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

• Status: No longer available
• Trial end date: 1969-12-31
• Target enrollment: 0
• Participant gender: Male

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.

Details

• Lead Sponsor: University of Minnesota; University of Minnesota - Clinical and Translational Science Institute

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