Conditions: Adrenoleukodystrophy
Keywords: adrenoleukodystrophy inborn errors of metabolism rare disease sphingolipidoses
Allocation: NA | Intervention model: SINGLE_GROUP
Enrollment: 126
Healthy volunteers: no

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 description:
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.

Arms:
arm 1: 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

Interventions: intervention 1: glyceryl trierucate/glyceryl trioleate
Intervention descriptions: intervention 1: Administration of glyceryl trierucate/glyceryl trioleate

Protocol document: