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

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00721214
First Posted: July 24, 2008
Last Update Posted: March 2, 2016
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Collaborator:
Celgene Corporation
Information provided by (Responsible Party):
Virginia Commonwealth University
  Purpose
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).

Condition Intervention Phase
Myelodysplastic Syndrome Drug: 5-azacytidine Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Virginia Commonwealth University:

Primary Outcome Measures:
  • 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%.

  • 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.

  • 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%.

  • 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 Outcome Measures:
  • 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.

  • 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.

  • 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%).

  • 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%).


Enrollment: 16
Study Start Date: July 2008
Study Completion Date: June 2014
Primary Completion Date: June 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Arm A: 5-azacytidine
5-azacytidine as pre-transplant cytoreduction prior to allogeneic stem cell transplantation for High Risk Myelodysplatic Syndromes.
Drug: 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.
Other Names:
  • Mylosar
  • Vidaza
  • 5-AC
  • 5-AZC
  • U-18496

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

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 70 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00721214


Locations
United States, Virginia
Massey Cancer Center / Virginia Commonwealth University
Richmond, Virginia, United States, 23298
Sponsors and Collaborators
Virginia Commonwealth University
Celgene Corporation
Investigators
Study Chair: John M. McCarty, MD Virginia Commonwealth University
  More Information

Responsible Party: Virginia Commonwealth University
ClinicalTrials.gov Identifier: NCT00721214     History of Changes
Other Study ID Numbers: MCC-11328
First Submitted: July 22, 2008
First Posted: July 24, 2008
Results First Submitted: April 29, 2015
Results First Posted: August 3, 2015
Last Update Posted: March 2, 2016
Last Verified: February 2016

Keywords provided by Virginia Commonwealth University:
Myelodysplastic Syndrome
5-azacytidine
allogeneic stem cell transplantation

Additional relevant MeSH terms:
Syndrome
Myelodysplastic Syndromes
Preleukemia
Disease
Pathologic Processes
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Neoplasms
Azacitidine
Antimetabolites, Antineoplastic
Antimetabolites
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Enzyme Inhibitors