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Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy

Primary Purpose

Adrenoleukodystrophy

Status
Completed
Phase
Not Applicable
Locations
Study Type
Interventional
Intervention
glyceryl trierucate
glyceryl trioleate
interferon beta
thalidomide
Sponsored by
FDA Office of Orphan Products Development
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Adrenoleukodystrophy focused on measuring adrenoleukodystrophy, inborn errors of metabolism, rare disease, sphingolipidoses

Eligibility Criteria

4 Years - undefined (Child, Adult, Older Adult)MaleDoes not accept healthy volunteers

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

Sites / Locations

    Outcomes

    Primary Outcome Measures

    Secondary Outcome Measures

    Full Information

    First Posted
    October 18, 1999
    Last Updated
    March 24, 2015
    Sponsor
    FDA Office of Orphan Products Development
    Collaborators
    Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
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    1. Study Identification

    Unique Protocol Identification Number
    NCT00004450
    Brief Title
    Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    January 2001
    Overall Recruitment Status
    Completed
    Study Start Date
    August 1998 (undefined)
    Primary Completion Date
    undefined (undefined)
    Study Completion Date
    November 2000 (undefined)

    3. Sponsor/Collaborators

    Name of the Sponsor
    FDA Office of Orphan Products Development
    Collaborators
    Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

    4. Oversight

    5. 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). 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.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Adrenoleukodystrophy
    Keywords
    adrenoleukodystrophy, inborn errors of metabolism, rare disease, sphingolipidoses

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Not Applicable
    Masking
    Double
    Allocation
    Randomized
    Enrollment
    60 (false)

    8. Arms, Groups, and Interventions

    Intervention Type
    Drug
    Intervention Name(s)
    glyceryl trierucate
    Intervention Type
    Drug
    Intervention Name(s)
    glyceryl trioleate
    Intervention Type
    Drug
    Intervention Name(s)
    interferon beta
    Intervention Type
    Drug
    Intervention Name(s)
    thalidomide

    10. Eligibility

    Sex
    Male
    Minimum Age & Unit of Time
    4 Years
    Accepts Healthy Volunteers
    No
    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
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Hugo Wolfgang Moser
    Organizational Affiliation
    Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
    Official's Role
    Study Chair

    12. IPD Sharing Statement

    Learn more about this trial

    Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy

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