Study of Bile Acids in Patients With Peroxisomal Disorders

The recruitment status of this study is unknown because the information has not been verified recently.
Verified November 2000 by FDA Office of Orphan Products Development.
Recruitment status was  Not yet recruiting
Sponsor:
Collaborator:
Children's Hospital Medical Center, Cincinnati
Information provided by:
FDA Office of Orphan Products Development
ClinicalTrials.gov Identifier:
NCT00004442
First received: October 18, 1999
Last updated: June 23, 2005
Last verified: November 2000

October 18, 1999
June 23, 2005
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Complete list of historical versions of study NCT00004442 on ClinicalTrials.gov Archive Site
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Study of Bile Acids in Patients With Peroxisomal Disorders
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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.

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.

Interventional
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Primary Purpose: Treatment
  • Infantile Refsum's Disease
  • Zellweger Syndrome
  • Bifunctional Enzyme Deficiency
  • Adrenoleukodystrophy
  • Drug: chenodeoxycholic acid
  • Drug: cholic acid
  • Drug: ursodiol
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*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Not yet recruiting
25
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Biochemically proven peroxisomal disorder, including:

  • Zellweger syndrome
  • Pseudo-Zellweger syndrome
  • Neonatal adrenoleukodystrophy
  • Bifunctional enzyme deficiency
  • Infantile Refsum's disease
Both
up to 5 Years
No
Contact: Kenneth Setchell 513-636-4548
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NCT00004442
199/13442, CHMC-C-FDR000995
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FDA Office of Orphan Products Development
Children's Hospital Medical Center, Cincinnati
Study Chair: Kenneth Setchell Children's Hospital Medical Center, Cincinnati
FDA Office of Orphan Products Development
November 2000

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP