Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy
Study Details
Study Description
Brief Summary
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).
- Evaluate the progress of the disease and possible side effects of the medication in these patients.
Condition or Disease | Intervention/Treatment | Phase |
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N/A |
Detailed Description
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.
Study Design
Outcome Measures
Primary Outcome Measures
Eligibility Criteria
Criteria
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
Locations
No locations specified.Sponsors and Collaborators
- FDA Office of Orphan Products Development
- Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Investigators
- Study Chair: Hugo Wolfgang Moser, Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- 199/13532
- KKI-94-06-16-01
- KKI-FDR001052