Symptoms and General Pathology

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Myelodysplastic Syndromes

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Azacitidine

Deferasirox

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azacitidine 75mg/m2 7 days/28 day cycle deferasirox 10 mg/kg/day

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

Azacitidine plus Deferasirox

Novartis Pharmaceuticals

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.

Phase II Study to Evaluate Overall Response in Patients With Higher Risk Myelodysplastic Syndromes (MDS) Treated With Azacitidine With or Without Deferasirox.

A 2-year, Multi-center, Phase II, Open-label, Fixed-dose, Randomized Comparative Trial of Azacitidine, With or Without Deferasirox in Patients With Higher Risk Myelodysplastic Syndromes

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 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.

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.

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.

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 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.

Incidence of adverse events (AEs) overall and by severity, and serious adverse events (SAEs).

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

Prevalence of the following mutations in the study population (TP53, EZH2, ETV6, RUNX1, ASXL1, other mutation that is present in ≥ 5% of patients)

Age, Categorical

Sex: Female, Male

No conclusion pertaining to efficacy and/or safety was drawn, only one patient was randomized to the study and their study participation was terminated prematurely due to fatal outcome of SAE.Only one patient in trial.No analysis will ever be done.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization	Link
Phase II Study to Evaluate Overall Response in Patients With Higher Risk Myelodysplastic Syndromes (MDS) Treated With Azacitidine With or Without Deferasirox.	NCT02159040	High Risk MDS	Azacitidine Azacitidine plu...	18 Years - 80 Years	Novartis
Phase II Study to Evaluate Overall Response in Patients With Higher Risk Myelodysplastic Syndromes (MDS) Treated With Azacitidine With or Without Deferasirox.	NCT02159040	High Risk MDS	Azacitidine Azacitidine plu...	18 Years - 80 Years	Novartis

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