Not applicableWithdrawnNCT00018694What this trial is testingCholestanol in HumansWho this might be right forCerebrotendinous Xanthomatosis US Department of Veterans Affairs
Large-scale testing (Phase 3)Study completedNCT01115582What this trial is testingEfficacy of To Be Marketed (TBM) Cholic Acid Capsules Used to Treat Children With Inborn Errors of Bile Acid SynthesisWho this might be right forInborn Errors of Bile Acid Synthesis Mirum Pharmaceuticals, Inc. 16
Large-scale testing (Phase 3)Study completedNCT01589523What this trial is testingGlycoCholic Acid Treatment for Patients With Inborn Errors in Bile Acid SynthesisWho this might be right forBile Acid Synthesis DefectInborn Error of Bile Acid MetabolismInborn Error of Bile Acid Conjugation Children's Hospital Medical Center, Cincinnati 5
Not applicableActive Not RecruitingNCT03655223What this trial is testingEarly Check: Expanded Screening in NewbornsWho this might be right forSpinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more RTI International 30,000
Large-scale testing (Phase 3)Study completedNCT01438411What this trial is testingOpen Label, Continuation Study of Cholic Acid in Subjects With Inborn Errors of Bile Acid SynthesisWho this might be right forBile Acid Synthesis Defect Mirum Pharmaceuticals, Inc. 53