N/A
Completed N=25
Pre-Transplant 5-Azacitidine In Patients With High-Risk Myelodysplastic Syndrome Who Are Candidates For Allogeneic Hematopoietic Cell Transplant
Source: ClinicalTrials.gov NCT00660400 ↗Enrolled (actual)
25
Serious AEs
32.0%
Results posted
Jul 2014
Primary outcomePrimary: Percentage of Participants With Relapse-free Survival (RFS) — 52 percentage of participants
Summary
The purpose of this study is to find out if treating people who have high-risk myelodysplastic syndrome (MDS) with 5-Azacitidine (Vidaza) prior to their allogeneic hematopoietic cell transplant (HCT) is helpful in preventing their myelodysplastic syndrome from coming back.
In previous research, 5-Azacitidine appeared to help the bone marrow of a patient with MDS begin to function more normally. This means bone marrow cells can grow and do their work the way they were meant to. 5-Azacitidine is approved by the Food and Drug Administration (FDA) for the treatment of MDS. The effect of 5-Azacitidine in patients receiving hematopoietic cell transplants have not been studied.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Participants With Relapse-free Survival (RFS) |
52 | — |
| SECONDARY Overall Response Rate (ORR) |
48; 0; 33; 19 | — |
| SECONDARY Percentage of Participants Who Proceed to Hematopoietic Cell Transplantation (HCT) |
84 | — |
| SECONDARY Percentage of Participants With Overall Survival (OS) |
62 | — |
Eligibility Criteria
Inclusion Criteria
- Potential candidate for HCT.
- Histologically confirmed diagnosis by pathologic review of previous diagnosis of high-risk myelodysplastic syndrome (MDS): International Prognostic Scoring System (IPSS) > 1 or AML-MDS or treatment related MDS.
- Serum bilirubin levels ≤1.5 times the upper limit of the normal (ULN) range for the laboratory. Higher levels are acceptable if these can be attributed to active hemolysis or ineffective erythropoiesis; Serum glutamic-oxaloacetic transaminase (SGOT) [aspartate aminotransferase (AST)] or serum glutamic pyruvic transaminase (SGPT) [alanine aminotransferase (ALT)] levels ≤2 x ULN.
- Serum creatinine levels ≤1.5 x ULN
- Karnofsky performance status greater or equal to 70%
- Signed informed consent form in accordance with institutional policies
Exclusion Criteria
- Known or suspected hypersensitivity to Vidaza or mannitol
- Pregnant or lactating women
- Human immunodeficiency virus (HIV) or seropositive, confirmed by nucleic acid amplification testing (NAT)
- Active central nervous system (CNS) malignancy
- Active infection
- History or presence of primary hepatoma
Data sourced from ClinicalTrials.gov (NCT00660400). 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. Informational only — not medical advice.