Study of Bile Acids in Patients With Peroxisomal Disorders

Not Applicable

Terminated

• Conditions: Adrenoleukodystrophy; Bifunctional Enzyme Deficiency; Infantile Refsum's Disease; Zellweger Syndrome

• Registration Number: NCT00004442
• Lead Sponsor: University of Cincinnati

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 Timeline

• Study Completion: 1999-04
• Study First Submitted: 1999-10-18
• Study First Submitted QC: 1999-10-18
• Study First Posted: 1999-10-19
• Status Verified: 2000-11
• Last Update Submitted: 2015-03-24
• Last Update Posted: 2015-03-25

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 25

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: Not specified