Sponsor study identifier
interventional
5
1 country
1
Study Start
February 2006
Date type not reportedThis date has not been verified as actual or estimated.
Primary Completion
January 22, 2019
Actual (reported)Study Completion
January 22, 2019
Actual (reported)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.
Registry-reported information. Elapsed time and enrollment totals alone do not measure study progress or recruitment pace.
January 22, 2019
Actual (reported)Click on a node to explore related trials.
Some registry-reported names shown below are not yet linked in this graph.
Dates and qualifiers reported by the registry. Estimated dates do not confirm that a milestone has occurred.
February 2006
Date type not reportedThis date has not been verified as actual or estimated.
April 3, 2012
Actual (reported)May 2, 2012
Actual (reported)January 22, 2019
Actual (reported)January 22, 2019
Actual (reported)May 12, 2022
Actual (reported)Conjugated Cholic Acid (GCA) for the Treatment of Inborn Errors in Bile Acid Synthesis Involving Side-chain Conjugation.
This is the number of participants with bile acid amidation defects treated with oral glycocholic acid (15 milligrams/kilograms (mg/kg) of body weight/day (bw/day))
Up to 10 years
Evaluation of Levels of Atypical Bile Acid Metabolites After GCA Treatment Compared
Semi-quantitative descriptive evaluation of the levels of atypical bile acids in urine measured by mass spectrometry (FAB MS) based on a scale of 0 = absent or traces levels, 1 = low levels, 2 = moderate levels, 3 = high levels using the signal/noise ratio and intensity of ions. Atypical bile acids evaluated included m/z 407 (unconjugated cholic acid), m/z 471 (dihydroxy-choleanoic-sulfate) and m/z 583 (trihydroxy-choleanoic glucuronide).
Average of 6 months, average 12 months, and average of after year 1 to 10 years
Changes in Liver Function Tests of ALT From Baseline to Post-treatment
Comparison between baseline and post-treatment (average of available timepoints after year 1 through year 10)
Change in Liver Function Test: AST From Baseline to Post-treatment
Comparison between baseline and post-treatment (average of available timepoints after year 1 through year 10)
Change in Vitamin D, 25-OH Measure From Baseline to Post-treatment
Pre-treatment and post treatment (average of available timepoints after year 1 through year 10)
An open label, single arm, non-randomized, non-comparative, treatment study of Glycocholic Acid in the treatment of defects of bile acid metabolism.
10-15mg/kg body weight/day taken orally. Supplied as either liquid or 50mg capsules.
Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
Setchell KD, Heubi JE, Shah S, Lavine JE, Suskind D, Al-Edreesi M, Potter C, Russell DW, O'Connell NC, Wolfe B, Jha P, Zhang W, Bove KE, Knisely AS, Hofmann AF, Rosenthal P, Bull LN. Genetic defects in bile acid conjugation cause fat-soluble vitamin deficiency. Gastroenterology. 2013 May;144(5):945-955.e6; quiz e14-5. doi: 10.1053/j.gastro.2013.02.004. Epub 2013 Feb 13.
Heubi JE, Setchell KD, Jha P, Buckley D, Zhang W, Rosenthal P, Potter C, Horslen S, Suskind D. Treatment of bile acid amidation defects with glycocholic acid. Hepatology. 2015 Jan;61(1):268-74. doi: 10.1002/hep.27401. Epub 2014 Dec 23.
Kenneth D. Setchell, Ph.D.
Children's Hospital Medical Center, Cincinnati
James E. Heubi, M.D.
Children's Hospital Medical Center, Cincinnati
5 participants (actual enrollment reported by the registry).
First Submitted
April 3, 2012
First Posted
May 2, 2012
Study Start
February 2006
Date type not reportedThis date has not been verified as actual or estimated.
Contact the study team to confirm eligibility.
Primary Completion
January 22, 2019
Actual (reported)Study Completion
January 22, 2019
Actual (reported)Last Updated
June 8, 2022
Results First Posted
May 12, 2022
Record last verified: 2022-05
Download the complete ClinicalTrials.gov record imported by Syfrah, including fields not shown in this page. The registry may contain a newer revision.
Source: ClinicalTrials.gov.
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