Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy
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|ClinicalTrials.gov Identifier: NCT00004418|
Recruitment Status : Suspended (No funding and moved to expanded access)
First Posted : October 19, 1999
Last Update Posted : September 18, 2014
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.
|Condition or disease||Intervention/treatment||Phase|
|Adrenoleukodystrophy||Drug: glyceryl trierucate Drug: glyceryl trioleate||Phase 2 Phase 3|
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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||30 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Study of Glyceryl Trierucate and Glyceryl Trioleate (Lorenzo's Oil) Therapy in Male Children With Adrenoleukodystrophy|
|Study Start Date :||April 1998|
|Estimated Primary Completion Date :||November 2014|
|Estimated Study Completion Date :||November 2014|
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
Drug: glyceryl trioleate
Patients receive 2-4 tablespoons of a mixture of glyceryl trierucate and glyceryl trioleate oil once daily.
- 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 ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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.|