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Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00004418
Recruitment Status : Terminated (No funding, move to expanded access)
First Posted : October 19, 1999
Results First Posted : October 23, 2020
Last Update Posted : October 23, 2020
Information provided by (Responsible Party):
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

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.

Condition or disease Intervention/treatment Phase
Adrenoleukodystrophy Drug: glyceryl trierucate/glyceryl trioleate Phase 2

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 they reach the age of 13 years or death.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 126 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Study of Glyceryl Trierucate and Glyceryl Trioleate (Lorenzo's Oil) Therapy in Male Children With Adrenoleukodystrophy
Study Start Date : April 1998
Actual Primary Completion Date : December 2014
Actual Study Completion Date : December 2014

Arm Intervention/treatment
Experimental: Glyceryl trierucate/glyceryl trioleate
Treatment of all enrolled participants. Dosage form is a liquid oil taken orally. Dose is to provide 20% of daily calories. Daily for duration of trial
Drug: glyceryl trierucate/glyceryl trioleate
Administration of glyceryl trierucate/glyceryl trioleate
Other Name: Lorenzo's oil

Primary Outcome Measures :
  1. Change From Baseline in Very Long Chain Fatty Acids (VLCFA) Blood Levels [ Time Frame: Baseline, an average of 10 years, up to age 13 ]
    we will assess the change in very long-chain fatty acids as determined by reduction of blood level baseline C26:0 level.

Secondary Outcome Measures :
  1. Number of Participants With T2 MRI Abnormality [ Time Frame: 10 years ]
    We used MRI to determine the number of participants with T2 MRI abnormality indicating childhood Adrenoleukodystrophy (ALD).

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Months to 8 Years   (Child)
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
Gender Eligibility Description:   males only
Accepts Healthy Volunteers:   No

Inclusion criteria.

  1. Boys between the age of 18 months and 8 years of age
  2. Biochemically proven asymptomatic X-linked adrenoleukodystrophy determined by elevation of very long chain fatty acids or DNA analysis.
  3. Platelet count in normal range

Exclusion criteria.

  1. Abnormal MRI consistent with childhood cerebral disease
  2. Boys who have undergone bone marrow transplantation
  3. Other medical condition which in the opinion of the investigator prevents evaluation or treatment

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00004418

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United States, Maryland
Kennedy Krieger Institute
Baltimore, Maryland, United States, 21205
Johns Hopkins Hospital
Baltimore, Maryland, United States, 21287-6681
Sponsors and Collaborators
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
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Principal Investigator: Gerald V Raymond, M.D. Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
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Responsible Party: Hugo W. Moser Research Institute at Kennedy Krieger, Inc. Identifier: NCT00004418    
Other Study ID Numbers: 199/13312
Nutricia-Loma Linda ( Other Grant/Funding Number: Nutricia-Loma Linda )
KKI-FDR000685 ( Other Identifier: FDA )
First Posted: October 19, 1999    Key Record Dates
Results First Posted: October 23, 2020
Last Update Posted: October 23, 2020
Last Verified: October 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Keywords provided by Hugo W. Moser Research Institute at Kennedy Krieger, Inc.:
inborn errors of metabolism
rare disease
Additional relevant MeSH terms:
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Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Hereditary Central Nervous System Demyelinating Diseases
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
Peroxisomal Disorders
Metabolic Diseases
Adrenal Insufficiency
Adrenal Gland Diseases
Endocrine System Diseases