Study of Glyceryl Trierucate and Glyceryl Trioleate (Lorenzo's Oil) Therapy in Male Children With Adrenoleukodystrophy - Tabular View

Tracking Information

First Received Date ^ICMJE

October 18, 1999

Last Updated Date

June 15, 2009

Start Date ^ICMJE

April 1998

Primary Completion Date

Current Primary Outcome Measures ^ICMJE

Original Primary Outcome Measures ^ICMJE

Change History

Complete list of historical versions of study NCT00004418 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures ^ICMJE

Original Secondary Outcome Measures ^ICMJE

Descriptive Information

Brief Title ^ICMJE

Study of Glyceryl Trierucate and Glyceryl Trioleate (Lorenzo's Oil) Therapy in Male Children With Adrenoleukodystrophy

Official Title ^ICMJE

Brief Summary

OBJECTIVES: I. Evaluate the clinical efficacy of combination glyceryl trierucate and glyceryl trioleate (Lorenzo's Oil) therapy in boys with X-linked adrenoleukodystrophy.

II. Compare the frequency and severity of neurological disability of study patients with untreated historical controls.

Detailed Description

PROTOCOL OUTLINE: This is an open label study. Patients must follow dietary instructions as provided by the investigator. Patients receive 2-4 tablespoons of a mixture of glyceryl trierucate and glyceryl trioleate oil once daily. Patients complete a neuropsychological scoring scale questionnaire to measure neurological disability. Patients undergo physical examinations including magnetic resonance imaging and magnetic resonance spectroscopy of the head.

Patients are followed monthly for 6 months, then every 3 months until death.

Study Phase

Study Type ^ICMJE

Interventional

Study Design ^ICMJE

Treatment, Open Label

Condition ^ICMJE

Adrenoleukodystrophy

Intervention ^ICMJE

Drug: glyceryl trierucate
Drug: glyceryl trioleate

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

Recruiting

Estimated Enrollment ^ICMJE

Completion Date

Primary Completion Date

Eligibility Criteria ^ICMJE

Biochemically proven asymptomatic X-linked adrenoleukodystrophy
Platelet count in normal range

Gender

Male

Ages

18 Months to 6 Years

Accepts Healthy Volunteers

Contacts ^ICMJE

Location Countries ^ICMJE

United States

Administrative Information

NCT ID ^ICMJE

NCT00004418

Responsible Party

Study ID Numbers ^ICMJE

199/13312, KKI-88-11-22-01, KKI-FDR000685

Study Sponsor ^ICMJE

FDA Office of Orphan Products Development

Collaborators ^ICMJE

Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

Investigators ^ICMJE

Study Chair:

Hugo W Moser

Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

Information Provided By

FDA Office of Orphan Products Development

Verification Date

September 2003

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