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Results for “Inborn Error of Bile Acid Metabolism”

7 trials

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Showing 7 of 7 results

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
Large-scale testing (Phase 3)Study completedNCT00007020
What this trial is testing

Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid

Who this might be right for
Infantile Refsum's DiseaseZellweger SyndromeAdrenoleukodystrophy+2 more
Mirum Pharmaceuticals, Inc. 85
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
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 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 applicableLooking for participantsNCT01793168
What this trial is testing

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

Who this might be right for
Rare DisordersUndiagnosed DisordersDisorders of Unknown Prevalence+340 more
Sanford Health 20,000
Not applicableEnded earlyNCT00004442
What this trial is testing

Study of Bile Acids in Patients With Peroxisomal Disorders

Who this might be right for
Infantile Refsum's DiseaseZellweger SyndromeBifunctional Enzyme Deficiency+1 more
University of Cincinnati 25