Trial Outcomes & Findings for Phase II Study of Azacitidine and Sargramostim as Maintenance Treatment for Poor-Risk AML or MDS (NCT NCT01700673)
NCT ID: NCT01700673
Last Updated: 2022-10-18
Results Overview
To evaluate the two-year relapse-free survival (RFS) of patients with poor-risk Acute Myeloid Leukemia (AML) or Myelodysplasia (MDS), who receive maintenance treatment with 5-Azacytidine (5AC) in combination with sargramostim (GM-CSF) during remission, following definitive therapy with either a stem cell transplant (SCT) or cytarabine-based consolidation chemotherapy.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
25 participants
Primary outcome timeframe
2 year
Results posted on
2022-10-18
Participant Flow
Participant milestones
| Measure |
Myeloablative BMT
Azacitidine and sargramostim after myeloablative stem cell transplant
Azacitidine: Azacitidine will be administered days 1-5 of a 28 day cycle. Treatment is planned for a total of 12 cycles.
Sargramostim: Sargramostim will be administered days 1-10 of a 28 day cycle. Treatment is planned for a total of 12 cycles.
|
Non-myeloablative BMT
Azacitidine and sargramostim after non-myeloablative stem cell transplant
Azacitidine: Azacitidine will be administered days 1-5 of a 28 day cycle. Treatment is planned for a total of 12 cycles.
Sargramostim: Sargramostim will be administered days 1-10 of a 28 day cycle. Treatment is planned for a total of 12 cycles.
|
Standard Consolidation
Azacitidine and sargramostim after standard consolidation
Azacitidine: Azacitidine will be administered days 1-5 of a 28 day cycle. Treatment is planned for a total of 12 cycles.
Sargramostim: Sargramostim will be administered days 1-10 of a 28 day cycle. Treatment is planned for a total of 12 cycles.
|
|---|---|---|---|
|
Overall Study
STARTED
|
1
|
23
|
1
|
|
Overall Study
COMPLETED
|
1
|
17
|
1
|
|
Overall Study
NOT COMPLETED
|
0
|
6
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Phase II Study of Azacitidine and Sargramostim as Maintenance Treatment for Poor-Risk AML or MDS
Baseline characteristics by cohort
| Measure |
Myeloablative BMT
n=1 Participants
Azacitidine and sargramostim after myeloablative stem cell transplant
Azacitidine: Azacitidine will be administered days 1-5 of a 28 day cycle. Treatment is planned for a total of 12 cycles.
Sargramostim: Sargramostim will be administered days 1-10 of a 28 day cycle. Treatment is planned for a total of 12 cycles.
|
Non-myeloablative BMT
n=23 Participants
Azacitidine and sargramostim after non-myeloablative stem cell transplant
Azacitidine: Azacitidine will be administered days 1-5 of a 28 day cycle. Treatment is planned for a total of 12 cycles.
Sargramostim: Sargramostim will be administered days 1-10 of a 28 day cycle. Treatment is planned for a total of 12 cycles.
|
Standard Consolidation
n=1 Participants
Azacitidine and sargramostim after standard consolidation
Azacitidine: Azacitidine will be administered days 1-5 of a 28 day cycle. Treatment is planned for a total of 12 cycles.
Sargramostim: Sargramostim will be administered days 1-10 of a 28 day cycle. Treatment is planned for a total of 12 cycles.
|
Total
n=25 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Customized
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Age, Customized
Between 18 and 72 years
|
1 Participants
n=5 Participants
|
23 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
25 Participants
n=4 Participants
|
|
Age, Customized
>=73 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
11 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
12 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=5 Participants
|
12 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
13 Participants
n=4 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
2 Participants
n=4 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
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Race (NIH/OMB)
White
|
1 Participants
n=5 Participants
|
18 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
20 Participants
n=4 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
PRIMARY outcome
Timeframe: 2 yearTo evaluate the two-year relapse-free survival (RFS) of patients with poor-risk Acute Myeloid Leukemia (AML) or Myelodysplasia (MDS), who receive maintenance treatment with 5-Azacytidine (5AC) in combination with sargramostim (GM-CSF) during remission, following definitive therapy with either a stem cell transplant (SCT) or cytarabine-based consolidation chemotherapy.
Outcome measures
| Measure |
Non-myeloablative BMT
n=17 Participants
Azacitidine and sargramostim after non-myeloablative stem cell transplant
|
Myeloablative BMT
n=1 Participants
Azacitidine and sargramostim after myeloablative stem cell transplant
|
Standard Consolidation
n=1 Participants
Azacitidine and sargramostim after standard consolidation
|
|---|---|---|---|
|
Two-year Relapse Free Survival of Patients
|
8 Participants
|
1 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: 1 yearPercentage of patients with anemia, the most commonly reported hematologic toxicity, after receiving the combination of Azacitidine and sargramostim
Outcome measures
| Measure |
Non-myeloablative BMT
n=17 Participants
Azacitidine and sargramostim after non-myeloablative stem cell transplant
|
Myeloablative BMT
n=1 Participants
Azacitidine and sargramostim after myeloablative stem cell transplant
|
Standard Consolidation
n=1 Participants
Azacitidine and sargramostim after standard consolidation
|
|---|---|---|---|
|
Hematologic Toxicity as Determined by Anemia
|
8 Participants
|
1 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: 1 yearWe will report the number of participants with one year RFS
Outcome measures
| Measure |
Non-myeloablative BMT
n=17 Participants
Azacitidine and sargramostim after non-myeloablative stem cell transplant
|
Myeloablative BMT
n=1 Participants
Azacitidine and sargramostim after myeloablative stem cell transplant
|
Standard Consolidation
n=1 Participants
Azacitidine and sargramostim after standard consolidation
|
|---|---|---|---|
|
One-year RFS
|
12 Participants
|
1 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: 2 yearsPercentage of participants with overall survival at 2 years.
Outcome measures
| Measure |
Non-myeloablative BMT
n=11 Participants
Azacitidine and sargramostim after non-myeloablative stem cell transplant
|
Myeloablative BMT
n=1 Participants
Azacitidine and sargramostim after myeloablative stem cell transplant
|
Standard Consolidation
n=1 Participants
Azacitidine and sargramostim after standard consolidation
|
|---|---|---|---|
|
Overall Survival
|
6 Participants
|
1 Participants
|
1 Participants
|
Adverse Events
Non-myeloablative BMT
Serious events: 4 serious events
Other events: 17 other events
Deaths: 8 deaths
Myeloablative BMT
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Standard Consolidation
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Non-myeloablative BMT
n=17 participants at risk
Azacitidine and sargramostim after non-myeloablative stem cell transplant
|
Myeloablative BMT
n=1 participants at risk
Azacitidine and sargramostim after myeloablative stem cell transplant
|
Standard Consolidation
n=1 participants at risk
Azacitidine and sargramostim after standard consolidation
|
|---|---|---|---|
|
Blood and lymphatic system disorders
anemia
|
11.8%
2/17 • 2 years
|
0.00%
0/1 • 2 years
|
0.00%
0/1 • 2 years
|
|
Blood and lymphatic system disorders
eosinophilia
|
5.9%
1/17 • 2 years
|
0.00%
0/1 • 2 years
|
0.00%
0/1 • 2 years
|
|
Skin and subcutaneous tissue disorders
Graft-vs-host disease
|
5.9%
1/17 • 2 years
|
0.00%
0/1 • 2 years
|
0.00%
0/1 • 2 years
|
|
Infections and infestations
infections
|
23.5%
4/17 • 2 years
|
0.00%
0/1 • 2 years
|
0.00%
0/1 • 2 years
|
|
Skin and subcutaneous tissue disorders
rash
|
5.9%
1/17 • 2 years
|
0.00%
0/1 • 2 years
|
0.00%
0/1 • 2 years
|
|
Blood and lymphatic system disorders
neutropenia
|
11.8%
2/17 • 2 years
|
0.00%
0/1 • 2 years
|
0.00%
0/1 • 2 years
|
|
Blood and lymphatic system disorders
leukopenia
|
11.8%
2/17 • 2 years
|
0.00%
0/1 • 2 years
|
0.00%
0/1 • 2 years
|
|
Blood and lymphatic system disorders
thrombocytopenia
|
5.9%
1/17 • 2 years
|
0.00%
0/1 • 2 years
|
0.00%
0/1 • 2 years
|
Other adverse events
| Measure |
Non-myeloablative BMT
n=17 participants at risk
Azacitidine and sargramostim after non-myeloablative stem cell transplant
|
Myeloablative BMT
n=1 participants at risk
Azacitidine and sargramostim after myeloablative stem cell transplant
|
Standard Consolidation
n=1 participants at risk
Azacitidine and sargramostim after standard consolidation
|
|---|---|---|---|
|
Gastrointestinal disorders
nausea
|
47.1%
8/17 • Number of events 13 • 2 years
|
100.0%
1/1 • 2 years
|
100.0%
1/1 • Number of events 2 • 2 years
|
|
Blood and lymphatic system disorders
ALT increase
|
17.6%
3/17 • Number of events 12 • 2 years
|
0.00%
0/1 • 2 years
|
100.0%
1/1 • 2 years
|
|
Blood and lymphatic system disorders
anemia
|
47.1%
8/17 • Number of events 21 • 2 years
|
100.0%
1/1 • 2 years
|
0.00%
0/1 • 2 years
|
|
Gastrointestinal disorders
anorexia
|
17.6%
3/17 • Number of events 6 • 2 years
|
0.00%
0/1 • 2 years
|
0.00%
0/1 • 2 years
|
|
Blood and lymphatic system disorders
edema
|
11.8%
2/17 • Number of events 5 • 2 years
|
0.00%
0/1 • 2 years
|
100.0%
1/1 • Number of events 2 • 2 years
|
|
Musculoskeletal and connective tissue disorders
myalgia
|
23.5%
4/17 • Number of events 6 • 2 years
|
100.0%
1/1 • 2 years
|
0.00%
0/1 • 2 years
|
|
Musculoskeletal and connective tissue disorders
bone pain
|
41.2%
7/17 • Number of events 8 • 2 years
|
0.00%
0/1 • 2 years
|
100.0%
1/1 • 2 years
|
|
Musculoskeletal and connective tissue disorders
chills
|
11.8%
2/17 • Number of events 3 • 2 years
|
0.00%
0/1 • 2 years
|
0.00%
0/1 • 2 years
|
|
Gastrointestinal disorders
constipation
|
23.5%
4/17 • Number of events 16 • 2 years
|
0.00%
0/1 • 2 years
|
100.0%
1/1 • 2 years
|
|
Blood and lymphatic system disorders
creatinine increase
|
11.8%
2/17 • Number of events 5 • 2 years
|
0.00%
0/1 • 2 years
|
0.00%
0/1 • 2 years
|
|
Gastrointestinal disorders
diarrhea
|
47.1%
8/17 • Number of events 15 • 2 years
|
0.00%
0/1 • 2 years
|
100.0%
1/1 • 2 years
|
|
Ear and labyrinth disorders
dizziness
|
23.5%
4/17 • Number of events 6 • 2 years
|
0.00%
0/1 • 2 years
|
100.0%
1/1 • 2 years
|
|
Respiratory, thoracic and mediastinal disorders
cough
|
47.1%
8/17 • Number of events 14 • 2 years
|
0.00%
0/1 • 2 years
|
100.0%
1/1 • 2 years
|
|
Gastrointestinal disorders
dyspepsia
|
23.5%
4/17 • Number of events 4 • 2 years
|
0.00%
0/1 • 2 years
|
100.0%
1/1 • 2 years
|
|
Respiratory, thoracic and mediastinal disorders
dyspnea
|
23.5%
4/17 • Number of events 12 • 2 years
|
0.00%
0/1 • 2 years
|
0.00%
0/1 • 2 years
|
|
Blood and lymphatic system disorders
eosiniphilia
|
11.8%
2/17 • Number of events 3 • 2 years
|
0.00%
0/1 • 2 years
|
0.00%
0/1 • 2 years
|
|
Skin and subcutaneous tissue disorders
injection site erythema
|
70.6%
12/17 • Number of events 17 • 2 years
|
100.0%
1/1 • 2 years
|
0.00%
0/1 • 2 years
|
|
General disorders
fatigue
|
52.9%
9/17 • Number of events 27 • 2 years
|
0.00%
0/1 • 2 years
|
100.0%
1/1 • 2 years
|
|
Skin and subcutaneous tissue disorders
graft-vs-host disease
|
23.5%
4/17 • Number of events 9 • 2 years
|
100.0%
1/1 • 2 years
|
0.00%
0/1 • 2 years
|
|
Nervous system disorders
headache
|
35.3%
6/17 • Number of events 8 • 2 years
|
0.00%
0/1 • 2 years
|
100.0%
1/1 • 2 years
|
|
Infections and infestations
infections
|
82.4%
14/17 • Number of events 44 • 2 years
|
0.00%
0/1 • 2 years
|
100.0%
1/1 • 2 years
|
|
Nervous system disorders
insomnia
|
23.5%
4/17 • Number of events 9 • 2 years
|
0.00%
0/1 • 2 years
|
0.00%
0/1 • 2 years
|
|
Skin and subcutaneous tissue disorders
rash
|
47.1%
8/17 • Number of events 19 • 2 years
|
0.00%
0/1 • 2 years
|
100.0%
1/1 • Number of events 3 • 2 years
|
|
Skin and subcutaneous tissue disorders
pruritis
|
35.3%
6/17 • Number of events 13 • 2 years
|
0.00%
0/1 • 2 years
|
100.0%
1/1 • Number of events 4 • 2 years
|
|
Blood and lymphatic system disorders
neutropenia
|
29.4%
5/17 • Number of events 13 • 2 years
|
0.00%
0/1 • 2 years
|
100.0%
1/1 • 2 years
|
|
Blood and lymphatic system disorders
leukopenia
|
23.5%
4/17 • Number of events 12 • 2 years
|
0.00%
0/1 • 2 years
|
100.0%
1/1 • 2 years
|
|
Blood and lymphatic system disorders
thrombocytopenia
|
47.1%
8/17 • Number of events 23 • 2 years
|
0.00%
0/1 • 2 years
|
0.00%
0/1 • 2 years
|
Additional Information
Jonathan Webster, MD
Johns Hopkins University School of Medicine, Division of Hematologic Malignancies
Phone: 4106149106
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place