CompletedPhase 3

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

United States5 participantsStarted 2006-02

Plain-language summary

The purpose of this research study is to determine the way (mechanisms) by which your defect in bile acid handling (metabolism) causes your liver disease or abnormality in absorption of vitamins and the effect of an investigational bile acid therapy (glycocholic acid) on your vitamin absorption and your liver disease. An investigational therapy is one that not approved by the United States Food and Drug Administration (FDA) and is being provided to you under an Investigational New Drug application from the FDA.

Who can participate

Age range1 Week – 85 Years

SexALL

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Inclusion criteria

✓. Confirmation of a diagnosis of an inborn error of bile acid synthesis/conjugation based upon urine analysis by FAB-MS.
✓. Any age
✓. Participant must be willing and able to comply with study assessments and procedures.
✓. The participant and/or parent/legal guardian must have signed the written informed consent document prior to study start.

What they're measuring

Conjugated Cholic Acid (GCA) for the Treatment of Inborn Errors in Bile Acid Synthesis Involving Side-chain Conjugation.

Timeframe: Up to 10 years

Evaluation of Levels of Atypical Bile Acid Metabolites After GCA Treatment Compared

Timeframe: Average of 6 months, average 12 months, and average of after year 1 to 10 years

Trial details

NCT IDNCT01589523

SponsorChildren's Hospital Medical Center, Cincinnati

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2019-01-22

Results submitted2022-03-15

View on ClinicalTrials.gov More Bile Acid Synthesis Defect trials