Completed

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

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What is being tested

5-azacitidine

+ Allogeneic Hematopoietic Cell Transplantation (HCT)

DrugProcedure
Who is being recruted

Hematologic Diseases+2

+ Hemic and Lymphatic Diseases

+ Leukemia

From 18 to 68 Years
+12 Eligibility Criteria
See all eligibility criteria
How is the trial designed

Treatment Study

Interventional
Study Start: March 2008
See protocol details

Summary

Principal SponsorH. Lee Moffitt Cancer Center and Research Institute
Last updated: January 27, 2026
Sourced from a government-validated database.Claim as a partner

Study start date: March 1, 2008

Actual date on which the first participant was enrolled.

RESEARCH PLAN * This will be a single-center prospective trial * Patients with high risk MDS that are potentially eligible for HCT will be enrolled. * A donor search will be initiated, and 5-Azacitidine will be given per standard practice. * 5-Azacitidine dose is 75 mg/M\^2/day subcutaneously by standard practice (generally this is 7 days per monthly cycle, but alterations occur depending on clinical and laboratory parameters). * Patients where a suitable donor is not found can continue with 5-Azacitidine per standard treatment. These patients will be followed until progression of MDS to acute myelogenous leukemia (AML) or death, for up to one year. * If a suitable donor is obtained, the patient will proceed to HCT. The HCT conditioning regimen will be dictated by the Blood and Marrow Transplant (BMT) physician. While waiting HCT, additional cycles 5-Azacitidine may be given. Pre-HCT conditioning regimen therapy will begin no more than 8 weeks and no less than 4 weeks after the last administration of 5-Azacitidine. * As the number of cycles of 5-Azacitidine is not standardized and the retrospective review of our patients noted above indicated a benefit to ANY exposure to 5-Azacitidine, the actual number of cycles of 5-Azacitidine delivered will not be specified. In addition, as high risk MDS patients have an average time to death of 0.4 years, any delay to HCT once it is available is to be avoided. * A bone marrow biopsy will be performed to reassess disease response to therapy after the last cycle of 5-Azacitidine before transplant, or after the fourth cycle of 5-Azacitidine, whichever comes first. Note that both the biopsy and the timing of the biopsy is a standard evaluation procedure. * Donor progenitor cell collection will be prescribed by the BMT Attending Physician. HCT * The patient will undergo HCT designated per attending BMT physician. * Supportive care will be based on institutional guidelines, Stem cell collections, processing and laboratory studies Stem cell collections, processing and laboratory studies * Graft assessment, processing, and characterization will be done as per institutional guidelines * Chimerism testing will be obtained to document post-transplant engraftment, per standard practice.

Official TitleA Pilot Study Of Pre-Transplant 5-Azacitidine (Vidaza) In Patients With High-Risk Myelodysplastic Syndrome (MDS) Who Are Candidates For Allogeneic Hematopoietic Cell Transplantation
NCT00660400
Principal SponsorH. Lee Moffitt Cancer Center and Research Institute
Last updated: January 27, 2026
Sourced from a government-validated database.Claim as a partner

Protocol

This section provides details of the study plan, including how the study is designed and what the study is measuring.
Design Details

25 patients to be enrolled

Total number of participants that the clinical trial aims to recruit.

Treatment Study

These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.



Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.
Conditions
Criteria

Any sex

Biological sex of participants that are eligible to enroll.

From 18 to 68 Years

Range of ages for which participants are eligible to join.

Healthy volunteers not allowed

If individuals who are healthy and do not have the condition being studied can participate.

Conditions

Pathology

Hematologic DiseasesHemic and Lymphatic DiseasesLeukemiaNeoplasmsNeoplasms by Histologic Type

Criteria

6 inclusion criteria required to participate
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

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6 exclusion criteria prevent from participating
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

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Study Plan

Find out more about all the medication administered in this study, their detailed description and what they involve.
Treatment Groups
Study Objectives

One single intervention group is designated in this study

This study does not include a placebo group 

Treatment Groups

Group I

Experimental
5-azacitidine therapy followed Allogeneic Hematopoietic Cell Transplantation (HCT).

Study Objectives

Primary Objectives

Secondary Objectives

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.

This study has 1 location

Suspended

H. Lee Moffitt Cancer Center & Research Institute

Tampa, United StatesOpen H. Lee Moffitt Cancer Center & Research Institute in Google Maps
CompletedOne Study Center