Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid
Status:
Completed
Trial end date:
2009-12-01
Target enrollment:
Participant gender:
Summary
OBJECTIVES:
I. To Evaluate the therapeutic efficacy of cholic acid during provision of compassionate
treatment to patients with identified inborn errors of bile acid synthesis and metabolism
II. To assess the safety and tolerability of cholic acid