Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy

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Brief Title

Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy


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).

      II. Evaluate the progress of the disease and possible side effects of the medication in these
      patients.
    

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 Type

Interventional




Condition

Adrenoleukodystrophy

Intervention

glyceryl trierucate


Publications

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

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

60

Start Date

August 1998

Completion Date

November 2000


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
      

Gender

Male

Ages

4 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Hugo Wolfgang Moser, , 



Administrative Informations


NCT ID

NCT00004450

Organization ID

199/13532

Secondary IDs

KKI-94-06-16-01


Study Sponsor

FDA Office of Orphan Products Development

Collaborators

 Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

Study Sponsor

Hugo Wolfgang Moser, Study Chair, Hugo W. Moser Research Institute at Kennedy Krieger, Inc.


Verification Date

January 2001