A Phase III Trial of Lorenzo's Oil in Adrenomyeloneuropathy

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Disease Information

Descriptive Information
Brief Title † A Phase III Trial of Lorenzo's Oil in Adrenomyeloneuropathy
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 Description 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.
Study Phase Phase 3
Study Type † Interventional
Study Design † Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Treatment
Primary Outcome Measure † Clinical Progression of the disorder
Secondary Outcome Measure † 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.
Condition † Adrenomyeloneuropathy Adrenoleukodystrophy
Intervention † DrugLorenzo's oil
Study Arms / Comparison Groups
Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information
Recruitment Status † Drug
Estimated Enrollment † 240
Start Date † March 2005
Completion Date October 2007
Primary Completion Date October 2007
Eligibility Criteria † Inclusion Criteria: - Males or females age 18 years or older in whom the diagnosis of X-ALD has been confirmed by VLCFA assay and/or mutation analysis. - Clinical evidence of spinal cord involvement with EDSS score between 1 and 6.5. Patients with an EDSS score of 6.5 are severely affected but have retained the capacity to walk 20 meters with the aid of a walker, crutch, or two canes. - Either a normal brain MRI, or a type 3 pattern of MRI abnormality in which the abnormality is considered to represent the centripetal extension of the distal axonopathy. - Adrenal function assessed by measurement of plasma ACTH and appropriate steroid replacement if adrenal insufficiency is present. Exclusion Criteria: - Kurtzke EDSS score of >6.5. - Cognitive or behavioral abnormalities that impair capacity to give informed consent or carry out procedures that form part of the protocol. - Current use, or use within 3 months, of Lorenzo's oil or other therapies that may alter the course of X-ALD. Bone marrow transplant will be a permanent exclusion criterion. - Contraindications for MRI procedure such as subjects with paramagnetic materials in the body, such as aneurysm clips, pacemakers, intraocular metal or cochlear implants. - Subjects who are pregnant. - Allergies to peppermint - Presence of non-specific conditions that may interfere with clinical assessment or participation in the protocol.
Gender Both
Ages 18 Years - N/A
Accepts Healthy Volunteers No
Contacts ††
Location Countries † United States
Administrative Information
NCT ID † NCT00545597
Organization ID R01 HD39276
Secondary IDs †† R01FD003030
Responsible Party Sponsor
Study Sponsor † Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Collaborators †† Food and Drug Administration (FDA)
Investigators † Principal Investigator: Gerald V Raymond, M.D., Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Information Provided By
Verification Date October 2007
First Received Date † October 16, 2007
Last Updated Date March 1, 2012
† Required WHO trial registration data element.
†† WHO trial registration data element that is required only if it exists.
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