Effect of Glycerol Trierucate on Clinical Course of 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.
|Adrenoleukodystrophy||Drug: glyceryl trierucate Drug: glyceryl trioleate||Phase 2 Phase 3|
|Study Design:||Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
|Official Title:||Study of Glyceryl Trierucate and Glyceryl Trioleate (Lorenzo's Oil) Therapy in Male Children With Adrenoleukodystrophy|
- neurological disability [ Time Frame: 6 months post intervention, then every 3 months until age 13 years ]
- magnetic resonance imaging of the head [ Time Frame: yearly ]
- very long chain fatty acids (VLCFA) [ Time Frame: every month during first year, then every 1-3 months ]
|Study Start Date:||April 1998|
|Estimated Study Completion Date:||November 2014|
|Estimated Primary Completion Date:||November 2014 (Final data collection date for primary outcome measure)|
Treatment with GTO/GTE orally; 30-60 ml daily for study period
Drug: glyceryl trierucate
Glyceryl trierucate is an oil to reduce very long chain fatty acids
Other Names:Drug: glyceryl trioleate
Patients receive 2-4 tablespoons of a mixture of glyceryl trierucate and glyceryl trioleate oil once daily.
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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00004418
|United States, Maryland|
|Kennedy Krieger Institute|
|Baltimore, Maryland, United States, 21205|
|Johns Hopkins Hospital|
|Baltimore, Maryland, United States, 21287-6681|
|Principal Investigator:||Gerald V Raymond, M.D.||Hugo W. Moser Research Institute at Kennedy Krieger, Inc.|