Open Label, Single Center, Nonrandomized Study Comparing Efficacy of To Be Marketed Cholic Acid With That of the Currently Used Formulation of Cholic Acid Capsules Used to Treat Children With Inborn Errors of Bile Acid Synthesis
Sponsor study identifier
interventional
16
1 country
1
Study Start
April 2010
Date type not reportedThis date has not been verified as actual or estimated.
Primary Completion
August 2010
Actual (reported)Study Completion
August 2010
Actual (reported)This is a study in a small population of children who have inborn errors of bile acid synthesis who are currently taking established doses of the currently used cholic acid capsules prepared at the Cincinnati Children's Hospital Pharmacy. The study is designed to compare the efficacy of these currently used capsules with the efficacy of the same treatment provided in a cholic acid capsule that is made by a company that will be marketed after FDA approval. At baseline, patients receive established doses of cholic acid capsules prepared at the Cincinnati Children's Hospital Medical Center Pharmacy. During the study, patients receive the same treatment provided in the to-be-marketed (TBM) cholic acid capsule. Hence, patients serve as their own controls, with baseline values presenting the reference value (CCHMC cholic acid capsule) and values after 30 days treatment presenting the value for the investigational treatment (TBM cholic acid capsule).
Registry-reported information. Elapsed time and enrollment totals alone do not measure study progress or recruitment pace.
August 2010
Actual (reported)Click on a node to explore related trials.
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Dates and qualifiers reported by the registry. Estimated dates do not confirm that a milestone has occurred.
January 19, 2010
Actual (reported)April 2010
Date type not reportedThis date has not been verified as actual or estimated.
May 4, 2010
Actual (reported)August 2010
Actual (reported)August 2010
Actual (reported)December 9, 2016
Actual (reported)Serum Transaminases
Concentration of serum alanine transaminase (ALT) and aspartate transaminase (AST)
At baseline and after 30 days of treatment
Serum and Urine Bile Acids
Concentration of bile acids in serum (S) and urine (U). (abbreviations: chol.=cholenoic; monohydro=monohydroxy; dihydro=monohydro)
At baseline (BL) and after 30 days of treatment (D30)
Adverse Events
Total of 30 days, i.e. from the time point the patients entered into the study up to the end of treatment
Blood Pressure
At baseline and after 30 days of treatment
Physical Examination
At baseline (BL) and after 30 days of treatment (D30)
Total Bilirubin
At baseline and after 30 days of treatment
Manufactured cholic acid capsules
The IUPAC name for cholic acid is 3 alpha,7alpha,12 alpha-trihydroxy-5 beta-cholanoic acid. The international nonproprietary name (INN) is cholic acid. Each patient will be given a box containing a 1 month supply of study drug. Each bottle will contain 90 capsules; each capsule will contain either 50 or 250 mg of manufactured cholic acid depending upon the child's weight. The study drug will be taken orally, in divided doses (as determined by the investigator), for a total daily dose of 10-15 mg/kg body weight. Parents of infants and young children who are unable to swallow the TBM cholic acid capsule will be instructed to sprinkle the contents of the capsule over 1-2 teaspoons of plain applesauce and feed it to the child.
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
Bove KE, Heubi JE, Balistreri WF, Setchell KD. Bile acid synthetic defects and liver disease: a comprehensive review. Pediatr Dev Pathol. 2004 Jul-Aug;7(4):315-34. doi: 10.1007/s10024-002-1201-8. Epub 2004 Jul 15.
Gonzales E, Gerhardt MF, Fabre M, Setchell KD, Davit-Spraul A, Vincent I, Heubi JE, Bernard O, Jacquemin E. Oral cholic acid for hereditary defects of primary bile acid synthesis: a safe and effective long-term therapy. Gastroenterology. 2009 Oct;137(4):1310-1320.e1-3. doi: 10.1053/j.gastro.2009.07.043. Epub 2009 Jul 19.
James E Heubi, MD
Children's Hospital Medical Center, Cincinnati
16 participants (actual enrollment reported by the registry).
First Submitted
January 19, 2010
First Posted
May 4, 2010
Study Start
April 2010
Date type not reportedThis date has not been verified as actual or estimated.
Study of Bile Acids in Patients With Peroxisomal Disorders
Contact the study team to confirm eligibility.
Heubi JE, Setchell KD, Bove KE. Inborn errors of bile acid metabolism. Semin Liver Dis. 2007 Aug;27(3):282-94. doi: 10.1055/s-2007-985073.
Jacquemin E., Gerhardt M, Cresteil D, Fabre M, Taburet AM, Hadchouel M, Trivin F, Stechell KDR and Bernard O. Long-term effects of bile acid therapy in children with defects of primary bile acid synthesis: 3ß-Hydroxy-C27-steroid dehydrogenase/isomerase and Delta4-3-Oxosteroid 5ß-reductase deficiencies. In: van Berge Henegouwen GP, et al. (eds): Falk Symposium No 120: Biology of Bile Acids in Health and Disease. Kluwer Academic Publishers, Dordrecht/Boston/London; 2001:278-282.
Setchell KD, Heubi JE. Defects in bile acid biosynthesis--diagnosis and treatment. J Pediatr Gastroenterol Nutr. 2006 Jul;43 Suppl 1:S17-22. doi: 10.1097/01.mpg.0000226386.79483.7b.
Setchell KDR, et al. A unique case of cerebrotendinous xantomatosis presenting in infancy with cholestatic liver disease further highlights bile acid synthetic defects as an important category of metabolic liver disease. In: van Berge Henegouwen GP et al. (ed): Falk Symposium No. 120: Biology of Bile Acids in Health and Disease. Boston: Kluwer Academic Publishers; 2001.
Primary Completion
August 2010
Actual (reported)Study Completion
August 2010
Actual (reported)Last Updated
October 3, 2023
Results First Posted
December 9, 2016
Record last verified: 2023-09
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Source: ClinicalTrials.gov.
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