Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy

Study Results

Results available

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2

Total Enrollment

126 participants

Study Classification

INTERVENTIONAL

Study Start Date

1998-04-30

Study Completion Date

2014-12-31

Brief Summary

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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.

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Detailed Description

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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 they reach the age of 13 years or death.

Conditions

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Adrenoleukodystrophy

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Glyceryl trierucate/glyceryl trioleate

Treatment of all enrolled participants. Dosage form is a liquid oil taken orally. Dose is to provide 20% of daily calories. Daily for duration of trial

Group Type EXPERIMENTAL

glyceryl trierucate/glyceryl trioleate

Intervention Type DRUG

Administration of glyceryl trierucate/glyceryl trioleate

Interventions

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glyceryl trierucate/glyceryl trioleate

Administration of glyceryl trierucate/glyceryl trioleate

Intervention Type DRUG

Other Intervention Names

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Lorenzo's oil

Eligibility Criteria

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Inclusion Criteria

1. Boys between the age of 18 months and 8 years of age
2. Biochemically proven asymptomatic X-linked adrenoleukodystrophy determined by elevation of very long chain fatty acids or DNA analysis.
3. Platelet count in normal range

Exclusion Criteria

1. Abnormal MRI consistent with childhood cerebral disease
2. Boys who have undergone bone marrow transplantation
3. Other medical condition which in the opinion of the investigator prevents evaluation or treatment

Minimum Eligible Age

18 Months

Maximum Eligible Age

8 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No