A Phase II Study of Intravenous Azacitidine Alone in Patients With Myelodysplastic Syndromes - Full Text View

Sponsored by:	Washington University School of Medicine
Information provided by:	Washington University School of Medicine
ClinicalTrials.gov Identifier:	NCT00384956

Purpose

The primary endpoint of this study is to estimate morphologic complete remission rate. Estimation of response rate is also a secondary objection.

Condition	Intervention	Phase
Myelodysplastic Syndromes	Drug: intravenous azacitidine	Phase II

Drug Information available for: Azacitidine

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Efficacy Study
Official Title:	A Phase II Study of Intravenous Azacitidine Alone in Patients With Myelodysplastic Syndromes

Further study details as provided by Washington University School of Medicine:

Primary Outcome Measures:

To estimate the rate of complete remission (CR) and partial remission (PR) after four cycles of therapy in patients with high risk myelodysplastic syndrome (MDS). [ Time Frame: After cycle 4 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

To determine the rate of Hematologic improvement [ Time Frame: throughout study ] [ Designated as safety issue: No ]
To determine the rate of transfusion independence [ Time Frame: throughout study ] [ Designated as safety issue: No ]
To determine the time to disease progression to AML [ Time Frame: throughout study ] [ Designated as safety issue: No ]
To determine the rate of cytogenetic response [ Time Frame: throughout study ] [ Designated as safety issue: No ]
To determine the rate of overall survival [ Time Frame: throughout study ] [ Designated as safety issue: No ]

Estimated Enrollment:	21
Study Start Date:	August 2006
Estimated Primary Completion Date:	December 2009 (Final data collection date for primary outcome measure)

Intervention Details:

Initially all patients will be given azacitidine 75 mg/m2 intravenously on days 1-5 of a 28 day cycle. Patients that do not respond to therapy after two cycles will have the dose increased to 100 mg/m2.

Detailed Description:

Primary Objective To estimate the rate of complete remission (CR) and partial remission (PR) after four cycles of therapy in patients with high risk myelodysplastic syndrome (MDS).

Secondary Objectives

The secondary objectives of the study are:

To determine the rate of Hematologic improvement
To determine the rate of transfusion independence
To determine the time to disease progression to AML
To determine the rate of cytogenetic response
To determine the rate of overall survival

Eligibility

Ages Eligible for Study:	18 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

MDS either de novo or secondary, fitting any of the WHO classifications[44, 45]. Patients with 5% bone marrow blasts must also meet one of the following criteria:
- Symptomatic anemia with either hemoglobin less than 10.0 g/dL or requiring RBC transfusion
- Thrombocytopenia with a history of two or more platelet counts more than 50,000 / µL or a significant hemorrhage requiring platelet transfusions, or
- Neutropenia with two or more absolute neutrophil counts less than 1,000 /µL.
ECOG performance status of 0-2.
Must give written informed consent indicating their awareness of the investigational nature of this study and its potential hazards.
Adequate renal and hepatic function (creatinine less than 2.0 mg/dl, total bilirubin less than 1.5 X institutional upper limit of normal, AST less than 2.0 X institutional upper limit of normal).
Life expectancy of at least 12 weeks.
Ineligible to receive or refused allogeneic stem cell transplant.
Have not received any chemotherapy within 4 weeks of study enrollment and must have recovered from any treatment-related toxicities.
Women of childbearing age must have a negative serum pregnancy test prior to initiating therapy.
Men must be willing to avoid fathering a new child while receiving therapy with azacitidine.
More than or equal to 18 years, no upper age limit

Exclusion Criteria:

Known CNS leukemia.
Previously received Azacitidine (Vidaza®, Pharmion Corp., Boulder CO).
Known or suspected hypersensitivity to azacitidine or mannitol.
Receiving any other investigational agents within 30 days of first dose of study drug.
Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, congestive heart failure of NYHA class 3 or 4, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situation that would limit compliance with study requirements.
Known positive serology for HIV.
Had radiotherapy within 14 days prior to study enrollment.
Known presence of hepatic tumors.
more than 18 years of age

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00384956

Locations

United States, Missouri
Washington University School of Medicine	Recruiting
St. Louis, Missouri, United States, 63110
Contact: Nicholas Fisher 314-454-5102 nfisher@dom.wustl.edu
Principal Investigator: Ravi Vij, M.D.

Sponsors and Collaborators

Washington University School of Medicine

Investigators

Principal Investigator:

Ravi Vij, M.D.

Washington University School of Medicine

More Information

Responsible Party:	Washington University School of Medicine ( Ravi Vij, M.D. )
Study ID Numbers:	06-0585
Study First Received:	October 4, 2006
Last Updated:	October 27, 2008
ClinicalTrials.gov Identifier:	NCT00384956
Health Authority:	United States: Institutional Review Board

Keywords provided by Washington University School of Medicine:

MDS either de novo or secondary, fitting any of the WHO classifications.

Study placed in the following topic categories:

Myelodysplastic syndromes
Preleukemia
Precancerous Conditions
Hematologic Diseases
Myelodysplasia

Myelodysplastic Syndromes
Azacitidine
Neoplasm Metastasis
Bone Marrow Diseases

Additional relevant MeSH terms:

Antimetabolites
Neoplasms
Antimetabolites, Antineoplastic
Pathologic Processes
Disease
Molecular Mechanisms of Pharmacological Action

Antineoplastic Agents
Therapeutic Uses
Syndrome
Enzyme Inhibitors
Pharmacologic Actions

ClinicalTrials.gov processed this record on January 16, 2009