Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy

Study Overview
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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.

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.

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

Conditions
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Adrenoleukodystrophy

Intervention / Treatment
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* Drug: glyceryl trierucate/glyceryl trioleate


Participation Criteria
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Eligibility Criteria
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Inclusion criteria. Boys between the age of 18 months and 8 years of age Biochemically proven asymptomatic X-linked adrenoleukodystrophy determined by elevation of very long chain fatty acids or DNA analysis. Platelet count in normal range Exclusion criteria. Abnormal MRI consistent with childhood cerebral disease Boys who have undergone bone marrow transplantation Other medical condition which in the opinion of the investigator prevents evaluation or treatment

Ages Eligible for Study
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Minimum Age: 18 Months
Maximum Age: 8 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Glyceryl trierucate/glyceryl trioleate<br>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 | Drug: glyceryl trierucate/glyceryl trioleate<br>* Administration of glyceryl trierucate/glyceryl trioleate<br>* Other names: Lorenzo's oil;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change From Baseline in Very Long Chain Fatty Acids (VLCFA) Blood Levels | we will assess the change in very long-chain fatty acids as determined by reduction of blood level baseline C26:0 level. | Baseline, an average of 10 years, up to age 13 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants With T2 MRI Abnormality | We used MRI to determine the number of participants with T2 MRI abnormality indicating childhood Adrenoleukodystrophy (ALD). | 10 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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adrenoleukodystrophy, inborn errors of metabolism, rare disease, sphingolipidoses