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
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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.
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) |
Primary Purpose: | Treatment |
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 |

Arm | Intervention/treatment |
---|---|
GTO/GTE treatment
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.
Other Names:
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- 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 ]

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Ages Eligible for Study: | 18 Months to 6 Years (Child) |
Sexes Eligible for Study: | Male |
Accepts Healthy Volunteers: | No |
- Biochemically proven asymptomatic X-linked adrenoleukodystrophy
- Platelet count in normal range

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. |
Responsible Party: | Gerald Raymond, Associate Professor of Neurology, Johns Hopkins University School of Medicine, Hugo W. Moser Research Institute at Kennedy Krieger, Inc. |
ClinicalTrials.gov Identifier: | NCT00004418 History of Changes |
Other Study ID Numbers: |
199/13312 Nutricia-Loma Linda ( Other Grant/Funding Number: Nutricia-Loma Linda ) KKI-FDR000685 ( Other Identifier: FDA ) |
First Posted: | October 19, 1999 Key Record Dates |
Last Update Posted: | September 18, 2014 |
Last Verified: | September 2014 |
Keywords provided by Gerald Raymond, Hugo W. Moser Research Institute at Kennedy Krieger, Inc.:
adrenoleukodystrophy inborn errors of metabolism rare disease sphingolipidoses |
Additional relevant MeSH terms:
Adrenoleukodystrophy Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic Brain Diseases Central Nervous System Diseases Nervous System Diseases Hereditary Central Nervous System Demyelinating Diseases Leukoencephalopathies Demyelinating Diseases Mental Retardation, X-Linked Intellectual Disability |
Neurobehavioral Manifestations Neurologic Manifestations Genetic Diseases, X-Linked Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Metabolism, Inborn Errors Peroxisomal Disorders Metabolic Diseases Adrenal Insufficiency Adrenal Gland Diseases Endocrine System Diseases |