Study of Bile Acids in Patients With Peroxisomal Disorders

• Status: Terminated
• Trial end date: 1999-04-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: N/A
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: University of Cincinnati
• Collaborator: Children's Hospital Medical Center, Cincinnati
• Treatments: Chenodeoxycholic Acid; Cholic Acids; Ursodeoxycholic Acid

Criteria

Biochemically proven peroxisomal disorder, including:

• Zellweger syndrome

• Pseudo-Zellweger syndrome

• Neonatal adrenoleukodystrophy

• Bifunctional enzyme deficiency

• Infantile Refsum's disease