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
18 Years
70 Years
Both
Myelodysplastic Syndrome
Trial Information
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
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.
Inclusion Criteria:
- Patients fulfilling the following criteria will be eligible for study entry:
1. Diagnosis of MDS according to WHO criteria
2. Intermediate-2 or high risk by IPSS score
3. Clinically able to receive 5-Azacytidine
4. Serum bilirubin levels laboratory (ULN). Higher levels are acceptable if these can be attributed to
active hemolysis or ineffective erythropoiesis
5. Serum glutamic-oxaloacetic transaminase (SGOT) or serum glutamic-pyruvic
transaminase (SGPT) levels
6. Serum creatinine levels
7. Negative serum pregnancy test prior to 5-Azacytidine treatment for women of
childbearing potential
8. Women and men of childbearing potential agree to use contraception while
receiving treatment with 5-Azacytidine
9. Potentially eligible for allogeneic transplantation
10. No prior allogeneic transplant
11. Age 18 to 70, inclusive.
Exclusion Criteria:
1. Known or suspected hypersensitivity to 5-azacytidine or mannitol
2. Patients previously treated with 5-azacytidine or deoxyazacytidine
3. Pregnant or breast feeding
4. Patients with advanced malignant hepatic tumors
Type of Study:
Interventional
Study Design:
Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Outcome Measure:
1 year and 2-year event free and overall survival (EFS and OS) from the time of transplant.
Outcome Time Frame:
3 years
Safety Issue:
No
Principal Investigator
John M. McCarty, MD
Investigator Role:
Study Chair
Investigator Affiliation:
Virginia Commonwealth University
Authority:
United States: Institutional Review Board
Study ID:
MCC-11328
NCT ID:
NCT00721214
Start Date:
July 2008
Completion Date:
December 2013
Related Keywords:
- Myelodysplastic Syndrome
- Myelodysplastic Syndrome
- 5-azacytidine
- allogeneic stem cell transplantation
- Myelodysplastic Syndromes
- Preleukemia
Name | Location |
|---|---|
| Massey Cancer Center / Virginia Commonwealth University | Richmond, Virginia 23298 |
