Study of Bile Acids in Patients With Peroxisomal Disorders
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.
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
Completion date provided represents the completion date of the grant per OOPD records
Infantile Refsum's Disease
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.