Cholic Acid
Sponsors
Amsterdam UMC Stichting, University of Cincinnati, University of Texas Southwestern Medical Center, Mirum Pharmaceuticals, Inc., University of Nebraska
Conditions
AdrenoleukodystrophyBifunctional Enzyme DeficiencyBile Acid Synthesis DefectBile acid synthesis defectsBile acid synthesis defects (BASD), 3ß-hydroxy-?5-C27-steroid oxidoreductase, ?4-3-oxosteroid-5ß-reductase, cholesterol 7a-hydroxylase (CYP7A1), and a-methylacyl-CoA racemase (AMACR), Zellweger spectrum disorderHepatic SteatosisInborn Errors of Bile Acid SynthesisInfantile Refsum's Disease
Phase 1
Phase 2
Phase 3
Efficacy of To Be Marketed (TBM) Cholic Acid Capsules Used to Treat Children With Inborn Errors of Bile Acid Synthesis
CompletedNCT01115582
Start: 2010-04-30End: 2010-08-31Updated: 2023-10-03
Open Label, Continuation Study of Cholic Acid in Subjects With Inborn Errors of Bile Acid Synthesis
CompletedNCT01438411
Start: 2010-01-31End: 2016-07-31Updated: 2023-10-03
Long-term safety study of personalized cholic acid treatment in patients with bile acid synthesis defects
CompletedCTIS2024-518652-23-00
End: 2025-03-17Target: 8Updated: 2024-11-25