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

Virginia Commonwealth University (VCU)

Status and phase: Completed; Phase 2

Conditions: Myelodysplastic Syndrome

Treatments: Drug: 5-azacytidine

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT00721214

MCC-11328

Details and patient eligibility

About

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).

Full 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.

Enrollment: 16 patients

Sex: All

Ages: 18 to 70 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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16 participants in 1 patient group

Arm A: 5-azacytidine

Experimental group

Description:

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

Treatment:

Drug: 5-azacytidine