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Study of Bile Acids in Patients With Peroxisomal Disorders

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Disease Information

Descriptive Information
Brief Title † Study of Bile Acids in Patients With Peroxisomal Disorders
Official Title †
Brief Summary OBJECTIVES: I. Determine the effectiveness of oral bile acid therapy with cholic acid, chenodeoxycholic acid, and ursodeoxycholic acid in patients with peroxisomal disorders involving impaired primary bile acid synthesis. II. Determine whether suppression of synthesis of atypical bile acids and enrichment of bile acid pool with this regimen is effective in treating this patient population and improving quality of life.
Detailed Description PROTOCOL OUTLINE: Patients receive oral cholic acid and oral chenodeoxycholic acid on day 1. On day 4, patients receive oral cholic and ursodeoxycholic acids. Patients are assessed at 3 and 6 months for liver function response, neurologic status, and nutritional status. Patients receive treatment until disease progression or unacceptable toxic effects are observed. Completion date provided represents the completion date of the grant per OOPD records
Study Phase N/A
Study Type † Interventional
Study Design † Primary Purpose: Treatment
Primary Outcome Measure †
Secondary Outcome Measure †
Condition † Infantile Refsum's Disease Zellweger Syndrome Bifunctional Enzyme Deficiency Adrenoleukodystrophy
Intervention † Drugchenodeoxycholic acid
Study Arms / Comparison Groups
Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information
Recruitment Status † Drug
Estimated Enrollment † 25
Start Date †
Completion Date April 1999
Primary Completion Date
Eligibility Criteria † Biochemically proven peroxisomal disorder, including: - Zellweger syndrome - Pseudo-Zellweger syndrome - Neonatal adrenoleukodystrophy - Bifunctional enzyme deficiency - Infantile Refsum's disease
Gender Both
Ages N/A - 5 Years
Accepts Healthy Volunteers No
Contacts ††
Location Countries †
Administrative Information
NCT ID † NCT00004442
Organization ID 199/13442
Secondary IDs †† CHMC-C-FDR000995
Responsible Party
Study Sponsor † Children's Hospital Research Foundation University of Cincinnati
Collaborators †† Children's Hospital Medical Center, Cincinnati
Investigators † Study Chair: Kenneth Setchell, Children's Hospital Medical Center, Cincinnati
Information Provided By
Verification Date November 2000
First Received Date † October 18, 1999
Last Updated Date March 24, 2015
† Required WHO trial registration data element.
†† WHO trial registration data element that is required only if it exists.
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