Clinical trial NCT00043381: Decitabine Versus Supportive Care in Adults With Advanced-stage MDS
Official title: A Randomized, Open-label, Phase III Trial of Decitabine (5-aza-2'Deoxycytidine) Versus Supportive Care in Adults With Advanced-stage Myelodysplastic Syndrome
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Myelodysplastic Syndrome
Keywords: myelodysplastic syndrome
MDS
chronic myelomonocytic leukemia
CMML
decitabine
5-aza-2'deoxycytidine
Lead sponsor: Astex Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2002-08-12
Enrollment: 160
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: To compare the safety and efficacy profiles of decitabine to those of supportive care in adults with advanced-stage myelodysplastic syndrome (MDS)

Detailed description: This experimental (investigational) study is intended to answer the question of whether decitabine is any better than supportive care alone in delaying progression (worsening) of the disease, prolonging survival or improving the overall quality of life for MDS patients who are not candidates for bone marrow transplant (BMT).

Eligibility criteria: Inclusion:

* MDS (de novo or secondary) fitting any of the recognized French-American-British, FAB, classifications or chronic myelomonocytic leukemia (CMML) with WBC \< 12,000/mm3, AND International Prognostic Scoring System (IPSS) \>/= 0.5 as determined by CBC, bone marrow assessment and bone marrow cytogenetics within 30 days of randomization
* 18 years or older
* Female patients of child-bearing potential must have a negative serum hCG within 24 hours prior to randomization, must practice a medically approved method of birth control for the past 30 days, and agree to continue this practice for the trial duration and must not be breast-feeding
* ECOG or WHO performance status of 0-2
* Written informed consent
* Normal renal and hepatic function (creatinine \</= 2 mg/dL, bilirubin \</= 1.5 mg/dL, SGPT \</= 2 times the upper limit of normal range)

Exclusion:

* Acute Myeloid Leukemia (AML) (\>/=30% bone marrow blasts) or other progressive malignant disease
* Patients must have recovered from the toxic effects of prior therapy and must be off all chemotherapy for a minimum of 4 weeks prior to study entry to the protocol (a minimum of six weeks for prior nitrosoureas and bone marrow transplantation)
* Ongoing treatment with androgenic hormones, danazol, colony-stimulating factors, or other agents used to treat MDS within 7 days of study initiation.
* Administration of any investigational agent within the 30 days preceding study initiation.
* Uncontrolled cardiac disease or congestive heart failure
* Uncontrolled restrictive or obstructive pulmonary disease
* Active viral or bacterial infection
* Superimposed autoimmune hemolytic anemia or thrombocytopenia
* Known positive serology for HIV
* Mental illness or other condition preventing full cooperation with the treatment and monitoring requirements of the study.

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Berkeley
country: United States
facility: Alta Bates Comprehensive Cancer Center
state: California
city: Duarte
country: United States
facility: City of Hope National Medical Center
state: California
city: Escondido
country: United States
facility: Scripps Clinic
state: California
city: Loma Linda
country: United States
facility: Loma Linda Univ. Cancer Center
state: California
city: San Francisco
country: United States
facility: Univ. California San Francisco Medical School
state: California
city: Gainesville
country: United States
facility: University of Florida
state: Florida
city: Tampa
country: United States
facility: H. Lee Moffitt Cancer Center
state: Florida
city: Tampa
country: United States
facility: James A. Haley Veteran's Hospital
state: Florida
city: Chicago
country: United States
facility: Rush Medical Center
state: Illinois
city: Chicago
country: United States
facility: University of Illinois at Chicago
state: Illinois
city: Boston
country: United States
facility: Dana Farber Cancer Institute
state: Massachusetts
city: Boston
country: United States
facility: New England Medical Center Hospital
state: Massachusetts
city: Worcester
country: United States
facility: University of Massachusetts Medical School
state: Massachusetts
city: Minneapolis
country: United States
facility: VA Medical Center
state: Minnesota
city: Saint Louis
country: United States
facility: Washington Univ. School of Medicine
state: Missouri
city: Buffalo
country: United States
facility: Roswell Park Cancer Institute
state: New York
city: New York
country: United States
facility: Memorial Sloan Kettering Cancer Center
state: New York
city: New York
country: United States
facility: Mount Sinai Medical Center
state: New York
city: Chapel Hill
country: United States
facility: UNC Lineberger Comprehensive Cancer Center
state: North Carolina
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Memphis
country: United States
facility: The Memphis Cancer Center
state: Tennessee
city: Austin
country: United States
facility: SW Regional Cancer Center (dba Central Texas Oncology Associates)
state: Texas
city: Dallas
country: United States
facility: Texas Oncology
state: Texas
city: Houston
country: United States
facility: MD Anderson Cancer Center
state: Texas
Study officials:
References:
citation: Jabbour E, Kantarjian H, O'Brien S, Kadia T, Malik A, Welch MA, Teng A, Cortes J, Ravandi F, Garcia-Manero G. Retrospective analysis of prognostic factors associated with response and overall survival by baseline marrow blast percentage in patients with myelodysplastic syndromes treated with decitabine. Clin Lymphoma Myeloma Leuk. 2013 Oct;13(5):592-6. doi: 10.1016/j.clml.2013.05.004. Epub 2013 Jun 20.
citation: Jabbour E, Garcia-Manero G, Ravandi F, Faderl S, O'Brien S, Fullmer A, Cortes JE, Wierda W, Kantarjian H. Prognostic factors associated with disease progression and overall survival in patients with myelodysplastic syndromes treated with decitabine. Clin Lymphoma Myeloma Leuk. 2013 Apr;13(2):131-8. doi: 10.1016/j.clml.2012.11.001. Epub 2012 Dec 21.