Trial Outcomes & Findings for Controlled Study of Post-transplant Azacitidine for Prevention of Acute Myelogenous Leukemia and Myelodysplastic Syndrome Relapse (VZ-AML-PI-0129) (NCT NCT00887068)

NCT ID: NCT00887068

Last Updated: 2020-01-14

Results Overview

The time that a participant survives without relapse of the disease.

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 187 participants
• Primary outcome timeframe: 3 years
• Results posted on: 2020-01-14

Participant Flow

Recruitment was from September 2009 to April 2017 for high risk subjects with acute myelogenous leukemia (AML) and myelodysplastic (MDS) patients who have undergone an allogeneic transplant.

Patients were randomized to receive azacitidine (AZA) or standard of care 40 to 100 days after allo SCT.

Participant milestones

Measure	AZA Group Subjects randomized to the AZA treatment will receive 32 mg/m2 for 5 consecutive days of each 28 day cycle and the maximum treatment will be 12 cycles. Each cycle will consist of approximately 28 days, allowing the possibility that treatment within a cycle may be delayed for up to 4 weeks due to organ toxicity or hematologic toxicity.	Standard of Care Group Best standard of care (ie, no maintenance)
Overall Study STARTED	93	94
Overall Study COMPLETED	87	94
Overall Study NOT COMPLETED	6	0

Reasons for withdrawal

Measure	AZA Group Subjects randomized to the AZA treatment will receive 32 mg/m2 for 5 consecutive days of each 28 day cycle and the maximum treatment will be 12 cycles. Each cycle will consist of approximately 28 days, allowing the possibility that treatment within a cycle may be delayed for up to 4 weeks due to organ toxicity or hematologic toxicity.	Standard of Care Group Best standard of care (ie, no maintenance)
Overall Study Withdrawal by Subject	2	0
Overall Study Didn't meet eligibility to start cycle 1	4	0

Baseline Characteristics

Controlled Study of Post-transplant Azacitidine for Prevention of Acute Myelogenous Leukemia and Myelodysplastic Syndrome Relapse (VZ-AML-PI-0129)

Baseline characteristics by cohort

Measure	AZA Group n=87 Participants Subjects randomized to the AZA treatment will receive 32 mg/m2 for 5 consecutive days of each 28 day cycle and the maximum treatment will be 12 cycles. Each cycle will consist of approximately 28 days, allowing the possibility that treatment within a cycle may be delayed for up to 4 weeks due to organ toxicity or hematologic toxicity.	Standard of Care Group n=94 Participants Best standard of care (ie, no maintenance)	Total n=181 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	71 Participants n=5 Participants	73 Participants n=7 Participants	144 Participants n=5 Participants
Age, Categorical >=65 years	16 Participants n=5 Participants	21 Participants n=7 Participants	37 Participants n=5 Participants
Sex: Female, Male Female	36 Participants n=5 Participants	37 Participants n=7 Participants	73 Participants n=5 Participants
Sex: Female, Male Male	51 Participants n=5 Participants	57 Participants n=7 Participants	108 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	2 Participants n=5 Participants	0 Participants n=7 Participants	2 Participants n=5 Participants
Race (NIH/OMB) White	79 Participants n=5 Participants	92 Participants n=7 Participants	171 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	6 Participants n=5 Participants	2 Participants n=7 Participants	8 Participants n=5 Participants
Region of Enrollment United States	87 participants n=5 Participants	94 participants n=7 Participants	181 participants n=5 Participants
Disease Strata AML	67 Participants n=5 Participants	67 Participants n=7 Participants	134 Participants n=5 Participants
Disease Strata MDS	26 Participants n=5 Participants	26 Participants n=7 Participants	52 Participants n=5 Participants
Disease Strata Biphenotypic Leukemia	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants

PRIMARY outcome

Timeframe: 3 years

The time that a participant survives without relapse of the disease.

Outcome measures

Measure	AZA Group n=87 Participants Subjects randomized to the AZA treatment will receive 32 mg/m2 for 5 consecutive days of each 28 day cycle and the maximum treatment will be 12 cycles. Each cycle will consist of approximately 28 days, allowing the possibility that treatment within a cycle may be delayed for up to 4 weeks due to organ toxicity or hematologic toxicity.	Standard of Care Group n=94 Participants Best standard of care (ie, no maintenance)
Relapse-free Survival (RFS)	2.1 years Standard Deviation .43	1.3 years Standard Deviation .43

SECONDARY outcome

Timeframe: 3 years

Outcome measures

Measure	AZA Group n=87 Participants Subjects randomized to the AZA treatment will receive 32 mg/m2 for 5 consecutive days of each 28 day cycle and the maximum treatment will be 12 cycles. Each cycle will consist of approximately 28 days, allowing the possibility that treatment within a cycle may be delayed for up to 4 weeks due to organ toxicity or hematologic toxicity.	Standard of Care Group n=94 Participants Best standard of care (ie, no maintenance)
Overall Survival (OS)	2.5 years Standard Deviation .85	2.6 years Standard Deviation .85

Adverse Events

AZA Group

Serious events: 0 serious events

Other events: 29 other events

Deaths: 0 deaths

Standard of Care Group

Serious events: 0 serious events

Other events: 19 other events

Deaths: 1 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	AZA Group n=87 participants at risk Subjects randomized to the AZA treatment will receive 32 mg/m2 for 5 consecutive days of each 28 day cycle and the maximum treatment will be 12 cycles. Each cycle will consist of approximately 28 days, allowing the possibility that treatment within a cycle may be delayed for up to 4 weeks due to organ toxicity or hematologic toxicity.	Standard of Care Group n=94 participants at risk Best standard of care (ie, no maintenance)
Blood and lymphatic system disorders Thrombocytopenia	33.3% 29/87 • From time of enrollment dependent on randomization would be one month from last day of study drug or one year if in standard of care group.	1.1% 1/94 • From time of enrollment dependent on randomization would be one month from last day of study drug or one year if in standard of care group.
Blood and lymphatic system disorders Poor Graft function	33.3% 29/87 • From time of enrollment dependent on randomization would be one month from last day of study drug or one year if in standard of care group.	2.1% 2/94 • From time of enrollment dependent on randomization would be one month from last day of study drug or one year if in standard of care group.
Infections and infestations Infection	0.00% 0/87 • From time of enrollment dependent on randomization would be one month from last day of study drug or one year if in standard of care group.	20.2% 19/94 • From time of enrollment dependent on randomization would be one month from last day of study drug or one year if in standard of care group.
Gastrointestinal disorders Gastrointestetinal	0.00% 0/87 • From time of enrollment dependent on randomization would be one month from last day of study drug or one year if in standard of care group.	12.8% 12/94 • From time of enrollment dependent on randomization would be one month from last day of study drug or one year if in standard of care group.
Hepatobiliary disorders Hepatic	9.2% 8/87 • From time of enrollment dependent on randomization would be one month from last day of study drug or one year if in standard of care group.	5.3% 5/94 • From time of enrollment dependent on randomization would be one month from last day of study drug or one year if in standard of care group.
Respiratory, thoracic and mediastinal disorders Pulmonary	4.6% 4/87 • From time of enrollment dependent on randomization would be one month from last day of study drug or one year if in standard of care group.	6.4% 6/94 • From time of enrollment dependent on randomization would be one month from last day of study drug or one year if in standard of care group.
Skin and subcutaneous tissue disorders Skin	2.3% 2/87 • From time of enrollment dependent on randomization would be one month from last day of study drug or one year if in standard of care group.	5.3% 5/94 • From time of enrollment dependent on randomization would be one month from last day of study drug or one year if in standard of care group.

Additional Information

Dr. Richard E. Champlin, MD/ Chair, Stem Cell Transplantation

UT MD Anderson Cancer Center

Phone: 713-792-3618

Email: rchampli@mdanderson.org

Results disclosure agreements

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