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

NCT00721214 | Completed Phase 2 Results DMC

• 1 other identifier: MCC-11328
• Study Type: interventional
• Target: 16
• Locations: 1 country
• Sites: 1

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).

Conditions

Myelodysplastic Syndrome

Keywords

Myelodysplastic Syndrome; 5-azacytidine; allogeneic stem cell transplantation

Outcome Measures

Primary Outcomes (4)

• One Year Overall Survival of Allogeneic Transplant Recipients After Transplantation

  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%.

  1 year

• Two Year Overall Survival of Allogeneic Transplant Recipients After Transplantation

  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.

  2 years

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

  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%.

  1 year

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

  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%.

  2 years

Secondary Outcomes (4)

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

  1 year

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

  2 years

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

  1 year

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

  2 years

Study Arms (1)

Arm A: 5-azacytidine

EXPERIMENTAL

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

Drug: 5-azacytidine

Interventions

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.

Also known as: Mylosar, Vidaza, 5-AC, 5-AZC, U-18496

Arm A: 5-azacytidine

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Virginia Commonwealth University: lead
• Celgene Corporation: collaborator

Study Sites (1)

Massey Cancer Center / Virginia Commonwealth University

Richmond, Virginia, 23298, United States

Location

MeSH Terms

Conditions

Myelodysplastic Syndromes

Interventions

Azacitidine

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Aza Compounds; Organic Chemicals; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Ribonucleosides

Results Point of Contact

• Title: John M. McCarty, MD
• Organization: Massey Cancer Center

jmccarty@vcu.edu

804-828-4360

Study Officials

• John M. McCarty, MD

  Virginia Commonwealth University

  STUDY CHAIR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 22, 2008
• First Posted: July 24, 2008
• Study Start: July 1, 2008
• Primary Completion: June 1, 2014
• Study Completion: June 1, 2014
• Last Updated: March 2, 2016
• Results First Posted: August 3, 2015

Record last verified: 2016-02

Locations

US (1)