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 they reach the age of 13 years or death.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
126
Administration of glyceryl trierucate/glyceryl trioleate
Kennedy Krieger Institute
Baltimore, Maryland, United States
Johns Hopkins Hospital
Baltimore, Maryland, United States
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.
Time frame: Baseline, an average of 10 years, up to age 13
Number of Participants With T2 MRI Abnormality
We used MRI to determine the number of participants with T2 MRI abnormality indicating childhood Adrenoleukodystrophy (ALD).
Time frame: 10 years
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