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| Sponsor: | FDA Office of Orphan Products Development |
|---|---|
| Collaborator: |
Hugo W. Moser Research Institute at Kennedy Krieger, Inc. |
| 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: Kim Hollandsworth 800-873-3377 ext 2772 | |
| Kennedy Krieger Institute | Recruiting |
| Baltimore, Maryland, United States, 21205 | |
| Contact: Kim Hollandsworth 443-923-2772 | |
| Study Chair: | Hugo W Moser | Hugo W. Moser Research Institute at Kennedy Krieger, Inc. |
More Information
| Study ID Numbers: | 199/13312, KKI-88-11-22-01, KKI-FDR000685 |
| Study First Received: | October 18, 1999 |
| Last Updated: | June 15, 2009 |
| ClinicalTrials.gov Identifier: | NCT00004418 History of Changes |
| Health Authority: | United States: Federal Government |
|
adrenoleukodystrophy inborn errors of metabolism rare disease sphingolipidoses |
|
Adrenal Insufficiency Autoimmune Diseases Metabolic Diseases Immune System Diseases Demyelinating Diseases Nervous System Diseases Endocrine System Diseases Adrenal Gland Diseases Central Nervous System Diseases Brain Diseases Hereditary Central Nervous System Demyelinating Diseases Mental Retardation |
Metabolism, Inborn Errors Heredodegenerative Disorders, Nervous System Genetic Diseases, Inborn Peroxisomal Disorders Adrenoleukodystrophy Genetic Diseases, X-Linked Addison Disease Neurologic Manifestations Brain Diseases, Metabolic, Inborn Mental Retardation, X-Linked Neurobehavioral Manifestations Brain Diseases, Metabolic |
