Clinical trial · Interventional
Pre-Transplant 5-Azacitidine In Patients With High-Risk Myelodysplastic Syndrome Who Are Candidates For Allogeneic Hematopoietic Cell Transplant
A Pilot Study Of Pre-Transplant 5-Azacitidine (Vidaza) In Patients With High-Risk Myelodysplastic Syndrome (MDS) Who Are Candidates For Allogeneic Hematopoietic Cell Transplantation
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Leukemia | Leukemia | ONTOLOGY_EXACT | 0.90 |
Interventions
Interventions (2)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| 5-azacitidine | Drug | Azacitidine | ALIAS |
| Allogeneic Hematopoietic Cell Transplantation (HCT) | Procedure | — | UNRESOLVED |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Combined Therapy
- description
- 5-azacitidine therapy followed Allogeneic Hematopoietic Cell Transplantation (HCT).
- interventionNames
- Drug: 5-azacitidine
- Procedure: Allogeneic Hematopoietic Cell Transplantation (HCT)
Primary outcomes (1)
- measure
- Percentage of Participants With Relapse-free Survival (RFS)
- timeFrame
- One year post allogeneic HCT
- description
- Relapse-free survival one year after allogeneic HCT in MDS patients receiving at least one complete cycle of 5-azacitidine (Vidaza) in the pre-transplantation setting. Relapsed disease: if with complete remission (CR) - greater than 5% blasts in bone marrow; if with partial response (PR) - greater than 30% increase in blasts in the marrow; if with stable disease (SD) - return to pretreatment peripheral blood levels and transfusion requirements due to disease.
Secondary outcomes (3)
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
- Maximum age
- 68 Years
Show eligibility criteria text
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
References
Publications (1)
- DERIVEDNishihori T, Perkins J, Mishra A, Komrokji R, Kim J, Kharfan-Dabaja MA, Perez L, Lancet J, Fernandez H, List A, Anasetti C, Field T. Pretransplantation 5-azacitidine in high-risk myelodysplastic syndrome. Biol Blood Marrow Transplant. 2014 Jun;20(6):776-80. doi: 10.1016/j.bbmt.2014.02.008. Epub 2014 Feb 15. PMID 24534108