- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00545597
A Phase III Trial of Lorenzo's Oil in Adrenomyeloneuropathy
Study Overview
Status
Conditions
Intervention / Treatment
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 Type
Enrollment (Anticipated)
Phase
- Phase 3
Contacts and Locations
Study Locations
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Maryland
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Baltimore, Maryland, United States, 21205
- Kennedy Krieger Institute
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
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.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: TREATMENT
- Allocation: RANDOMIZED
- Interventional Model: PARALLEL
- Masking: DOUBLE
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Clinical Progression of the disorder
Time Frame: 48 months
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48 months
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Secondary Outcome Measures
Outcome Measure |
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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.
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Collaborators and Investigators
Publications and helpful links
Study record dates
Study Major Dates
Study Start
Primary Completion (ACTUAL)
Study Completion (ACTUAL)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (ESTIMATE)
Study Record Updates
Last Update Posted (ESTIMATE)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Metabolic Diseases
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Demyelinating Diseases
- Neurologic Manifestations
- Neurobehavioral Manifestations
- Endocrine System Diseases
- Genetic Diseases, Inborn
- Genetic Diseases, X-Linked
- Metabolism, Inborn Errors
- Mental Retardation, X-Linked
- Intellectual Disability
- Heredodegenerative Disorders, Nervous System
- Brain Diseases, Metabolic
- Brain Diseases, Metabolic, Inborn
- Leukoencephalopathies
- Adrenal Gland Diseases
- Hereditary Central Nervous System Demyelinating Diseases
- Peroxisomal Disorders
- Adrenal Insufficiency
- Adrenoleukodystrophy
Other Study ID Numbers
- R01HD039276 (NIH)
- R01FD003030 (FDA)
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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