Trial Outcomes & Findings for Maintenance Low Dose 5'-Azacitidine Post T Cell Depleted Allogeneic Stem Cell Transplantation for Patients With Myelodysplastic Syndrome and Acute Myelogenous Leukemia With High Risk for Post-Transplant Relapse (NCT NCT01995578)
NCT ID: NCT01995578
Last Updated: 2024-05-22
Results Overview
Relapse of MDS or AML will be analyzed as to type and genetic origin of the leukemic cells. These will be defined by morphologic and/or cytogenetic criteria: an increasing number of blasts in the marrow over 5%, by presence of circulating blasts, or by presence of blasts in any extramedullary site as well as presence of previous cytogenetic abnormalities. Other studies assessing for MRD, FACS and FISH assays will be evaluated but would not be considered disease relapse if positive since they are experimental.
COMPLETED
PHASE2
32 participants
2 years
2024-05-22
Participant Flow
Participant milestones
| Measure |
Low Dose 5'-Azacitidine
This is a single arm phase II trial to assess the efficacy and confirm the safety of maintenance therapy with 5'-azacitadine compared to historical control after TCD allogeneic hematopoietic stem cell transplant for patients with MDS and AML who are at high risk of relapse.
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|---|---|
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Overall Study
STARTED
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32
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Overall Study
COMPLETED
|
32
|
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Overall Study
NOT COMPLETED
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0
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Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Maintenance Low Dose 5'-Azacitidine Post T Cell Depleted Allogeneic Stem Cell Transplantation for Patients With Myelodysplastic Syndrome and Acute Myelogenous Leukemia With High Risk for Post-Transplant Relapse
Baseline characteristics by cohort
| Measure |
Low Dose 5'-Azacitidine
n=32 Participants
This is a single arm phase II trial to assess the efficacy and confirm the safety of maintenance therapy with 5'-azacitadine compared to historical control after TCD allogeneic hematopoietic stem cell transplant for patients with MDS and AML who are at high risk of relapse.
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|---|---|
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Age, Continuous
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60 years
n=93 Participants
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Sex: Female, Male
Female
|
15 Participants
n=93 Participants
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Sex: Female, Male
Male
|
17 Participants
n=93 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=93 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
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30 Participants
n=93 Participants
|
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Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
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Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Black or African American
|
2 Participants
n=93 Participants
|
|
Race (NIH/OMB)
White
|
30 Participants
n=93 Participants
|
|
Race (NIH/OMB)
More than one race
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0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
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Region of Enrollment
United States
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32 Participants
n=93 Participants
|
PRIMARY outcome
Timeframe: 2 yearsRelapse of MDS or AML will be analyzed as to type and genetic origin of the leukemic cells. These will be defined by morphologic and/or cytogenetic criteria: an increasing number of blasts in the marrow over 5%, by presence of circulating blasts, or by presence of blasts in any extramedullary site as well as presence of previous cytogenetic abnormalities. Other studies assessing for MRD, FACS and FISH assays will be evaluated but would not be considered disease relapse if positive since they are experimental.
Outcome measures
| Measure |
Low Dose 5'-Azacitidine
n=32 Participants
This is a single arm phase II trial to assess the efficacy and confirm the safety of maintenance therapy with 5'-azacitadine compared to historical control after TCD allogeneic hematopoietic stem cell transplant for patients with MDS and AML who are at high risk of relapse.
|
|---|---|
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Relapse Rate
Progression Free
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20 Participants
|
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Relapse Rate
Complete Response
|
2 Participants
|
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Relapse Rate
Not Entered
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10 Participants
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SECONDARY outcome
Timeframe: 2 yearsKaplan-Meier methodology will be used to compare overall survival.
Outcome measures
| Measure |
Low Dose 5'-Azacitidine
n=32 Participants
This is a single arm phase II trial to assess the efficacy and confirm the safety of maintenance therapy with 5'-azacitadine compared to historical control after TCD allogeneic hematopoietic stem cell transplant for patients with MDS and AML who are at high risk of relapse.
|
|---|---|
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Overall Survival
Alive
|
20 Participants
|
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Overall Survival
Dead
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12 Participants
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SECONDARY outcome
Timeframe: 2 yearsThe safety will be described by tabulating the number of transfusions, frequencies of bleeding and serious infections, and the use of G-CSF support.
Outcome measures
| Measure |
Low Dose 5'-Azacitidine
n=32 Participants
This is a single arm phase II trial to assess the efficacy and confirm the safety of maintenance therapy with 5'-azacitadine compared to historical control after TCD allogeneic hematopoietic stem cell transplant for patients with MDS and AML who are at high risk of relapse.
|
|---|---|
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Number of Participants Evaluated for Treatment Safety
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32 Participants
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Adverse Events
Low Dose 5'-Azacitidine
Serious adverse events
| Measure |
Low Dose 5'-Azacitidine
n=32 participants at risk
This is a single arm phase II trial to assess the efficacy and confirm the safety of maintenance therapy with 5'-azacitadine compared to historical control after TCD allogeneic hematopoietic stem cell transplant for patients with MDS and AML who are at high risk of relapse.
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|---|---|
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Respiratory, thoracic and mediastinal disorders
Bronchopulmonary hemorrhage
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3.1%
1/32 • 2 years
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|
Respiratory, thoracic and mediastinal disorders
Dyspnea
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3.1%
1/32 • 2 years
|
|
General disorders
Fever
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3.1%
1/32 • 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
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3.1%
1/32 • 2 years
|
|
Infections and infestations
Infections and infestations - Other, specify
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3.1%
1/32 • 2 years
|
|
Nervous system disorders
Intracranial hemorrhage
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6.2%
2/32 • 2 years
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|
Infections and infestations
Lung infection
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12.5%
4/32 • 2 years
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|
Investigations
Platelet count decreased
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6.2%
2/32 • 2 years
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Treatment related secondary malignancy
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3.1%
1/32 • 2 years
|
Other adverse events
| Measure |
Low Dose 5'-Azacitidine
n=32 participants at risk
This is a single arm phase II trial to assess the efficacy and confirm the safety of maintenance therapy with 5'-azacitadine compared to historical control after TCD allogeneic hematopoietic stem cell transplant for patients with MDS and AML who are at high risk of relapse.
|
|---|---|
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Gastrointestinal disorders
Nausea
|
37.5%
12/32 • 2 years
|
|
Gastrointestinal disorders
Vomiting
|
28.1%
9/32 • 2 years
|
|
Investigations
Alanine aminotransferase increased
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15.6%
5/32 • 2 years
|
|
Investigations
Alkaline phosphatase increased
|
15.6%
5/32 • 2 years
|
|
Investigations
Lymphocyte count decreased
|
15.6%
5/32 • 2 years
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|
Investigations
Neutrophil count decreased
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15.6%
5/32 • 2 years
|
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Blood and lymphatic system disorders
Anemia
|
12.5%
4/32 • 2 years
|
|
Investigations
Platelet count decreased
|
12.5%
4/32 • 2 years
|
|
Investigations
Aspartate aminotransferase increased
|
9.4%
3/32 • 2 years
|
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Gastrointestinal disorders
Constipation
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9.4%
3/32 • 2 years
|
|
General disorders
Fatigue
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9.4%
3/32 • 2 years
|
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Investigations
White blood cell decreased
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9.4%
3/32 • 2 years
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Additional Information
Dr. Roni Tamari MD
Memorial Sloan Kettering Cancer Center
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place