Clinical Trial: A Phase III Trial of Lorenzo's Oil in Adrenomyeloneuropathy
Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional
Official Title: A Phase III Trial of Lorenzo's Oil in Adrenomyeloneuropathy
Brief Summary: Study of the use of Lorenzo's oil in adults with adrenomyeloneuropathy, the adult form of Lorenzo's oil.
Detailed Summary:
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.
Sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Current Primary Outcome: Clinical Progression of the disorder [ Time Frame: 48 months ]
Original Primary Outcome: Same as current
Current Secondary Outcome: Determine the degree to which newly developed methods to assess spinal cord function and structure in adrenomyeloneuropathy, namely quantitative sensorimotor tests and spinal cord imaging, can act as early surrogate markers of disease progression.
Original Secondary Outcome: Same as current
Information By: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Dates:
Date Received: October 16, 2007
Date Started: March 2005
Date Completion:
Last Updated: March 1, 2012
Last Verified: October 2007
