This trial is active, not recruiting.

Condition myelodysplastic syndrome
Treatment 5-azacytidine
Phase phase 2
Sponsor Virginia Commonwealth University
Collaborator Celgene Corporation
Start date July 2008
End date June 2014
Trial size 75 participants
Trial identifier NCT00721214, MCC-11328


Patients with high risk myelodysplastic syndrome (MDS) responding to 5-azacytidine prior to allogeneic transplant have improved event free and overall survival.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
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 Outcomes

1 year and 2-year event free and overall survival (EFS and OS) from the time of transplant.
time frame: 3 years

Secondary Outcomes

Secondary endpoints will include intent to treat analysis of 1-year and 2-year EFS and OS for all patients receiving 5-Azacytidine from the time of initial therapy as well as engraftment of WBC and platelets, graft failure, relapse and GVHD.
time frame: 3 years

Eligibility Criteria

Male or female participants from 18 years up to 70 years old.

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

Additional Information

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
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.
Trial information was received from ClinicalTrials.gov and was last updated in March 2015.
Information provided to ClinicalTrials.gov by Virginia Commonwealth University.