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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




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