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NCT00004442 Clinical Trial

Study of Bile Acids in Patients With Peroxisomal Disorders

Adrenoleukodystrophy Clinical Trial

Clinical Trial Details — Status: Terminated

Administrative data
NCT number NCT00004442
Other study ID # 199/13442
Secondary ID CHMC-C-FDR000995
Status Terminated
Phase N/A
First received October 18, 1999
Last updated March 24, 2015
Est. completion date April 1999

Study information
Verified date November 2000
Source FDA Office of Orphan Products Development
Contact n/a
Is FDA regulated No
Health authority United States: Federal Government
Study type Interventional

Clinical Trial 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.

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

Recruitment information / eligibility
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

Study Design

Primary Purpose: Treatment

Related Conditions & MeSH terms

Intervention

Drug:
• chenodeoxycholic acid

• cholic acid

• ursodiol

Locations
Country Name City State
n/a

Sponsors (2)
Lead Sponsor Collaborator
University of Cincinnati Children's Hospital Medical Center, Cincinnati
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