A Multicenter, Randomized, Open-label, Parallel-group, Phase 3 Trial of Subcutaneous Azacitidine Plus Best Supportive Care Versus Conventional Care Regimens Plus Best Supportive Care for the Treatment of Myelodysplastic Syndromes (MDS)
Azacitidine
+ Physician Choice
Bone Marrow Diseases+6
+ Disease
+ Hematologic Diseases
Other Study
Summary
Study start date: November 1, 2003
Actual date on which the first participant was enrolled.Comparison/Control Interventions offered the physician three options: * Best supportive care (BSC) alone, * Low-dose cytarabine subcutaneously for 14 days every 28 to 42 days, or * Standard chemotherapy administered for induction as a continuous intravenous infusion of cytarabine over 7 days plus an anthracycline (daunorubicin, idarubicin, or mitoxantrone) on Days 1, 2, and 3; and, for those eligible, 1 or 2 consolidation cycles administered as continuous intravenous infusions of cytarabine for 3 to 7 days with the same anthracycline that was used at induction on Days 1 and 2 (each cycle between 28 to 70 days from the start of the previous cycle). All three options included best supportive care. Neither the experimental group (azacitidine) nor any of the comparison/control options allowed use of erythropoietin. Duration of Intervention: Patients will be treated until death, withdrawal, unacceptable toxicity or conclusion of the study.
Protocol
This section provides details of the study plan, including how the study is designed and what the study is measuring.358 patients to be enrolled
Total number of participants that the clinical trial aims to recruit.Other Study
Eligibility
Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.Any sex
Biological sex of participants that are eligible to enroll.Over 18 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
Criteria
Inclusion Criteria: * Have a diagnosis of refractory anemia with excess blasts or refractory anemia with excess blasts in transformation according to the French-American-British classification system for myelodysplastic syndromes (MDS) and a relatively high risk of acute myeloid leukemia (AML) transformation, with an International Prognostic Scoring System score of INT-2 or High. * Be 18 years of age or older * Have a life expectancy of at least 3 months * Be unlikely to proceed to bone marrow or stem cell transplantation therapy following remission * Have serum bilirubin levels less than or equal to 1.5 times the upper limit of normal range for the laboratory * Have serum glutamic-oxaloacetic transaminase (aspartate aminotransferase) or serum glutamic-pyruvic transaminase (alanine aminotransferase) levels less than or equal to 2 times the upper limit of normal (unless these are considered to be related to transfusion-induced secondary hemosiderosis) * Have serum creatinine levels less than or equal to 1.5 times the upper limit of normal Exclusion Criteria: * Secondary myelodysplastic syndromes (MDS) * Prior treatment with azacitidine; * Prior history of acute myeloid leukemia (AML); * Malignant disease diagnosed within prior 12 months; * Metastatic disease; * Hepatic tumors; * Radiation, chemotherapy, cytotoxic therapy for non-MDS conditions within prior 12 months; * Prior transplantation or cytotoxic therapy to treat MDS; * Serious medical illness likely to limit survival to 12 months or less; * Treatment with erythropoietin or myeloid growth factors during prior 21 days or androgenic hormones during prior 13 days; * Active HIV, viral hepatitis type B or C; * Treatment with investigational drugs during prior 30 days; * Within the 28-day screening period, documented red cell folate deficiency, as evidenced by red blood cell folate (not serum folate) or vitamin B12 deficiency
Study Plan
Find out more about all the medication administered in this study, their detailed description and what they involve.2 intervention groups are designated in this study
This study does not include a placebo group
Treatment Groups
Group I
ExperimentalGroup II
Active ComparatorStudy 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 108 locations
National Centre of Hematology and Transfusiology, Sofia
Sofia, BulgariaMultiprofile Hospital for Active Treatment (MHAT), "St. Marina" Clinic of Hematology
Varna, BulgariaUniversity Multiprofile Hospital for Active Treatment "Sveta Marina"
Varna, Bulgaria