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Cholestanol in Humans

NCT ID: NCT00018694

Last Updated: 2013-10-14

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

WITHDRAWN

Clinical Phase

NA

Study Classification

INTERVENTIONAL

Study Start Date

1999-10-31

Study Completion Date

2009-12-31

Brief Summary

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The treatment of cerebrotendinous xanthomatosis an in born error of bile acid synthesis with chenodeoxycholic acid. Patients with this disease over produce cholestanol and bile acid precursors because of the block in synthesis. Replacement with chenodeoxycholic acid shut down abnormal pathway and reduces elevated level of cholestanol and improves the clinical syndrome.

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Detailed Description

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Cerebrotendinous xanthomatosis is a recessively inherited in born of bile acid synthesis due to a mutation in sterol 27-hydroxylase (CYP27A1). Patients with this disease suffer from xanthomas located in the brain and tendon, accelerated atherosclerosis progression neurologic disease and cataracts. Plasma cholesterol levels are normal but cholestanol and C-27 bile alcohol that precursor of bile acid synthesis accumulate and are believe are responsible for the atherosclerosis, xanthomas and neurologic disease. Analysis of the bile reveal a severe sufficiency of the primary bile acid chenodeoxycholic acid that can not be produce because of the inherited defect. However, replacement of chenodeoxycholic acid in the enterohepatic pool inhibit abnormal bile acid synthesis and reduces the elevated level of cholestanol and C-27 bile alcohol this therapy halt the neurologic disease and prevents symptomatic atherosclerosis developing.

Conditions

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Cerebrotendinous Xanthomatosis

Study Design

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Allocation Method

NON_RANDOMIZED

Blinding Strategy

NONE

Study Groups

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Arm 1

Group Type OTHER

Chenodeoxycholic Acid

Intervention Type DRUG

Interventions

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Chenodeoxycholic Acid

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

Patients with clinical and biochemical findings of cerebrotendinous xanthomatosis. Elevated levels of serum cholestanol and bile acid precursors.
Minimum Eligible Age

5 Years

Maximum Eligible Age

80 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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US Department of Veterans Affairs

FED

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Gerald Salen

Role: PRINCIPAL_INVESTIGATOR

VA New Jersey Health Care System, East Orange

Locations

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VA New Jersey Health Care System, East Orange

East Orange, New Jersey, United States

Site Status

Countries

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United States

Other Identifiers

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GAST-007-99S

Identifier Type: -

Identifier Source: org_study_id

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