Pre-Transplant 5-Azacitidine In Patients With High-Risk Myelodysplastic Syndrome Who Are Candidates For Allogeneic Hematopoietic Cell Transplant

NCT00660400 | Completed Results

• 2 other identifiers: MCC-15158106349
• Study Type: interventional
• Target: 25
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Leukemia

Keywords

Myeloid; Monocytic

Outcome Measures

Primary Outcomes (1)

• Percentage of Participants With Relapse-free Survival (RFS)

  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.

  One year post allogeneic HCT

Secondary Outcomes (3)

• Overall Response Rate (ORR)

  At the end of up to six (28 day) cycles of 5-azacitidine

• Percentage of Participants Who Proceed to Hematopoietic Cell Transplantation (HCT)

  Up to 3 years

• Percentage of Participants With Overall Survival (OS)

  One year

Study Arms (1)

Combined Therapy

EXPERIMENTAL

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

Drug: 5-azacitidine; Procedure: Allogeneic Hematopoietic Cell Transplantation (HCT)

Interventions

5-azacitidine DRUG

Once enrolled, the patients will receive pre-transplant 5-azacitidine (Vidaza) 75 mg/M\^2/day subcutaneously for 5-7 days every 28 days). Adjustments in dose and timing may occur based on clinical and hematological parameters.

Also known as: Vidaza

Combined Therapy

Allogeneic Hematopoietic Cell Transplantation (HCT) PROCEDURE

Patients will receive transplantation if there is either a suitable sibling or an unrelated donor. * Response will be evaluated by bone marrow biopsy after 4 cycles of 5-azacitidine or prior to HCT whichever comes first. Due to the very high risk of progression to AML or death in this patient population, the HCT will be done as soon as possible. * The patients may have additional cycles of 5-azacitidine per standard hematology practice until scheduled for transplant or until progression of disease. * All patients will be followed until time to progression of MDS, AML or death or a maximum of 1 year. For patients that are transplanted, follow up will be to one year post-transplant.

Also known as: HCT

Combined Therapy

Eligibility Criteria

• Age: 18 Years - 68 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• H. Lee Moffitt Cancer Center and Research Institute: lead
• Celgene Corporation: collaborator

Study Sites (1)

H. Lee Moffitt Cancer Center & Research Institute

Tampa, Florida, 33612, United States

Location

Related Publications (1)

• Nishihori 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 DERIVED

MeSH Terms

Conditions

Leukemia

Interventions

Azacitidine

Condition Hierarchy (Ancestors)

Neoplasms by Histologic Type; Neoplasms; 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

Limitations and Caveats

Limitation: Small single center pilot study to show the use of pre-treatment 5-Azacitidine and successfully proceeding to transplant.

Results Point of Contact

• Title: Teresa Field, M.D., Ph.D.
• Organization: H. Lee Moffitt Cancer Center and Research Institute

teresa.field@moffitt.org

813-745-7202

Study Officials

• Teresa Field, M.D., Ph.D.

  H. Lee Moffitt Cancer Center and Research Institute

  PRINCIPAL INVESTIGATOR

• Janelle Perkins, Pharm.D.

  H. Lee Moffitt Cancer Center and Research Institute

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 16, 2008
• First Posted: April 17, 2008
• Study Start: March 1, 2008
• Primary Completion: December 1, 2013
• Study Completion: June 1, 2014
• Last Updated: September 22, 2014
• Results First Posted: July 16, 2014

Record last verified: 2014-09

Locations

US (1)