Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy
Adrenoleukodystrophy
About this trial
This is an interventional treatment trial for Adrenoleukodystrophy focused on measuring adrenoleukodystrophy, inborn errors of metabolism, rare disease, sphingolipidoses
Eligibility 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