Clinical Trial: Expanded Access for Lorenzo's Oil (GTO/GTE) in Adrenoleukodystrophy
Study Status: Available
Recruit Status: Available
Study Type: Expanded Access
Official Title: Expanded Access for Lorenzo's Oil (GTO/GTE) in Adrenoleukodystrophy
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.
Detailed Summary:
Expanded Access for Lorenzo's Oil in X-linked Adrenoleukodystrophy to Intermediate Size Group
Introduction/Rationale: A diet that consists predominantly of long-chain monounsaturated fatty acids has been demonstrated to reduce the levels of very long chain fatty acids (VLCA) in individuals with X-linked adrenoleukodystrophy (ALD). VLCFA are the primary biochemical abnormality in this genetic disorder and have been implicated in the pathogenesis of the cerebral disease. It has been demonstrated in an open study that reduction of VLCFA for more than a year is protective for childhood cerebral disease.
The purpose of this expanded access proposal is to transition an open label study presently ongoing in boys between the ages of 18 months and 13 years of age to an expanded access study. The reason for this transition is lack of funding for the investigator to continue to support this as a study. A study requires investigator involvement including the assistance of research associates to monitor and track outcomes, perform neuropsychological assessments, monitor adverse events, and coordinate periodic evaluations.
A move to expanded access would decentralize the coordination and monitoring of evaluations which are clinically indicated even for participants who are not participating in a study. This role would be undertaken by the treating physician.
Data collected in such an open study could serve as safety material for a later filing.
Study Population:
- Males with X-linked adrenoleukodystrophy; see inclusion/exclusion criteria below.
Procedure:
Sponsor: University of Minnesota - Clinical and Translational Science Institute
Current Primary Outcome:
Original Primary Outcome:
Current Secondary Outcome:
Original Secondary Outcome:
Information By: University of Minnesota - Clinical and Translational Science Institute
Dates:
Date Received: May 5, 2014
Date Started:
Date Completion:
Last Updated: December 8, 2016
Last Verified: December 2016
