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

NO_LONGER_AVAILABLE

Conditions studied: X-linked Adrenoleukodystrophy

In brief

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.

Key facts

Study ID

NCT02233257

Run by

University of Minnesota

Last updated by the study team

2018-01-23

Who can join

Age: 2 and older, up to 18. Sex: male. Healthy volunteers: not accepted.

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

Where it is running

• University of Minnesota Medical Center — Minneapolis, Minnesota, United States

Full record on ClinicalTrials.gov

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