A Phase III Trial of Lorenzo's Oil in Adrenomyeloneuropathy

NCT00545597 | Terminated Phase 3 DMC

• 2 other identifiers: R01HD039276R01FD003030
• Study Type: interventional
• Target: 240
• Locations: 1 country
• Sites: 1

Brief Summary

Study of the use of Lorenzo's oil in adults with adrenomyeloneuropathy, the adult form of Lorenzo's oil.

Conditions

Adrenomyeloneuropathy; Adrenoleukodystrophy

Keywords

adrenoleukodystrophy; adrenomyeloneuropathy

Outcome Measures

Primary Outcomes (1)

• Clinical Progression of the disorder

  48 months

Secondary Outcomes (1)

• 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.

Interventions

Lorenzo's oil DRUG

4:1 glyceryl trioleate-glyceryl trierucate (Lorenzo's oil)given orally, daily as approximately 10% of nutritional calories with supplementation.

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD): lead
• Food and Drug Administration (FDA): collaborator

Study Sites (1)

Kennedy Krieger Institute

Baltimore, Maryland, 21205, United States

Location

Related Links

• Adrenomyeloneuropathy (AMN) Research Study
• United Leukodystrophy Foundation
• Myelin Project

MeSH Terms

Conditions

Adrenoleukodystrophy

Interventions

Lorenzo's oil

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Hereditary Central Nervous System Demyelinating Diseases; Leukoencephalopathies; Demyelinating Diseases; X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Heredodegenerative Disorders, Nervous System; Metabolism, Inborn Errors; Peroxisomal Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Adrenal Insufficiency; Adrenal Gland Diseases; Endocrine System Diseases

Study Officials

• Gerald V Raymond, M.D.

  Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 16, 2007
• First Posted: October 17, 2007
• Study Start: March 1, 2005
• Primary Completion: October 1, 2007
• Study Completion: October 1, 2007
• Last Updated: March 2, 2012

Record last verified: 2007-10

Locations

US (1)