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

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

This study is currently recruiting participants.
Verified by FDA Office of Orphan Products Development, September 2003

Sponsors and Collaborators:	FDA Office of Orphan Products Development Kennedy-Krieger Research Institute
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

Genetics Home Reference related topics:

beta-ketothiolase deficiency familial encephalopathy with neuroserpin inclusion bodies L1 syndrome leukoencephalopathy with vanishing white matter megalencephalic leukoencephalopathy with subcortical cysts X-linked adrenoleukodystrophy

MedlinePlus related topics:

Addison's Disease

Drug Information available for:

Triolein

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Open Label

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: Hugo W Moser 410-502-9405
	Kennedy Krieger Institute	Recruiting
	Baltimore, Maryland, United States, 21205
	Contact: Hugo W Moser 410-502-9405

Sponsors and Collaborators

FDA Office of Orphan Products Development

Kennedy-Krieger Research Institute

Investigators


Study Chair:	Hugo W Moser	Kennedy-Krieger Research Institute

More Information

Study ID Numbers:	199/13312, KKI-88-11-22-01, KKI-FDR000685
First Received:	October 18, 1999
Last Updated:	June 23, 2005
ClinicalTrials.gov Identifier:	NCT00004418
Health Authority:	United States: Federal Government

Keywords provided by FDA Office of Orphan Products Development:

adrenoleukodystrophy

inborn errors of metabolism

rare disease

sphingolipidoses

Study placed in the following topic categories:

Sphingolipidoses

Addison's disease

Adrenal Gland Diseases

Demyelinating diseases

Hypoadrenalism

Brain Diseases

Metabolism, Inborn Errors

Heredodegenerative Disorders, Nervous System

Peroxisomal Disorders

Adrenoleukodystrophy

Addison Disease

Genetic Diseases, X-Linked

Brain Diseases, Metabolic, Inborn

Neurobehavioral Manifestations

X-linked adrenoleukodystrophy

Adrenal Insufficiency

Metabolic Diseases

Autoimmune Diseases

Demyelinating Diseases

Rare Diseases

Central Nervous System Diseases

Endocrine System Diseases

Sphingolipidosis

Adrenal gland hypofunction

Mental Retardation

Genetic Diseases, Inborn

Neurologic Manifestations

Endocrinopathy

Metabolic disorder

Brain Diseases, Metabolic

Additional relevant MeSH terms:

Immune System Diseases

Nervous System Diseases

Mental Retardation, X-Linked

Hereditary Central Nervous System Demyelinating Diseases

ClinicalTrials.gov processed this record on November 20, 2008