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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: | NCT00004450 |
Purpose
OBJECTIVES: I. Evaluate the efficacy of interferon beta and thalidomide in male patients with adrenoleukodystrophy who show evidence of brain inflammatory response and are receiving concurrent glyceryl trierucate and glyceryl trioleate (Lorenzo's oil).
II. Evaluate the progress of the disease and possible side effects of the medication in these patients.
| Condition | Intervention |
|---|---|
|
Adrenoleukodystrophy |
Drug: glyceryl trierucate Drug: glyceryl trioleate Drug: interferon beta Drug: thalidomide |
| Study Type: | Interventional |
| Study Design: | Treatment, Randomized, Double-Blind, Placebo Control, Efficacy Study |
| Estimated Enrollment: | 60 |
| Study Start Date: | August 1998 |
| Estimated Study Completion Date: | November 2000 |
PROTOCOL OUTLINE: This is randomized, double blind, placebo controlled study. Patients are randomized to receive beta interferon and thalidomide placebo (arm I), thalidomide and beta interferon placebo (arm II), or placebo for both beta interferon and thalidomide (arm III). Patients receive interferon beta by subcutaneous injection and thalidomide orally. All patients are maintained on glyceryl trierucate and glyceryl trioleate (Lorenzo's oil) therapy.
Patients are followed at 3, 6, and 12 months and then may be followed every 6 months thereafter.
Eligibility| Ages Eligible for Study: | 4 Years and older |
| Genders Eligible for Study: | Male |
| Accepts Healthy Volunteers: | No |
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics-- Adrenoleukodystrophy (ALD) diagnosis based on history and examination, MRI, and biochemical assay
Clinical evidence of rapidly progressive phase of cerebral ALD must include 2 or more of the following during the preceding year:
Significant and progressive impairment of school performance Significant loss of cognitive function leading to an IQ of 75 or less Progressive impairment of the ability to understand spoken words Progressive impairment of vision Progressive deterioration of handwriting Progressive difficulty in walking Progressive impairment in speech articulation, and vocabulary Progressive weakness of one or more limbs
Must have MRI abnormalities characteristic of cerebral ALD, especially evidence of the breakdown of the blood-brain barrier using gadolinium contrast medium and magnetization transfer technique Evidence of brain white matter inflammatory response Must not meet criteria for bone marrow transplantation Not in an apparent vegetative state --Prior/Concurrent Therapy-- Concurrent glyceryl trierucate and glyceryl trioleate (Lorenzo's oil) therapy required --Patient Characteristics-- Effective contraception required of all patients
Contacts and Locations More Information
| Study ID Numbers: | 199/13532, KKI-94-06-16-01, KKI-FDR001052 |
| Study First Received: | October 18, 1999 |
| Last Updated: | June 23, 2005 |
| ClinicalTrials.gov Identifier: | NCT00004450 History of Changes |
| Health Authority: | United States: Federal Government |
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adrenoleukodystrophy inborn errors of metabolism rare disease sphingolipidoses |
|
Addison's Disease Sphingolipidoses Immunologic Factors Thalidomide Adrenal Gland Diseases Hypoadrenalism Brain Diseases X-linked Adrenoleukodystrophy Anti-Bacterial Agents Metabolism, Inborn Errors Heredodegenerative Disorders, Nervous System Peroxisomal Disorders Adrenoleukodystrophy Addison Disease Genetic Diseases, X-Linked |
Brain Diseases, Metabolic, Inborn Metabolic Disorder Neurobehavioral Manifestations Adrenal Insufficiency Metabolic Diseases Autoimmune Diseases Adrenal Gland Hypofunction Demyelinating Diseases Interferons Interferon-beta Rare Diseases Central Nervous System Diseases Endocrine System Diseases Sphingolipidosis Angiogenesis Inhibitors |
|
Anti-Infective Agents Immunologic Factors Thalidomide Antineoplastic Agents Physiological Effects of Drugs Adrenal Gland Diseases Brain Diseases Hereditary Central Nervous System Demyelinating Diseases Anti-Bacterial Agents Metabolism, Inborn Errors Heredodegenerative Disorders, Nervous System Peroxisomal Disorders Therapeutic Uses Adrenoleukodystrophy Addison Disease |
Genetic Diseases, X-Linked Growth Inhibitors Angiogenesis Modulating Agents Brain Diseases, Metabolic, Inborn Neurobehavioral Manifestations Adrenal Insufficiency Metabolic Diseases Autoimmune Diseases Demyelinating Diseases Immune System Diseases Growth Substances Interferons Nervous System Diseases Interferon-beta Central Nervous System Diseases |
