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Study of Bile Acids in Patients With Peroxisomal Disorders
This study is not yet open for patient recruitment.
Sponsored by: | FDA Office of Orphan Products Development Children's Hospital Medical Center - Cincinnati
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Information provided by: | FDA Office of Orphan Products Development |
Purpose
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 | Treatment or Intervention |
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Infantile Refsum's Disease Zellweger Syndrome Bifunctional Enzyme Deficiency Adrenoleukodystrophy |
Drug: chenodeoxycholic acid Drug: cholic acid Drug: ursodiol |
MedlinePlus related topics: Birth Defects; Genetic Brain Disorders; Genetic Disorders; Kidney Diseases; Leukodystrophies; Liver Diseases; Metabolic Disorders
Study Type: Interventional
Study Design: Treatment
Expected Total Enrollment: 25
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.
Eligibility
Ages Eligible for Study: up to 5 Years, Genders Eligible for Study: Both
Criteria
Biochemically proven peroxisomal disorder, including:
Location Information
More Information
U.S. National Library of Medicine, Contact NLM Customer Service | ||||||||||||||
National Institutes of Health, Department of Health & Human Services | ||||||||||||||
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