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|Title:||A phase 2 study of the safety, tolerability, and pharmacodynamics of FBS0701, a novel oral iron chelator, in transfusional iron overload|
|Authors:||Ellis J. Neufeld|
Gian Luca Forni
Farrukh T. Shah
Rachael F. Grace
John B. Porter
John C. Wood
Hugh Young Rienhoff
Universita degli Studi di Cagliari
Ege University Medical School
Scienze Cliniche e Biologiche
UCSF Benioff Children's Hospital Oakland
E.O. Ospedali Galliera
Children's Hospital Los Angeles
FerroKin BioSciences, Inc.
|Keywords:||Biochemistry, Genetics and Molecular Biology;Immunology and Microbiology;Medicine|
|Citation:||Blood. Vol.119, No.14 (2012), 3263-3268|
|Abstract:||This was a 24-week, multicenter phase- 2 study designed to assess safety, tolerability, and pharmacodynamics of FBS0701, a novel oral chelator, in adults with transfusional iron overload. Fifty-one patients, stratified by transfusional iron intake, were randomized to FBS0701 at either 14.5 or 29 mg/kg/d (16 and 32 mg/kg/d salt form). FBS0701 was generally well tolerated at both doses. Forty-nine patients (96%) completed the study. There were no drug-related serious adverse events. No adverse events (AEs) showed dose-dependency in frequency or severity. Treatment-related nausea, vomiting, abdominal pain, and diarrhea were each noted in < 5% of patients. Mean serum creatinine did not change significantly from Baseline or between dose groups. Transaminases wer increased in 8 (16%), three of whom acquired HCV on-study from a single blood bank while five had an abnormal baselineALT. The 24 week mean change in liver iron concentration (ΔLIC) at 14.5 mg/kg/d was +3.1 mg/g (dw); 29% achieved a decrease in LIC. Mean ΔLIC at 29 mg/kg/d was -0.3 mg/g (dw); 44% achieved a decrease in LIC (P < .03 for ΔLIC between doses). The safety and tolerability profile at therapeutic doses compare favorably to other oral chelators. This trialwasregistered atwww.clinicaltrials. gov as NCT01186419. © 2012 by The American Society of Hematology.|
|Appears in Collections:||Scopus 2011-2015|
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