Study of Bile Acids in Patients With Peroxisomal Disorders

• Sponsor: University of Cincinnati (Other)
• Overall Status: Terminated
• CT.gov ID: NCT00004442
• Collaborator: Children's Hospital Medical Center, Cincinnati (Other)
• Enrollment: 25

Study Details

Study Description

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.

1. 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 or Disease	Intervention/Treatment	Phase
Infantile Refsum's Disease; Zellweger Syndrome; Bifunctional Enzyme Deficiency; Adrenoleukodystrophy	Drug: chenodeoxycholic acid; Drug: cholic acid; Drug: ursodiol	N/A

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 Design

• Study Type: Interventional
• Anticipated Enrollment: 25 participants
• Primary Purpose: Treatment
• Study Completion Date: Apr 1, 1999

Outcome Measures

Primary Outcome Measures

Eligibility Criteria

Criteria

• Ages Eligible for Study: N/A to 5 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Biochemically proven peroxisomal disorder, including:

• Zellweger syndrome

• Pseudo-Zellweger syndrome

• Neonatal adrenoleukodystrophy

• Bifunctional enzyme deficiency

• Infantile Refsum's disease

Contacts and Locations

Locations

No locations specified.

Sponsors and Collaborators

• University of Cincinnati
• Children's Hospital Medical Center, Cincinnati

Investigators

• Study Chair: Kenneth Setchell, Children's Hospital Medical Center, Cincinnati

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• ClinicalTrials.gov Identifier: NCT00004442
• Other Study ID Numbers: 199/13442; CHMC-C-FDR000995
• First Posted: Oct 19, 1999
• Last Update Posted: Mar 25, 2015
• Last Verified: Nov 1, 2000

• Keywords provided by , ,: Zellweger syndrome; adrenoleukodystrophy; bifunctional enzyme deficiency; inborn errors of metabolism; infantile Refsum's disease; peroxisomal disorders; pseudo-Zellweger syndrome; rare disease; sphingolipidoses
• Additional relevant MeSH terms: Zellweger Syndrome; Adrenoleukodystrophy; Refsum Disease; Refsum Disease, Infantile; Peroxisomal Disorders; Syndrome; Ursodeoxycholic Acid; Chenodeoxycholic Acid; Cholic Acids

Study Results

No Results Posted as of Mar 25, 2015