Study of Bile Acids in Patients With Peroxisomal Disorders - Tabular View

Tracking Information

First Received Date ^ICMJE

October 18, 1999

Last Updated Date

June 23, 2005

Start Date ^ICMJE

Primary Completion Date

Current Primary Outcome Measures ^ICMJE

Original Primary Outcome Measures ^ICMJE

Change History

Complete list of historical versions of study NCT00004442 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures ^ICMJE

Original Secondary Outcome Measures ^ICMJE

Descriptive Information

Brief Title ^ICMJE

Study of Bile Acids in Patients With Peroxisomal Disorders

Official Title ^ICMJE

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.

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.

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.

Study Phase

Study Type ^ICMJE

Interventional

Study Design ^ICMJE

Treatment

Condition ^ICMJE

Infantile Refsum's Disease
Zellweger Syndrome
Bifunctional Enzyme Deficiency
Adrenoleukodystrophy

Intervention ^ICMJE

Drug: chenodeoxycholic acid
Drug: cholic acid
Drug: ursodiol

Study Arms / Comparison Groups

Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status ^ICMJE

Not yet recruiting

Enrollment ^ICMJE

Completion Date

Primary Completion Date

Eligibility Criteria ^ICMJE

Biochemically proven peroxisomal disorder, including:

Zellweger syndrome
Pseudo-Zellweger syndrome
Neonatal adrenoleukodystrophy
Bifunctional enzyme deficiency
Infantile Refsum's disease

Gender

Both

Ages

up to 5 Years

Accepts Healthy Volunteers

Contacts ^ICMJE

Contact: Kenneth Setchell

513-636-4548

Location Countries ^ICMJE

Administrative Information

NCT ID ^ICMJE

NCT00004442

Responsible Party

Study ID Numbers ^ICMJE

199/13442, CHMC-C-FDR000995

Study Sponsor ^ICMJE

FDA Office of Orphan Products Development

Collaborators ^ICMJE

Children's Hospital Medical Center, Cincinnati

Investigators ^ICMJE

Study Chair:

Kenneth Setchell

Children's Hospital Medical Center, Cincinnati

Information Provided By

FDA Office of Orphan Products Development

Verification Date

November 2000

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