Publication: A phase 2 study of the safety, tolerability, and pharmacodynamics of FBS0701, a novel oral iron chelator, in transfusional iron overload
Issued Date
2012-04-05
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ISSN
15280020
00064971
00064971
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2-s2.0-84859604828
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Mahidol University
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SCOPUS
Bibliographic Citation
Blood. Vol.119, No.14 (2012), 3263-3268
Suggested Citation
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 A phase 2 study of the safety, tolerability, and pharmacodynamics of FBS0701, a novel oral iron chelator, in transfusional iron overload. Blood. Vol.119, No.14 (2012), 3263-3268. doi:10.1182/blood-2011-10-386268 Retrieved from: https://repository.li.mahidol.ac.th/handle/20.500.14594/13761
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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
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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.