Study of Bile Acids in Patients With Peroxisomal Disorders
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|ClinicalTrials.gov Identifier: NCT00004442|
Recruitment Status : Terminated
First Posted : October 19, 1999
Last Update Posted : March 25, 2015
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.
|Condition or disease||Intervention/treatment||Phase|
|Infantile Refsum's Disease Zellweger Syndrome Bifunctional Enzyme Deficiency Adrenoleukodystrophy||Drug: chenodeoxycholic acid Drug: cholic acid Drug: ursodiol||Not Applicable|
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 Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||25 participants|
|Study Completion Date :||April 1999|
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00004442
|Study Chair:||Kenneth Setchell||Children's Hospital Medical Center, Cincinnati|