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

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.