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Phase 2 N=16 Treatment

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

Myelodysplastic Syndrome

Enrolled (actual)
16
Serious AEs
81.3%
Results posted
Aug 2015
Primary outcome: Primary: One Year Overall Survival of Allogeneic Transplant Recipients After Transplantation — 50 percentage of participants

Study Design & Population

Study type
Interventional
Phase
Phase 2
Interventions
5-azacytidine (Drug)
Age
Adult, Older Adult · 18+ yrs
Sex
All
Sponsor
Virginia Commonwealth University
Primary completion
Jun 2014

Outcome Measures

OutcomeResultp-value
PRIMARY
One Year Overall Survival of Allogeneic Transplant Recipients After Transplantation
50
PRIMARY
Two Year Overall Survival of Allogeneic Transplant Recipients After Transplantation
50
PRIMARY
One Year Event Free Survival (EFS) for Allogeneic Transplant Recipients After Transplantation
50
PRIMARY
Two Year Event Free Survival (EFS) for Allogeneic Transplant Recipients After Transplantation
50
SECONDARY
One-year Overall Survival From Time of Treatment Initiation With 5-Azacytidine for All Study Cohorts
47
SECONDARY
Two-year Overall Survival From Time of Treatment Initiation With 5-Azacytidine for All Study Cohorts.
37
SECONDARY
One-year Event-free Survival From Time of Treatment Initiation With 5-Azacytidine for All Study Cohorts
47
SECONDARY
Two-year Event-free Survival From Time of Treatment Initiation With 5-Azacytidine for All Study Cohorts
37

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).

Eligibility Criteria

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
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00721214). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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