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Glycocholic Acid for Inborn Errors of Bile Acid Synthesis
NCT01589523Phase 3
Key facts
- Status
- Completed
- Start
- Feb 2006
- Primary completion
- Jan 2019
- Countries
- 1
- Sponsor
- Cincinnati Children's Hospital Medical Center
- Modality
- Small Molecules
- Drugs
- glycocholate
- Conditions
- inborn errors of bile acid synthesis
01
Overview
Phase 3, open-label, single-arm trial of glycocholate in inborn errors of bile acid synthesis, sponsored by Cincinnati Children's Hospital Medical Center. Primary endpoints: Conjugated Cholic Acid (GCA) for the Treatment of Inborn Errors in Bile Acid Synthesis Involving Side-chain Conjugation up to 10 years and Evaluation of Levels of Atypical Bile Acid Metabolites After GCA Treatment Compared. Started Feb 2006; status: completed.
02
Design
1 arm- Study design
- Non-randomizedOpen-label
GlycoCholic Acid
Oral Glycocholic Acid treatment
03
Who it enrols
Patients of any age with a confirmed diagnosis of an inborn error of bile acid synthesis/conjugation based on urine analysis by FAB-MS.
04
Endpoints
Primary
Conjugated Cholic Acid (GCA) for the Treatment of Inborn Errors in Bile Acid Synthesis Involving Side-chain Conjugation
Evaluation of Levels of Atypical Bile Acid Metabolites After GCA Treatment Compared
Secondary
Changes in Liver Function Tests of ALT From Baseline to Post-treatment
Change in Liver Function Test: AST From Baseline to Post-treatment
Change in Vitamin D, 25-OH Measure From Baseline to Post-treatment
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