Back to results5-Azacytidine Prior to Allogeneic Stem Cell Transplant in High Risk Myelodysplastic Syndrome
Primary Purpose
Myelodysplastic Syndrome
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
5-azacytidine
Sponsored by
About this trial
This is an interventional treatment trial for Myelodysplastic Syndrome focused on measuring Myelodysplastic Syndrome, 5-azacytidine, allogeneic stem cell transplantation
Eligibility Criteria
Inclusion Criteria:
Patients fulfilling the following criteria will be eligible for study entry:
- Diagnosis of MDS according to WHO criteria
- Intermediate-2 or high risk by IPSS score
- Clinically able to receive 5-Azacytidine
- Serum bilirubin levels </=1.5 times the upper limit of the normal range for the laboratory (ULN). Higher levels are acceptable if these can be attributed to active hemolysis or ineffective erythropoiesis
- Serum glutamic-oxaloacetic transaminase (SGOT) or serum glutamic-pyruvic transaminase (SGPT) levels </=2 x ULN
- Serum creatinine levels </=1.5 x ULN
- Negative serum pregnancy test prior to 5-Azacytidine treatment for women of childbearing potential
- Women and men of childbearing potential agree to use contraception while receiving treatment with 5-Azacytidine
- Potentially eligible for allogeneic transplantation
- No prior allogeneic transplant
- Age 18 to 70, inclusive.
Exclusion Criteria:
- Known or suspected hypersensitivity to 5-azacytidine or mannitol
- Patients previously treated with 5-azacytidine or deoxyazacytidine
- Pregnant or breast feeding
- Patients with advanced malignant hepatic tumors
Sites / Locations
- Massey Cancer Center / Virginia Commonwealth University
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Arm A: 5-azacytidine
Arm Description
5-azacytidine as pre-transplant cytoreduction prior to allogeneic stem cell transplantation for High Risk Myelodysplatic Syndromes.
Outcomes
Primary Outcome Measures
One Year Overall Survival of Allogeneic Transplant Recipients After Transplantation
Percentage of patients alive one year after their transplantation, as estimated by the Kaplan-Meier survival curve. The estimated one year survival rate from this curve is 50%, while the estimated two year survival rate is 50%.
Two Year Overall Survival of Allogeneic Transplant Recipients After Transplantation
Percentage of patients alive two years after their transplantation, as estimated by the Kaplan-Meier survival curve. The estimated two year survival rate is 50%, the same as one year survival rate.
One Year Event Free Survival (EFS) for Allogeneic Transplant Recipients After Transplantation
Percentage of participants that received allogeneic transplant and had event free survival, as estimated by the Kaplan-Meier survival curve. The events analyzed are evidence of molecular, cytogenetic or histologic relapse or death from any cause. The estimated one-year event-free survival rate is the same as overall survival, 50%.
Two Year Event Free Survival (EFS) for Allogeneic Transplant Recipients After Transplantation
Percentage of participants that received allogeneic transplant and had event free survival. The percentage of patients was estimated by the Kaplan-Meier survival curve. The events analyzed are evidence of molecular, cytogenetic or histologic relapse or death from any cause. The estimated two-year event-free survival rate is the same as overall survival, 50%.
Secondary Outcome Measures
One-year Overall Survival From Time of Treatment Initiation With 5-Azacytidine for All Study Cohorts
Percentage of participants alive one year after their first treatment was estimated by the Kaplan-Meier survival curve. The events analyzed are evidence of molecular, cytogenetic or histologic relapse or death from any cause. Patients alive at the time of last observation will be censored. The estimated one year overall survival rate from this curve is 47%. The one year overall survival is the same as one year event free survival rate.
Two-year Overall Survival From Time of Treatment Initiation With 5-Azacytidine for All Study Cohorts.
Percentage of participants alive two years after their first treatment was estimated by the Kaplan-Meier survival curve. The events analyzed are evidence of molecular, cytogenetic or histologic relapse or death from any cause. Patients alive at the time of last observation will be censored.The estimated two year survival rate is 37% .The estimated two-year overall survival rate is the same as two-year event free survival.
One-year Event-free Survival From Time of Treatment Initiation With 5-Azacytidine for All Study Cohorts
Percentage of participants with one year event free survival after their first treatment was estimated by the Kaplan-Meier survival curve. The events analyzed are evidence of molecular, cytogenetic or histologic relapse or death from any cause. Patients alive at the time of last observation will be censored.The estimated one-year event-free survival rate is the same as for overall survival, 47% (SE = 13.6%).
Two-year Event-free Survival From Time of Treatment Initiation With 5-Azacytidine for All Study Cohorts
Percentage of participants with two year event free survival after their first treatment was estimated by the Kaplan-Meier survival curve. The events analyzed are evidence of molecular, cytogenetic or histologic relapse or death from any cause. Patients alive at the time of last observation will be censored.The estimated two- year event-free survival rate is the same as for overall survival, 37% (SE = 14.3%).
Full Information
NCT ID
NCT00721214
First Posted
July 22, 2008
Last Updated
February 3, 2016
Sponsor
Virginia Commonwealth University
Collaborators
Celgene Corporation
1. Study Identification
Unique Protocol Identification Number
NCT00721214
Brief Title
5-Azacytidine Prior to Allogeneic Stem Cell Transplant in High Risk Myelodysplastic Syndrome
Official Title
A Phase II Study of the Use of 5-Azacytidine as Pre-Transplant Cytoreduction Prior to Allogeneic Stem Cell Transplantation for High Risk Myelodysplastic Syndromes
Study Type
Interventional
2. Study Status
Record Verification Date
February 2016
Overall Recruitment Status
Completed
Study Start Date
July 2008 (undefined)
Primary Completion Date
June 2014 (Actual)
Study Completion Date
June 2014 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Virginia Commonwealth University
Collaborators
Celgene Corporation
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
The purpose of this study is to examine the feasibility and efficacy of using the demethylating agent 5-Azacytidine prior to allogeneic stem cell transplantation in patients with high risk myelodysplastic syndrome (MDS).
Detailed Description
The study drug, 5-azacytidine, is given daily intravenously for 7 days. After every 2 cycles study participants will have a bone marrow test to evaluate the effect of the 5-azacytidine on the Myelodysplastic Syndrome (MDS). Participants continue to get cycles of 5-Azacytidine until 2 bone marrow tests show the MDS has stopped responding to the treatment. At that time they will undergo a transplant if a donor is available.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Myelodysplastic Syndrome
Keywords
Myelodysplastic Syndrome, 5-azacytidine, allogeneic stem cell transplantation
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
16 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Arm A: 5-azacytidine
Arm Type
Experimental
Arm Description
5-azacytidine as pre-transplant cytoreduction prior to allogeneic stem cell transplantation for High Risk Myelodysplatic Syndromes.
Intervention Type
Drug
Intervention Name(s)
5-azacytidine
Other Intervention Name(s)
Mylosar, Vidaza, 5-AC, 5-AZC, U-18496
Intervention Description
The recommended starting dose for the first treatment cycle, for all patients regardless of baseline hematology laboratory values, is 75 mg/m2 subcutaneously or intravenously, daily for 7 days.
Primary Outcome Measure Information:
Title
One Year Overall Survival of Allogeneic Transplant Recipients After Transplantation
Description
Percentage of patients alive one year after their transplantation, as estimated by the Kaplan-Meier survival curve. The estimated one year survival rate from this curve is 50%, while the estimated two year survival rate is 50%.
Time Frame
1 year
Title
Two Year Overall Survival of Allogeneic Transplant Recipients After Transplantation
Description
Percentage of patients alive two years after their transplantation, as estimated by the Kaplan-Meier survival curve. The estimated two year survival rate is 50%, the same as one year survival rate.
Time Frame
2 years
Title
One Year Event Free Survival (EFS) for Allogeneic Transplant Recipients After Transplantation
Description
Percentage of participants that received allogeneic transplant and had event free survival, as estimated by the Kaplan-Meier survival curve. The events analyzed are evidence of molecular, cytogenetic or histologic relapse or death from any cause. The estimated one-year event-free survival rate is the same as overall survival, 50%.
Time Frame
1 year
Title
Two Year Event Free Survival (EFS) for Allogeneic Transplant Recipients After Transplantation
Description
Percentage of participants that received allogeneic transplant and had event free survival. The percentage of patients was estimated by the Kaplan-Meier survival curve. The events analyzed are evidence of molecular, cytogenetic or histologic relapse or death from any cause. The estimated two-year event-free survival rate is the same as overall survival, 50%.
Time Frame
2 years
Secondary Outcome Measure Information:
Title
One-year Overall Survival From Time of Treatment Initiation With 5-Azacytidine for All Study Cohorts
Description
Percentage of participants alive one year after their first treatment was estimated by the Kaplan-Meier survival curve. The events analyzed are evidence of molecular, cytogenetic or histologic relapse or death from any cause. Patients alive at the time of last observation will be censored. The estimated one year overall survival rate from this curve is 47%. The one year overall survival is the same as one year event free survival rate.
Time Frame
1 year
Title
Two-year Overall Survival From Time of Treatment Initiation With 5-Azacytidine for All Study Cohorts.
Description
Percentage of participants alive two years after their first treatment was estimated by the Kaplan-Meier survival curve. The events analyzed are evidence of molecular, cytogenetic or histologic relapse or death from any cause. Patients alive at the time of last observation will be censored.The estimated two year survival rate is 37% .The estimated two-year overall survival rate is the same as two-year event free survival.
Time Frame
2 years
Title
One-year Event-free Survival From Time of Treatment Initiation With 5-Azacytidine for All Study Cohorts
Description
Percentage of participants with one year event free survival after their first treatment was estimated by the Kaplan-Meier survival curve. The events analyzed are evidence of molecular, cytogenetic or histologic relapse or death from any cause. Patients alive at the time of last observation will be censored.The estimated one-year event-free survival rate is the same as for overall survival, 47% (SE = 13.6%).
Time Frame
1 year
Title
Two-year Event-free Survival From Time of Treatment Initiation With 5-Azacytidine for All Study Cohorts
Description
Percentage of participants with two year event free survival after their first treatment was estimated by the Kaplan-Meier survival curve. The events analyzed are evidence of molecular, cytogenetic or histologic relapse or death from any cause. Patients alive at the time of last observation will be censored.The estimated two- year event-free survival rate is the same as for overall survival, 37% (SE = 14.3%).
Time Frame
2 years
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
70 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Patients fulfilling the following criteria will be eligible for study entry:
Diagnosis of MDS according to WHO criteria
Intermediate-2 or high risk by IPSS score
Clinically able to receive 5-Azacytidine
Serum bilirubin levels </=1.5 times the upper limit of the normal range for the laboratory (ULN). Higher levels are acceptable if these can be attributed to active hemolysis or ineffective erythropoiesis
Serum glutamic-oxaloacetic transaminase (SGOT) or serum glutamic-pyruvic transaminase (SGPT) levels </=2 x ULN
Serum creatinine levels </=1.5 x ULN
Negative serum pregnancy test prior to 5-Azacytidine treatment for women of childbearing potential
Women and men of childbearing potential agree to use contraception while receiving treatment with 5-Azacytidine
Potentially eligible for allogeneic transplantation
No prior allogeneic transplant
Age 18 to 70, inclusive.
Exclusion Criteria:
Known or suspected hypersensitivity to 5-azacytidine or mannitol
Patients previously treated with 5-azacytidine or deoxyazacytidine
Pregnant or breast feeding
Patients with advanced malignant hepatic tumors
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
John M. McCarty, MD
Organizational Affiliation
Virginia Commonwealth University
Official's Role
Study Chair
Facility Information:
Facility Name
Massey Cancer Center / Virginia Commonwealth University
City
Richmond
State/Province
Virginia
ZIP/Postal Code
23298
Country
United States
12. IPD Sharing Statement
Learn more about this trial
5-Azacytidine Prior to Allogeneic Stem Cell Transplant in High Risk Myelodysplastic Syndrome
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