Study of Glyceryl Trierucate and Glyceryl Trioleate (Lorenzo's Oil) Therapy in Male Children With Adrenoleukodystrophy

The recruitment status of this study is unknown because the information has not been verified recently.

Verified September 2003 by FDA Office of Orphan Products Development. Recruitment status was Recruiting

First Received on October 18, 1999. Last Updated on June 15, 2009 History of Changes

Sponsor:	FDA Office of Orphan Products Development
Collaborator:	Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Information provided by:	FDA Office of Orphan Products Development
ClinicalTrials.gov Identifier:	NCT00004418

Purpose

OBJECTIVES: I. Evaluate the clinical efficacy of combination glyceryl trierucate and glyceryl trioleate (Lorenzo's Oil) therapy in boys with X-linked adrenoleukodystrophy.

II. Compare the frequency and severity of neurological disability of study patients with untreated historical controls.

Condition	Intervention
Adrenoleukodystrophy	Drug: glyceryl trierucate Drug: glyceryl trioleate

Study Type:	Interventional
Study Design:	Masking: Open Label Primary Purpose: Treatment

Resource links provided by NLM:

Genetics Home Reference related topics: beta-ketothiolase deficiency leukoencephalopathy with vanishing white matter MECP2 duplication syndrome megalencephalic leukoencephalopathy with subcortical cysts PPM-X syndrome succinic semialdehyde dehydrogenase deficiency X-linked adrenoleukodystrophy Zellweger spectrum Help Me Understand Genetics

Drug Information available for: Triolein

U.S. FDA Resources

Further study details as provided by FDA Office of Orphan Products Development:

Estimated Enrollment:	30
Study Start Date:	April 1998

Detailed Description:

PROTOCOL OUTLINE: This is an open label study. Patients must follow dietary instructions as provided by the investigator. Patients receive 2-4 tablespoons of a mixture of glyceryl trierucate and glyceryl trioleate oil once daily. Patients complete a neuropsychological scoring scale questionnaire to measure neurological disability. Patients undergo physical examinations including magnetic resonance imaging and magnetic resonance spectroscopy of the head.

Patients are followed monthly for 6 months, then every 3 months until death.

Eligibility

Ages Eligible for Study:	18 Months to 6 Years
Genders Eligible for Study:	Male
Accepts Healthy Volunteers:	No

Criteria

Biochemically proven asymptomatic X-linked adrenoleukodystrophy
Platelet count in normal range

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00004418

Locations

United States, Maryland
Johns Hopkins Hospital	Recruiting
Baltimore, Maryland, United States, 21287-6681
Contact: Kim Hollandsworth 800-873-3377 ext 2772
Kennedy Krieger Institute	Recruiting
Baltimore, Maryland, United States, 21205
Contact: Kim Hollandsworth 443-923-2772

Sponsors and Collaborators

FDA Office of Orphan Products Development

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

Investigators

Study Chair:

Hugo W Moser

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

More Information

No publications provided

ClinicalTrials.gov Identifier:	NCT00004418 History of Changes
Other Study ID Numbers:	199/13312, KKI-88-11-22-01, KKI-FDR000685
Study First Received:	October 18, 1999
Last Updated:	June 15, 2009
Health Authority:	United States: Federal Government

Keywords provided by FDA Office of Orphan Products Development:

adrenoleukodystrophy
inborn errors of metabolism
rare disease
sphingolipidoses

Additional relevant MeSH terms:

Adrenoleukodystrophy
Hereditary Central Nervous System Demyelinating Diseases
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Peroxisomal Disorders
Leukoencephalopathies
Demyelinating Diseases
Mental Retardation, X-Linked

Mental Retardation
Neurobehavioral Manifestations
Neurologic Manifestations
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Metabolism, Inborn Errors
Metabolic Diseases
Adrenal Insufficiency
Adrenal Gland Diseases
Endocrine System Diseases

ClinicalTrials.gov processed this record on February 12, 2012