Trial record 5 of 23 for:    Adrenoleukodystrophy

Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy

This study is currently recruiting participants.
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
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:
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 April 15, 2014