The primary objective of the study is to compare the overall response rate (inclusive of complete response, partial response and hematologic improvement) per IWG 2006 criteria in patients with higher risk MDS treated with azacitidine with or without deferasirox achieved over the course of one year. Hematologic improvement must be maintained for at least 8 weeks.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
1
SC or IV AZA at 75mg/m2 7 days/28 day cycle
SC or IV AZA at 75mg/m2 7 days/28 day cycle DFX 10mg/kg/day
Hematology Oncology Services of Arkansas HOSA 2
Little Rock, Arkansas, United States
City of Hope National Medical Center Oncology
Duarte, California, United States
University of Maryland Medical Center UM Greenbaum Cancer Ctr (2)
Baltimore, Maryland, United States
Rochester General Hospital / Lipson Cancer Center Lipson Cancer Center
Rochester, New York, United States
Overall Response Rate Per IWG 2006 Criteria
ORR (inclusive of CR, PR and HI) per IWG 2006 criteria including erythroid response, platelet response and neutrophil response over the course of one year. Hematologic improvement must be maintained for at least 8 weeks in order to count as HI.
Time frame: 1 year
Time to Response
Time to response is defined as time from the date of the first dose of study treatment to the date of the first documented hematologic improvement.
Time frame: up to 24 months
Duration of Response
Duration of response is defined as time from the date of the first observed hematologic improvement to the date of the first subsequent documented disease progression or relapse per IWG 2006 criteria.
Time frame: up to 24 months
Progression Free Survival
Progression free survival is defined as time from the date of the first dose of study treatment to the date of the first documented disease progression or relapse per IWG 2006 criteria.
Time frame: Up to 24 months
Overall Survival
Overall survival is defined as time from the date of the first dose of study treatment to the date of death from any cause.
Time frame: up to 24 months
Time to AML Transformation
Time to AML transformation is defined as time from the date of the first dose of study treatment to the date of the first documented bone marrow blast count ≥ 20% per WHO classification 1999.
Time frame: Up to 24 months
Change in Serum Ferritin
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The Jones Clinic
Germantown, Tennessee, United States
Utah Cancer Specialists IHO Corp
Salt Lake City, Utah, United States
Change in Serum Ferritin
Time frame: up to 24 months
Incidence of Adverse Events
Incidence of adverse events (AEs) overall and by severity, and serious adverse events (SAEs).
Time frame: up to 24 months
Rate of Infection
Median number of infections (positive bacterial, viral or fungal culture, or infection requiring IV antimicrobial, or infection resulting in hospitalization or death) in patients treated with azacitidine alone vs. azacitidine + deferasirox
Time frame: up to 24 months
Prevalence of MDS/AML Related Gene Mutations
Prevalence of the following mutations in the study population (TP53, EZH2, ETV6, RUNX1, ASXL1, other mutation that is present in ≥ 5% of patients)
Time frame: Baseline