Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy
Information source: FDA Office of Orphan Products Development
Information obtained from ClinicalTrials.gov on December 31, 2007
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Adrenoleukodystrophy
Intervention: glyceryl trierucate (Drug); glyceryl trioleate (Drug); interferon beta (Drug); thalidomide (Drug)
Phase: N/A
Status: Completed
Sponsored by: FDA Office of Orphan Products Development Official(s) and/or principal investigator(s):
Hugo Wolfgang Moser, Study Chair, Affiliation: Kennedy-Krieger Research Institute
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).
II. Evaluate the progress of the disease and possible side effects of the medication in
these patients.
Clinical Details
Study design: Treatment, Randomized, Double-Blind, Placebo Control, Efficacy Study
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.
Eligibility
Minimum age: 4 Years.
Maximum age: N/A.
Gender(s): Male.
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
Locations and Contacts
Additional Information
Starting date: August 1998
Ending date: November 2000
Last updated: June 23, 2005
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