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Results for “Inborn Errors of Bile Acid Synthesis”

5 trials

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Enrolling by Invitation

Showing 5 of 5 results

Not applicableEnrolling By InvitationNCT03655223
What this trial is testing

Early Check: Expanded Screening in Newborns

Who this might be right for
Spinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more
RTI International 30,000
Large-scale testing (Phase 3)Study completedNCT01115582
What this trial is testing

Efficacy of To Be Marketed (TBM) Cholic Acid Capsules Used to Treat Children With Inborn Errors of Bile Acid Synthesis

Who this might be right for
Inborn Errors of Bile Acid Synthesis
Mirum Pharmaceuticals, Inc. 16
Large-scale testing (Phase 3)Study completedNCT01438411
What this trial is testing

Open Label, Continuation Study of Cholic Acid in Subjects With Inborn Errors of Bile Acid Synthesis

Who this might be right for
Bile Acid Synthesis Defect
Mirum Pharmaceuticals, Inc. 53
Not applicableWithdrawnNCT00018694
What this trial is testing

Cholestanol in Humans

Who this might be right for
Cerebrotendinous Xanthomatosis
US Department of Veterans Affairs
Large-scale testing (Phase 3)Study completedNCT01589523
What this trial is testing

GlycoCholic Acid Treatment for Patients With Inborn Errors in Bile Acid Synthesis

Who this might be right for
Bile Acid Synthesis DefectInborn Error of Bile Acid MetabolismInborn Error of Bile Acid Conjugation
Children's Hospital Medical Center, Cincinnati 5