Efficacy of To Be Marketed (TBM) Cholic Acid Capsules Used to Treat Children With Inborn Errors of Bile Acid Synthesis
Status:
Completed
Trial end date:
2010-08-01
Target enrollment:
Participant gender:
Summary
This is a study in a small population of children who have inborn errors of bile acid
synthesis who are currently taking established doses of the currently used cholic acid
capsules prepared at the Cincinnati Children's Hospital Pharmacy. The study is designed to
compare the efficacy of these currently used capsules with the efficacy of the same treatment
provided in a cholic acid capsule that is made by a company that will be marketed after FDA
approval.
At baseline, patients receive established doses of cholic acid capsules prepared at the
Cincinnati Children's Hospital Medical Center Pharmacy. During the study, patients receive
the same treatment provided in the to-be-marketed (TBM) cholic acid capsule. Hence, patients
serve as their own controls, with baseline values presenting the reference value (CCHMC
cholic acid capsule) and values after 30 days treatment presenting the value for the
investigational treatment (TBM cholic acid capsule).