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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
Renzo Galanello
Vip Viprakasit
Yesim Aydinok
Antonio Piga
Paul Harmatz
Gian Luca Forni
Farrukh T. Shah
Rachael F. Grace
John B. Porter
John C. Wood
Jennifer Peppe
Amber Jones
Hugh Young Rienhoff
Harvard University
Universita degli Studi di Cagliari
Mahidol University
Ege University Medical School
Scienze Cliniche e Biologiche
UCSF Benioff Children's Hospital Oakland
E.O. Ospedali Galliera
Whittington Hospital
Children's Hospital Los Angeles
FerroKin BioSciences, Inc.
Keywords: Biochemistry, Genetics and Molecular Biology;Immunology and Microbiology;Medicine
Issue Date: 5-Apr-2012
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.
ISSN: 15280020
Appears in Collections:Scopus 2011-2015

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