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

Effects of Diet and Medication in Patients With Cerebrotendinous Xanthomatosis (CTX)

Status

UNKNOWN

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT00004346

Enrollment

Registered

1999-10-19

Start date

1996-01-31

Completion date

Unknown

Last updated

2005-06-24

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Cerebrotendinous Xanthomatosis

Keywords

cerebrotendinous xanthomatosis, inborn errors of metabolism, rare disease

Brief summary

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.

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.

Interventions

DRUGchenodeoxycholic acid

DRUGlovastatin

Study design

Primary purpose

TREATMENT

Eligibility

Sex/Gender

ALL

Age

0 Years to No maximum

Healthy volunteers

Inclusion criteria

PROTOCOL ENTRY CRITERIA: Cerebrotendinous xanthomatosis

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026