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| Study 4 of 27 for search of: | "Adrenoleukodystrophy" |
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| Sponsors and Collaborators: |
FDA Office of Orphan Products Development Kennedy-Krieger Research Institute |
|---|---|
| Information provided by: | FDA Office of Orphan Products Development |
| ClinicalTrials.gov Identifier: | NCT00004418 |
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 |
|---|---|
|
Adrenoleukodystrophy |
Drug: glyceryl trierucate Drug: glyceryl trioleate |
| Study Type: | Interventional |
| Study Design: | Treatment, Open Label |
| Estimated Enrollment: | 30 |
| Study Start Date: | April 1998 |
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 |
Contacts and Locations| United States, Maryland | |
| Johns Hopkins Hospital | Recruiting |
| Baltimore, Maryland, United States, 21287-6681 | |
| Contact: Hugo W Moser 410-502-9405 | |
| Kennedy Krieger Institute | Recruiting |
| Baltimore, Maryland, United States, 21205 | |
| Contact: Hugo W Moser 410-502-9405 | |
| Study Chair: | Hugo W Moser | Kennedy-Krieger Research Institute |
More Information
| Study ID Numbers: | 199/13312, KKI-88-11-22-01, KKI-FDR000685 |
| Study First Received: | October 18, 1999 |
| Last Updated: | June 23, 2005 |
| ClinicalTrials.gov Identifier: | NCT00004418 |
| Health Authority: | United States: Federal Government |
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adrenoleukodystrophy inborn errors of metabolism rare disease sphingolipidoses |
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Sphingolipidoses Addison's disease Adrenal Gland Diseases Demyelinating diseases Hypoadrenalism Brain Diseases Metabolism, Inborn Errors Heredodegenerative Disorders, Nervous System Peroxisomal Disorders Adrenoleukodystrophy Addison Disease Genetic Diseases, X-Linked Brain Diseases, Metabolic, Inborn Neurobehavioral Manifestations X-linked adrenoleukodystrophy |
Adrenal Insufficiency Metabolic Diseases Autoimmune Diseases Demyelinating Diseases Rare Diseases Central Nervous System Diseases Endocrine System Diseases Sphingolipidosis Adrenal gland hypofunction Mental Retardation Genetic Diseases, Inborn Neurologic Manifestations Endocrinopathy Metabolic disorder Brain Diseases, Metabolic |
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Immune System Diseases Nervous System Diseases Mental Retardation, X-Linked Hereditary Central Nervous System Demyelinating Diseases |
