Clinical Trial: Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy

Study Status: Suspended
Recruit Status: Suspended
Study Type: Interventional

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

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 Summary:

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.

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

Current Primary Outcome: neurological disability [ Time Frame: 6 months post intervention, then every 3 months until age 13 years ]

Original Primary Outcome:

Current Secondary Outcome: magnetic resonance imaging of the head [ Time Frame: yearly ]

Original Secondary Outcome:

Information By: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

Dates:
Date Received: October 18, 1999
Date Started: April 1998
Date Completion: November 2014
Last Updated: September 17, 2014
Last Verified: September 2014