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Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy | Clinical Trials | Clareo Health

TERMINATEDPHASE2

Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy

Study of Glyceryl Trierucate and Glyceryl Trioleate (Lorenzo's Oil) Therapy in Male Children With Adrenoleukodystrophy

NCT00004418•Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

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 they reach the age of 13 years or death.

Eligibility

Age

1 - 8 years

Sex

MALE

Healthy Volunteers

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

Locations (2)

Kennedy Krieger Institute

Baltimore, Maryland, United States

Johns Hopkins Hospital

Baltimore, Maryland, United States

Key Dates

Start Date

April 1, 1998

Primary Completion Date

December 1, 2014

Completion Date

December 1, 2014

Last Updated

October 23, 2020

Enrollment

126

ACTUAL participants

Conditions

Adrenoleukodystrophy

Interventions

glyceryl trierucate/glyceryl trioleate

DRUG

The Myelin Disorders Biorepository Project

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RECRUITINGPHASE1

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: October 23, 2020Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy

Inclusion Criteria

Exclusion Criteria

The Myelin Disorders Biorepository Project

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells

Adrenoleukodystrophy National Registry Study

Quality of Life in Women with X-linked Adrenoleukodystrophy

Precision Exercise in Children With Malignant Hemopathies

Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product