Hugh Young RienhoffVip ViprakasitLay TayPaul HarmatzElliott VichinskyDeborah ChirnomasJanet L. KwiatkowskiAmy TapperWilliam KramerJohn B. PorterEllis J. NeufeldFerroKin BioSciences, Inc.Mahidol UniversityInstitute of Medical and Veterinary Science AustraliaUCSF Benioff Children's Hospital OaklandChildren's Hospital BostonThe Children's Hospital of PhiladelphiaUCL2018-05-032018-05-032011-04-01Haematologica. Vol.96, No.4 (2011), 521-52515928721039060782-s2.0-79953853812https://repository.li.mahidol.ac.th/handle/20.500.14594/12583Background: There is still a clinical need for a well-tolerated and safe iron chelator for the treatment of trans-fusional iron overload. We describe the pharmacokinetic properties and safety data after 7 days of dosing of FBS0701, a novel oral, once-daily iron chelator. Design and Methods: This phase 1b dose-escalation study to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of FBS0701, a novel oral iron chelator for the treatment of transfusional iron overload, was conducted in 16 adult patients with iron overloaded consequent to transfusions. FBS0701 was given daily for 7 days at doses up to 32 mg/kg and was well tolerated at all dose levels. Results: Pharmacokinetics showed dose-proportionality. The maxium plasma concentration (Cmax) was reached within 60-90 minutes of dosing and the drug was rapidly distributed at the predicted therapeutic doses. The plasma elimination half-life (t1/2) was approximately 19 hours. There were no serious adverse events associated with the drug. Conclusions: On the basis of these safety and pharmacokinetic data, FBS0701 warrants further clinical evaluation in patients with transfusional iron overload. © 2011 Ferrata Storti Foundation.Mahidol UniversityMedicineArticleSCOPUS10.3324/haematol.2010.034405