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

NCT02233257 | Unknown

• 1 other identifier: 1401M47561
• Study Type: expanded_access
• Target: N/A
• Locations: 1 country
• Sites: 1

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.

Conditions

X-linked Adrenoleukodystrophy

Keywords

Adrenoleukodystrophy; Lorenzo's oil; erucic acid

Interventions

Lorenzo's Oil DRUG

Oral use of Lorenzo's oil to lower very long chain fatty acids

Also known as: Glyceryl trierucate

Eligibility Criteria

• Age: 18 Months - 18 Years
• Sex: male
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

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

Sponsors & Collaborators

• University of Minnesota: lead

Study Sites (1)

University of Minnesota Medical Center

Minneapolis, Minnesota, 55455, United States

Location

MeSH Terms

Conditions

Adrenoleukodystrophy

Interventions

Lorenzo's oil; trierucate

Condition Hierarchy (Ancestors)

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; X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Heredodegenerative Disorders, Nervous System; Metabolism, Inborn Errors; Peroxisomal Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Adrenal Insufficiency; Adrenal Gland Diseases; Endocrine System Diseases

Study Officials

• Gerald V Raymond, MD

  University of Minnesota

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: expanded access
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 5, 2014
• First Posted: September 8, 2014
• Last Updated: January 23, 2018

Record last verified: 2018-01

Locations

US (1)