Phase II Study of Cholesterol- and Cholestanol-Free Diet, Lovastatin, and Chenodeoxycholic Acid for Cerebrotendinous Xanthomatosis

This study is currently recruiting participants.

Verified by National Center for Research Resources (NCRR), December 2003

First Received: October 18, 1999 Last Updated: June 23, 2005 History of Changes

Sponsor:	National Center for Research Resources (NCRR)
Collaborator:	Oregon Health and Science University
Information provided by:	National Center for Research Resources (NCRR)
ClinicalTrials.gov Identifier:	NCT00004346

Purpose

OBJECTIVES: I. Assess the biosynthesis of cholesterol and cholestanol, and measure the turnover of individual sterols and bile acids in patients with cerebrotendinous xanthomatosis before and after a cholesterol- and cholestanol-free diet.

II. Assess the biosynthesis of cholesterol and cholestanol, and measure the turnover of individual sterols and bile acids in these patients before and after lovastatin and chenodeoxycholic acid.

Condition	Intervention	Phase
Cerebrotendinous Xanthomatosis	Drug: chenodeoxycholic acid Drug: lovastatin	Phase II

Study Type:	Interventional
Study Design:	Treatment
Official Title:	Effects of Diet and Medication in Patients With Cerebrotendinous Xanthomatosis (CTX)

Resource links provided by NLM:

Genetics Home Reference related topics: cerebrotendinous xanthomatosis Chanarin-Dorfman syndrome cholesteryl ester storage disease Farber lipogranulomatosis primary carnitine deficiency

MedlinePlus related topics: Cholesterol Diets

Drug Information available for: Lovastatin Cholestanol Chenodeoxycholic acid

U.S. FDA Resources

Further study details as provided by National Center for Research Resources (NCRR):

Estimated Enrollment:	5
Study Start Date:	January 1996

Detailed Description:

PROTOCOL OUTLINE: Following a 2-week baseline assessment, patients participate in a feeding study for up to 3 weeks. The diet is free of cholesterol and cholestanol.

For the next 4 weeks, patients return to their typical diet and are medicated with daily lovastatin and chenodeoxycholic acid.

The feeding study is repeated for an additional 3 weeks, with the patient taking either lovastatin or chenodeoxycholic acid.

Eligibility

Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

PROTOCOL ENTRY CRITERIA: Cerebrotendinous xanthomatosis

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00004346

Locations

United States, Oregon
Oregon Health and Science University	Recruiting
Portland, Oregon, United States, 97239-3098
Contact: Sonja Connor 503-494-7775 connors@ohsu.edu

Sponsors and Collaborators

National Center for Research Resources (NCRR)

Oregon Health and Science University

Investigators

Study Chair:

William Connor

Oregon Health and Science University

More Information

No publications provided

Study ID Numbers:	NCRR-M01RR00334-0067, OHSU-4008
Study First Received:	October 18, 1999
Last Updated:	June 23, 2005
ClinicalTrials.gov Identifier:	NCT00004346 History of Changes
Health Authority:	United States: Federal Government

Keywords provided by National Center for Research Resources (NCRR):

cerebrotendinous xanthomatosis
inborn errors of metabolism
rare disease

Additional relevant MeSH terms:

Antimetabolites
Lipid Metabolism, Inborn Errors
Metabolic Diseases
Molecular Mechanisms of Pharmacological Action
Antilipemic Agents
Gastrointestinal Agents
Enzyme Inhibitors
Cathartics
Anticholesteremic Agents
Xanthomatosis, Cerebrotendinous

Hydroxymethylglutaryl-CoA Reductase Inhibitors
Pharmacologic Actions
Xanthomatosis
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Chenodeoxycholic Acid
Therapeutic Uses
Lovastatin
Lipid Metabolism Disorders

ClinicalTrials.gov processed this record on November 22, 2009