Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy

Descriptive Information
Brief Title † Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy
Official Title † Study of Glyceryl Trierucate and Glyceryl Trioleate (Lorenzo's Oil) Therapy in Male Children With Adrenoleukodystrophy
Brief Summary 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.
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.
Study Phase 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
Primary Outcome Measure † neurological disability
Secondary Outcome Measure † magnetic resonance imaging of the head
Condition † Adrenoleukodystrophy
Intervention † Drugglyceryl trierucate
Study Arms / Comparison Groups GTO/GTE treatment Treatment with GTO/GTE orally; 30-60 ml daily for study period
Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information
Recruitment Status † Drug
Estimated Enrollment † 30
Start Date † April 1998
Completion Date November 2014
Primary Completion Date November 2014
Eligibility Criteria † - Biochemically proven asymptomatic X-linked adrenoleukodystrophy - Platelet count in normal range
Gender Male
Ages 18 Months - 6 Years
Accepts Healthy Volunteers No
Contacts ††
Location Countries † United States
Administrative Information
NCT ID † NCT00004418
Organization ID 199/13312
Secondary IDs †† Nutricia-Loma Linda, KKI-FDR000685
Responsible Party Principal Investigator
Study Sponsor † Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Collaborators ††
Investigators † Principal Investigator: Gerald V Raymond, M.D., Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Information Provided By
Verification Date September 2014
First Received Date † October 18, 1999
Last Updated Date September 17, 2014
† Required WHO trial registration data element.
†† WHO trial registration data element that is required only if it exists.
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