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Completed

NCT Number: NCT00721214

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

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).

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Key information

Conditions

Myelodysplastic Syndrome Bone Marrow Diseases Hematologic Diseases Hemic and Lymphatic Diseases Myelodysplastic Syndromes

Age range

18 year–70 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Massey Cancer Center / Virginia Commonwealth University

Richmond, Virginia, 23298, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Patients fulfilling the following criteria will be eligible for study entry:
  • Diagnosis of MDS according to WHO criteria
  • Intermediate-2 or high risk by IPSS score
  • Clinically able to receive 5-Azacytidine
  • 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
  • Serum glutamic-oxaloacetic transaminase (SGOT) or serum glutamic-pyruvic transaminase (SGPT) levels </=2 x ULN
  • Serum creatinine levels </=1.5 x ULN
  • Negative serum pregnancy test prior to 5-Azacytidine treatment for women of childbearing potential
  • Women and men of childbearing potential agree to use contraception while receiving treatment with 5-Azacytidine
  • Potentially eligible for allogeneic transplantation
  • No prior allogeneic transplant
  • Age 18 to 70, inclusive.

Exclusion criteria

  • Known or suspected hypersensitivity to 5-azacytidine or mannitol
  • Patients previously treated with 5-azacytidine or deoxyazacytidine
  • Pregnant or breast feeding
  • Patients with advanced malignant hepatic tumors

Treatment and study plan

5-Azacytidine

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.

Other names: Mylosar, Vidaza, 5-AC, 5-AZC, U-18496

Primary outcomes

  1. One Year Overall Survival of Allogeneic Transplant Recipients After Transplantation

    Time frame: 1 year

    Percentage of patients alive one year after their transplantation, as estimated by the Kaplan-Meier survival curve. The estimated one year survival rate from this curve is 50%, while the estimated two year survival rate is 50%.

  2. Two Year Overall Survival of Allogeneic Transplant Recipients After Transplantation

    Time frame: 2 years

    Percentage of patients alive two years after their transplantation, as estimated by the Kaplan-Meier survival curve. The estimated two year survival rate is 50%, the same as one year survival rate.

  3. One Year Event Free Survival (EFS) for Allogeneic Transplant Recipients After Transplantation

    Time frame: 1 year

    Percentage of participants that received allogeneic transplant and had event free survival, as estimated by the Kaplan-Meier survival curve. The events analyzed are evidence of molecular, cytogenetic or histologic relapse or death from any cause. The estimated one-year event-free survival rate is the same as overall survival, 50%.

  4. Two Year Event Free Survival (EFS) for Allogeneic Transplant Recipients After Transplantation

    Time frame: 2 years

    Percentage of participants that received allogeneic transplant and had event free survival. The percentage of patients was estimated by the Kaplan-Meier survival curve. The events analyzed are evidence of molecular, cytogenetic or histologic relapse or death from any cause. The estimated two-year event-free survival rate is the same as overall survival, 50%.

Secondary outcomes

  1. One-year Overall Survival From Time of Treatment Initiation With 5-Azacytidine for All Study Cohorts

    Time frame: 1 year

    Percentage of participants alive one year after their first treatment was estimated by the Kaplan-Meier survival curve. The events analyzed are evidence of molecular, cytogenetic or histologic relapse or death from any cause. Patients alive at the time of last observation will be censored. The estimated one year overall survival rate from this curve is 47%. The one year overall survival is the same as one year event free survival rate.

  2. Two-year Overall Survival From Time of Treatment Initiation With 5-Azacytidine for All Study Cohorts.

    Time frame: 2 years

    Percentage of participants alive two years after their first treatment was estimated by the Kaplan-Meier survival curve. The events analyzed are evidence of molecular, cytogenetic or histologic relapse or death from any cause. Patients alive at the time of last observation will be censored.The estimated two year survival rate is 37% .The estimated two-year overall survival rate is the same as two-year event free survival.

  3. One-year Event-free Survival From Time of Treatment Initiation With 5-Azacytidine for All Study Cohorts

    Time frame: 1 year

    Percentage of participants with one year event free survival after their first treatment was estimated by the Kaplan-Meier survival curve. The events analyzed are evidence of molecular, cytogenetic or histologic relapse or death from any cause. Patients alive at the time of last observation will be censored.The estimated one-year event-free survival rate is the same as for overall survival, 47% (SE = 13.6%).

  4. Two-year Event-free Survival From Time of Treatment Initiation With 5-Azacytidine for All Study Cohorts

    Time frame: 2 years

    Percentage of participants with two year event free survival after their first treatment was estimated by the Kaplan-Meier survival curve. The events analyzed are evidence of molecular, cytogenetic or histologic relapse or death from any cause. Patients alive at the time of last observation will be censored.The estimated two- year event-free survival rate is the same as for overall survival, 37% (SE = 14.3%).

Sponsors and collaborators

Lead sponsor

Virginia Commonwealth University

Other

Collaborators

  • Celgene Corporation

Registry information

Official study 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

Important dates

Study start
2008
Primary completion
2014
Study completion
2014
First posted
Jul 24, 2008
Registry last updated
Mar 2, 2016

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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