Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy

This study is currently recruiting participants. (see Contacts and Locations)
Verified February 2013 by Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Sponsor:
Information provided by (Responsible Party):
Gerald Raymond, Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
ClinicalTrials.gov Identifier:
NCT00004418
First received: October 18, 1999
Last updated: February 5, 2013
Last verified: February 2013
  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 Phase
Adrenoleukodystrophy
Drug: glyceryl trierucate
Drug: glyceryl trioleate
Phase 2
Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Study of Glyceryl Trierucate and Glyceryl Trioleate (Lorenzo's Oil) Therapy in Male Children With Adrenoleukodystrophy

Resource links provided by NLM:


Further study details as provided by Hugo W. Moser Research Institute at Kennedy Krieger, Inc.:

Primary Outcome Measures:
  • neurological disability [ Time Frame: 6 months post intervention, then every 3 months until age 13 years ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • magnetic resonance imaging of the head [ Time Frame: yearly ] [ Designated as safety issue: No ]

Other Outcome Measures:
  • very long chain fatty acids (VLCFA) [ Time Frame: every month during first year, then every 1-3 months ] [ Designated as safety issue: No ]

Estimated Enrollment: 30
Study Start Date: April 1998
Estimated Primary Completion Date: January 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
glyceryl trierucate /glyceryl trioleate mixture
glyceryl trierucate and glyceryl trioleate mixture, 2-4 tablespoons once daily
Drug: glyceryl trierucate Drug: glyceryl trioleate
Patients receive 2-4 tablespoons of a mixture of glyceryl trierucate and glyceryl trioleate oil once daily.
Other Name: Lorenzo's Oil

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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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 Active, not recruiting
Baltimore, Maryland, United States, 21205
Sponsors and Collaborators
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Investigators
Principal Investigator: Gerald V Raymond, M.D. Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
  More Information

No publications provided

Responsible Party: Gerald Raymond, Associate Professor of Neurology, Johns Hopkins University School of Medicine, Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
ClinicalTrials.gov Identifier: NCT00004418     History of Changes
Other Study ID Numbers: 199/13312, Nutricia-Loma Linda, KKI-FDR000685
Study First Received: October 18, 1999
Last Updated: February 5, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by Hugo W. Moser Research Institute at Kennedy Krieger, Inc.:
adrenoleukodystrophy
inborn errors of metabolism
rare disease
sphingolipidoses

Additional relevant MeSH terms:
Adrenal Insufficiency
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
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Metabolism, Inborn Errors
Metabolic Diseases
Adrenal Gland Diseases
Endocrine System Diseases

ClinicalTrials.gov processed this record on September 14, 2014