration on cycle 1 /day 1, through 28 days following the last dose of azacitidine.
Ear and labyrinth disorders Auditory/Ear - Other	8.3% 2/24 • Number of events 3 • 6 years, 1 month overall Adverse event observation from after study drug administration on cycle 1 /day 1, through 28 days following the last dose of azacitidine.
Cardiac disorders Cardiac Arrhythmia - Other	8.3% 2/24 • Number of events 2 • 6 years, 1 month overall Adverse event observation from after study drug administration on cycle 1 /day 1, through 28 days following the last dose of azacitidine.

Additional Information

Jeffrey Lancet, M.D.

H. Lee Moffitt Cancer Center and Research Institute

Phone: 813-745-6841

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place