PMID- 22251482
OWN - NLM
STAT- MEDLINE
DCOM- 20120619
LR  - 20231120
IS  - 1528-0020 (Electronic)
IS  - 0006-4971 (Print)
IS  - 0006-4971 (Linking)
VI  - 119
IP  - 14
DP  - 2012 Apr 5
TI  - A phase 2 study of the safety, tolerability, and pharmacodynamics of FBS0701, a 
      novel oral iron chelator, in transfusional iron overload.
PG  - 3263-8
LID - 10.1182/blood-2011-10-386268 [doi]
AB  - 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 baseline ALT. The 24 week mean change in liver iron 
      concentration (DeltaLIC) at 14.5 mg/kg/d was +3.1 mg/g (dw); 29% achieved a decrease 
      in LIC. Mean DeltaLIC at 29 mg/kg/d was -0.3 mg/g (dw); 44% achieved a decrease in 
      LIC (P < .03 for DeltaLIC between doses). The safety and tolerability profile at 
      therapeutic doses compare favorably to other oral chelators.
FAU - Neufeld, Ellis J
AU  - Neufeld EJ
AD  - Children's Hospital, Harvard University, Boston, MA, USA.
FAU - Galanello, Renzo
AU  - Galanello R
FAU - Viprakasit, Vip
AU  - Viprakasit V
FAU - Aydinok, Yesim
AU  - Aydinok Y
FAU - Piga, Antonio
AU  - Piga A
FAU - Harmatz, Paul
AU  - Harmatz P
FAU - Forni, Gian Luca
AU  - Forni GL
FAU - Shah, Farrukh T
AU  - Shah FT
FAU - Grace, Rachael F
AU  - Grace RF
FAU - Porter, John B
AU  - Porter JB
FAU - Wood, John C
AU  - Wood JC
FAU - Peppe, Jennifer
AU  - Peppe J
FAU - Jones, Amber
AU  - Jones A
FAU - Rienhoff, Hugh Young Jr
AU  - Rienhoff HY Jr
LA  - eng
SI  - ClinicalTrials.gov/NCT01186419
GR  - UL1 RR024131/RR/NCRR NIH HHS/United States
GR  - UL1 RR025758/RR/NCRR NIH HHS/United States
GR  - UL1RR025758/RR/NCRR NIH HHS/United States
GR  - UL1RR024131/RR/NCRR NIH HHS/United States
PT  - Clinical Trial, Phase II
PT  - Journal Article
PT  - Multicenter Study
PT  - Randomized Controlled Trial
PT  - Research Support, N.I.H., Extramural
DEP - 20120117
PL  - United States
TA  - Blood
JT  - Blood
JID - 7603509
RN  - 0 
      (4,5-dihydro-2-(2-hydroxy-3-(3,6,9-trioxadecyloxy)phenyl)-4-methyl-4-thiazolecarboxylic 
      acid)
RN  - 0 (Ethyl Ethers)
RN  - 0 (Iron Chelating Agents)
RN  - 0 (Thiazoles)
RN  - AYI8EX34EU (Creatinine)
RN  - E1UOL152H7 (Iron)
SB  - IM
CIN - Blood. 2012 Apr 5;119(14):3191-2. PMID: 22493210
MH  - Adolescent
MH  - Adult
MH  - Creatinine/metabolism
MH  - Dose-Response Relationship, Drug
MH  - Ethyl Ethers/adverse effects/pharmacology/*therapeutic use
MH  - Female
MH  - Hemoglobinopathies/complications/therapy
MH  - Humans
MH  - Iron/analysis/metabolism
MH  - Iron Chelating Agents/adverse effects/pharmacology/*therapeutic use
MH  - Iron Overload/diagnosis/*drug therapy/*etiology
MH  - Liver/metabolism
MH  - Male
MH  - Middle Aged
MH  - Thiazoles/adverse effects/pharmacology/*therapeutic use
MH  - *Transfusion Reaction
MH  - Treatment Outcome
MH  - Young Adult
PMC - PMC3321852
EDAT- 2012/01/19 06:00
MHDA- 2012/06/20 06:00
PMCR- 2012/04/05
CRDT- 2012/01/19 06:00
PHST- 2012/01/19 06:00 [entrez]
PHST- 2012/01/19 06:00 [pubmed]
PHST- 2012/06/20 06:00 [medline]
PHST- 2012/04/05 00:00 [pmc-release]
AID - S0006-4971(20)48025-5 [pii]
AID - 2011/386268 [pii]
AID - 10.1182/blood-2011-10-386268 [doi]
PST - ppublish
SO  - Blood. 2012 Apr 5;119(14):3263-8. doi: 10.1182/blood-2011-10-386268. Epub 2012 
      Jan 17.