Adrenoleukodystrophy Clinical Trial
Official title:
A Phase III Trial of Lorenzo's Oil in Adrenomyeloneuropathy
Study of the use of Lorenzo's oil in adults with adrenomyeloneuropathy, the adult form of Lorenzo's oil.
This is a double-masked placebo controlled study of glyceryl trioleate-glyceryl trierucate
(Lorenzo's Oil (LO)) therapy in adrenomyeloneuropathy (AMN), the adult form of X-linked
adrenoleukodystrophy (X-ALD). AMN is a slowly progressive distal axonopathy that involves
the long tracts of the spinal cord and differs from the rapidly progressive inflammatory
cerebral forms that most commonly affect boys and adolescents. All forms of X-ALD are
associated with the abnormal accumulation of very long chain fatty acids (VLCFA) in plasma
and tissues. The oral administration of LO normalizes plasma VLCFA levels within 4 weeks.
While previous therapeutic trials of LO therapy in patients with the cerebral forms of X-ALD
have been disappointing, recent studies suggest that it is beneficial in two types of X-ALD:
1) as a preventive of neurological involvement in asymptomatic boys; and 2) in AMN, where it
appears to slow the rate of progression. None of the previous studies have been controlled,
and we are now conducting the first placebo-controlled trial.
The 4-year study will include 120 men with AMN who do not have evidence of cerebral
involvement, and 120 women who are heterozygous for X-ALD and have an AMN-like syndrome. The
rate of progression will be compared in the LO and placebo groups using the Kurtzke EDSS
score as the primary outcome and a variety of secondary outcomes.
Adrenomyeloneuropathy affects about 1 in 40,000 men and 1 in 30,000 women. It is a
progressive disorder that leads to inability to walk and other severe deficits. This study
will provide definitive information whether Lorenzo's Oil therapy can slow progression.
;
Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Treatment
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