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Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy

This study has suspended participant recruitment.
(No funding and moved to expanded access)
Sponsor:
ClinicalTrials.gov Identifier:
NCT00004418
First Posted: October 19, 1999
Last Update Posted: September 18, 2014
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Gerald Raymond, Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
  Purpose

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 Intervention Phase
Adrenoleukodystrophy Drug: glyceryl trierucate Drug: glyceryl trioleate Phase 2 Phase 3

Study Type: Interventional
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

Resource links provided by NLM:


Further study details as provided by Gerald Raymond, Hugo W. Moser Research Institute at Kennedy Krieger, Inc.:

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 ]

Estimated Enrollment: 30
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)
Arms Assigned Interventions
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

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.

  Eligibility

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 Submitted: October 18, 1999
First Posted: October 19, 1999
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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