Sponsor study identifier
interventional
53
1 country
1
Study Start
January 2010
Date type not reportedThis date has not been verified as actual or estimated.
Primary Completion
July 2016
Actual (reported)Study Completion
July 2016
Actual (reported)The primary purpose of the study is to evaluate the therapeutic efficacy and safety of cholic acid in subjects with identified inborn errors of bile acid synthesis.
Registry-reported information. Elapsed time and enrollment totals alone do not measure study progress or recruitment pace.
July 2016
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.
January 2010
Date type not reportedThis date has not been verified as actual or estimated.
September 20, 2011
Actual (reported)September 22, 2011
Actual (reported)July 2016
Actual (reported)July 2016
Actual (reported)August 21, 2020
Actual (reported)Change in Atypical Urinary Bile Acid Excretion by FAB-MS (Fast-Atom-Bombardment Ionization-Mass Spectrometry)
The level of atypical urinary bile acid secretion was scored using a scale of: 0, normal; 1, slight; 2, significant; or 3, marked. A Cochran-Mantel-Haenszel (CMH) test with modified ridit scoring was used to compare the difference between the score at baseline and the worst post-baseline score during treatment with cholic acid in this single-arm trial.
At baseline, then every 12 months for an average of 3.5 years
Evaluation of Serum Transaminases: ALT
At baseline, then every 12 months for an average of 3.5 years
Evaluation of Serum Transaminases: AST
At baseline, then every 12 months for an average of 3.5 years
Clinical Laboratory Results: Bilirubin
At baseline, then every 12 months for an average of 3.5 years
Clinical Laboratory Results: Gamma Glutamyl Transferase (GGT)
At baseline, then every 12 months for an average of 3.5 years
Clinical Laboratory Results: Alkaline Phosphatase
At baseline, then every 12 months for an average of 3.5 years
Active drug
10-15 mg/kg body weight/day supplied in 50 or 250 mg Cholic Acid 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
Heubi JE, Setchell KDR. Open-label Phase 3 Continuation Study of Cholic Acid in Patients With Inborn Errors of Bile Acid Synthesis. J Pediatr Gastroenterol Nutr. 2020 Apr;70(4):423-429. doi: 10.1097/MPG.0000000000002618.
James E Heubi, MD
Children's Hospital Medical Center, Cincinnati
Kenneth Setchell, PhD
Children's Hospital Medical Center, Cincinnati
53 participants (actual enrollment reported by the registry).
First Submitted
September 20, 2011
First Posted
September 22, 2011
Study Start
January 2010
Date type not reportedThis date has not been verified as actual or estimated.
Smith-Lemli-Opitz Syndrome and Cholic Acid
Contact the study team to confirm eligibility.
Primary Completion
July 2016
Actual (reported)Study Completion
July 2016
Actual (reported)Last Updated
October 3, 2023
Results First Posted
August 21, 2020
Record last verified: 2023-09
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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