Adrenoleukodystrophy Clinical Trial
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.
Status | Terminated |
Enrollment | 25 |
Est. completion date | April 1999 |
Est. primary completion date | |
Accepts healthy volunteers | No |
Gender | Both |
Age group | N/A to 5 Years |
Eligibility |
Biochemically proven peroxisomal disorder, including: - Zellweger syndrome - Pseudo-Zellweger syndrome - Neonatal adrenoleukodystrophy - Bifunctional enzyme deficiency - Infantile Refsum's disease |
Primary Purpose: Treatment
Country | Name | City | State |
---|---|---|---|
n/a |
Lead Sponsor | Collaborator |
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University of Cincinnati | Children's Hospital Medical Center, Cincinnati |
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