Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy

This study has suspended participant recruitment.

(No funding and moved to expanded access)

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: September 17, 2014

Last verified: September 2014

History of Changes

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:

Genetics Home Reference related topics: CASK-related intellectual disability D-bifunctional protein deficiency MECP2 duplication syndrome PPM-X syndrome Renpenning syndrome SYNGAP1-related intellectual disability X-linked adrenoleukodystrophy alpha-methylacyl-CoA racemase deficiency familial glucocorticoid deficiency leukoencephalopathy with vanishing white matter megalencephalic leukoencephalopathy with subcortical cysts peroxisomal acyl-CoA oxidase deficiency succinic semialdehyde dehydrogenase deficiency

Drug Information available for: Glyceryl trioleate

Genetic and Rare Diseases Information Center resources: X-linked Adrenoleukodystrophy Adrenomyeloneuropathy Sphingolipidosis Peroxisomal Biogenesis Disorders Hypoadrenalism

U.S. FDA Resources

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 Study Completion Date:	November 2014
Estimated Primary Completion Date:	November 2014 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
GTO/GTE treatment Treatment with GTO/GTE orally; 30-60 ml daily for study period	Drug: glyceryl trierucate Glyceryl trierucate is an oil to reduce very long chain fatty acids Other Names: Lorenzo's oil GTE Drug: glyceryl trioleate Patients receive 2-4 tablespoons of a mixture of glyceryl trierucate and glyceryl trioleate oil once daily. Other Names: Lorenzo's Oil GTO

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 (Child)
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
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.

Investigators


Principal Investigator:	Gerald V Raymond, M.D.	Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

More Information


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:	September 17, 2014
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 Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic Brain Diseases Central Nervous System Diseases Nervous System Diseases Hereditary Central Nervous System Demyelinating Diseases 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 Peroxisomal Disorders Metabolic Diseases Adrenal Insufficiency Adrenal Gland Diseases Endocrine System Diseases

ClinicalTrials.gov processed this record on September 28, 2016

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