Clinical trial NCT00721214: 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
Overall status: COMPLETED
Phase: PHASE2
Conditions: Myelodysplastic Syndrome
Keywords: Myelodysplastic Syndrome
5-azacytidine
allogeneic stem cell transplantation
Lead sponsor: Virginia Commonwealth University
Sponsor class: OTHER
Collaborators: name: Celgene Corporation
First posted: 2008-07-24
Enrollment: 16
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: 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 \</=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
  5. Serum glutamic-oxaloacetic transaminase (SGOT) or serum glutamic-pyruvic transaminase (SGPT) levels \</=2 x ULN
  6. Serum creatinine levels \</=1.5 x ULN
  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

Primary outcomes:
measure: One Year Overall Survival of Allogeneic Transplant Recipients After Transplantation
timeFrame: 1 year
measure: Two Year Overall Survival of Allogeneic Transplant Recipients After Transplantation
timeFrame: 2 years
measure: One Year Event Free Survival (EFS) for Allogeneic Transplant Recipients After Transplantation
timeFrame: 1 year
measure: Two Year Event Free Survival (EFS) for Allogeneic Transplant Recipients After Transplantation
timeFrame: 2 years
Secondary outcomes:
measure: One-year Overall Survival From Time of Treatment Initiation With 5-Azacytidine for All Study Cohorts
timeFrame: 1 year
measure: Two-year Overall Survival From Time of Treatment Initiation With 5-Azacytidine for All Study Cohorts.
timeFrame: 2 years
measure: One-year Event-free Survival From Time of Treatment Initiation With 5-Azacytidine for All Study Cohorts
timeFrame: 1 year
measure: Two-year Event-free Survival From Time of Treatment Initiation With 5-Azacytidine for All Study Cohorts
timeFrame: 2 years
Other outcomes:
Locations:
city: Richmond
country: United States
facility: Massey Cancer Center / Virginia Commonwealth University
state: Virginia
Study officials:
affiliation: Virginia Commonwealth University
name: John M. McCarty, MD
role: STUDY_CHAIR
References: