Safety and Pharmacokinetic Study of Escalating Multiple Doses of an Iron Chelator in Patients With Iron Overload
Status:
Completed
Trial end date:
1969-12-31
Target enrollment:
Participant gender:
Summary
The purpose of this research study is to study the safety of increasing doses of FBS0701, and
to see how quickly the study medication is absorbed and how quickly it disappears from the
bloodstream. FBS0701 is a new, oral iron chelator - a medication taken by mouth that
increases the body's elimination of iron. Iron chelators are used in patients who develop
iron overload from their transfusions. Four increasing doses of FBS0701 will be tested during
this study. The study will start with the lowest dose given to 4 patients (3 mg/kg/day. The
next group of 4 patients will receive the next high dose (8mg/kg/day only after the results
of the first 4 patients are examined and it is determined safe to continue. Participating
patients will take the study medication for 7 days and be followed for 28 days after their
last dose to determine if they have any reactions to the study medication - therefore a total
of 35 days on study. Patients will need to give up to 17 blood samples over the screening
period and first 15 days of the study (a total of about 9 tablespoons). Patients will not
need to stay overnight in the clinic but will need to visit the clinic 10 times for screening
and on-study visits over the 35 days. Patients currently taking an iron chelator will need to
stop that treatment for up to 22 days (up to 5 days before they start the study and for 15
days during the study). The results of this study will be helpful in determining the safety
of the drug and the best doses of FBS0701 to be used in the next study which will assess the
effectiveness of this new iron chelator.