Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy



Status:Suspended
Conditions:Endocrine
Therapuetic Areas:Endocrinology
Healthy:No
Age Range:Any - 6
Updated:10/14/2017
Start Date:April 1998
End Date:November 2014

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Trial Summary
Trial Overview
Inclusion Criteria

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

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.

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.

- Biochemically proven asymptomatic X-linked adrenoleukodystrophy

- Platelet count in normal range
We found this trial at
2
sites
Johns Hopkins Hospital
1800 Orleans St.
Baltimore, Maryland 21287
410-955-5000
Johns Hopkins Hospital Patients are the focus of everything we do at The Johns Hopkins...
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Kennedy Krieger Institute
707 North Broadway
Baltimore, Maryland 21205
443-923-9200
Kennedy Krieger Institute While not officially part of Johns Hopkins Medicine, Kennedy Krieger Institute is...
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