5-Azacytidine Prior to Allogeneic Stem Cell Transplant in High Risk Myelodysplastic Syndrome

NCT ID: NCT00721214

Last Updated: 2016-03-02

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 16 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2008-07-31
• Study Completion Date: 2014-06-30

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.

Conditions

Myelodysplastic Syndrome

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Arm A: 5-azacytidine

5-azacytidine as pre-transplant cytoreduction prior to allogeneic stem cell transplantation for High Risk Myelodysplatic Syndromes.

Group Type: EXPERIMENTAL

5-azacytidine

Intervention Type: DRUG

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.

Interventions

5-azacytidine

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.

Intervention Type: DRUG

Other Intervention Names

Mylosar; Vidaza; 5-AC; 5-AZC; U-18496

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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 70 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Celgene Corporation

INDUSTRY

Sponsor Role: collaborator

Virginia Commonwealth University

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

John M. McCarty, MD

Role: STUDY_CHAIR

Virginia Commonwealth University

Locations

Massey Cancer Center / Virginia Commonwealth University

Richmond, Virginia, United States

Countries

United States

Other Identifiers

MCC-11328

Identifier Type: -

Identifier Source: org_study_id