NCT02038816.

Trial name or title	Azacitidine Plus Deferasirox (ICL670) in Higher Risk MDS
Methods	An open‐label, phase II, randomised efficacy study in patients with higher risk MDS
Participants	Inclusion criteria: Adults > 18 years of age WHO defined MDS with Higher risk MDS (IPSS int‐2/high) Azacitidine X at least 6 cycles with no hematologic improvement/no disease progression as per IWG 2006 criteria Ferritin > 500 µg/L If transfusion independent, must have Hb < 110 g/L or neutrophils < 1000/mL or platelets < 100,000/mL ECOG ≤ 2 CrCl > 40 mL/min Exclusion criteria: Increased ALT (> 300 U/L) Uncontrolled infection HIV+ Pregnant or breast‐feeding Previous and concurrent iron chelation Concurrent use of hematopoietic growth factors including erythropoietin, darbepoietin and granulocyte colony stimulating factor Concurrent use of other disease modifying agents including cytotoxic chemotherapy, histone deacetylase inhibitors, other hypomethylating agents or lenalidomide
Interventions	Experimental: Deferasirox plus azacitidine Active comparator: Azacitidine
Outcomes	Primary outcome measures: Difference in proportion of patients with hematologic improvement as defined by the IWG criteria 30 with the addition of deferasirox to azacitidine compared with azacitidine alone in higher risk non‐responding MDS patients after 6 cycles of azacitidine Secondary outcome measures: Tolerability (defined by the percentage of patients able to remain on deferasirox for 6 cycles concurrent with azacitidine) and safety (type, using CTCAE version 4.0, frequency, severity and relationship of adverse events to study therapy Percentage and absolute change in serum ferritin and labile plasma iron (LPI) between baseline and end of study Percentage change in CD34 cell intracellular reactive oxygen species (ROS) from baseline to end of study Percentage change in erythroid colony forming units (BFU‐E and CFU‐E) from baseline to end of study Percentage change in markers of DNA damage (lipid peroxidation, GSH content and gH2AX expression), and activity of NFkappaB and Akt signaling pathways between baseline and end of study
Starting date	Study start date: March 2014; Estimated primary completion date: October 2016 (Final data collection date for primary measure)
Contact information	Odette Cancer Centre, Sunnybrook Health Sciences Centre Toronto, Ontario, Canada, M4N 3M5 Contact: Claudia Li (claudia.li@sunnybrook.ca)
Notes	Planned sample size: 26 ClinicalTrials.gov Identifier: NCT02038816 Study ID Number: CICL670ACA02T Registry entry last updated on 15/01/2014