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Expanded Access for Lorenzo's Oil (GTO/GTE) in Adrenoleukodystrophy

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ClinicalTrials.gov Identifier: NCT02233257
Expanded Access Status : No longer available
First Posted : September 8, 2014
Last Update Posted : January 23, 2018
Sponsor:
Information provided by (Responsible Party):
University of Minnesota - Clinical and Translational Science Institute

Brief Summary:

X-linked adrenoleukodystrophy (ALD) is a genetic disorder affecting the brain and adrenal glands. Approximately one third of boys who are at risk will develop cerebral disease. Using a specific diet and the compound Lorenzo's oil, it can be shown that very long chain fatty acids may be lowered in the blood, but it is not known to what degree that may prevent the onset of childhood disease.

This proposal makes available Lorenzo's oil to individuals with ALD, a life threatening disorder for which there are presently no other therapies.


Condition or disease Intervention/treatment
X-linked Adrenoleukodystrophy Drug: Lorenzo's Oil

  Show Detailed Description

Study Type : Expanded Access
Official Title: Expanded Access for Lorenzo's Oil (GTO/GTE) in Adrenoleukodystrophy



Intervention Details:
  • Drug: Lorenzo's Oil
    Oral use of Lorenzo's oil to lower very long chain fatty acids
    Other Name: Glyceryl trierucate

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Months to 18 Years   (Child, Adult)
Sexes Eligible for Study:   Male
Criteria

Inclusion Criteria:

  • Males with X-linked adrenoleukodystrophy as determined by biochemical or genetic determination.
  • Greater than 18 months of age through 18 years of age
  • Normal cerebral MRI at baseline.

Exclusion Criteria:

  • Medical issues which preclude the administration of Lorenzo's oil

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): NCT02233257


Locations
United States, Minnesota
University of Minnesota Medical Center
Minneapolis, Minnesota, United States, 55455
Sponsors and Collaborators
University of Minnesota - Clinical and Translational Science Institute
Investigators
Principal Investigator: Gerald V Raymond, MD University of Minnesota - Clinical and Translational Science Institute

Responsible Party: University of Minnesota - Clinical and Translational Science Institute
ClinicalTrials.gov Identifier: NCT02233257     History of Changes
Other Study ID Numbers: 1401M47561
First Posted: September 8, 2014    Key Record Dates
Last Update Posted: January 23, 2018
Last Verified: January 2018

Keywords provided by University of Minnesota - Clinical and Translational Science Institute:
Adrenoleukodystrophy
Lorenzo's oil
erucic acid

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
Demyelinating Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Metabolism, Inborn Errors
Peroxisomal Disorders
Metabolic Diseases
Adrenal Gland Diseases
Endocrine System Diseases
Leukoencephalopathies
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Adrenal Insufficiency