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Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy
This study has been completed.
Study NCT00004450   Information provided by FDA Office of Orphan Products Development
First Received: October 18, 1999   Last Updated: June 23, 2005   History of Changes

October 18, 1999
June 23, 2005
August 1998
 
 
 
Complete list of historical versions of study NCT00004450 on ClinicalTrials.gov Archive Site
 
 
 
Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy
 

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.

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.

 
Interventional
Treatment, Randomized, Double-Blind, Placebo Control, Efficacy Study
Adrenoleukodystrophy
  • Drug: glyceryl trierucate
  • Drug: glyceryl trioleate
  • Drug: interferon beta
  • Drug: thalidomide
 
 

*   Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
 
Completed
60
November 2000
 

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

Male
4 Years and older
No
Contact information is only displayed when the study is recruiting subjects
 
 
NCT00004450
 
199/13532, KKI-94-06-16-01, KKI-FDR001052
FDA Office of Orphan Products Development
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Study Chair: Hugo Wolfgang Moser Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
FDA Office of Orphan Products Development
January 2001

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP