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

Sponsor:

Information provided by (Responsible Party):

Gerald Raymond, Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

Study Description

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.

Condition or disease	Intervention/treatment	Phase
Adrenoleukodystrophy	Drug: glyceryl trierucate Drug: glyceryl trioleate	Phase 2 Phase 3

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 death.

Study Design


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

Resource links provided by the National Library of Medicine

Genetics Home Reference related topics: RNAse T2-deficient leukoencephalopathy X-linked adrenoleukodystrophy Leukoencephalopathy with vanishing white matter Megalencephalic leukoencephalopathy with subcortical cysts

Genetic and Rare Diseases Information Center resources: X-linked Adrenoleukodystrophy Adrenomyeloneuropathy Sphingolipidosis Peroxisomal Biogenesis Disorders Hypoadrenalism

U.S. FDA Resources

Arms and Interventions

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: Lorenzo's oil GTE Drug: glyceryl trioleate Patients receive 2-4 tablespoons of a mixture of glyceryl trierucate and glyceryl trioleate oil once daily. Other Names: Lorenzo's Oil GTO

Outcome Measures

Primary Outcome Measures :

neurological disability [ Time Frame: 6 months post intervention, then every 3 months until age 13 years ]

Secondary Outcome Measures :

magnetic resonance imaging of the head [ Time Frame: yearly ]

Other Outcome Measures:

very long chain fatty acids (VLCFA) [ Time Frame: every month during first year, then every 1-3 months ]

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:	18 Months to 6 Years (Child)
Sexes Eligible for Study:	Male
Accepts Healthy Volunteers:	No

Criteria

Biochemically proven asymptomatic X-linked adrenoleukodystrophy
Platelet count in normal range

Contacts and Locations

Information from the National Library of Medicine

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

Locations


United States, Maryland
Kennedy Krieger Institute
Baltimore, Maryland, United States, 21205
Johns Hopkins Hospital
Baltimore, Maryland, United States, 21287-6681

Sponsors and Collaborators

Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

Investigators


Principal Investigator:	Gerald V Raymond, M.D.	Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

More Information


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

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